WO2004020631A3 - Genetic suppression and replacement - Google Patents
Genetic suppression and replacement Download PDFInfo
- Publication number
- WO2004020631A3 WO2004020631A3 PCT/GB2003/003793 GB0303793W WO2004020631A3 WO 2004020631 A3 WO2004020631 A3 WO 2004020631A3 GB 0303793 W GB0303793 W GB 0303793W WO 2004020631 A3 WO2004020631 A3 WO 2004020631A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- suppression
- nucleic acid
- gene
- replacement
- replacement nucleic
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New breeds of animals
- A01K67/027—New breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2207/00—Modified animals
- A01K2207/15—Humanized animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/121—Hammerhead
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
Abstract
Priority Applications (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CA2496904A CA2496904C (en) | 1996-04-02 | 2003-09-01 | Genetic suppression and replacement |
EP03753679.4A EP1532246B1 (en) | 1996-04-02 | 2003-09-01 | Genetic suppression and replacement |
AU2003271841A AU2003271841A1 (en) | 1996-04-02 | 2003-09-01 | Genetic suppression and replacement |
Applications Claiming Priority (8)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
GBGB9606961.2A GB9606961D0 (en) | 1996-04-02 | 1996-04-02 | Genetic strategy III |
US09/155,708 US7138378B1 (en) | 1996-04-02 | 1997-04-02 | Genetic suppression and replacement |
US40738902P | 2002-08-30 | 2002-08-30 | |
US60/407,389 | 2002-08-30 | ||
US41469802P | 2002-09-30 | 2002-09-30 | |
US60/414,698 | 2002-09-30 | ||
US10/651,754 US20040234999A1 (en) | 1996-04-02 | 2003-08-29 | Genetic suppression and replacement |
US10/651,754 | 2003-08-29 |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2004020631A2 WO2004020631A2 (en) | 2004-03-11 |
WO2004020631A3 true WO2004020631A3 (en) | 2004-07-29 |
Family
ID=37670462
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/GB2003/003793 WO2004020631A2 (en) | 1996-04-02 | 2003-09-01 | Genetic suppression and replacement |
Country Status (5)
Country | Link |
---|---|
US (1) | US20040234999A1 (en) |
EP (1) | EP1532246B1 (en) |
AU (1) | AU2003271841A1 (en) |
CA (1) | CA2496904C (en) |
WO (1) | WO2004020631A2 (en) |
Families Citing this family (16)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US8551970B2 (en) * | 1996-04-02 | 2013-10-08 | Optigen Patents Limited | Genetic suppression and replacement |
RU2348695C2 (en) | 2006-05-23 | 2009-03-10 | Закрытое акционерное общество "Молекулярно-медицинские технологии" | Differentiating and specific oligonucleotids for dna sequence identification for infection agents in biological materials, method of species identification of infection agents, biochip and method implementation kit |
JP2010500011A (en) | 2006-08-08 | 2010-01-07 | ガンサー ハートマン | Structure and use of 5 'phosphate oligonucleotides |
CA2678055C (en) * | 2007-04-10 | 2016-02-16 | Qiagen Gmbh | Rna interference tags |
CA2683469C (en) | 2007-04-12 | 2019-11-26 | The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
WO2009141146A1 (en) | 2008-05-21 | 2009-11-26 | Gunther Hartmann | 5' triphosphate oligonucleotide with blunt end and uses thereof |
EP2218781A1 (en) * | 2009-02-11 | 2010-08-18 | Qiagen GmbH | RNAi based selection system |
WO2010124231A2 (en) * | 2009-04-24 | 2010-10-28 | The Board Of Regents Of The University Of Texas System | Modulation of gene expression using oligomers that target gene regions downstream of 3' untranslated regions |
WO2011019793A2 (en) * | 2009-08-13 | 2011-02-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer, National Institutes Of Health | Individualized therapy of single gene disorders with genetically modified adult stem cells |
CA3102008A1 (en) | 2010-06-02 | 2011-12-08 | Alnylam Pharmaceuticals, Inc. | Compositions and methods directed to treating liver fibrosis |
EP2508530A1 (en) | 2011-03-28 | 2012-10-10 | Rheinische Friedrich-Wilhelms-Universität Bonn | Purification of triphosphorylated oligonucleotides using capture tags |
EP2712870A1 (en) | 2012-09-27 | 2014-04-02 | Rheinische Friedrich-Wilhelms-Universität Bonn | Novel RIG-I ligands and methods for producing them |
CN115896106A (en) | 2016-03-01 | 2023-04-04 | 佛罗里达大学研究基金会有限公司 | AAV vectors for the treatment of dominant retinitis pigmentosa |
WO2022019706A1 (en) * | 2020-07-24 | 2022-01-27 | 알지노믹스 주식회사 | Trans-splicing ribozyme that is specific to rhodopsin transcriptome, and use thereof |
