DE69221730T2 - Sequenzspezifische bindende Polymere für Duplex Nucleinsäuren - Google Patents

Sequenzspezifische bindende Polymere für Duplex Nucleinsäuren

Info

Publication number
DE69221730T2
DE69221730T2 DE69221730T DE69221730T DE69221730T2 DE 69221730 T2 DE69221730 T2 DE 69221730T2 DE 69221730 T DE69221730 T DE 69221730T DE 69221730 T DE69221730 T DE 69221730T DE 69221730 T2 DE69221730 T2 DE 69221730T2
Authority
DE
Germany
Prior art keywords
sequence
nucleic acids
specific binding
duplex nucleic
binding polymers
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
DE69221730T
Other languages
English (en)
Other versions
DE69221730D1 (de
Inventor
James Summerton
Dwight Weller
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sarepta Therapeutics Inc
Original Assignee
Antivirals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Antivirals Inc filed Critical Antivirals Inc
Publication of DE69221730D1 publication Critical patent/DE69221730D1/de
Application granted granted Critical
Publication of DE69221730T2 publication Critical patent/DE69221730T2/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/545—Heterocyclic compounds
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/548—Phosphates or phosphonates, e.g. bone-seeking
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6813—Hybridisation assays
    • C12Q1/6839—Triple helix formation or other higher order conformations in hybridisation assays
    • B—PERFORMING OPERATIONS; TRANSPORTING
    • B01—PHYSICAL OR CHEMICAL PROCESSES OR APPARATUS IN GENERAL
    • B01J—CHEMICAL OR PHYSICAL PROCESSES, e.g. CATALYSIS OR COLLOID CHEMISTRY; THEIR RELEVANT APPARATUS
    • B01J2219/00—Chemical, physical or physico-chemical processes in general; Their relevant apparatus
    • B01J2219/00274—Sequential or parallel reactions; Apparatus and devices for combinatorial chemistry or for making arrays; Chemical library technology
    • B01J2219/00718—Type of compounds synthesised
    • B01J2219/0072—Organic compounds
DE69221730T 1991-06-20 1992-06-18 Sequenzspezifische bindende Polymere für Duplex Nucleinsäuren Expired - Fee Related DE69221730T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US07/719,732 US5166315A (en) 1989-12-20 1991-06-20 Sequence-specific binding polymers for duplex nucleic acids
PCT/US1992/005208 WO1993000352A1 (en) 1991-06-20 1992-06-18 Sequence-specific binding polymers for duplex nucleic acids

Publications (2)

Publication Number Publication Date
DE69221730D1 DE69221730D1 (de) 1997-09-25
DE69221730T2 true DE69221730T2 (de) 1998-02-26

Family

ID=24891147

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69221730T Expired - Fee Related DE69221730T2 (de) 1991-06-20 1992-06-18 Sequenzspezifische bindende Polymere für Duplex Nucleinsäuren

Country Status (10)

Country Link
US (1) US5166315A (de)
EP (1) EP0592511B1 (de)
JP (1) JPH06508526A (de)
KR (1) KR100225090B1 (de)
AT (1) ATE157098T1 (de)
AU (1) AU665560B2 (de)
CA (1) CA2110988C (de)
DE (1) DE69221730T2 (de)
TW (1) TW466243B (de)
WO (1) WO1993000352A1 (de)

Families Citing this family (990)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5405938A (en) * 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) * 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US6441130B1 (en) 1991-05-24 2002-08-27 Isis Pharmaceuticals, Inc. Linked peptide nucleic acids
US6451968B1 (en) * 1991-05-24 2002-09-17 Isis Pharmaceuticals, Inc. Peptide nucleic acids
US5714331A (en) * 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5766855A (en) * 1991-05-24 1998-06-16 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity and sequence specificity
US6414112B1 (en) 1991-05-24 2002-07-02 Ole Buchardt Peptide nucleic acids having 2,6-diaminopurine nucleobases
US6713602B1 (en) * 1991-05-24 2004-03-30 Ole Buchardt Synthetic procedures for peptide nucleic acids
US7223833B1 (en) * 1991-05-24 2007-05-29 Isis Pharmaceuticals, Inc. Peptide nucleic acid conjugates
US5719262A (en) * 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
DK51092D0 (da) * 1991-05-24 1992-04-15 Ole Buchardt Oligonucleotid-analoge betegnet pna, monomere synthoner og fremgangsmaade til fremstilling deraf samt anvendelser deraf
US5726014A (en) * 1991-06-27 1998-03-10 Genelabs Technologies, Inc. Screening assay for the detection of DNA-binding molecules
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
US5473060A (en) * 1993-07-02 1995-12-05 Lynx Therapeutics, Inc. Oligonucleotide clamps having diagnostic applications
AU679566B2 (en) 1993-09-03 1997-07-03 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
US6710164B1 (en) 1993-11-22 2004-03-23 Peter E. Nielsen Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
GB2284208A (en) * 1993-11-25 1995-05-31 Pna Diagnostics As Nucleic acid analogues with a chelating functionality for metal ions
AU1602995A (en) * 1994-01-11 1995-08-01 Affymax Technologies N.V. Methods for the solid phase synthesis of glycoconjugates
FR2728264B1 (fr) * 1994-12-16 1997-01-31 Rhone Poulenc Rorer Sa Purification d'adn par formation de triple helice avec un oligonucleotide immobilise
US7038026B2 (en) 2000-05-26 2006-05-02 Centelion Purification of a triple heli formation with an immobilized oligonucleotide
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
JPH11506605A (ja) * 1995-06-07 1999-06-15 アボツト・ラボラトリーズ 増幅反応におけるバックグラウンドを減少させるためにプローブをマスキングする方法
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
ATE311402T1 (de) * 1996-07-24 2005-12-15 Buchardt Dorte Peptidnukleinsäuren mit erhöhter bindungsaffinität, sequenzspezifität und löslichkeit
US6251591B1 (en) 1997-02-27 2001-06-26 Lorne Park Research, Inc. Quantitative method for detecting nucleotide concentration
US6046004A (en) * 1997-02-27 2000-04-04 Lorne Park Research, Inc. Solution hybridization of nucleic acids with antisense probes having modified backbones
AU731909B2 (en) 1997-07-01 2001-04-05 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US6383808B1 (en) 2000-09-11 2002-05-07 Isis Pharmaceuticals, Inc. Antisense inhibition of clusterin expression
WO2000018885A1 (en) 1998-09-29 2000-04-06 Gamida Cell Ltd. Methods of controlling proliferation and differentiation of stem and progenitor cells
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US7321828B2 (en) 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
CA2329252A1 (en) 1998-05-21 1999-11-25 Isis Pharmaceuticals Inc. Compositions and methods for topical delivery of oligonucleotides
AU745880B2 (en) * 1998-05-21 2002-04-11 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6255050B1 (en) 1998-05-22 2001-07-03 Lorne Park Research, Inc. Dynamic hybridization system
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6242589B1 (en) 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6225293B1 (en) 1998-09-02 2001-05-01 Isis Pharmaceuticals, Inc. Methods and compounds for tracking the biodistribution of macromolecule-carrier combinations
US6335194B1 (en) 1998-09-29 2002-01-01 Isis Pharmaceuticals, Inc. Antisense modulation of survivin expression
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
US6127124A (en) * 1999-01-20 2000-10-03 Isis Pharmaceuticals, Inc. Fluorescence based nuclease assay
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US7534605B2 (en) * 1999-06-08 2009-05-19 Yissum Research Development Company Of The Hebrew University Of Jerusalem CD44 polypeptides, polynucleotides encoding same, antibodies directed thereagainst and method of using same for diagnosing and treating inflammatory diseases
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6593466B1 (en) 1999-07-07 2003-07-15 Isis Pharmaceuticals, Inc. Guanidinium functionalized nucleotides and precursors thereof
US7332275B2 (en) 1999-10-13 2008-02-19 Sequenom, Inc. Methods for detecting methylated nucleotides
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
AU3087801A (en) * 2000-02-04 2001-08-14 Molecular Dynamics Inc Human genome-derived single exon nucleic acid probes useful for analysis of geneexpression in human breast and hbl 100 cells
AU5340801A (en) * 2000-04-13 2001-10-30 Thomas N Wight Therapeutic compounds and methods
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US6686188B2 (en) * 2000-05-26 2004-02-03 Amersham Plc Polynucleotide encoding a human myosin-like polypeptide expressed predominantly in heart and muscle
US6656700B2 (en) 2000-05-26 2003-12-02 Amersham Plc Isoforms of human pregnancy-associated protein-E
EP3020804A1 (de) 2000-07-06 2016-05-18 Sarepta Therapeutics, Inc. Umwandlung der wachstumsfaktor-beta (tgf-beta) blockierungsmittelbehandelten stammzellenzusammensetzung und verfahren
US6958214B2 (en) * 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
EP2336166A1 (de) 2000-10-12 2011-06-22 University Of Rochester Zusammensetzungen die die Proliferation von Krebszellen hemmen
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US6573051B2 (en) * 2001-03-09 2003-06-03 Molecular Staging, Inc. Open circle probes with intramolecular stem structures
EP1992643A3 (de) 2001-06-20 2008-12-10 Genentech, Inc. Zusammensetzungen und Verfahren zur Behandlung und Diagnose von Tumoren
US7803915B2 (en) 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
CA2790034A1 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
WO2003013437A2 (en) * 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
US20040096880A1 (en) * 2001-08-07 2004-05-20 Kmiec Eric B. Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
WO2003024392A2 (en) 2001-09-18 2003-03-27 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
NZ566396A (en) 2001-10-09 2009-07-31 Isis Pharmaceuticals Inc Antisense modulation of insulin-like growth factor binding protein 5 expressions
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
WO2003088808A2 (en) 2002-04-16 2003-10-30 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP1575571A4 (de) 2002-01-02 2008-06-25 Genentech Inc Zusammensetzungen und verfahren zur diagnose und behandlung eines tumors
IL152904A0 (en) 2002-01-24 2003-06-24 Gamida Cell Ltd Utilization of retinoid and vitamin d receptor antagonists for expansion of renewable stem cell populations
WO2003062404A1 (en) 2002-01-25 2003-07-31 Gamida-Cell Ltd. Methods of expanding stem and progenitor cells and expanded cell populations obtained thereby
US7553619B2 (en) 2002-02-08 2009-06-30 Qiagen Gmbh Detection method using dissociated rolling circle amplification
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
WO2003093296A2 (en) 2002-05-03 2003-11-13 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof, and related methods
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
AU2003231912A1 (en) * 2002-06-12 2003-12-31 Tel Aviv Medical Center Research Development Fund Methods of detecting and treating prostate cancer
AU2003257181A1 (en) 2002-08-05 2004-02-23 University Of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
EP1537208A1 (de) 2002-09-13 2005-06-08 Replicor, Inc. Nicht-sequenzkomplementäre antivirale oligonukleotide
CA2499770A1 (en) * 2002-09-20 2004-04-01 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches.
