WO1997032991A3 - Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors - Google Patents

Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors Download PDF

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Publication number
WO1997032991A3
WO1997032991A3 PCT/US1997/003535 US9703535W WO9732991A3 WO 1997032991 A3 WO1997032991 A3 WO 1997032991A3 US 9703535 W US9703535 W US 9703535W WO 9732991 A3 WO9732991 A3 WO 9732991A3
Authority
WO
WIPO (PCT)
Prior art keywords
gene
treatment
associated virus
solid tumors
recombinant adeno
Prior art date
Application number
PCT/US1997/003535
Other languages
French (fr)
Other versions
WO1997032991A2 (en
Inventor
Gary J Kurtzman
Peter C Colosi
Original Assignee
Avigen Inc
Yoshida Jun
Mizuno Masaaki
Okada Hideho
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 Avigen Inc, Yoshida Jun, Mizuno Masaaki, Okada Hideho filed Critical Avigen Inc
Priority to EP97914890A priority Critical patent/EP0885306A2/en
Priority to JP53194997A priority patent/JP2002514900A/en
Publication of WO1997032991A2 publication Critical patent/WO1997032991A2/en
Publication of WO1997032991A3 publication Critical patent/WO1997032991A3/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/54Interleukins [IL]
    • C07K14/55IL-2
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/555Interferons [IFN]
    • C07K14/565IFN-beta
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Abstract

The use of recombinant adeno-associated virus (AAV) virions for the treatment of solid tumors is disclosed. The invention provides for the use of recombinant AAV virions to deliver an AAV vector containing a drug-susceptibility gene and a second gene capable of providing an ancillary effect to solid tumor cells. The second gene can be used to enhance the immunogenicity of the transduced tumor cell. Alternatively, the second gene can be used to provide a tumorstatic effect. The invention also provides for the use of recombinant AAV virions to deliver an interferon gene, or a tumor suppressor gene to provide a therapeutic effect in a transduced tumor cell.
PCT/US1997/003535 1996-03-06 1997-03-06 Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors WO1997032991A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP97914890A EP0885306A2 (en) 1996-03-06 1997-03-06 Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors
JP53194997A JP2002514900A (en) 1996-03-06 1997-03-06 Gene therapy for treatment of solid tumors using recombinant adeno-associated virus vectors

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US1320996P 1996-03-06 1996-03-06
US60/013,209 1996-03-06
US08/812,102 US5952221A (en) 1996-03-06 1997-03-05 Adeno-associated virus vectors comprising a first and second nucleic acid sequence
US08/812,102 1997-03-05

Publications (2)

Publication Number Publication Date
WO1997032991A2 WO1997032991A2 (en) 1997-09-12
WO1997032991A3 true WO1997032991A3 (en) 1998-02-05

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1997/003535 WO1997032991A2 (en) 1996-03-06 1997-03-06 Gene therapy for the treatment of solid tumors using recombinant adeno-associated virus vectors

Country Status (5)

Country Link
US (4) US5952221A (en)
EP (1) EP0885306A2 (en)
JP (1) JP2002514900A (en)
CA (1) CA2247806A1 (en)
WO (1) WO1997032991A2 (en)

