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)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- Virology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (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 (de) |
| EP (1) | EP1496944B1 (de) |
| AT (1) | ATE405295T1 (de) |
| AU (1) | AU2003295312B2 (de) |
| CA (1) | CA2483624A1 (de) |
| DE (1) | DE60323078D1 (de) |
| DK (1) | DK1496944T3 (de) |
| NZ (2) | NZ561656A (de) |
| SI (1) | SI1496944T1 (de) |
| WO (1) | WO2004027019A2 (de) |
Families Citing this family (84)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004027019A2 (en) | 2002-05-01 | 2004-04-01 | University Of Florida Research Foundation, Inc. | Improved raav expression systems for genetic modification of specific capsid proteins |
| US20060093589A1 (en) * | 2004-02-19 | 2006-05-04 | Warrington Kenneth H | Vp2-modified raav vector compositions and uses therefor |
| 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 (de) * | 2003-05-01 | 2006-01-25 | University of Florida Research Foundation, Inc. | Vp2-modifizierte raav-vektoren und verwendungen davon |
| EP1486567A1 (de) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Verbesserter Adeno-assoziierter Virus (AAV)-Vektor für Gentherapie |
| 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. |
| US9611302B2 (en) | 2007-04-09 | 2017-04-04 | University Of Florida Research Foundation, Inc. | High-transduction-efficiency RAAV vectors, compositions, and methods of use |
| DK2191001T3 (en) | 2007-04-09 | 2016-09-19 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| 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 |
| JP2011512417A (ja) * | 2008-02-20 | 2011-04-21 | ジェンザイム・コーポレーション | 血管形成の阻害 |
| EP2910637A1 (de) | 2008-05-20 | 2015-08-26 | University of Florida Research Foundation, Inc. | Vektoren für die übertragung licht-empfindlicher proteine und deren verwendung |
| EP2396343B1 (de) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modifizierte virusvektoren sowie verfahren zu ihrer herstellung und verwendung |
| PL2403867T3 (pl) | 2009-03-04 | 2019-12-31 | Deutsches Krebsforschungszentrum | Białko aktywujące składanie (aap) i jego zastosowanie do wytwarzania cząstek parwowirusa zasadniczo składających się z vp3 |
| US9078878B2 (en) | 2010-12-01 | 2015-07-14 | Alderbio Holdings Llc | Anti-NGF antibodies that selectively inhibit the association of NGF with TrkA, without affecting the association of NGF with p75 |
| US9884909B2 (en) | 2010-12-01 | 2018-02-06 | Alderbio Holdings Llc | Anti-NGF compositions and use thereof |
| US11214610B2 (en) | 2010-12-01 | 2022-01-04 | H. Lundbeck A/S | High-purity production of multi-subunit proteins such as antibodies in transformed microbes such as Pichia pastoris |
| US9539324B2 (en) | 2010-12-01 | 2017-01-10 | Alderbio Holdings, Llc | Methods of preventing inflammation and treating pain using anti-NGF compositions |
| US9067988B2 (en) | 2010-12-01 | 2015-06-30 | Alderbio Holdings Llc | Methods of preventing or treating pain using anti-NGF antibodies |
| JP6190723B2 (ja) | 2010-12-01 | 2017-08-30 | アルダーバイオ ホールディングス エルエルシー | 抗ｎｇｆ組成物およびその使用 |
| EP2500434A1 (de) * | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsidfreie AAV-Vektoren, Zusammensetzungen und Verfahren zur Vektorherstellung und Genlieferung |
| JP6348064B2 (ja) | 2011-11-22 | 2018-06-27 | ザ チルドレンズ ホスピタル オブ フィラデルフィア | 効率の高いトランスジーン送達のためのウイルスベクター |
| US10294281B2 (en) | 2012-05-15 | 2019-05-21 | University Of Florida Research Foundation, Incorporated | High-transduction-efficiency rAAV vectors, compositions, and methods of use |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
| KR20240090694A (ko) | 2013-07-22 | 2024-06-21 | 더 칠드런스 호스피탈 오브 필라델피아 | 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도 |