WO2004031350A2 (en) 2002-09-26 2004-04-15 Amgen, Inc. Modulation of forkhead box o1a expression
WO2004044139A2 (en) 2002-11-05 2004-05-27 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US9150606B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
CA2504720C (en) 2002-11-05 2013-12-24 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
CA2505801A1 (en) 2002-11-13 2004-05-27 Rosanne Crooke Antisense modulation of apolipoprotein b expression
DK1569695T3 (da) 2002-11-13 2013-08-05 Genzyme Corp Antisense-modulering af apolipoprotein-b-ekspression
US20060009378A1 (en) * 2002-11-14 2006-01-12 Itshak Golan Novel galectin sequences and compositions and methods utilizing same for treating or diagnosing arthritis and other chronic inflammatory diseases
EP2292259A3 (de) 2002-11-15 2011-03-23 MUSC Foundation For Research Development Komplement-Modulatoren für Komplement-Rezeptor 2
WO2004046330A2 (en) 2002-11-15 2004-06-03 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
AU2003294462C1 (en) 2002-11-21 2011-06-30 University Of Utah Research Foundation Purinergic modulation of smell
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
CA2510587A1 (en) 2002-12-20 2004-07-15 Qiagen Gmbh Nucleic acid amplification
US9487823B2 (en) 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
US6977153B2 (en) * 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
NZ541637A (en) 2003-02-11 2008-07-31 Antisense Therapeutics Pty Ltd Modulation of insulin like growth factor I receptor
US7002006B2 (en) * 2003-02-12 2006-02-21 Isis Pharmaceuticals, Inc. Protection of nucleosides
AU2004215133B2 (en) 2003-02-27 2010-10-14 Yeda Research And Development Co. Ltd. Nucleic acid molecules, polypeptides, antibodies and compositions containing same useful for treating and detecting influenza virus infection
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US8043834B2 (en) 2003-03-31 2011-10-25 Qiagen Gmbh Universal reagents for rolling circle amplification and methods of use
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
CA2523672C (en) 2003-04-29 2012-07-17 Avi Biopharma, Inc. Compositions for enhancing transport of molecules into cells
US7276599B2 (en) 2003-06-02 2007-10-02 Isis Pharmaceuticals, Inc. Oligonucleotide synthesis with alternative solvents
CN1984921B (zh) 2003-06-03 2010-06-16 Isis药物公司 存活蛋白表达的调节
DK3604537T3 (da) 2003-06-13 2022-02-28 Alnylam Europe Ag Dobbeltstrenget ribonukleinsyre med forøget effektivitet i en organisme
CA2533701A1 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20050153935A1 (en) * 2003-09-12 2005-07-14 Iversen Patrick L. Compound and method for treating androgen-independent prostate cancer
EP1668130A2 (de) 2003-09-18 2006-06-14 Isis Pharmaceuticals, Inc. Modulation der eif4e-expression
US20050222068A1 (en) * 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
EP1689432B1 (de) 2003-11-17 2009-12-30 Genentech, Inc. Zusammensetzungen und verfahren zur behandlung von tumoren hämatopoetischen ursprungs
WO2006054296A2 (en) 2004-11-17 2006-05-26 Spectrum Dynamics Llc Methods of detecting prostate cancer
EP2363480A3 (de) 2004-01-20 2015-10-07 Isis Pharmaceuticals, Inc. Modulierung der Glukokortikoid-Rezeptor-Expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8778900B2 (en) 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
WO2005072527A2 (en) * 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20060147946A1 (en) * 2004-01-27 2006-07-06 Pinchas Akiva Novel calcium channel variants and methods of use thereof
US7842459B2 (en) * 2004-01-27 2010-11-30 Compugen Ltd. Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
WO2005089268A2 (en) 2004-03-15 2005-09-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of rna by rnase h
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP1737878A2 (de) 2004-04-05 2007-01-03 Alnylam Pharmaceuticals Inc. Verfahren und reagentien für die synthese und aufreinigung von oligonukleotiden
EP1750776A2 (de) 2004-04-30 2007-02-14 Alnylam Pharmaceuticals Inc. Oligonukleotide mit c5-modifiziertem pyrimidin
NZ550772A (en) 2004-05-21 2009-10-30 Uab Research Foundation Variable lymphocyte receptors, related polypeptides and nucleic acids, and uses thereof
US20090048192A1 (en) * 2004-06-03 2009-02-19 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
EP4272748A3 (de) 2004-06-28 2024-03-27 The University Of Western Australia Antisense-oligonukleotide zur induzierung von exon-skipping und verfahren zur verwendung davon
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
DK1766012T3 (da) 2004-07-02 2011-09-19 Avi Biopharma Inc Antisense-antibakteriel fremgangsmåde og forbindelse
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
EP1799812A4 (de) 2004-09-16 2009-09-09 Gamida Cell Ltd Verfahren zur ex-vivo-expansion von progenitor- und stammzellen durch gemeinsame kultivierung mit mesenchymzellen
US8129352B2 (en) * 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
ES2729826T3 (es) * 2004-09-23 2019-11-06 Arc Medical Devices Inc Composiciones farmacéuticas y métodos relacionados para inhibir adherencias fibrosas o enfermedad inflamatoria usando fucanos con bajo contenido de sulfato
US7524829B2 (en) * 2004-11-01 2009-04-28 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
WO2006050999A2 (en) * 2004-11-15 2006-05-18 Obe Therapy Biotechnology S.A.S Methods of reducing body fat
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
CN101175769A (zh) 2005-03-10 2008-05-07 健泰科生物技术公司 用于调控血管完整性的方法和组合物
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
US20060240032A1 (en) * 2005-03-31 2006-10-26 Hinrichs David J Immunomodulating compositions and methods for use in the treatment of human autoimmune diseases
EP1863908B1 (de) 2005-04-01 2010-11-17 Qiagen GmbH Reverse transkription und amplifikation von rna bei simultaner degradierung von dna
US9505867B2 (en) 2005-05-31 2016-11-29 Ecole Polytechmique Fédérale De Lausanne Triblock copolymers for cytoplasmic delivery of gene-based drugs
WO2006133022A2 (en) 2005-06-03 2006-12-14 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
US8252756B2 (en) 2005-06-14 2012-08-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
WO2007015168A2 (en) * 2005-07-01 2007-02-08 Dako Denmark A/S Monomeric and polymeric linkers useful for conjugating biological molecules and other substances
WO2007007317A1 (en) 2005-07-07 2007-01-18 Yissum Research Development Company Of The Hebrew University Of Jerusalem Nucleic acid agents for downregulating h19, and methods of using same
US7790694B2 (en) * 2005-07-13 2010-09-07 Avi Biopharma Inc. Antisense antibacterial method and compound
AU2006267051B2 (en) 2005-07-13 2013-03-07 Sarepta Therapeutics, Inc. Antisense antibacterial method and compound
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7776532B2 (en) 2005-08-11 2010-08-17 Synthetic Genomics, Inc. Method for in vitro recombination
AU2006281569A1 (en) 2005-08-17 2007-02-22 Medexis S.A. Composition and method for determination of CK19 expression
JP5523705B2 (ja) 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Ｍｉｒ−１２２ａをモジュレートする使用方法
EP1937278B1 (de) * 2005-09-08 2012-07-25 AVI BioPharma, Inc. Antivirale antisense-verbindung und verfahren zur behandlung von picornavirus-infektion
WO2007030691A2 (en) * 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
US8906609B1 (en) 2005-09-26 2014-12-09 Arrowhead Center, Inc. Label-free biomolecule sensor based on surface charge modulated ionic conductance
IL172297A (en) 2005-10-03 2016-03-31 Compugen Ltd Soluble vegfr-1 variants for the diagnosis of preeclampsia
EP2189522A1 (de) 2005-10-14 2010-05-26 MUSC Foundation For Research Development PAX2 als Ziel zur Induktion einer Immunität gegen DEFB1-vermittelte Tumore und bei der Krebstherapie
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
CN101365801B (zh) 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
CA2626690A1 (en) 2005-11-09 2007-05-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
WO2007062380A2 (en) 2005-11-21 2007-05-31 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
US8846393B2 (en) 2005-11-29 2014-09-30 Gamida-Cell Ltd. Methods of improving stem cell homing and engraftment
US8313901B2 (en) * 2005-12-21 2012-11-20 Yale University Methods and compositions related to the modulation of riboswitches
CN101437933B (zh) 2005-12-28 2013-11-06 斯克里普斯研究所 作为药物靶标的天然反义和非编码的rna转录物
EP2216339A1 (de) 2006-01-16 2010-08-11 Compugen Ltd. Neuartiges Nukleotid und neuartige Aminosäuresequenzen und Verfahren zu deren Verwendung zur Diagnose
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
US8129515B2 (en) 2006-01-27 2012-03-06 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
KR20130042043A (ko) 2006-01-27 2013-04-25 아이시스 파마수티컬즈 인코포레이티드 6-변형된 바이시클릭 핵산 유사체
ATE467688T1 (de) * 2006-03-07 2010-05-15 Avi Biopharma Inc Antivirale antisense-verbindung und verfahren zur behandlung von arenavirus-infektion
NZ571568A (en) 2006-03-31 2010-11-26 Alnylam Pharmaceuticals Inc Double-stranded RNA molecule compositions and methods for inhibiting expression of Eg5 gene
WO2007125173A2 (en) 2006-05-03 2007-11-08 Baltic Technology Development, Ltd. Antisense agents combining strongly bound base - modified oligonucleotide and artificial nuclease
US20090326042A1 (en) 2006-05-05 2009-12-31 Isis Pharmaceuticals, Inc Compounds and methods for modulating expression of crp
JP2010505741A (ja) * 2006-05-10 2010-02-25 エイブイアイ バイオファーマ， インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
US8785407B2 (en) * 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
DK2066684T3 (da) 2006-05-11 2012-10-22 Isis Pharmaceuticals Inc 5´-Modificerede bicycliske nukleinsyreanaloge
US20070265215A1 (en) * 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
CN103614375A (zh) 2006-05-11 2014-03-05 阿尔尼拉姆医药品有限公司 抑制pcsk9基因表达的组合物和方法
CA2652770A1 (en) 2006-05-19 2007-11-29 Alnylam Pharmaceuticals, Inc. Rnai modulation of aha and therapeutic uses thereof
WO2007137220A2 (en) 2006-05-22 2007-11-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
WO2008033866A2 (en) * 2006-09-11 2008-03-20 Yale University Methods and compositions for the use of lysine riboswitches
AU2007299629C1 (en) 2006-09-21 2012-05-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the HAMP gene
DK2068886T3 (da) 2006-10-03 2013-11-18 Tekmira Pharmaceuticals Corp Lipidholdige præparater
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
JP2010512327A (ja) 2006-12-11 2010-04-22 ユニヴァーシティー オブ ユタ リサーチ ファウンデーション 病的血管形成および脈管透過性の処置用の組成物および方法
US7928083B2 (en) * 2007-01-16 2011-04-19 Yissum Research Development Company Of The Hebrew University Of Jerusalem H19 silencing nucleic acid agents for treating rheumatoid arthritis
US20100196403A1 (en) * 2007-01-29 2010-08-05 Jacob Hochman Antibody conjugates for circumventing multi-drug resistance
WO2009045469A2 (en) 2007-10-02 2009-04-09 Amgen Inc. Increasing erythropoietin using nucleic acids hybridizable to micro-rna and precursors thereof
MX2009008470A (es) 2007-02-09 2009-11-26 Univ Northwestern Particulas para detectar objetivos intracelulares.
JP2010521977A (ja) 2007-03-22 2010-07-01 イェール ユニバーシティー オルタナティブスプライシングを調節するリボスイッチに関する方法および組成物
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
AP3018A (en) 2007-03-29 2014-10-31 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expressionof a gene from the ebola
EP2162552A4 (de) 2007-05-11 2010-06-30 Univ Johns Hopkins Biomarker für melanome
JP2010528617A (ja) 2007-05-29 2010-08-26 イェール ユニバーシティー リボスイッチならびにリボスイッチを使用するためのおよびリボスイッチと共てに使用するための方法および組成物
JP2010528616A (ja) * 2007-05-29 2010-08-26 イェール ユニバーシティー オルタナティブスプライシングとｒｎａのスプライシングを調節するリボスイッチに関連する方法と組成物
AU2008259907B2 (en) 2007-05-30 2014-12-04 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
ES2798758T3 (es) * 2007-06-06 2020-12-14 Sarepta Therapeutics Inc Proteínas variantes de empalme her2 y her3 solubles, oligonucleótidos de cambio de empalme y su uso en el tratamiento de enfermedades
WO2008154401A2 (en) 2007-06-08 2008-12-18 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
US20100184823A1 (en) 2007-07-05 2010-07-22 Mark Aron Labow dsRNA For Treating Viral Infection
WO2009006478A2 (en) 2007-07-05 2009-01-08 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
EP2188298B1 (de) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Tetrahydropyrannukleinsäureanaloga
JP2010537638A (ja) 2007-08-28 2010-12-09 ユーエービー リサーチ ファウンデーション 合成アポリポ蛋白質ｅ模倣ポリペプチドおよび使用方法
WO2009032693A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
US8415455B2 (en) 2007-09-04 2013-04-09 Compugen Ltd Polypeptides and polynucleotides, and uses thereof as a drug target for producing drugs and biologics
US8445217B2 (en) 2007-09-20 2013-05-21 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
WO2009039442A1 (en) 2007-09-21 2009-03-26 California Institute Of Technology Nfia in glial fate determination, glioma therapy and astrocytoma treatment
US8097712B2 (en) 2007-11-07 2012-01-17 Beelogics Inc. Compositions for conferring tolerance to viral disease in social insects, and the use thereof
ES2641290T3 (es) 2007-11-20 2017-11-08 Ionis Pharmaceuticals, Inc Modulación de la expresión de CD40
US8546556B2 (en) * 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
EP2245159A2 (de) 2007-12-10 2010-11-03 Alnylam Pharmaceuticals Inc. Zusammensetzungen und verfahren zur hemmung der expression des faktor-vii-gens
AU2008345033B2 (en) * 2007-12-28 2014-04-03 Sarepta Therapeutics, Inc. Immunomodulatory agents and methods of use
JP5749494B2 (ja) 2008-01-02 2015-07-15 テクミラ ファーマシューティカルズ コーポレイション 核酸の送達のための改善された組成物および方法
EP2265627A2 (de) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclische cyclohexitolnukleinsäureanaloga
CA2716793A1 (en) 2008-03-05 2009-09-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 and vegf genes
EP2282744B1 (de) 2008-03-21 2018-01-17 Ionis Pharmaceuticals, Inc. Oligomere verbindungen mit trizyklischen nukleosiden und anwendungsverfahren dafür
EP2285819B1 (de) 2008-04-04 2013-10-16 Isis Pharmaceuticals, Inc. Oligomere verbindungen, die neutral gebundene, terminal bicyclische nukleoside enthalten
EP2982753B1 (de) 2008-04-18 2018-06-06 Baxter International Inc. Zusammensetzung auf mikrokügelchenbasis zur vorbeugung und/oder abwendung einer neuerkrankung an autoimmunem diabetes
EP3081648A1 (de) 2008-08-25 2016-10-19 Excaliard Pharmaceuticals, Inc. Gegen den bindegewebe-wachstumsfaktor gerichtete antisense-oligonukleotide und verwendungen davon
EP3208337A1 (de) 2008-09-02 2017-08-23 Alnylam Pharmaceuticals, Inc. Zusammensetzungen zur kombinationshemmung der expression eines mutanten egfr und il-6
US8604192B2 (en) 2008-09-24 2013-12-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acids analogs
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
JP5529142B2 (ja) 2008-09-25 2014-06-25 アルナイラム ファーマシューティカルズ， インコーポレイテッド 血清アミロイドａ遺伝子の発現を阻害するための脂質製剤組成物および方法
AU2009303345B2 (en) 2008-10-09 2015-08-20 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
CA2966011C (en) 2008-10-15 2021-10-19 Ionis Pharmaceuticals, Inc. Modulation of factor 11 expression
US8168775B2 (en) 2008-10-20 2012-05-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
CN102264374B (zh) 2008-10-24 2015-01-07 Isis制药公司 5′和2′双取代的核苷和由其制备的低聚化合物
JP5864257B2 (ja) 2008-10-24 2016-02-17 サレプタ セラピューティクス， インコーポレイテッド Ｄｍｄのための複数のエキソンスキッピング組成物
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
AU2009316286B2 (en) 2008-11-24 2016-05-26 Northwestern University Polyvalent RNA-nanoparticle compositions
WO2010061393A1 (en) 2008-11-30 2010-06-03 Compugen Ltd. He4 variant nucleotide and amino acid sequences, and methods of use thereof
ES2629630T3 (es) 2008-12-04 2017-08-11 Curna, Inc. Tratamiento de enfermedades relacionadas con eritropoyetina (EPO) mediante inhibición del transcrito antisentido natural a EPO
US20110294870A1 (en) 2008-12-04 2011-12-01 Opko Curna, Llc Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
CN102307997B (zh) 2008-12-04 2018-03-30 库尔纳公司 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1(sirt1)相关的疾病
EP2633854B1 (de) 2008-12-05 2015-09-16 Yeda Research And Development Co. Ltd. miRNA-9 or miRNA-9* zur Verwendung in der Behandlung von ALS
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
CA2746508A1 (en) * 2008-12-17 2010-07-15 Avi Biopharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
US20100294952A1 (en) * 2009-01-15 2010-11-25 Northwestern University Controlled agent release and sequestration
US20120101148A1 (en) 2009-01-29 2012-04-26 Alnylam Pharmaceuticals, Inc. lipid formulation
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
WO2010090969A1 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
ES2762610T3 (es) 2009-02-12 2020-05-25 Curna Inc Tratamiento de enfermedades relacionadas con el factor neurotrófico derivado de cerebro (BDNF) por inhibición de transcrito antisentido natural para BDNF
CN102439149B (zh) 2009-02-12 2018-01-02 库尔纳公司 通过抑制针对胶质细胞衍生神经营养因子(gdnf)的天然反义转录物来治疗gdnf相关的疾病
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
AU2010221419B2 (en) 2009-03-02 2015-10-01 Alnylam Pharmaceuticals, Inc. Nucleic acid chemical modifications
ES2845644T3 (es) 2009-03-04 2021-07-27 Curna Inc Tratamiento de enfermedades relacionadas con sirtuina1 (SIRT1) mediante inhibición del transcrito del antisentido natural a sirtuina 1
US20100267806A1 (en) 2009-03-12 2010-10-21 David Bumcrot LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF Eg5 AND VEGF GENES
MX2011009751A (es) 2009-03-16 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2.