Families Citing this family (125)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030027250A1 (en) * 1995-12-15 2003-02-06 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20020193580A1 (en) * 1995-12-15 2002-12-19 Mitchell Lloyd G. Methods and compositions for use in spliceosome mediated RNA trans-splicing
US20060088938A1 (en) * 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
US6242212B1 (en) * 1996-02-09 2001-06-05 Thomas Jefferson University Fragile histidine triad (FHIT) nucleic acids and methods of producing FHIT proteins
US5952221A (en) * 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence
DE19608751B4 (en) 1996-03-06 2006-05-18 Medigene Ag Use of an adeno-associated virus vector to increase the immunogenicity of cells
US6361938B1 (en) * 1996-11-08 2002-03-26 Elan Corporation, Plc Peptides which enhance transport across tissues and methods of identifying and using the same
US6696423B1 (en) 1997-08-29 2004-02-24 Biogen, Inc. Methods and compositions for therapies using genes encoding secreted proteins such as interferon-beta
SI1007717T1 (en) * 1997-08-29 2006-10-31 Biogen Idec Inc Methods and compositions for cancer therapies using genes encoding interferon-beta
BR9812138A (en) * 1997-08-29 2000-07-18 Biogen Inc Methods and compositions for therapies employing genes encoding secreted proteins such as interferon-beta
DE69834813T2 (en) * 1998-10-14 2007-05-24 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Parvovirus vectors and their use
US6759237B1 (en) * 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
AU780231B2 (en) * 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6303362B1 (en) * 1998-11-19 2001-10-16 The Board Of Trustees Of The Leland Stanford Junior University Adenoviral vector and methods for making and using the same
US20040043488A1 (en) * 1999-02-17 2004-03-04 University Of Pittsburgh Of The Commonwealth System Of Higher Education Adeno-associated viral gene-transfer vector system
US7141417B1 (en) * 1999-02-25 2006-11-28 Thomas Jefferson University Compositions, kits, and methods relating to the human FEZ1 gene, a novel tumor suppressor gene
US8063022B1 (en) 1999-06-08 2011-11-22 The Children's Hospital Of Philadelphia Methods for preventing formation of inhibitory antibodies in the setting of gene therapy
JP5557974B2 (en) * 1999-10-07 2014-07-23 アグイラ−コルドバ,カルロス,エストアルド Method of treating solid tumors and metastases by gene therapy
US6716824B1 (en) * 1999-10-22 2004-04-06 F. Charles Brunicardi Treatment of pancreatic adenocarcinoma by cytotoxic gene therapy
US6582692B1 (en) 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
AU2001240018A1 (en) 2000-03-03 2001-09-17 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral compositions and methods
EP1276879A4 (en) * 2000-04-11 2004-12-22 Univ Jefferson MUIR-TORRE-LIKE SYNDROME IN Fhit DEFICIENT MICE
US20040126774A1 (en) * 2001-01-08 2004-07-01 Mitchell Lioyd G. Correction of factor VIII genetic defects using spliceosome mediated RNA trans splicing
US20030138405A1 (en) * 2001-04-17 2003-07-24 Juan Fueyo Conditionally replicative adenovirus to target the Rb and Rb-related pathways
US20040002058A1 (en) * 2001-06-21 2004-01-01 Uab Research Foundation Chimeric capsid proteins and uses thereof
WO2003000855A2 (en) * 2001-06-21 2003-01-03 Uab Research Foundation Chimeric capsid proteins and uses thereof
US6838285B2 (en) 2001-09-18 2005-01-04 Becton Dickinson Site specific recombinase based method for producing adenoviral vectors
CA2469623C (en) * 2001-12-12 2012-05-29 F H Faulding & Co Limited Composition for the preservation of viruses
US20040115800A1 (en) * 2002-01-16 2004-06-17 Momparler Richard L Selectable marker for genetically engineered cells and tissues
AU2003239120B2 (en) * 2002-02-21 2008-04-03 Morphotek, Inc. Regulated vectors for controlling DNA hypermutability in eukariotic cells
US7399753B2 (en) * 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
WO2003074561A1 (en) * 2002-03-02 2003-09-12 University Of South Florida A method for treating allergic disease and asthma by recombinant adenovirus-and adeno-associated virus-mediated ifn-ϝ gene
WO2003086451A1 (en) * 2002-04-05 2003-10-23 Centocor, Inc. Asthma-related anti-il-13 immunoglobulin derived proteins, compositions, methods and uses
AU2003225281A1 (en) 2002-04-30 2003-11-17 University Of South Florida Materials and methods for prevention and treatment of rna viral diseases
US20060127358A1 (en) * 2002-05-01 2006-06-15 Nicholas Muzyczka Raav expression systems and methods for enhancing transduction of mammalian neural cells
AU2003302216A1 (en) * 2002-05-08 2004-06-23 Virxsys Corporation Use of spliceosome mediated rna trans-splicing to confer cell selective replication to adenoviruses
US20050208023A1 (en) * 2002-07-19 2005-09-22 Krissansen Geoffrey W Tumor treating composition and methods
US7595303B1 (en) * 2002-09-05 2009-09-29 University Of South Florida Genetic adjuvants for immunotherapy
JP2006512908A (en) * 2002-10-11 2006-04-20 トーマス ジェファーソン ユニバーシティー Tumor suppressor genes and compositions and methods for their preparation and use
AU2003285009B2 (en) * 2002-10-23 2010-10-07 Virxsys Corporation Screening methods for identification of efficient pre-trans-splicing molecules
JP4939055B2 (en) * 2002-11-13 2012-05-23 トマス ジェファソン ユニバーシティ Compositions and methods for diagnosis and treatment of cancer
JP4754219B2 (en) * 2002-12-02 2011-08-24 アムジエン・フレモント・インコーポレイテツド Antibodies directed against tumor necrosis factor and their use
CA2520279C (en) 2003-03-27 2012-09-04 Ottawa Health Research Institute Mutant vesicular stomatitis viruses and use thereof
AU2003286741A1 (en) * 2003-05-02 2004-11-26 Thomas Jefferson University Methods and compositions for diagnosis and therapy of parkin-associated disorders
US20040248826A1 (en) * 2003-06-03 2004-12-09 Alberto Auricchio Treatment of cancer by in vivo gene-transfer induced TIMP-3 expression
WO2005021768A1 (en) 2003-09-01 2005-03-10 Academisch Medisch Centrum Aav vectors for in vivo gene therapy of rheumatoid arthritis
US7968334B2 (en) * 2004-01-23 2011-06-28 Virxsys Corporation Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing
JP2007518423A (en) * 2004-01-23 2007-07-12 イントロン、インコーポレイテッド Expression of ApoA-1 and its variants using spliceosome-mediated RNA trans-splicing
WO2005070948A1 (en) * 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US20050266093A1 (en) * 2004-04-27 2005-12-01 Mohapatra Shyam S Nanogene therapy for cell proliferation disorders
CA2574802A1 (en) 2004-07-20 2006-02-02 Isogenis, Inc. Specific inhibition of autoimmunity and diseases associated with autoantigens
US20060094110A1 (en) * 2004-07-30 2006-05-04 Mcgarrity Gerard J Use of spliceosome mediated RNA trans-splicing for immunotherapy
US20060134658A1 (en) * 2004-08-09 2006-06-22 Garcia-Blanco Mariano A Use of RNA trans-splicing for generation of interfering RNA molecules
DE102004047492B4 (en) * 2004-09-23 2006-07-20 Jost-Werke Gmbh & Co. Kg Method for transmitting electrical, pneumatic or hydraulic energy and a power transmission system
AU2005326784B2 (en) * 2004-10-08 2012-03-15 Virxsys Corporation Use of RNA trans-splicing for antibody gene transfer and antibody polypeptide production
US7871795B2 (en) 2004-10-08 2011-01-18 Virxsys Corporation Targeted trans-splicing of highly abundant transcripts for in vivo production of recombinant proteins