| 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 |
| EP3485964B1 (de) * | 2013-12-06 | 2022-09-21 | Johnson Matthey Public Limited Company | Kaltstartkatalysator und dessen verwendung in abgassystemen |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| ES2768763T3 (es) | 2014-03-04 | 2020-06-23 | Univ Florida | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| BR112017005892A2 (pt) * | 2014-09-24 | 2017-12-12 | Hope City | variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| CA3193811A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN119876138A (zh) | 2014-11-14 | 2025-04-25 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| EP3031820A1 (de) | 2014-12-08 | 2016-06-15 | Life Science Inkubator | JC Polyomavirus virus-ähnliche Partikeln (VLP) mit einem Targeting-Peptid |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| WO2016126857A1 (en) | 2015-02-03 | 2016-08-11 | University Of Florida Research Foundation, Inc. | Recombinant aav1, aav5, and aav6 capsid mutants and uses thereof |
| US10648000B2 (en) | 2015-02-16 | 2020-05-12 | University Of Florida Research Foundation, Incorporated | rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes |
| US10081659B2 (en) | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| WO2016186772A2 (en) | 2015-05-16 | 2016-11-24 | Genzyme Corporation | Gene editing of deep intronic mutations |
| WO2016210170A1 (en) | 2015-06-23 | 2016-12-29 | The Children's Hospital Of Philadelphia | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| WO2017147477A1 (en) * | 2016-02-26 | 2017-08-31 | University Of Florida Research Foundation, Inc. | Aav heparin mutants that display significantly improved eye and brain transduction |
| EP3448987A4 (de) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | Zusammensetzungen zur behandlung einer erkrankung |
| EP3448874A4 (de) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | Zusammensetzungen zur behandlung einer erkrankung |
| FI3445773T3 (fi) | 2016-05-13 | 2023-03-30 | 4D Molecular Therapeutics Inc | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| NZ791267A (en) | 2016-08-15 | 2025-11-28 | Genzyme Corp | Methods for detecting AAV |
| 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 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| BR112019016769A2 (pt) * | 2017-02-15 | 2020-05-26 | The University Of North Carolina At Chapel Hill | Métodos e composições para transferência genética através da vasculatura |
| CN110392607A (zh) * | 2017-03-13 | 2019-10-29 | 三菱化学株式会社 | 过渡金属负载沸石和其制造方法以及氮氧化物净化用催化剂和其使用方法 |
| US10550405B2 (en) | 2017-03-15 | 2020-02-04 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| CN110770346B (zh) * | 2017-03-15 | 2024-01-12 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
| JP2020518259A (ja) | 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． | ハンチントン病治療組成物および方法 |
| EP3618839A4 (de) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Zusammensetzungen und verfahren zur behandlung von amyotropher lateralsklerose (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7229989B2 (ja) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | 軌道アレイガイドシステム |
| SG11202002276VA (en) | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| TW202413649A (zh) | 2017-10-16 | 2024-04-01 | 美商航海家醫療公司 | 肌萎縮性脊髓側索硬化症(als)之治療 |
| DK3717636T5 (da) | 2017-11-27 | 2024-08-26 | 4D Molecular Therapeutics Inc | Adeno-associeret-virus-variantcapsider og anvendelse til inhibering af angiogenese |