WO2010107740A2 (en) 2009-03-17 2010-09-23 Curna, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
KR20170072367A (ko) 2009-04-15 2017-06-26 노오쓰웨스턴 유니버시티 올리고뉴클레오티드 관능화된 나노입자의 전달
EP3524275A1 (de) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Angeborene immununterdrückung ermöglicht die wiederholte verabreichung von langen rna-molekülen
EP2424987B1 (de) 2009-05-01 2017-11-15 CuRNA, Inc. Behandlung von hämoglobin (hbf / hbg) verwandten krankheiten durch die inhibition des natürlichen antisense transcripts zu hbf / hbg
JP5769701B2 (ja) 2009-05-05 2015-08-26 テクミラ ファーマシューティカルズ コーポレイションＴｅｋｍｉｒａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｃｏｒｐｏｒａｔｉｏｎ 脂質組成物
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
JP6250930B2 (ja) 2009-05-06 2017-12-20 クルナ・インコーポレーテッド トリステトラプロリン（ｔｔｐ）に対する天然アンチセンス転写物の抑制によるｔｔｐ関連疾患の治療
CN102459596B (zh) 2009-05-06 2016-09-07 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
US20120107331A1 (en) 2009-05-15 2012-05-03 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
CN102575251B (zh) 2009-05-18 2018-12-04 库尔纳公司 通过抑制针对重编程因子的天然反义转录物来治疗重编程因子相关的疾病
KR101703695B1 (ko) 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의해 ｔｆｅ3 및 인슐린 수용체 기질 2(ｉｒｓ2)의 치료
CN103221541B (zh) 2009-05-28 2017-03-01 库尔纳公司 通过抑制抗病毒基因的天然反义转录物来治疗抗病毒基因相关疾病
WO2010141511A2 (en) 2009-06-01 2010-12-09 Halo-Bio Rnai Therapeutics, Inc. Polynucleotides for multivalent rna interference, compositions and methods of use thereof
DK2440183T3 (en) 2009-06-10 2018-10-01 Arbutus Biopharma Corp Improved lipid formulation
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Ｐｏｎ1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(ｐｏｎ1) 관련된 질환의 치료
WO2010148050A2 (en) 2009-06-16 2010-12-23 Curna, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
CA2765889A1 (en) 2009-06-24 2010-12-29 Opko Curna, Llc Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
CA2765815A1 (en) 2009-06-26 2010-12-29 Opko Curna, Llc Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
US9512164B2 (en) 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
US9234199B2 (en) 2009-08-05 2016-01-12 Curna, Inc. Treatment of insulin gene (INS) related diseases by inhibition of natural antisense transcript to an insulin gene (INS)
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011020023A2 (en) 2009-08-14 2011-02-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
WO2011022420A1 (en) 2009-08-17 2011-02-24 Yale University Methylation biomarkers and methods of use
CN102482671B (zh) 2009-08-25 2017-12-01 库尔纳公司 通过抑制‘含有iq模体的gtp酶激活蛋白’(iqgap)的天然反义转录物而治疗iqgap相关疾病
US9321823B2 (en) 2009-09-02 2016-04-26 Genentech, Inc. Mutant smoothened and methods of using the same
EP2488646B1 (de) 2009-10-14 2017-12-06 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Zusammensetzungen zur steuerung vpn varroa-milben bei bienen
US8962584B2 (en) 2009-10-14 2015-02-24 Yissum Research Development Company Of The Hebrew University Of Jerusalem, Ltd. Compositions for controlling Varroa mites in bees
BR112012009409A2 (pt) 2009-10-22 2017-02-21 Genentech Inc método de identificação de uma substância inibidora, molécula antagonista, ácido nucleico isolado, vetor, célula hospedeira, método para fabricar a molécula, composição, artigo de fabricação, método de inibição de uma atividade biológica, método de tratamento de uma condição patológica, método para detectar msp em uma amostra e método para detectar hepsina em uma amostra
JP6147502B2 (ja) 2009-10-27 2017-06-14 スウィフト バイオサイエンシーズ， インコーポレイテッド ポリヌクレオチドプライマー及びプローブ
CN102666879B (zh) 2009-10-30 2016-02-24 西北大学 模板化的纳米缀合物
EP2496716A1 (de) 2009-11-03 2012-09-12 University Of Virginia Patent Foundation Vielseitiges sichtbares verfahren für den nachweis von polymeranalyten
CN105838714B (zh) 2009-11-12 2020-07-17 西澳大利亚大学 反义分子和治疗疾病的方法
WO2011058555A1 (en) 2009-11-12 2011-05-19 Yeda Research And Development Co. Ltd. A method of editing dna in a cell and constructs capable of same
EP3199634A1 (de) 2009-11-13 2017-08-02 Sarepta Therapeutics, Inc. Antivirale antisense-verbindung und verfahren zur behandlung von influenzavirusinfektion
WO2011063403A1 (en) 2009-11-23 2011-05-26 Swift Biosciences, Inc. Devices to extend single stranded target molecules
AR079217A1 (es) 2009-11-30 2012-01-04 Genentech Inc Composiciones y metodos para el diagnostico y el tratamiento de tumores
ES2661813T3 (es) 2009-12-16 2018-04-04 Curna, Inc. Tratamiento de enfermedades relacionadas con la peptidasa del factor de transcripción de membrana, sitio 1 (mbtps1) mediante inhibición del transcrito antisentido natural al gen mbtps1
CA2782373C (en) 2009-12-23 2019-03-26 Opko Curna, Llc Treatment of hepatocyte growth factor (hgf) related diseases by inhibition of natural antisense transcript to hgf
US9068183B2 (en) 2009-12-23 2015-06-30 Curna, Inc. Treatment of uncoupling protein 2 (UCP2) related diseases by inhibition of natural antisense transcript to UCP2
WO2011090740A2 (en) 2009-12-29 2011-07-28 Opko Curna, Llc Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
ES2585829T3 (es) 2009-12-29 2016-10-10 Curna, Inc. Tratamiento de las enfermedades relacionadas con la proteína tumoral 63 (p63) por inhibición de transcripción antisentido natural a p63
KR101878501B1 (ko) 2010-01-04 2018-08-07 큐알엔에이, 인크. 인터페론 조절 인자 8 (ｉｒｆ8)에 대한 자연 안티센스 전사체의 저해에 의한 인터페론 조절 인자 8 (ｉｒｆ8) 관련된 질환의 치료
EP2521785B1 (de) 2010-01-06 2022-03-09 CuRNA, Inc. Inhibition des natürlichen antisense-transkripts des pankreasentwicklungsgens zur verwendung in der behandlung von pankreatischen entwicklungsgenbezogenen krankheiten
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
DK2524039T3 (en) 2010-01-11 2018-03-12 Curna Inc TREATMENT OF GENDER HORMON-BINDING GLOBULIN (SHBG) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTS TO SHBG
SG182365A1 (en) 2010-01-12 2012-08-30 Univ Yale Structured rna motifs and compounds and methods for their use
CN102782135A (zh) 2010-01-25 2012-11-14 库尔纳公司 通过抑制rna酶h1的天然反义转录物而治疗rna酶h1相关疾病
WO2011097407A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
CN102844435B (zh) 2010-02-22 2017-05-10 库尔纳公司 通过抑制吡咯啉‑5‑羧酸还原酶1(pycr1)的天然反义转录物而治疗pycr1相关疾病
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
MX2012009215A (es) 2010-02-23 2012-11-23 Genentech Inc Composiciones y metodos para el diagnostico y tratamiento de tumores.