US7892809B2 (en) * 2004-12-15 2011-02-22 The University Of North Carolina At Chapel Hill Chimeric vectors
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
AU2008314486B2 (en) * 2007-10-15 2014-06-26 Jingang Medicine (Australia) Pty Ltd Construct system and uses therefor
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2266588A1 (en) * 2009-06-04 2010-12-29 Deutsches Krebsforschungszentrum, Stiftung des öffentlichen Rechts Method for cancer therapy based on co-administration of a parovirus and a cytokine
US9795658B2 (en) 2010-04-20 2017-10-24 Admedus Vaccines Pty Ltd Expression system for modulating an immune response
US9309534B2 (en) 2010-07-12 2016-04-12 Universidad Autonoma De Barcelona Gene therapy composition for use in diabetes treatment
US8808684B2 (en) 2010-09-10 2014-08-19 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
US9943574B2 (en) 2012-06-27 2018-04-17 Arthrogen B.V. Combination for treating an inflammatory disorder
EP2692868A1 (en) 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
US10131910B2 (en) 2014-07-10 2018-11-20 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Usher syndrome type 2
CA2960912A1 (en) 2014-09-16 2016-03-24 Universitat Autonoma De Barcelona Adeno-associated viral vectors for the gene therapy of metabolic diseases
CN112553229A (en) 2014-11-05 2021-03-26 沃雅戈治疗公司 AADC polynucleotides for treating Parkinson's disease
CN107207556B (en) 2014-11-14 2020-12-08 沃雅戈治疗公司 Regulatory polynucleotides
RU2020108189A (en) 2014-11-14 2020-03-11 Вояджер Терапьютикс, Инк. COMPOSITIONS AND METHODS OF TREATMENT OF LATERAL AMYOTROPHIC SCLEROSIS (ALS)
WO2016086227A2 (en) 2014-11-26 2016-06-02 The Regents Of The University Of California Therapeutic compositions comprising transcription factors and methods of making and using the same
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
EP3242945B1 (en) 2015-01-07 2021-09-01 Universitat Autònoma de Barcelona Single-vector gene construct comprising insulin and glucokinase genes
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
EA036393B1 (en) 2015-05-21 2020-11-05 Вингс Терапьютикс, Инк. Antisense oligonucleotides to treat dystrophic epidermolysis bullosa
US11266748B2 (en) * 2015-07-02 2022-03-08 University Of Florida Research Foundation, Incorporated Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
CA3000046A1 (en) 2015-10-16 2017-04-20 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating alzheimer's disease
WO2017096162A1 (en) 2015-12-02 2017-06-08 Voyager Therapeutics, Inc. Assays for the detection of aav neutralizing antibodies
JP7110108B2 (en) 2015-12-09 2022-08-01 ジンガン メディスン(オーストラリア) プロプライアタリー リミティド Therapeutic immunomodulatory compositions
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. Compositions for the treatment of disease
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
JP7066635B2 (en) 2016-05-18 2022-05-13 ボイジャー セラピューティクス インコーポレイテッド Modulatory polynucleotide
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
JP2020500524A (en) 2016-12-13 2020-01-16 スティヒティング カソリーケ ユニベルシタイトStichting Katholieke Universiteit Antisense oligonucleotides for the treatment of Stargardt disease
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
JP2020518258A (en) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. Amyotrophic lateral sclerosis (ALS) treatment composition and method
JP2020530977A (en) 2017-05-24 2020-11-05 ウニベルシダッド アウトノマ デ バルセロナ Viral expression construct containing fibroblast growth factor 21 (FGF21) coding sequence
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN111132626B (en) 2017-07-17 2024-01-30 沃雅戈治疗公司 Track array guidance system
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3759126A1 (en) 2018-02-28 2021-01-06 Stichting Katholieke Universiteit Minigene for the treatment of usher syndrome type 2a and ush2a-associated retinitis pigmentosa
WO2019191232A2 (en) 2018-03-27 2019-10-03 University Of Rochester Nucleic acid molecules for pseudouridylation
EP3824086A1 (en) 2018-07-19 2021-05-26 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4
MX2021005824A (en) 2018-11-26 2021-10-13 UNIV AUTòNOMA DE BARCELONA Fibroblast growth factor 21 (fgf21) gene therapy.
EA202191469A1 (en) 2018-12-04 2021-10-19 Стихтинг Католике Университет RESTORATION USING ANTI-SENSE OLIGONUCLEOTIDES ABCA4 WITH ABERRANT SPLICING
WO2020148400A1 (en) 2019-01-16 2020-07-23 Stichting Katholieke Universiteit Antisense oligonucleotides for use in the treatment of crpc
US20220098584A1 (en) 2019-01-28 2022-03-31 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber`s congenital amaurosis
CA3124493A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
GB201902735D0 (en) 2019-02-28 2019-04-17 Proqr Therapeutics Ii Bv Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa
WO2020201144A1 (en) 2019-04-02 2020-10-08 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
US20220213478A1 (en) 2019-04-18 2022-07-07 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
CN113891726A (en) 2019-05-31 2022-01-04 巴塞罗那自治大学 Insulin gene therapy
US20220290154A1 (en) 2019-08-08 2022-09-15 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides rescue aberrant splicing of ABCA4
WO2021108050A2 (en) * 2019-10-23 2021-06-03 Yale University Compositions and methods comprising viral vector systems for multiplexed activation of endogenous genes as immunotherapy and viral-based immune-gene therapy
EP4051794A1 (en) 2019-10-31 2022-09-07 Stichting Radboud universitair medisch centrum Allele-specific silencing therapy for dfna9 using antisense oligonucleotides
AU2020414395A1 (en) 2019-12-23 2022-07-21 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of Stargardt disease
US20230134677A1 (en) 2020-03-04 2023-05-04 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
AU2021281506A1 (en) 2020-05-26 2023-02-02 Universitat Autònoma De Barcelona Fibroblast growth factor 21 (FGF21) gene therapy for central nervous system disorders
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
EP4284440A1 (en) 2021-01-30 2023-12-06 Universitat Autònoma De Barcelona Gene therapy for monogenic diabetes
EP4089171A1 (en) 2021-05-12 2022-11-16 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Recombinant tert-encoding viral genomes and vectors
WO2022269016A1 (en) 2021-06-25 2022-12-29 Stichting Radboud Universitair Medisch Centrum Allele-specific silencing therapy for dfna21 using antisense oligonucleotides
WO2023285700A1 (en) 2021-07-16 2023-01-19 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Oligonucleotide for inhibiting quaking activity
WO2023152371A1 (en) 2022-02-14 2023-08-17 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
GB202211123D0 (en) 2022-07-29 2022-09-14 Univ Edinburgh Cancer treatment
WO2024052413A1 (en) 2022-09-07 2024-03-14 Universitat Autònoma De Barcelona Beta-hexosaminidase vectors
DE102022124232A1 (en) 2022-09-21 2024-03-21 Carl von Ossietzky Universität Oldenburg, Körperschaft des öffentlichen Rechts Antisense oligonucleotides for the treatment of Joubert syndrome
WO2024074668A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exons 30-31
WO2024074670A1 (en) 2022-10-06 2024-04-11 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for treatment of usher 2a. exon 68

Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS63141588A (en) * 1986-12-05 1988-06-14 Toray Ind Inc Recombinant dna and transformant
WO1992005262A1 (en) * 1990-09-14 1992-04-02 The John Hopkins University Methods and compositions for genetic therapy and potentiation of anti-tumor immunity
WO1994021115A1 (en) * 1993-03-25 1994-09-29 Baylor College Of Medicine Broad spectrum tumor suppressor genes, gene products and methods for tumor suppression gene therapy
WO1995007105A1 (en) * 1993-09-07 1995-03-16 Sidney Kimmel Cancer Center Haplotype-matched cytokine-secreting cells and methods of using to stimulate an immune response
WO1995009655A1 (en) * 1993-10-06 1995-04-13 The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Treatment of tumors by genetic transformation of tumor cells with genes encoding negative selective markers and cytokines
WO1995013392A1 (en) * 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
WO1995014771A1 (en) * 1993-11-24 1995-06-01 GOVERNMENT OF THE UNITED STATES, represented by THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES: National Institutes of Health Vector systems for the generation of adeno-associated virus particles
WO1995028493A1 (en) * 1994-04-13 1995-10-26 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
WO1995028948A1 (en) * 1994-04-25 1995-11-02 Board Of Regents, The University Of Texas System Compositions comprising dna damaging agents and p53
DE4431401A1 (en) * 1994-08-24 1996-02-29 Max Delbrueck Centrum Live vaccine against tumor diseases