KR102396200B1 (en) * | 2020-07-24 | 2022-05-12 | 알지노믹스 주식회사 | A trans-splicing ribozyme specific for rhodopsin transcript and uses thereof |
US20220098615A1 (en) * | 2020-09-30 | 2022-03-31 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
Citations (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1995019448A1 (en) * | 1994-01-14 | 1995-07-20 | The Johns Hopkins University School Of Medicine | Nucleic acid mutation detection in histologic tissue |
WO1997032024A1 (en) * | 1996-03-01 | 1997-09-04 | Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin | Allele suppression |
WO1997037014A1 (en) * | 1996-04-02 | 1997-10-09 | Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin | Genetic suppression and replacement |
WO1998048027A2 (en) * | 1997-04-21 | 1998-10-29 | University Of Florida | Materials and methods for treatment of retinal diseases |
WO2003048362A2 (en) * | 2001-11-30 | 2003-06-12 | Optigen Patents Limited | Suppression of polymeric alleles |
Family Cites Families (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5240846A (en) * | 1989-08-22 | 1993-08-31 | The Regents Of The University Of Michigan | Gene therapy vector for cystic fibrosis |
US5223391A (en) * | 1990-02-21 | 1993-06-29 | President And Fellows Of Harvard College | Inhibitors of herpes simplex virus replication |
DK51092D0 (en) * | 1991-05-24 | 1992-04-15 | Ole Buchardt | OLIGONUCLEOTIDE ANALOGUE DESCRIBED BY PEN, MONOMERIC SYNTHONES AND PROCEDURES FOR PREPARING THEREOF, AND APPLICATIONS THEREOF |
US5807718A (en) * | 1994-12-02 | 1998-09-15 | The Scripps Research Institute | Enzymatic DNA molecules |
US6482803B1 (en) * | 1995-09-01 | 2002-11-19 | Board Of Regents, The University Of Texas System | Modification of mutated P53 gene in tumors by retroviral delivery of ribozyme A |
GB9519299D0 (en) * | 1995-09-21 | 1995-11-22 | Farrar Gwyneth J | Genetic strategy |
-
2003
- 2003-08-29 US US10/651,754 patent/US20040234999A1/en not_active Abandoned
- 2003-09-01 CA CA2496904A patent/CA2496904C/en not_active Expired - Lifetime
- 2003-09-01 WO PCT/GB2003/003793 patent/WO2004020631A2/en not_active Application Discontinuation
- 2003-09-01 EP EP03753679.4A patent/EP1532246B1/en not_active Expired - Lifetime
- 2003-09-01 AU AU2003271841A patent/AU2003271841A1/en not_active Abandoned
Patent Citations (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1995019448A1 (en) * | 1994-01-14 | 1995-07-20 | The Johns Hopkins University School Of Medicine | Nucleic acid mutation detection in histologic tissue |
WO1997032024A1 (en) * | 1996-03-01 | 1997-09-04 | Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin | Allele suppression |
WO1997037014A1 (en) * | 1996-04-02 | 1997-10-09 | Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin | Genetic suppression and replacement |
WO1998048027A2 (en) * | 1997-04-21 | 1998-10-29 | University Of Florida | Materials and methods for treatment of retinal diseases |
WO2003048362A2 (en) * | 2001-11-30 | 2003-06-12 | Optigen Patents Limited | Suppression of polymeric alleles |
Non-Patent Citations (5)
Title |
---|
BRUMMELKAMP T R ET AL: "A system for stable expression of short interfering RNAs in mammalian cells", SCIENCE, vol. 296, no. 5567, 19 April 2002 (2002-04-19), pages 550 - 553, XP002225638, ISSN: 0036-8075 * |
LEWIN A S ET AL: "Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa", NATURE MEDICINE, vol. 4, no. 8, 1 August 1998 (1998-08-01), pages 967 - 971, XP002090346, ISSN: 1078-8956 * |
MILLINGTON-WARD S ET AL: "Strategems in vitro for gene therapies directed to dominant mutations", NUCLEIC ACIDS RESEARCH, vol. 6, no. 9, September 1997 (1997-09-01), pages 1415 - 1426, XP002254680, ISSN: 0305-1048 * |
O'NEILL BRIAN ET AL: "Ribozyme-based therapeutic approaches for autosomal dominant retinitis pigmentosa", IOVS, vol. 41, no. 10, September 2000 (2000-09-01), pages 2863 - 2869, XP002281307 * |
TOUDJARSKA I ET AL: "Delivery of a hammerhead ribozyme specifically downregulates mutant type I collagen mRNA in a murine model of osteogenesis imperfecta", ANTISENSE & NUCLEIC ACID DRUG DEVELOPMENT, vol. 11, no. 5, October 2001 (2001-10-01), pages 341 - 346, XP002254679, ISSN: 1087-2906 * |
Also Published As
Publication number | Publication date |
---|---|
US20040234999A1 (en) | 2004-11-25 |
AU2003271841A8 (en) | 2004-03-19 |
EP1532246B1 (en) | 2018-07-11 |
CA2496904C (en) | 2014-12-09 |
EP1532246A2 (en) | 2005-05-25 |
AU2003271841A1 (en) | 2004-03-19 |
WO2004020631A2 (en) | 2004-03-11 |
CA2496904A1 (en) | 2004-03-11 |
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