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
US9121022B2 (en) 2010-03-08 2015-09-01 Monsanto Technology Llc Method for controlling herbicide-resistant plants
WO2011113054A2 (en) 2010-03-12 2011-09-15 Aurasense Llc Crosslinked polynucleotide structure
EP2545173A2 (de) 2010-03-12 2013-01-16 Sarepta Therapeutics, Inc. Antisense-modulation von nukleären hormonrezeptoren
EP3210611B1 (de) 2010-03-12 2019-08-21 The Brigham and Women's Hospital, Inc. Verfahren zur behandlung von gefässentzündungserkrankungen
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
US8889350B2 (en) 2010-03-26 2014-11-18 Swift Biosciences, Inc. Methods and compositions for isolating polynucleotides
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
RU2610661C2 (ru) 2010-04-09 2017-02-14 Курна, Инк. Лечение заболеваний, связанных с фактором роста фибробластов 21 (fgf21), путем ингибирования природного антисмыслового транскрипта к fgf21
US20110269194A1 (en) 2010-04-20 2011-11-03 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
AU2011248625B2 (en) 2010-04-26 2017-01-05 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of cysteinyl-tRNA synthetase
JP6294074B2 (ja) 2010-04-27 2018-03-14 エータイアー ファーマ， インコーポレイテッド イソロイシルｔＲＮＡ合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
EP2601204B1 (de) 2010-04-28 2016-09-07 Ionis Pharmaceuticals, Inc. Modifizierte nukleoside und daraus hergestellte oligomere verbindungen
WO2011139853A2 (en) 2010-04-28 2011-11-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of alanyl trna synthetases
EP3091027B1 (de) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5'-modifizierte nukleoside und daraus hergestellte oligomere verbindungen
US9068177B2 (en) 2010-04-29 2015-06-30 Atyr Pharma, Inc Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glutaminyl-tRNA synthetases
CA2797374C (en) 2010-04-29 2021-02-16 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of asparaginyl trna synthetases
WO2011139917A1 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression
CA2797393C (en) 2010-04-29 2020-03-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of valyl trna synthetases
MA34291B1 (fr) 2010-05-03 2013-06-01 Genentech Inc Compositions et méthodes de diagnostic et de traitement d'une tumeur
CA2797978C (en) 2010-05-03 2019-12-03 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of methionyl-trna synthetases
CN107988228B (zh) 2010-05-03 2022-01-25 库尔纳公司 通过抑制沉默调节蛋白(sirt)的天然反义转录物而治疗沉默调节蛋白(sirt)相关疾病
CN103096912A (zh) 2010-05-03 2013-05-08 Atyr医药公司 与苯丙氨酰-α-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
CN103096925A (zh) 2010-05-03 2013-05-08 Atyr医药公司 与精氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
CN102985103A (zh) 2010-05-04 2013-03-20 Atyr医药公司 与p38多-tRNA合成酶复合物相关的治疗、诊断和抗体组合物的创新发现
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
CA3090304A1 (en) 2010-05-13 2011-11-17 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
JP6396656B2 (ja) 2010-05-14 2018-09-26 エータイアー ファーマ， インコーポレイテッド フェニルアラニルβｔＲＮＡ合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
JP6027965B2 (ja) 2010-05-17 2016-11-16 エータイアー ファーマ， インコーポレイテッド ロイシルｔＲＮＡ合成酵素のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
WO2011150226A1 (en) 2010-05-26 2011-12-01 Landers James P Method for detecting nucleic acids based on aggregate formation
US8895528B2 (en) 2010-05-26 2014-11-25 Curna, Inc. Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to ATOH1
AU2011257980B2 (en) 2010-05-28 2016-06-30 Sarepta Therapeutics, Inc. Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups
WO2011153323A2 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156713A1 (en) 2010-06-11 2011-12-15 Vanderbilt University Multiplexed interferometric detection system and method
WO2011163466A1 (en) 2010-06-23 2011-12-29 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Regulation of skin pigmentation by neuregulin-1 (nrg-1)
US8678238B2 (en) 2010-06-30 2014-03-25 Actamax Surgical Materials, Llc Self-contained hand-held yoke-connected device for dispensing a two-part adhesive aerosol
CN107441480A (zh) 2010-06-30 2017-12-08 卡姆普根有限公司 多肽及其作为用于治疗多发性硬化、类风湿性关节炎以及其他自身免疫病症的药物的用途
US9211554B2 (en) 2010-06-30 2015-12-15 Actamax Surgical Materials, Llc Self-contained hand-held direct drive device for dispensing a two-part adhesive aerosol
CA2804416C (en) 2010-07-12 2020-04-28 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-trna synthetases
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
US8198429B2 (en) 2010-08-09 2012-06-12 Avi Biopharma, Inc. Antisense antiviral compounds and methods for treating a filovirus infection
US20130143955A1 (en) 2010-08-09 2013-06-06 Yale University Cyclic di-GMP-II Riboswitches, Motifs, and Compounds, and Methods for Their Use
AU2011293294B2 (en) 2010-08-25 2016-03-24 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of Tyrosyl-tRNA synthetases
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
US20130210901A1 (en) 2010-09-20 2013-08-15 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Method of treating neurodegenerative diseases
WO2012047968A2 (en) 2010-10-05 2012-04-12 Genentech, Inc. Mutant smoothened and methods of using the same
EP2625274B1 (de) 2010-10-06 2017-07-19 CuRNA, Inc. Behandlung sialidase-4-(neu4)-vermittelter erkrankungen durch hemmung des natürlichen antisense-transkripts gegen neu4
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
WO2012052872A2 (en) 2010-10-17 2012-04-26 Yeda Research And Development Co. Ltd. Methods and compositions for the treatment of insulin-associated medical conditions
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
CA2815212A1 (en) 2010-10-22 2012-04-26 Curna, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
JP6073795B2 (ja) 2010-10-27 2017-02-01 カッパーアールエヌエー，インコーポレイテッド インターフェロン関連発生制御因子１（ｉｆｒｄ１）への天然アンチセンス転写物の阻害によるｉｆｒｄ１関連疾患の治療
CN103370054A (zh) 2010-11-09 2013-10-23 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因的表达的脂质配制的组合物和方法
CA2817090A1 (en) 2010-11-12 2012-05-18 Sarepta Therapeutics, Inc. Antisense antibacterial compounds and methods
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN103459599B (zh) 2010-11-23 2017-06-16 库尔纳公司 通过抑制nanog的天然反义转录物而治疗nanog相关疾病
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
EP2649182A4 (de) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur erhöhung einer erythropoietin (epo)-herstellung
US9045749B2 (en) 2011-01-14 2015-06-02 The General Hospital Corporation Methods targeting miR-128 for regulating cholesterol/lipid metabolism
DK2670404T3 (en) 2011-02-02 2018-11-19 Univ Princeton CIRCUIT MODULATORS AS VIRUS PRODUCTION MODULATORS
WO2012106508A1 (en) 2011-02-02 2012-08-09 Pfizer Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (ctgf)
JP2014507143A (ja) 2011-02-08 2014-03-27 ザ シャーロット−メクレンバーグ ホスピタル オーソリティ ドゥーイング／ビジネス／アズ キャロライナズ ヘルスケア システム アンチセンスオリゴヌクレオチド
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
KR102481317B1 (ko) 2011-03-29 2022-12-26 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
EP2694660B1 (de) 2011-04-03 2018-08-08 The General Hospital Corporation Effiziente in-vivo-proteinexpression mittels modifizierter rna (mod-rna)
WO2012140627A1 (en) 2011-04-15 2012-10-18 Compugen Ltd. Polypeptides and polynucleotides, and uses thereof for treatment of immune related disorders and cancer
WO2012149154A1 (en) 2011-04-26 2012-11-01 Swift Biosciences, Inc. Polynucleotide primers and probes
WO2012151268A1 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system for high throughput optical and label free detection of analytes
WO2012151289A2 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system to detect aggregate formation on a substrate
US9353371B2 (en) 2011-05-02 2016-05-31 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
JP6188686B2 (ja) 2011-06-09 2017-08-30 カッパーアールエヌエー，インコーポレイテッド フラタキシン（ｆｘｎ）への天然アンチセンス転写物の阻害によるｆｘｎ関連疾患の治療
EP2717923B1 (de) 2011-06-10 2017-09-27 Ionis Pharmaceuticals, Inc. Verfahren zur modulierung der kallikrein (klkb1)-expression
US9068184B2 (en) 2011-06-21 2015-06-30 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein C (PROC) genes
KR102540778B1 (ko) 2011-06-21 2023-06-07 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
MX345095B (es) 2011-06-21 2017-01-17 Alnylam Pharmaceuticals Inc Composiciones de arni similares a angiopoyetina 3 (angptl3) y metodos para su uso.
EP3597750B1 (de) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1-sirnas: materialzusammensetzungen und behandlungsverfahren
CA2840614A1 (en) 2011-06-29 2013-01-03 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
AU2012277376B2 (en) 2011-06-30 2016-11-24 Compugen Ltd. Polypeptides and uses thereof for treatment of autoimmune disorders and infection
AU2012284259A1 (en) 2011-07-15 2014-03-06 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
US20140328811A1 (en) 2011-08-01 2014-11-06 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
ES2632212T3 (es) 2011-08-04 2017-09-11 Yeda Research And Development Co. Ltd. miR-135 y composiciones que lo comprenden para el tratamiento de afecciones médicas asociadas con la serotonina
AU2012308659B2 (en) 2011-09-13 2017-05-04 Monsanto Technology Llc Methods and compositions for weed control
US9840715B1 (en) 2011-09-13 2017-12-12 Monsanto Technology Llc Methods and compositions for delaying senescence and improving disease tolerance and yield in plants
US10760086B2 (en) 2011-09-13 2020-09-01 Monsanto Technology Llc Methods and compositions for weed control
US10806146B2 (en) 2011-09-13 2020-10-20 Monsanto Technology Llc Methods and compositions for weed control
AU2012308694B2 (en) 2011-09-13 2018-06-14 Monsanto Technology Llc Methods and compositions for weed control
US10829828B2 (en) 2011-09-13 2020-11-10 Monsanto Technology Llc Methods and compositions for weed control
UA116090C2 (uk) 2011-09-13 2018-02-12 Монсанто Текнолоджи Ллс Спосіб та композиція для боротьби з бур'янами (варіанти)
AU2012308686B2 (en) 2011-09-13 2018-05-10 Monsanto Technology Llc Methods and compositions for weed control
US9920326B1 (en) 2011-09-14 2018-03-20 Monsanto Technology Llc Methods and compositions for increasing invertase activity in plants
CA2848753C (en) 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
AU2012308302A1 (en) 2011-09-14 2014-03-20 Northwestern University Nanoconjugates able to cross the blood-brain barrier
WO2013040548A2 (en) 2011-09-17 2013-03-21 Yale University Fluoride-responsive riboswitchs, fluoride transporters, and methods of use
US9801948B2 (en) 2011-09-21 2017-10-31 Yale University Antimicrobial compositions and methods of use thereof
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
AU2012322788B2 (en) 2011-10-11 2018-01-04 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
WO2013061328A2 (en) 2011-10-27 2013-05-02 Yeda Research And Development Co. Ltd. Method of treating cancer
KR102271212B1 (ko) 2011-11-18 2021-07-01 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
WO2013082548A1 (en) 2011-11-30 2013-06-06 Sarepta Therapeutics, Inc. Oligonucleotides for treating expanded repeat diseases
AU2012345638C1 (en) 2011-11-30 2018-10-18 Sarepta Therapeutics, Inc. Induced exon inclusion in spinal muscle atrophy
WO2013082529A1 (en) 2011-12-02 2013-06-06 Yale University Enzymatic synthesis of poly(amine-co-esters) and methods of use thereof for gene delivery
US10465042B2 (en) 2011-12-02 2019-11-05 Yale University Poly(amine-co-ester) nanoparticles and methods of use thereof
US9895451B2 (en) 2011-12-02 2018-02-20 Yale University Formulations for targeted release of agents to low pH tissue environments or cellular compartments and methods of use thereof
HUE038369T2 (hu) 2011-12-08 2018-10-29 Sarepta Therapeutics Inc Humán LMNA-t célzó oligonukleotid-analógok
EP2790736B1 (de) 2011-12-12 2018-01-31 Oncoimmunin, Inc. In-vivo-verabreichung von oligonucleotiden
AU2012358238B2 (en) 2011-12-22 2017-12-07 C. Frank Bennett Methods for modulating Metastasis-Associated-in-Lung-Adenocarcinoma-Transcript-1(MALAT-1) expression
ES2842938T3 (es) 2012-01-11 2021-07-15 Ionis Pharmaceuticals Inc Composiciones y métodos para la modulación del empalme de IKBKAP
AU2013216320A1 (en) 2012-02-01 2014-04-03 Compugen Ltd. C10RF32 antibodies, and uses thereof for treatment of cancer
EP2812342B1 (de) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation von rna durch wiederholtes targeting
BR112014020119A2 (pt) 2012-02-13 2020-10-27 Gamida-Cell Ltd cultura de células-tronco mesenquimais
JP6329911B2 (ja) 2012-02-22 2018-05-23 ブレインステム バイオテック リミテッド 星状膠細胞作製のためのミクロｒｎａ
EP3401394A1 (de) 2012-02-22 2018-11-14 Exostem Biotec Ltd Erzeugung von neuralen stammzellen
CA2866625C (en) 2012-03-13 2020-12-08 Swift Biosciences, Inc. Methods and compositions for size-controlled homopolymer tailing of substrate polynucleotides by a nucleic acid polymerase
EP2639238A1 (de) 2012-03-15 2013-09-18 Universität Bern Tricyclische Nukleoside und daraus hergestellte oligomere Verbindungen
US20150031750A1 (en) 2012-03-15 2015-01-29 The Scripps Research Institute Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2013142514A1 (en) 2012-03-19 2013-09-26 Isis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
NZ700399A (en) 2012-03-20 2016-07-29 Sarepta Therapeutics Inc Boronic acid conjugates of oligonucleotide analogues
WO2013148260A1 (en) 2012-03-30 2013-10-03 Washington University Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome
EP3563872A1 (de) 2012-04-05 2019-11-06 Massachusetts Institute Of Technology Immunstimulationszusammensetzungen und verfahren zur verwendung davon
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (de) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclische nukleinsäureanaloga
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP2839005B1 (de) 2012-04-20 2021-01-06 Aptamir Therapeutics, Inc. Mirna-modulatoren von thermogenese
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
CN104583398A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节基因表达的组合物和方法
CA2873794A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating smn gene family expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
US9518261B2 (en) 2012-05-22 2016-12-13 Ionis Pharmaceuticals, Inc. Modulation of enhancer RNA mediated gene expression
AR091143A1 (es) 2012-05-24 2015-01-14 Seeds Ltd Ab Composiciones y metodos para silenciar la expresion genetica
WO2013181665A1 (en) 2012-06-01 2013-12-05 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
US9487780B2 (en) 2012-06-01 2016-11-08 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
ES2688831T3 (es) 2012-06-25 2018-11-07 Ionis Pharmaceuticals, Inc. Modulación de la expresión de UBE3A-ATS
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
US9567569B2 (en) 2012-07-23 2017-02-14 Gamida Cell Ltd. Methods of culturing and expanding mesenchymal stem cells
US9175266B2 (en) 2012-07-23 2015-11-03 Gamida Cell Ltd. Enhancement of natural killer (NK) cell proliferation and activity
EP3693460A1 (de) 2012-07-27 2020-08-12 Ionis Pharmaceuticals, Inc. Modulation von mit dem reninangiotensinsystem (ras) assoziierten erkrankungen durch angiotensinogen
US20150216892A1 (en) 2012-08-03 2015-08-06 Aptamir Therapeutics, Inc. Cell-specific delivery of mirna modulators for the treatment of obesity and related disorders
US8603470B1 (en) 2012-08-07 2013-12-10 National Cheng Kung University Use of IL-20 antagonists for treating liver diseases
KR102237882B1 (ko) 2012-08-15 2021-04-07 아이오니스 파마수티컬즈, 인코포레이티드 변형된 캡핑 프로토콜을 이용하는 올리고머 화합물 제조 방법
EP2885313A4 (de) 2012-08-20 2016-03-09 Univ California Polynukleotide mit bioreversiblen gruppen
KR102240217B1 (ko) 2012-09-25 2021-04-14 젠자임 코포레이션 근긴장성 이영양증의 치료용 펩티드-결합 모르폴리노 안티센스 올리고뉴클레오티드
EP4052709A1 (de) 2012-10-11 2022-09-07 Ionis Pharmaceuticals, Inc. Verfahren zur behandlung der kennedy-krankheit
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
EP2906255B1 (de) 2012-10-12 2023-02-22 Ionis Pharmaceuticals, Inc. Antisense-verbindungen und verwendungen davon
US20150275208A1 (en) 2012-10-12 2015-10-01 Isis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
MX364070B (es) 2012-10-18 2019-04-10 Monsanto Technology Llc Métodos y composiciones para el control de plagas vegetales.
