DK1496944T3 - Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteiner - Google Patents
Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteinerInfo
- Publication number
- DK1496944T3 DK1496944T3 DK03786493T DK03786493T DK1496944T3 DK 1496944 T3 DK1496944 T3 DK 1496944T3 DK 03786493 T DK03786493 T DK 03786493T DK 03786493 T DK03786493 T DK 03786493T DK 1496944 T3 DK1496944 T3 DK 1496944T3
- Authority
- DK
- Denmark
- Prior art keywords
- vectors
- expression systems
- proteins
- sapsid
- raav
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 238000000034 method Methods 0.000 abstract 3
- 239000013598 vector Substances 0.000 abstract 3
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 abstract 2
- 102000004196 processed proteins & peptides Human genes 0.000 abstract 2
- 108090000765 processed proteins & peptides Proteins 0.000 abstract 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 102000053642 Catalytic RNA Human genes 0.000 abstract 1
- 108090000994 Catalytic RNA Proteins 0.000 abstract 1
- 229920002971 Heparan sulfate Polymers 0.000 abstract 1
- 241000124008 Mammalia Species 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 238000001476 gene delivery Methods 0.000 abstract 1
- 229920000669 heparin Polymers 0.000 abstract 1
- 229960002897 heparin Drugs 0.000 abstract 1
- -1 host cells Substances 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 210000000056 organ Anatomy 0.000 abstract 1
- 239000002245 particle Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 229920001184 polypeptide Polymers 0.000 abstract 1
- 239000013608 rAAV vector Substances 0.000 abstract 1
- 108091092562 ribozyme Proteins 0.000 abstract 1
- 230000001225 therapeutic effect Effects 0.000 abstract 1
- 210000002845 virion Anatomy 0.000 abstract 1
- 230000003612 virological effect Effects 0.000 abstract 1
Classifications
-
- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
-
- 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
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
- C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
- C12N2810/858—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from apolipopeptides
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US37731502P | 2002-05-01 | 2002-05-01 | |
| PCT/US2003/013583 WO2004027019A2 (en) | 2002-05-01 | 2003-05-01 | Improved raav expression systems for genetic modification of specific capsid proteins |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK1496944T3 true DK1496944T3 (da) | 2008-12-01 |
Family
ID=32030574
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK03786493T DK1496944T3 (da) | 2002-05-01 | 2003-05-01 | Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteiner |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US8802080B2 (da) |
| EP (1) | EP1496944B1 (da) |
| AT (1) | ATE405295T1 (da) |
| AU (1) | AU2003295312B2 (da) |
| CA (1) | CA2483624A1 (da) |
| DE (1) | DE60323078D1 (da) |
| DK (1) | DK1496944T3 (da) |
| NZ (2) | NZ536274A (da) |
| SI (1) | SI1496944T1 (da) |
| WO (1) | WO2004027019A2 (da) |
Families Citing this family (84)
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| DE60323078D1 (de) | 2002-05-01 | 2008-10-02 | Univ Florida | Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine |
| US20040179028A1 (en) * | 2003-03-12 | 2004-09-16 | Fuji Photo Film Co., Ltd. | Pixel defect correcting method, color mura correcting method and image display device |
| EP1618201A1 (en) * | 2003-05-01 | 2006-01-25 | University of Florida Research Foundation, Inc. | Vp2 - modified raav vectors and uses thereof |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| ES2329636B2 (es) | 2006-02-17 | 2010-07-26 | Universitat De Valencia, Estudi General (Participa Con El 70%) | Uso del factor pedf para inducir la auto-renovacion de celulas madre. |
| US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
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| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
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| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
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| CN104592364B (zh) * | 2013-10-30 | 2018-05-01 | 北京大学 | 定点突变和定点修饰的腺相关病毒、其制备方法及应用 |
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| WO2024006433A1 (en) * | 2022-06-30 | 2024-01-04 | Carnegie Mellon University | Method for production of virus-like particles for gene delivery |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0702722B1 (en) * | 1993-06-07 | 2005-08-03 | Vical Incorporated | Plasmids suitable for gene therapy |
| US6204059B1 (en) * | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5741683A (en) * | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| EP0933997A4 (en) * | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT AAV VECTORS |
| US6221646B1 (en) * | 1997-07-31 | 2001-04-24 | Chiron Corporation | Materials and methods for simplified AAV production |
| CA2367375A1 (en) | 1999-03-15 | 2000-09-21 | Chiron Corporation | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6943153B1 (en) * | 1999-03-15 | 2005-09-13 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6329181B1 (en) * | 2000-08-07 | 2001-12-11 | Neurologix, Inc. | Helper functions for recombinant vector production |
| WO2002024234A2 (en) | 2000-09-20 | 2002-03-28 | The Regents Of The University Of California | Use of recombinant gene delivery vectors for treating or preventing diseases of the eye |
| US6962815B2 (en) * | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
| US6692815B2 (en) * | 2001-06-15 | 2004-02-17 | Vinylex Corporation | Extruded plastic lumber and method of manufacture |
| US6723551B2 (en) * | 2001-11-09 | 2004-04-20 | The United States Of America As Represented By The Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| AU2003221733A1 (en) * | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| DE60323078D1 (de) | 2002-05-01 | 2008-10-02 | Univ Florida | Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine |
| WO2004020600A2 (en) * | 2002-08-28 | 2004-03-11 | University Of Florida | Modified aav |
| EP1618201A1 (en) | 2003-05-01 | 2006-01-25 | University of Florida Research Foundation, Inc. | Vp2 - modified raav vectors and uses thereof |
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2003
- 2003-05-01 DE DE60323078T patent/DE60323078D1/de not_active Expired - Fee Related
- 2003-05-01 NZ NZ536274A patent/NZ536274A/en unknown
- 2003-05-01 SI SI200331424T patent/SI1496944T1/sl unknown
- 2003-05-01 AT AT03786493T patent/ATE405295T1/de not_active IP Right Cessation
- 2003-05-01 DK DK03786493T patent/DK1496944T3/da active
- 2003-05-01 US US10/513,059 patent/US8802080B2/en active Active
- 2003-05-01 AU AU2003295312A patent/AU2003295312B2/en not_active Ceased
- 2003-05-01 EP EP03786493A patent/EP1496944B1/en not_active Expired - Lifetime
- 2003-05-01 NZ NZ561656A patent/NZ561656A/en unknown
- 2003-05-01 CA CA002483624A patent/CA2483624A1/en not_active Abandoned
- 2003-05-01 WO PCT/US2003/013583 patent/WO2004027019A2/en not_active Ceased
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2014
- 2014-07-08 US US14/326,293 patent/US20150005369A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1496944A4 (en) | 2006-03-22 |
| EP1496944A2 (en) | 2005-01-19 |
| US20150005369A1 (en) | 2015-01-01 |
| AU2003295312B2 (en) | 2008-08-14 |
| ATE405295T1 (de) | 2008-09-15 |
| EP1496944B1 (en) | 2008-08-20 |
| DE60323078D1 (de) | 2008-10-02 |
| WO2004027019A2 (en) | 2004-04-01 |
| HK1071518A1 (en) | 2005-07-22 |
| WO2004027019A3 (en) | 2004-08-05 |
| NZ561656A (en) | 2009-03-31 |
| SI1496944T1 (sl) | 2009-02-28 |
| CA2483624A1 (en) | 2004-04-01 |
| US20060088936A1 (en) | 2006-04-27 |
| AU2003295312A1 (en) | 2004-04-08 |
| US8802080B2 (en) | 2014-08-12 |
| NZ536274A (en) | 2008-04-30 |
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