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8919607D0 (en) * 1989-08-30 1989-10-11 Wellcome Found Novel entities for cancer therapy
CA2113990A1 (en) * 1991-07-26 1993-02-18 Frederick L. Moolten Cancer therapy utilizing malignant cells
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5834441A (en) * 1993-09-13 1998-11-10 Rhone-Poulenc Rorer Pharmaceuticals Inc. Adeno-associated viral (AAV) liposomes and methods related thereto
WO1995027494A1 (en) * 1994-04-11 1995-10-19 Sloan-Kettering Institute For Cancer Research DEFECTIVE HERPES AND DEFECTIVE ADENO-ASSOCIATED VIRUS VECTORS WITH p53 FOR THE TREATMENT OF CANCER
US5952221A (en) * 1996-03-06 1999-09-14 Avigen, Inc. Adeno-associated virus vectors comprising a first and second nucleic acid sequence

Patent Citations (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS63141588A (en) * 1986-12-05 1988-06-14 Toray Ind Inc Recombinant dna and transformant
WO1992005262A1 (en) * 1990-09-14 1992-04-02 The John Hopkins University Methods and compositions for genetic therapy and potentiation of anti-tumor immunity
WO1994021115A1 (en) * 1993-03-25 1994-09-29 Baylor College Of Medicine Broad spectrum tumor suppressor genes, gene products and methods for tumor suppression gene therapy
WO1995007105A1 (en) * 1993-09-07 1995-03-16 Sidney Kimmel Cancer Center Haplotype-matched cytokine-secreting cells and methods of using to stimulate an immune response
WO1995009655A1 (en) * 1993-10-06 1995-04-13 The Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Treatment of tumors by genetic transformation of tumor cells with genes encoding negative selective markers and cytokines
WO1995013392A1 (en) * 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
WO1995014771A1 (en) * 1993-11-24 1995-06-01 GOVERNMENT OF THE UNITED STATES, represented by THE SECRETARY OF THE DEPARTMENT OF HEALTH AND HUMAN SERVICES: National Institutes of Health Vector systems for the generation of adeno-associated virus particles
WO1995028493A1 (en) * 1994-04-13 1995-10-26 The Rockefeller University Aav-mediated delivery of dna to cells of the nervous system
WO1995028948A1 (en) * 1994-04-25 1995-11-02 Board Of Regents, The University Of Texas System Compositions comprising dna damaging agents and p53
DE4431401A1 (en) * 1994-08-24 1996-02-29 Max Delbrueck Centrum Live vaccine against tumor diseases