CA2890207A1 (en) 2012-11-05 2014-05-08 Foundation Medicine, Inc. Novel ntrk1 fusion molecules and uses thereof
KR20200143739A (ko) 2012-12-20 2020-12-24 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
US10683505B2 (en) 2013-01-01 2020-06-16 Monsanto Technology Llc Methods of introducing dsRNA to plant seeds for modulating gene expression
BR112015015975A2 (pt) 2013-01-01 2018-11-06 A. B. Seeds Ltd. moléculas de dsrna isoladas e métodos de uso das mesmas para silenciamento das moléculas alvo de interesse.
WO2014113540A1 (en) 2013-01-16 2014-07-24 Iowa State University Research Foundation, Inc. A deep intronic target for splicing correction on spinal muscular atrophy gene
CA3150658A1 (en) 2013-01-18 2014-07-24 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
US10000767B2 (en) 2013-01-28 2018-06-19 Monsanto Technology Llc Methods and compositions for plant pest control
US9701708B2 (en) 2013-01-31 2017-07-11 Ionis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
AU2014216137B2 (en) 2013-02-14 2018-05-10 Ionis Pharmaceuticals, Inc. Modulation of Apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
KR102384693B1 (ko) 2013-03-12 2022-04-07 유니버시티 오브 유타 리서치 파운데이션 세포사멸 유도용 조성물 및 방법
UY35385A (es) 2013-03-13 2014-09-30 Monsanto Technology Llc ?métodos y composiciones para el control de malezas?.
AU2014249015B2 (en) 2013-03-13 2020-04-16 Monsanto Technology Llc Methods and compositions for weed control
BR112015022156A2 (pt) 2013-03-14 2017-11-14 Isis Pharmaceuticals Inc composições e métodos para modular a expressão de tau
DK2970964T3 (en) 2013-03-14 2019-04-01 Sarepta Therapeutics Inc EXON SKIPPING COMPOSITIONS FOR TREATMENT OF MUSCLE DYROPHY
EA035882B1 (ru) 2013-03-14 2020-08-27 Сарепта Терапьютикс, Инк. Антисмысловые олигонуклеотиды, обеспечивающие пропуск экзонов, для лечения мышечной дистрофии
KR102342916B1 (ko) 2013-03-14 2021-12-24 알닐람 파마슈티칼스 인코포레이티드 보체 성분 Ｃ5 ｉＲＮＡ 조성물 및 그 이용 방법
US20140283211A1 (en) 2013-03-14 2014-09-18 Monsanto Technology Llc Methods and Compositions for Plant Pest Control
US10568328B2 (en) 2013-03-15 2020-02-25 Monsanto Technology Llc Methods and compositions for weed control
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
AU2014259759B2 (en) 2013-05-01 2020-06-18 Ionis Pharmaceuticals, Inc. Compositions and methods
PT2999785T (pt) 2013-05-22 2018-07-09 Alnylam Pharmaceuticals Inc Composições de irna de serpina1 e métodos de uso das mesmas
EP3587578A1 (de) 2013-05-22 2020-01-01 Alnylam Pharmaceuticals, Inc. Tmprss6-irna-zusammensetzungen und verfahren zur verwendung davon
WO2014197835A2 (en) 2013-06-06 2014-12-11 The General Hospital Corporation Methods and compositions for the treatment of cancer
CA2918787A1 (en) 2013-06-13 2014-12-18 George Tachas Combination therapy
WO2014205449A2 (en) 2013-06-21 2014-12-24 Isis Pharmaceuticals, Inc. Compounds and methods for modulating apolipoprotein c-iii expression for improving a diabetic profile
RU2700244C2 (ru) 2013-07-02 2019-09-13 Ионис Фармасьютикалз, Инк. Модуляторы рецептора гормона роста
JP6617702B2 (ja) 2013-07-15 2019-12-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア Ｆｔｙ７２０のアザサイクリック拘束アナログ
RU2703498C2 (ru) 2013-07-19 2019-10-17 Монсанто Текнолоджи Ллс Композиции и способы борьбы с leptinotarsa
TWI657819B (zh) 2013-07-19 2019-05-01 美商Ionis製藥公司 用於調節τ蛋白表現之組合物
US9850496B2 (en) 2013-07-19 2017-12-26 Monsanto Technology Llc Compositions and methods for controlling Leptinotarsa
US10894963B2 (en) 2013-07-25 2021-01-19 Exicure, Inc. Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
AU2014306271A1 (en) 2013-08-08 2016-03-24 The Scripps Research Institute A method for the site-specific enzymatic labelling of nucleic acids in vitro by incorporation of unnatural nucleotides
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
JP6652922B2 (ja) 2013-08-28 2020-02-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． プレカリクレイン（ｐｋｋ）発現の調節
US20150197534A1 (en) 2013-09-05 2015-07-16 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
US10059947B2 (en) 2013-09-11 2018-08-28 Synthena Ag Nucleic acids and methods for the treatment of Pompe disease
EP3791862A1 (de) 2013-09-11 2021-03-17 Eagle Biologics, Inc. Flüssige proteinformulierungen mit viskositätssenkenden mitteln
PE20190354A1 (es) 2013-09-13 2019-03-07 Ionis Pharmaceuticals Inc Moduladores del factor del complemento b
US9708360B2 (en) 2013-09-30 2017-07-18 Geron Corporation Phosphorodiamidate backbone linkage for oligonucleotides
WO2015050990A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US10119143B2 (en) 2013-10-04 2018-11-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
WO2015054451A1 (en) 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US9758546B2 (en) 2013-10-21 2017-09-12 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
CA2928779A1 (en) 2013-10-21 2015-04-30 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
WO2015066708A1 (en) 2013-11-04 2015-05-07 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (sna)
AU2014341879B2 (en) 2013-11-04 2020-07-23 Beeologics, Inc. Compositions and methods for controlling arthropod parasite and pest infestations
DK3077510T3 (da) 2013-12-02 2020-06-08 Ionis Pharmaceuticals Inc Antisense-forbindelser og anvendelser deraf
WO2015126502A2 (en) 2013-12-03 2015-08-27 Northwestern University Liposomal particles, methods of making same and uses thereof
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
US10385388B2 (en) 2013-12-06 2019-08-20 Swift Biosciences, Inc. Cleavable competitor polynucleotides
UA119253C2 (uk) 2013-12-10 2019-05-27 Біолоджикс, Інк. Спосіб боротьби із вірусом у кліща varroa та у бджіл
CN105814205B (zh) 2013-12-12 2019-11-19 阿尔尼拉姆医药品有限公司 补体成分iRNA组合物及其使用方法
JP6599334B2 (ja) 2013-12-20 2019-10-30 ザ ジェネラル ホスピタル コーポレイション 血中循環腫瘍細胞に関する方法およびアッセイ
AU2014369900B2 (en) 2013-12-24 2021-05-20 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
AU2015206585A1 (en) 2014-01-15 2016-07-21 Monsanto Technology Llc Methods and compositions for weed control using EPSPS polynucleotides
WO2015120075A2 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
WO2015118537A2 (en) 2014-02-05 2015-08-13 Yeda Research And Development Co. Ltd. Micro-rnas and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
EA201691587A1 (ru) 2014-02-11 2017-01-30 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US10036019B2 (en) 2014-03-17 2018-07-31 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
DK3119888T3 (da) 2014-03-19 2021-09-06 Ionis Pharmaceuticals Inc Sammensætninger til at modulere ataxin-2-ekspression
EP3127907A4 (de) * 2014-03-31 2018-04-04 Boston Biomedical, Inc. Neues tricyclisches chinonderivat
WO2015153339A2 (en) 2014-04-01 2015-10-08 Monsanto Technology Llc Compositions and methods for controlling insect pests
EP3757214B1 (de) 2014-04-01 2022-06-15 Biogen MA Inc. Zusammensetzungen zur modulierung der sod-1-expression
DK3129493T3 (da) 2014-04-09 2021-09-27 Scripps Research Inst Import af unaturlige eller modificerede nukleosidtriphosphater ind i celler via nukleinsyre-triphosphat-transportører
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
PL3608406T3 (pl) 2014-05-01 2023-05-22 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji czynnika B dopełniacza
JP6667453B2 (ja) 2014-05-01 2020-03-18 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 成長ホルモン受容体発現を調節するための組成物及び方法
WO2015168514A1 (en) 2014-05-01 2015-11-05 Isis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
MX2016014140A (es) 2014-05-01 2017-09-15 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de pkk.
EP3845547A1 (de) 2014-05-01 2021-07-07 Ionis Pharmaceuticals, Inc. Galnac3-modifizierte oligonukleotid-konjugat zur modulierung der angiopoietin-like-3-expression
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
EP3143141B1 (de) 2014-05-16 2019-10-02 Oregon State University Antibakterielle antisense-verbindungen und verfahren
AU2015264449B2 (en) 2014-05-19 2022-05-05 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
KR20220087576A (ko) 2014-05-22 2022-06-24 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐 (ａｇｔ) ｉｒｎａ 조성물 및 이의 사용 방법
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US20170182189A1 (en) 2014-05-23 2017-06-29 Genzyme Corporation Inhibiting or downregulating glycogen synthase by creating premature stop codons using antisense oligonucleotides
WO2015187541A1 (en) 2014-06-02 2015-12-10 Children's Medical Center Corporation Methods and compositions for immunomodulation
WO2015190922A1 (en) 2014-06-10 2015-12-17 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease
AU2015280252A1 (en) 2014-06-23 2017-01-12 Monsanto Technology Llc Compositions and methods for regulating gene expression via RNA interference
EP3161159B1 (de) 2014-06-25 2020-08-05 The General Hospital Corporation Targeting von humanem satellit ii (hsatii)
US11807857B2 (en) 2014-06-25 2023-11-07 Monsanto Technology Llc Methods and compositions for delivering nucleic acids to plant cells and regulating gene expression
HUE049261T2 (hu) 2014-07-15 2020-09-28 Yissum Research And Development Company Of The Hebrew Univ Of Jerusalem Ltd CD44 izolált polipeptidek és alkalmazásaik
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
RU2021123470A (ru) 2014-07-29 2021-09-06 Монсанто Текнолоджи Ллс Композиции и способы борьбы с насекомыми-вредителями
AU2015298263B2 (en) 2014-07-31 2020-05-14 Anji Pharmaceuticals, Inc. ApoE mimetic peptides and higher potency to clear plasma cholesterol
US20170232109A1 (en) 2014-08-19 2017-08-17 Northwestern University Protein/oligonucleotide core-shell nanoparticle therapeutics
WO2016030899A1 (en) 2014-08-28 2016-03-03 Yeda Research And Development Co. Ltd. Methods of treating amyotrophic lateral scleroses
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
PL3185957T3 (pl) 2014-08-29 2022-11-14 Alnylam Pharmaceuticals, Inc. Patisiran do zastosowania w leczeniu amyloidozy związanej z transtyretyną
CA2959386A1 (en) 2014-08-29 2016-03-03 Lee Adam Wheeler Methods and compositions for the treatment of cancer
EP3191591A1 (de) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
US10556020B2 (en) 2014-09-26 2020-02-11 University Of Massachusetts RNA-modulating agents
SG11201702614SA (en) 2014-10-01 2017-04-27 Eagle Biolog Inc Polysaccharide and nucleic acid formulations containing viscosity-lowering agents
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3207138B1 (de) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Gegen aminolävulinsäuresynthase-1 (alas1) gerichtete polynukleotidmittel und verwendungen davon
EP3212794B1 (de) 2014-10-30 2021-04-07 Genzyme Corporation Polynukleotidwirkstoffe mit abzielung auf serpinc1 (at3) und verfahren zur verwendung davon
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3221451A1 (de) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein-c3 (apoc3)-irna-zusammensetzungen und verfahren zur verwendung davon
WO2016081621A1 (en) 2014-11-18 2016-05-26 Yale University Formulations for targeted release of agents under low ph conditions and methods of use thereof
US10682422B2 (en) 2014-11-18 2020-06-16 Yale University Formulations for targeted release of agents under low pH conditions and methods of use thereof
JP2017537619A (ja) 2014-11-21 2017-12-21 ノースウェスタン ユニバーシティ 球状核酸ナノ粒子複合体の配列特異的細胞内取込
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
JP6997623B2 (ja) 2014-12-12 2022-02-04 エム． ウルフ、トッド オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
CA2972653A1 (en) 2014-12-31 2016-07-07 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US9434947B2 (en) 2015-01-20 2016-09-06 Oregon Health & Science University Modulation of KCNH2 isoform expression by oligonucleotides as a therapeutic approach for long QT syndrome
WO2016118762A1 (en) 2015-01-22 2016-07-28 Monsanto Technology Llc Compositions and methods for controlling leptinotarsa
EP3247988A4 (de) 2015-01-23 2018-12-19 Vanderbilt University Robustes interferometer und verfahren zur verwendung davon
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
JP2018510621A (ja) 2015-02-13 2018-04-19 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
US10450342B2 (en) 2015-02-23 2019-10-22 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
AU2016222546B2 (en) 2015-02-26 2020-01-23 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
US10781445B2 (en) 2015-03-11 2020-09-22 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Decoy oligonucleotides for the treatment of diseases
US10036066B2 (en) 2015-03-17 2018-07-31 Dako Denmark A/S In situ hybridization detection method
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
KR20180020125A (ko) 2015-03-27 2018-02-27 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 변형된 t 세포 및 이의 제조 및 사용 방법
US20180064748A1 (en) 2015-03-27 2018-03-08 Yeda Research And Development Co. Ltd. Methods of treating motor neuron diseases
EP3283502A4 (de) 2015-04-07 2019-04-03 The General Hospital Corporation Verfahren zur reaktivierung von genen auf dem inaktiven x-chromosom
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US20180126014A1 (en) 2015-04-15 2018-05-10 Yale University Compositions for enhancing delivery of agents across the blood brain barrier and methods of use thereof
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
PL3283080T3 (pl) 2015-04-16 2020-07-27 Ionis Pharmaceuticals, Inc. Kompozycja do modulowania ekspresji c9orf72
EP3291677A4 (de) 2015-05-04 2019-02-13 Monsanto Technology LLC Zusammensetzungen und verfahren zur bekämpfung eines befalls durch arthropoden und andere schädlinge
US20180161300A1 (en) 2015-05-11 2018-06-14 Yeda Research And Development Co., Ltd. Citrin inhibitors for the treatment of cancer
WO2016187425A1 (en) 2015-05-19 2016-11-24 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP3851531A1 (de) 2015-06-01 2021-07-21 Sarepta Therapeutics, Inc. Antisense-induzierte exonexklusion in typ-vii-kollagen
UY36703A (es) 2015-06-02 2016-12-30 Monsanto Technology Llc Composiciones y métodos para la administración de un polinucleótido en una planta
WO2016196782A1 (en) 2015-06-03 2016-12-08 Monsanto Technology Llc Methods and compositions for introducing nucleic acids into plants
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (de) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Gegen hydroxysäureoxidase (glycolat-oxidase, hao1) gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
EP3314250A4 (de) 2015-06-26 2018-12-05 Beth Israel Deaconess Medical Center, Inc. Auf tetraspanin 33 (tspann33) in myeloidabgeleiteten suppressorzellen abzielende krebstherapie
WO2017004243A1 (en) 2015-06-29 2017-01-05 Caris Science, Inc. Therapeutic oligonucleotides
JP2018519811A (ja) 2015-06-29 2018-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ｃｒｉｓｐｒ ｒｎａ及び修飾単一ｃｒｉｓｐｒ ｒｎａならびにその使用
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
WO2017011276A1 (en) 2015-07-10 2017-01-19 Ionis Pharmaceuticals, Inc. Modulators of diacyglycerol acyltransferase 2 (dgat2)
WO2017019918A1 (en) 2015-07-28 2017-02-02 Caris Science, Inc. Targeted oligonucleotides
US20180221393A1 (en) 2015-08-03 2018-08-09 Biokine Therapeutics Ltd. Cxcr4 binding agents for treatment of diseases
WO2017021961A1 (en) 2015-08-04 2017-02-09 Yeda Research And Development Co. Ltd. Methods of screening for riboswitches and attenuators
MX2018002090A (es) 2015-08-24 2018-09-12 Halo Bio Rnai Therapeutics Inc Nanoparticulas de polinucleótido para modulación de expresión génica y sus usos.
WO2017040078A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA2999177A1 (en) 2015-09-24 2017-03-30 The Regents Of The University Of California Synthetic sphingolipid-like molecules, drugs, methods of their synthesis and methods of treatment
CA2998287A1 (en) 2015-09-24 2017-04-20 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
EP3353328A4 (de) 2015-09-24 2019-06-12 Ionis Pharmaceuticals, Inc. Modulatoren der kras-expression
WO2017053781A1 (en) 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
US20190054113A1 (en) 2015-09-30 2019-02-21 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
PE20181085A1 (es) 2015-10-08 2018-07-05 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de angiotensinogeno
JP2018530560A (ja) 2015-10-09 2018-10-18 サレプタ セラピューティクス， インコーポレイテッド デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法
JP2019507579A (ja) 2015-10-28 2019-03-22 クリスパー セラピューティクス アーゲー デュシェンヌ型筋ジストロフィーの処置のための材料および方法
EP3370734B1 (de) 2015-11-05 2023-01-04 Children's Hospital Los Angeles Antisense-oligo zur verwendung bei der behandlung von akuter myeloischer leukämie
BR112018008971A2 (pt) 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
US20190046555A1 (en) 2015-11-06 2019-02-14 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds for use in therapy
US10557137B2 (en) 2015-11-06 2020-02-11 Ionis Pharmaceuticals, Inc. Modulating apolipoprotein (a) expression
WO2017081686A1 (en) 2015-11-10 2017-05-18 B. G. Negev Technologies And Applications Ltd., At Ben-Gurion University Means and methods for reducing tumorigenicity of cancer stem cells
CA3005878A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
CA3005968A1 (en) 2015-11-23 2017-06-01 The Regents Of The University Of California Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
EP3389670A4 (de) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. Verfahren zur behandlung von brustkrebs
WO2017099579A1 (en) 2015-12-07 2017-06-15 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
CA3006748A1 (en) 2015-12-15 2017-06-22 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
CA3009308A1 (en) 2015-12-23 2017-06-29 Chad Albert COWAN Materials and methods for treatment of amyotrophic lateral sclerosis and/or frontal temporal lobular degeneration
AU2016379402B2 (en) 2015-12-23 2023-01-12 Board Of Regents, The University Of Texas System Antisense antibacterial compounds and methods
EP3394261A4 (de) 2015-12-23 2019-11-27 Oregon State University Antibakterielle antisense-verbindungen und verfahren
CA3006599A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
US20190038771A1 (en) 2016-02-02 2019-02-07 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
CA3019952A1 (en) 2016-02-04 2017-08-10 Curis, Inc. Mutant smoothened and methods of using the same
EP3416689B1 (de) 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von schwerer kombinierter immundefizienz (scid) oder omenn syndrome
US11234996B2 (en) 2016-02-25 2022-02-01 The Brigham And Women's Hospital, Inc. Treatment methods for fibrosis targeting SMOC2
CN109072240A (zh) 2016-02-26 2018-12-21 耶鲁大学 使用piRNA诊断和治疗癌症的组合物和方法
US20210189062A1 (en) 2016-03-01 2021-06-24 Alexion Pharmaceuticals, Inc. Biodegradable activated polymers for therapeutic delivery
SG11201807729YA (en) 2016-03-08 2018-10-30 Kemyth Biotech Co Ltd Use of pneumolysin peptides as antagonists against toll-like receptor 4 and methods of treating toll-like receptor 4 related diseases
US11136577B2 (en) 2016-03-09 2021-10-05 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting PMP22 expression
WO2017158422A1 (en) 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
WO2017161172A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Methods of modulating keap1
EP4339288A2 (de) 2016-03-18 2024-03-20 Caris Science, Inc. Oligonukleotidsonden und verwendungen davon
WO2017173453A1 (en) 2016-04-01 2017-10-05 The Brigham And Women's Hospital, Inc. Stimuli-responsive nanoparticles for biomedical applications
EP3443094B1 (de) 2016-04-13 2022-10-19 Ionis Pharmaceuticals, Inc. Verfahren zur reduzierung der c9orf72-expression
KR102522059B1 (ko) 2016-04-18 2023-04-14 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 올리고머, 및 산성 알파-글루코시다제 유전자와 연관된 질환을 치료하기 위한 이의 사용 방법
US20200330609A1 (en) 2016-04-18 2020-10-22 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
KR102329187B1 (ko) 2016-04-29 2021-11-22 사렙타 쎄러퓨틱스, 인코퍼레이티드 인간 lmna를 표적화하는 올리고뉴클레오타이드 유사체
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017197128A1 (en) 2016-05-11 2017-11-16 Yale University Poly(amine-co-ester) nanoparticles and methods of use thereof
CN109311920B (zh) 2016-05-24 2021-11-09 萨勒普塔医疗公司 制备磷酸二酰胺吗啉代寡聚物的方法
WO2017205879A2 (en) 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
WO2017205880A1 (en) 2016-05-24 2017-11-30 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
CN109563114B (zh) 2016-05-24 2022-08-12 萨勒普塔医疗公司 用于制备寡聚物的方法
MA45158A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Composition pharmaceutique comprenant de l'éteplirsen
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
AU2017271579B2 (en) 2016-05-25 2023-10-19 Caris Science, Inc. Oligonucleotide probes and uses thereof
EP3469083A1 (de) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung von paroxysmaler nächtlicher hämoglobinurie (pnh)
US10925973B2 (en) 2016-06-15 2021-02-23 University Of Utah Research Foundation Compositions and methods for using albumin-based nanomedicines
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
ES2929047T3 (es) 2016-06-24 2022-11-24 Scripps Research Inst Transportador de nucleósido trifosfato novedoso y usos del mismo
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
CA3029119A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
JP2019525742A (ja) 2016-06-30 2019-09-12 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
CA3029132A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
JP2019520079A (ja) 2016-07-06 2019-07-18 クリスパー セラピューティクス アクチェンゲゼルシャフト 疼痛関連障害を処置するための物質及び方法
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
LT3484524T (lt) 2016-07-15 2022-12-27 Ionis Pharmaceuticals, Inc. Junginiai ir būdai, skirti smn2 moduliavimui
KR20190031306A (ko) 2016-07-21 2019-03-25 맥스시티 인코포레이티드 게놈 dna를 변경하기 위한 방법 및 조성물
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
EP4101859A1 (de) 2016-09-02 2022-12-14 Dicerna Pharmaceuticals, Inc. 4'-oxymethylphosphonat-nukleotidanaloga und oligonukleotide damit
CN116804031A (zh) 2016-09-20 2023-09-26 科罗拉多州立大学董事会法人团体 使用亚磷酰胺化学法合成主链修饰的吗啉代寡核苷酸和嵌合体
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau
WO2018067900A1 (en) 2016-10-06 2018-04-12 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
US11459568B2 (en) 2016-10-31 2022-10-04 University Of Massachusetts Targeting microRNA-101-3p in cancer therapy
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
CA3045306A1 (en) 2016-11-29 2018-06-07 Boston Biomedical, Inc. Naphthofuran derivatives, preparation, and methods of use thereof
EP3330276A1 (de) 2016-11-30 2018-06-06 Universität Bern Neuartige bicyclische nukleoside und daraus hergestellte oligomere
EP3548620A4 (de) 2016-12-02 2020-07-22 Cold Spring Harbor Laboratory Modulation der lnc05-expression
AU2017378153B2 (en) 2016-12-13 2024-03-28 Seattle Children's Hospital (dba Seattle Children's Research Institute) Methods of exogenous drug activation of chemical-induced signaling complexes expressed in engineered cells in vitro and in vivo
WO2018112470A1 (en) 2016-12-16 2018-06-21 The Brigham And Women's Hospital, Inc. Co-delivery of nucleic acids for simultaneous suppression and expression of target genes
SG10201913552UA (en) 2016-12-16 2020-03-30 Alnylam Pharmaceuticals Inc Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
WO2018118627A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
AU2017382773A1 (en) 2016-12-19 2019-08-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
BR112019012647A2 (pt) 2016-12-19 2019-11-19 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
MX2019008675A (es) 2017-01-23 2019-09-18 Regeneron Pharma Variantes de hidroxiesteroide 17-beta deshidrogenasa 13 (hsd17b13) y usos de estas.