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
DATABASE WPI Section Ch Week 8829, Derwent World Patents Index; Class B04, AN 88-202841, XP002047796 *
H. OKADA ET AL.: "GENE THERAPY AGAINST AN EXPERIMENTAL GLIOMA USING ADENO-ASSOCIATED VIRUS VECTORS", GENE THERAPY, vol. 3, no. 11, November 1996 (1996-11-01), pages 957 - 964, XP000614698 *
LAURET E ET AL.: "DEVELOPMENT OF METHODS FOR SOMATIC CELL GENE THERAPY DIRECTED AGAINST VIRAL DISEASES, USING RETROVIRAL VECTORS CARRYING THE MURINE OR HUMAN INTERFERON-BETA CODING SEQUENCE:ESTABLISHEMENT OF THE ANTIVIRAL STATE IN HUMAN CELLS", HUMAN GENE THERAPY, vol. 4, no. 5, October 1993 (1993-10-01), NEW YORK US, pages 567 - 577, XP002047794 *
YAGI K ET AL.: "INTERFERON-BETA ENDOGENOUSLY PRODUCED BY INTRATUMORAL INJECTION OF CATIONIC LIPOSOME- ENCAPSULATED GENE: CYTOCIDAL EFFECT ON GLIOMA TRANSPLANTED INTO NUDE MOUSE BRAIN", BIOCHEM MOL BIOL INT, vol. 32, no. 1, January 1994 (1994-01-01), SYDNEY AU, pages 167 - 171, XP002047795 *
ZHANG J F ET AL: "GENE THERAPY WITH AN ADENO-ASSOCIATED VIRUS CARRYING AN INTERFERON GENE RESULTS IN TUMOR GROWTH SUPPRESSION AND REGRESSION", CANCER GENE THERAPY, vol. 3, no. 1, January 1996 (1996-01-01), US, pages 31 - 38, XP002038701 *

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