WO2018154459A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
US20200216857A1 (en) 2017-02-22 2020-07-09 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
WO2018154439A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
JP2020508056A (ja) 2017-02-22 2020-03-19 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ 遺伝子編集のための組成物および方法
WO2018154418A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
US20180284123A1 (en) 2017-03-30 2018-10-04 California Institute Of Technology Barcoded rapid assay platform useful for efficient analysis of candidate molecules and methods of making and using the platform
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
EP3612546B1 (de) 2017-04-20 2022-07-13 Synthena AG Modifizierte oligomere verbindungen mit tricyclischen dna-nukleosiden und verwendungen davon
JP2020517638A (ja) 2017-04-20 2020-06-18 エータイアー ファーマ， インコーポレイテッド 肺の炎症を治療するための組成物および方法
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
EP3612232A1 (de) 2017-04-21 2020-02-26 The Broad Institute, Inc. Gezielte abgabe an beta-zellen
CA3062595A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
JP7356354B2 (ja) 2017-05-12 2023-10-04 クリスパー セラピューティクス アクチェンゲゼルシャフト 細胞の操作のための材料及び方法並びに免疫腫瘍学におけるその使用
JP7325341B2 (ja) 2017-07-11 2023-08-14 シンソークス，インク． 非天然ヌクレオチドの組み込み及びその方法
KR20200028997A (ko) 2017-07-13 2020-03-17 노오쓰웨스턴 유니버시티 올리고뉴클레오타이드-작용화된 금속-유기 프레임워크 나노입자를 제조하는 일반적이고 직접적인 방법
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
AU2018301442A1 (en) 2017-07-13 2020-01-30 Massachusetts Institute Of Technology Targeting the HDAC2-Sp3 complex to enhance synaptic function
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
NZ761430A (en) 2017-08-03 2024-03-22 Synthorx Inc Cytokine conjugates for the treatment of proliferative and infectious diseases
NL2019390B1 (en) 2017-08-04 2019-02-21 Univ Leiden Screening Method
WO2019032827A1 (en) 2017-08-09 2019-02-14 Massachusetts Institute Of Technology PEPTIDE ALBUMIN BINDING CONJUGATES AND METHODS THEREOF
WO2019036613A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATHWAY FOR THE TREATMENT OF RESPIRATORY DISORDERS
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
MA50267A (fr) 2017-09-19 2020-07-29 Alnylam Pharmaceuticals Inc Compositions et méthodes de traitement de l'amylose médiée par la transthyrétine (ttr)
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3687519A1 (de) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Kombinationstherapien zur behandlung von muskeldystrophie
WO2019067981A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
US20220080055A9 (en) 2017-10-17 2022-03-17 Crispr Therapeutics Ag Compositions and methods for gene editing for hemophilia a
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019089922A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
MA50579A (fr) 2017-11-09 2020-09-16 Crispr Therapeutics Ag Systèmes crispr/cas ou crispr/cpf1 à auto-inactivation (sin) et leurs utilisations
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
US20200385719A1 (en) 2017-11-16 2020-12-10 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
US10953036B2 (en) 2017-11-20 2021-03-23 University Of Georgia Research Foundation, Inc. Compositions and methods of modulating HIF-2A to improve muscle generation and repair
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
CA3082450A1 (en) 2017-11-21 2019-05-31 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
CN111629747A (zh) 2017-12-05 2020-09-04 沃泰克斯药物股份有限公司 Crispr-cas9修饰的cd34+人血色素干细胞和祖细胞及其用途
EP3724206B1 (de) 2017-12-14 2023-06-28 Ionis Pharmaceuticals, Inc. Konjugierte antisense-verbindungen und deren verwendung
CA3084825A1 (en) 2017-12-14 2019-06-20 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and their use in genome editing and other applications
US20200308588A1 (en) 2017-12-18 2020-10-01 Alnylam Pharmaceuticals, Inc. High mobility group box-1 (hmgb1) irna compositions and methods of use thereof
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
EP3728595A1 (de) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materialien und verfahren zur behandlung des usher-syndroms des typs 2a und/oder von nichtsyndromaler autosomaler rezessiver retinitis pigmentosa (arrp)
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
CA3088180A1 (en) 2018-01-12 2019-07-18 Crispr Therapeutics Ag Compositions and methods for gene editing by targeting transferrin
US20200392510A1 (en) 2018-01-15 2020-12-17 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
EP3740472A1 (de) 2018-01-19 2020-11-25 Synthena AG Tricyclo-dna-nukleosidvorläufer und verfahren zu ihrer herstellung
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP3749767A1 (de) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materialien und verfahren zur behandlung von hämoglobinopathien
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749368A1 (de) 2018-02-08 2020-12-16 Yeda Research and Development Co. Ltd Verfahren zur identifizierung und verwendung von mitteln zur behandlung von mit darmbarrierefunktionsstörungen einhergehenden krankheiten
US11332733B2 (en) 2018-02-12 2022-05-17 lonis Pharmaceuticals, Inc. Modified compounds and uses thereof
MA51869A (fr) 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
JP2021514974A (ja) 2018-02-26 2021-06-17 シンソークス， インコーポレイテッド Ｉｌ−１５コンジュゲートおよびその使用
JP7239597B2 (ja) 2018-03-02 2023-03-14 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｉｒｆ４発現の調節因子
EP3759127A4 (de) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation des amyloid-beta-vorläuferproteins
CN112105625A (zh) 2018-03-07 2020-12-18 赛诺菲 核苷酸前体、核苷酸类似物以及含其的寡聚化合物
AU2019239957A1 (en) 2018-03-19 2020-09-10 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and uses thereof
EP3768694A4 (de) 2018-03-22 2021-12-29 Ionis Pharmaceuticals, Inc. Verfahren zur modulierung der fmr1-expression
EP4051799A2 (de) 2018-03-30 2022-09-07 Rheinische Friedrich-Wilhelms-Universität Bonn Aptamere zur gezielten aktivierung von t-zellvermittelter immunität
US20210155959A1 (en) 2018-04-06 2021-05-27 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
JP7275164B2 (ja) 2018-04-11 2023-05-17 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｅｚｈ２発現の調節因子
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
KR20210008497A (ko) 2018-05-09 2021-01-22 아이오니스 파마수티컬즈, 인코포레이티드 Atxn3 발현 감소용 화합물 및 방법
BR112020020957B1 (pt) 2018-05-09 2022-05-10 Ionis Pharmaceuticals, Inc Compostos oligoméricos, população e composição farmacêutica dos mesmos e seus usos
TW202016304A (zh) 2018-05-14 2020-05-01 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (de) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. Exon-skipping-oligomere für muskeldystrophie
JP2021526823A (ja) 2018-06-14 2021-10-11 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． Ｓｔｍｎ２発現を増加させるための化合物及び方法
JP7315594B2 (ja) 2018-06-27 2023-07-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｌｒｒｋ２発現を低減するための化合物及び方法
SG11202012499RA (en) 2018-06-28 2021-01-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
EP3826645A4 (de) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur verringerung der atxn2-expression
US10857174B2 (en) 2018-07-27 2020-12-08 United States Government As Represented By The Department Of Veterans Affairs Morpholino oligonucleotides useful in cancer treatment
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
US11939593B2 (en) 2018-08-01 2024-03-26 University Of Georgia Research Foundation, Inc. Compositions and methods for improving embryo development
JP2021533767A (ja) 2018-08-13 2021-12-09 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. Ｂ型肝炎ウイルス（ＨＢＶ）ｄｓＲＮＡ物質組成物およびその使用方法
US20210348162A1 (en) 2018-08-16 2021-11-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
CN113329739A (zh) 2018-08-20 2021-08-31 罗贡股份有限公司 用于治疗scn1a脑病的靶向scn2a的反义寡核苷酸
EP3843845A4 (de) 2018-08-29 2022-05-11 University Of Massachusetts Hemmung von proteinkinasen zur behandlung von friedreich-ataxie
WO2020051507A1 (en) 2018-09-06 2020-03-12 The Broad Institute, Inc. Nucleic acid assemblies for use in targeted delivery
EP3620520A1 (de) 2018-09-10 2020-03-11 Universidad del Pais Vasco Neuartiges target zur behandlung einer stoffwechselerkrankung in einem individuum
CN112912422A (zh) 2018-09-14 2021-06-04 西北大学 用dna编程蛋白质聚合
CN112424355A (zh) 2018-09-18 2021-02-26 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
TW202023573A (zh) 2018-09-19 2020-07-01 美商Ionis製藥公司 Pnpla3表現之調節劑
JP2022505173A (ja) 2018-10-17 2022-01-14 クリスパー・セラピューティクス・アクチェンゲゼルシャフト 導入遺伝子を送達するための組成物および方法
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
BR112021008967A2 (pt) 2018-11-15 2021-08-17 Ionis Pharmaceuticals, Inc. moduladores da expressão de irf5
JP7455831B2 (ja) 2018-11-21 2024-03-26 アイオーニス ファーマシューティカルズ， インコーポレーテッド プリオン発現を低減するための化合物及び方法
IL263184A (en) 2018-11-21 2020-05-31 Yarden Yosef Method of treating cancer and compositions for same
US20210332495A1 (en) 2018-12-06 2021-10-28 Northwestern University Protein Crystal Engineering Through DNA Hybridization Interactions
KR20210104759A (ko) 2018-12-13 2021-08-25 사렙타 쎄러퓨틱스 인코퍼레이티드 근 이영양증을 위한 엑손 스키핑 올리고머 접합체
CN113543791A (zh) 2018-12-20 2021-10-22 维尔生物科技有限公司 组合hbv疗法
CN113631709A (zh) 2018-12-20 2021-11-09 普拉克西斯精密药物股份有限公司 用于治疗kcnt1相关病症的组合物和方法
SG11202107669WA (en) 2019-01-16 2021-08-30 Genzyme Corp Serpinc1 irna compositions and methods of use thereof
EP3918069A1 (de) 2019-01-31 2021-12-08 Bar Ilan University Durch aberrant-induziertes spleissen erzeugte neoantigene und deren verwendung zur verstärkung einer immuntherapie
MX2021008918A (es) 2019-01-31 2021-08-24 Ionis Pharmaceuticals Inc Moduladores de expresion yap1.
BR112021014415A2 (pt) 2019-02-06 2021-09-21 Synthorx, Inc. Conjugados de il-2 e métodos de uso dos mesmos
WO2020168362A1 (en) 2019-02-15 2020-08-20 Crispr Therapeutics Ag Gene editing for hemophilia a with improved factor viii expression
WO2020171889A1 (en) 2019-02-19 2020-08-27 University Of Rochester Blocking lipid accumulation or inflammation in thyroid eye disease
EP3927378A1 (de) 2019-02-21 2021-12-29 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Verfahren zur reduktion von arzneimittelinduzierter nephrotoxizität
EP3931328A4 (de) 2019-02-27 2023-09-13 Ionis Pharmaceuticals, Inc. Modulatoren der malat1-expression
US20220145274A1 (en) 2019-03-12 2022-05-12 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
WO2020198268A1 (en) 2019-03-28 2020-10-01 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy with casimersen
MX2021011928A (es) 2019-03-29 2022-01-04 Dicerna Pharmaceuticals Inc Composiciones y metodos para el tratamiento de enfermedades o trastornos asociados con kras.
CA3198063A1 (en) 2019-03-29 2020-10-08 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for modulating expression of dux4
AU2020253821A1 (en) 2019-03-29 2021-10-28 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating UBE3A-ATS
WO2020214763A1 (en) 2019-04-18 2020-10-22 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
US11814464B2 (en) 2019-04-29 2023-11-14 Yale University Poly(amine-co-ester) polymers and polyplexes with modified end groups and methods of use thereof
US20220177880A1 (en) 2019-05-03 2022-06-09 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
WO2020225606A1 (en) 2019-05-08 2020-11-12 Crispr Therapeutics Ag Crispr/cas all-in-two vector systems for treatment of dmd
SG11202112240VA (en) 2019-05-13 2021-12-30 Vir Biotechnology Inc Compositions and methods for treating hepatitis b virus (hbv) infection
US20220243203A1 (en) 2019-05-28 2022-08-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fus expression
TW202113078A (zh) 2019-06-14 2021-04-01 美商史基普研究協會 於半合成生物體中複製、轉錄及轉譯之試劑及方法
WO2020254872A2 (en) 2019-06-17 2020-12-24 Crispr Therapeutics Ag Methods and compositions for improved homology directed repair
WO2020257489A1 (en) 2019-06-19 2020-12-24 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
EP3956450A4 (de) 2019-07-26 2022-11-16 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation von gfap
WO2021022108A2 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. CARBOXYPEPTIDASE B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021025899A1 (en) 2019-08-02 2021-02-11 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer pharmaceutical compositions
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
CN114555621A (zh) 2019-08-15 2022-05-27 Ionis制药公司 键修饰的寡聚化合物及其用途
MX2022001776A (es) 2019-08-15 2022-03-17 Synthorx Inc Terapias de combinacion de inmuno oncologia con conjugados de il-2.
MX2022002053A (es) 2019-08-23 2022-03-17 Synthorx Inc Conjugados de il-15 y sus usos.
CA3149421A1 (en) 2019-08-30 2021-03-04 Yale University Compositions and methods for delivery of nucleic acids to cells
CN114616331A (zh) 2019-09-03 2022-06-10 阿尔尼拉姆医药品有限公司 抑制lect2基因表达的组合物和方法
CN114728999A (zh) 2019-09-05 2022-07-08 赛诺菲 含有核苷酸类似物的寡核苷酸
US20210070827A1 (en) 2019-09-10 2021-03-11 Synthorx, Inc. Il-2 conjugates and methods of use to treat autoimmune diseases
WO2021067747A1 (en) 2019-10-04 2021-04-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
EP4045062A1 (de) 2019-10-14 2022-08-24 Astrazeneca AB Modulatoren der pnpla3-expression
EP4045652A1 (de) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen eines elements der solute-carrier-familie und verwendungsverfahren dafür
CN115176004A (zh) 2019-10-22 2022-10-11 阿尔尼拉姆医药品有限公司 补体成分C3 iRNA组合物及其使用方法
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
TW202131952A (zh) 2019-11-04 2021-09-01 美商欣爍克斯公司 介白素10接合物及其用途
BR112022009216A2 (pt) 2019-11-13 2022-08-02 Alnylam Pharmaceuticals Inc Métodos e composições para tratar um distúrbio associado a angiotensinogênio (agt)
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
CA3159501A1 (en) 2019-11-27 2021-06-03 Brian Joseph CAFFERTY Methods of synthesizing rna molecules
BR112022011417A2 (pt) 2019-12-13 2022-08-30 Alnylam Pharmaceuticals Inc Composições do agente de irna da fase de leitura aberta 72 do cromossomo humano 9 (c9orf72) e métodos de uso das mesmas
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法
WO2021122944A1 (en) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
MX2022008738A (es) 2020-01-15 2022-09-23 Dicerna Pharmaceuticals Inc Acidos nucleicos de fosfonato de 4'-o-metileno y analogos de estos.
WO2021154705A1 (en) 2020-01-27 2021-08-05 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rab13 and net1 antisense oligonucleotides to treat metastatic cancer
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
KR20220140593A (ko) 2020-02-10 2022-10-18 알닐람 파마슈티칼스 인코포레이티드 Vegf-a 발현을 사일런싱하기 위한 조성물 및 방법
WO2021163281A1 (en) 2020-02-12 2021-08-19 Accutar Biotechnology Inc. Antisense oligonucleotides and their use for treating pendred syndrome
IL295496A (en) 2020-02-18 2022-10-01 Alnylam Pharmaceuticals Inc Apolipoprotein c3 (apoc3) irna preparations and methods of using them
US20220064638A1 (en) 2020-02-28 2022-03-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating smn2
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
AU2021232014A1 (en) 2020-03-06 2022-10-06 Alnylam Pharmaceuticals, Inc. Ketohexokinase (KHK) IRNA compositions and methods of use thereof
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
JP2023519274A (ja) 2020-03-26 2023-05-10 アルナイラム ファーマシューティカルズ， インコーポレイテッド コロナウイルスｉＲＮＡ組成物およびその使用方法
US20230190785A1 (en) 2020-03-30 2023-06-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
US20230295622A1 (en) 2020-04-06 2023-09-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
JP2023521094A (ja) 2020-04-07 2023-05-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｓｃｎ９ａ発現をサイレンシングするための組成物および方法
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
EP4133076A1 (de) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen aus angiotensin-konvertierendem enzym 2 (ace2) und verfahren zur verwendung davon
JP2023523993A (ja) 2020-04-27 2023-06-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド アポリポタンパク質Ｅ（ＡｐｏＥ）ｉＲＮＡ剤組成物およびその使用方法
CN116096381A (zh) 2020-04-30 2023-05-09 阿尔尼拉姆医药品有限公司 补体因子B（CFB）iRNA组合物及其使用方法
IL297435A (en) 2020-05-01 2022-12-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating atxn1
US20230227824A1 (en) 2020-05-12 2023-07-20 Mitsubishi Tanabe Pharma Corporation Compound, method and pharmaceutical composition for regulating expression of ataxin 3
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150078A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten editierung von argininosuccinat-lyase (asl)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150089A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von retinosin 1 (rs1)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150076A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von methyl-cpg-bindendem protein 2 (mecp2)
EP4150086A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von leucinreicher repeat-kinase 2 (lrrk2)
EP4150077A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von transmembrankanalähnlichem protein 1 (tmc1)
EP4153746A1 (de) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der marc1-genexpression
AU2021274944A1 (en) 2020-05-22 2022-12-15 Wave Life Sciences Ltd. Double stranded oligonucleotide compositions and methods relating thereto
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
JP2023530234A (ja) 2020-06-05 2023-07-14 ザ・ブロード・インスティテュート・インコーポレイテッド 新生物を治療するための組成物および方法
EP4162050A1 (de) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai-zusammensetzungen und verfahren zur verwendung davon zur verabreichung durch inhalation
CA3184289A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
US20230233693A1 (en) 2020-06-19 2023-07-27 Yale University Poly(amine-co-ester) polymers with modified end groups and enhanced pulmonary delivery
CA3182458A1 (en) 2020-06-24 2021-12-30 Laura ROSEN Engineered hepatitis b virus neutralizing antibodies and uses thereof
KR20230027235A (ko) 2020-06-25 2023-02-27 신톡스, 인크. Il-2 콘쥬게이트 및 항-egfr 항체를 사용한 면역 종양학 병용 요법
US11732263B2 (en) 2020-06-29 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating PLP1
EP4192505A1 (de) 2020-08-04 2023-06-14 Dicerna Pharmaceuticals, Inc. Systemische verabreichung von oligonukleotiden
EP4204002A1 (de) 2020-08-31 2023-07-05 Yale University Zusammensetzungen und verfahren zur abgabe von nukleinsäuren an zellen
TW202227102A (zh) 2020-09-22 2022-07-16 瑞典商阿斯特捷利康公司 治療脂肪肝病之方法
EP4217489A1 (de) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidylpeptidase 4 (dpp4)-irna-zusammensetzungen und verfahren zur verwendung davon
US20230392134A1 (en) 2020-09-30 2023-12-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
EP3978608A1 (de) 2020-10-05 2022-04-06 SQY Therapeutics Oligomere verbindung zur dystrophinrettung in dmd-patienten während des skipping exon-51
MX2023004029A (es) 2020-10-09 2023-04-27 Synthorx Inc Terapia de combinacion de inmunoncologia con conjugados de il-2 y pembrolizumab.
AU2021356610A1 (en) 2020-10-09 2023-06-15 Synthorx, Inc. Immuno oncology therapies with il-2 conjugates
EP4228637A1 (de) 2020-10-15 2023-08-23 Yeda Research and Development Co. Ltd Verfahren zur behandlung von myeloidmalignitäten
EP4232455A2 (de) 2020-10-20 2023-08-30 Sanofi Neue liganden für den asialoglycoproteinrezeptor
AU2021365822A1 (en) 2020-10-21 2023-06-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (de) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin-5b (muc5b)-irna-zusammensetzungen und verfahren zur verwendung davon
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
AU2021381363A1 (en) 2020-11-18 2023-06-15 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
WO2022106695A1 (en) 2020-11-23 2022-05-27 Alpha Anomeric Sas Nucleic acid duplexes
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP4259795A1 (de) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Gerinnungsfaktor x (f10)-irna-zusammensetzungen und verfahren zur verwendung davon
GB2603454A (en) 2020-12-09 2022-08-10 Ucl Business Ltd Novel therapeutics for the treatment of neurodegenerative disorders
WO2022140702A1 (en) 2020-12-23 2022-06-30 Flagship Pioneering, Inc. Compositions of modified trems and uses thereof
WO2022140535A1 (en) 2020-12-23 2022-06-30 Sarepta Therapeutics, Inc. Compositions comprising exon skipping oligonucleotide conjugates for treating muscular dystrophy
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
WO2022174102A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
WO2022174000A2 (en) 2021-02-12 2022-08-18 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
AU2022226098A1 (en) 2021-02-26 2023-08-24 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
IL305418A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
WO2022192038A1 (en) 2021-03-12 2022-09-15 Northwestern University Antiviral vaccines using spherical nucleic acids
AR125230A1 (es) 2021-03-29 2023-06-28 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
EP4314293A1 (de) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Prolindehydrogenase 2 (prodh2)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4330392A1 (de) 2021-04-26 2024-03-06 Alnylam Pharmaceuticals, Inc. Transmembranprotease, serin 6 (tmprss6)-irna-zusammensetzungen und verfahren zur verwendung davon
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4330395A1 (de) 2021-04-30 2024-03-06 Sarepta Therapeutics, Inc. Behandlungsverfahren für muskeldystrophie
WO2022235537A1 (en) 2021-05-03 2022-11-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4341405A1 (de) 2021-05-20 2024-03-27 Korro Bio, Inc. Verfahren und zusammensetzungen für adar-vermittelte bearbeitung
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）的ｉＲＮＡ組成物及其使用方法
WO2022256538A1 (en) 2021-06-03 2022-12-08 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and cetuximab
IL308743A (en) 2021-06-04 2024-01-01 Alnylam Pharmaceuticals Inc Compositions of human chromosome 9 open reading frame 72 iRNA factor (C9ORF72) and methods of using them
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
IL308896A (en) 2021-06-11 2024-01-01 Bayer Ag Programmable type V RNA endoclase systems
EP4101928A1 (de) 2021-06-11 2022-12-14 Bayer AG Programmierbare typ-v-rna-endonukleasesysteme
IL309217A (en) 2021-06-18 2024-02-01 Ionis Pharmaceuticals Inc Compounds and Methods for Reducing IFNAR1 Expression
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
KR20240026203A (ko) 2021-06-30 2024-02-27 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐(agt)-관련 장애를 치료하는 방법 및 조성물
WO2023285431A1 (en) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions and methods for allele specific treatment of retinitis pigmentosa
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
IL309905A (en) 2021-07-23 2024-03-01 Alnylam Pharmaceuticals Inc IRNA compositions in β-catenin (CTNNB1) and methods of using them
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
WO2023014677A1 (en) 2021-08-03 2023-02-09 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof
TW202337474A (zh) 2021-08-04 2023-10-01 美商艾拉倫製藥股份有限公司 用於緘默血管收縮素原（ＡＧＴ）的ｉＲＮＡ組成物及方法
TW202334413A (zh) 2021-08-13 2023-09-01 美商艾拉倫製藥股份有限公司 第十二因子（Ｆ１２）ｉＲＮＡ組成物及其使用方法
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
WO2023034515A2 (en) 2021-09-03 2023-03-09 Sarepta Therapeutics, Inc. Delivery of anitsense oligomers by mirror image peptides
EP4144841A1 (de) 2021-09-07 2023-03-08 Bayer AG Neue programmierbare rna-endonuklease-systeme mit verbesserter pam-spezifität und deren verwendung
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
WO2023044094A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CA3233242A1 (en) 2021-09-30 2023-04-06 Sarepta Therapeutics, Inc. Antisense oligonucleotides having one or more abasic units
WO2023070086A1 (en) 2021-10-22 2023-04-27 Sarepta Therapeutics, Inc. Morpholino oligomers for treatment of peripheral myelin protein 22 related diseases
CA3234835A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023086292A2 (en) 2021-11-10 2023-05-19 University Of Rochester Gata4-targeted therapeutics for treatment of cardiac hypertrophy
WO2023086295A2 (en) 2021-11-10 2023-05-19 University Of Rochester Antisense oligonucleotides for modifying protein expression
GB202117758D0 (en) 2021-12-09 2022-01-26 Ucl Business Ltd Therapeutics for the treatment of neurodegenerative disorders
WO2023122573A1 (en) 2021-12-20 2023-06-29 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023118349A1 (en) 2021-12-21 2023-06-29 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023159189A1 (en) 2022-02-18 2023-08-24 Yale University Branched poly(amine-co-ester) polymers for more efficient nucleic expression
WO2023168427A1 (en) 2022-03-03 2023-09-07 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
WO2023178230A1 (en) 2022-03-17 2023-09-21 Sarepta Therapeutics, Inc. Phosphorodiamidate morpholino oligomer conjugates
WO2023177866A1 (en) 2022-03-18 2023-09-21 Dicerna Pharmaceuticals, Inc. Decarboxylative acetoxylation using mn(ii) or mn(iii) reagent for synthesis of 4'-acetoxy- nucleoside and use thereof for synthesis of corresponding 4'-(dimethoxyphosphoryl)methoxy- nucleotide
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
WO2023205451A1 (en) 2022-04-22 2023-10-26 Entrada Therapeutics, Inc. Cyclic peptides for delivering therapeutics
WO2023237587A1 (en) 2022-06-10 2023-12-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2024015924A2 (en) 2022-07-15 2024-01-18 Entrada Therapeutics, Inc. Hybrid oligonucleotides
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024050261A1 (en) 2022-08-29 2024-03-07 University Of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
WO2024081736A2 (en) 2022-10-11 2024-04-18 Yale University Compositions and methods of using cell-penetrating antibodies

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8311228D0 (en) * 1983-04-25 1983-06-02 Fujisawa Pharmaceutical Co Morpholine derivatives
EP0215942B1 (de) * 1985-03-15 1995-07-12 Antivirals Inc. Immunotestmittel für polynukleotid und verfahren
DE69033986T2 (de) * 1989-12-20 2003-03-13 Antivirals Inc Ungeladene, auf Morpholin basierende Polymere mit Chiralen, Phosphor enthaltenden Brücken zwischen den Untereinheiten
JPH09118898A (ja) * 1995-08-23 1997-05-06 Kao Corp 硬質表面用漂白剤組成物

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