| CN112105630A (zh) * | 2018-02-15 | 2020-12-18 | T·比约尔克隆德 | 经修饰的病毒衣壳 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| WO2019216932A1 (en) * | 2018-05-07 | 2019-11-14 | The University Of North Carolina At Chapel Hill | Rational polyploid adeno-associated virus vectors and methods of making and using the same |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| US11821009B2 (en) | 2018-05-15 | 2023-11-21 | Cornell University | Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| EP3841109A4 (de) * | 2018-08-21 | 2022-09-21 | Massachusetts Eye and Ear Infirmary | Zusammensetzungen und verfahren zur modulation der transduktionseffizienz von adeno-assoziierten viren |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| JP2022520875A (ja) | 2019-02-25 | 2022-04-01 | ノバルティス アーゲー | Ｂｉｅｔｔｉクリスタリン網膜症を治療するための組成物及び方法 |
| AU2020229085A1 (en) | 2019-02-25 | 2021-08-19 | Friedrich Miescher Institute For Biomedical Research | Compositions and methods to treat bietti crystalline dystrophy |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| US20210082541A1 (en) | 2019-08-31 | 2021-03-18 | Wyatt Technology Corporation | Measuring attributes of a viral gene delivery vehicle sample via separation |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4200408A1 (de) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-assoziierte virusvektoren zur behandlung des rett-syndroms |
| JP2023548746A (ja) | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| 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 |
|---|---|---|---|---|
| CA2164088C (en) * | 1993-06-07 | 2005-06-14 | Gary J. Nabel | 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 |
| US6521225B1 (en) | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| US6221646B1 (en) | 1997-07-31 | 2001-04-24 | Chiron Corporation | Materials and methods for simplified AAV production |
| 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 |
| AU3755900A (en) | 1999-03-15 | 2000-10-04 | Chiron Corporation | 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 |
| AU2002248297A1 (en) * | 2001-01-05 | 2002-07-16 | Children's Hospital, 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 |
| WO2004027019A2 (en) | 2002-05-01 | 2004-04-01 | University Of Florida Research Foundation, Inc. | Improved raav expression systems for genetic modification of specific capsid proteins |
| AU2003265855A1 (en) * | 2002-08-28 | 2004-03-19 | University Of Florida | Modified aav |
| EP1618201A1 (de) | 2003-05-01 | 2006-01-25 | University of Florida Research Foundation, Inc. | Vp2-modifizierte raav-vektoren und verwendungen davon |
-
2003
- 2003-05-01 WO PCT/US2003/013583 patent/WO2004027019A2/en not_active Ceased
- 2003-05-01 US US10/513,059 patent/US8802080B2/en active Active
- 2003-05-01 AT AT03786493T patent/ATE405295T1/de not_active IP Right Cessation
- 2003-05-01 SI SI200331424T patent/SI1496944T1/sl unknown
- 2003-05-01 AU AU2003295312A patent/AU2003295312B2/en not_active Ceased
- 2003-05-01 DK DK03786493T patent/DK1496944T3/da active
- 2003-05-01 NZ NZ561656A patent/NZ561656A/en unknown
- 2003-05-01 CA CA002483624A patent/CA2483624A1/en not_active Abandoned
- 2003-05-01 NZ NZ536274A patent/NZ536274A/en unknown
- 2003-05-01 EP EP03786493A patent/EP1496944B1/de not_active Expired - Lifetime
- 2003-05-01 DE DE60323078T patent/DE60323078D1/de not_active Expired - Fee Related
-
2014
- 2014-07-08 US US14/326,293 patent/US20150005369A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1496944A4 (de) | 2006-03-22 |
| US20060088936A1 (en) | 2006-04-27 |
| NZ536274A (en) | 2008-04-30 |
| US20150005369A1 (en) | 2015-01-01 |
| WO2004027019A2 (en) | 2004-04-01 |
| SI1496944T1 (sl) | 2009-02-28 |
| EP1496944A2 (de) | 2005-01-19 |
| ATE405295T1 (de) | 2008-09-15 |
| AU2003295312A1 (en) | 2004-04-08 |
| AU2003295312B2 (en) | 2008-08-14 |
| NZ561656A (en) | 2009-03-31 |
| DE60323078D1 (de) | 2008-10-02 |
| CA2483624A1 (en) | 2004-04-01 |
| EP1496944B1 (de) | 2008-08-20 |
| WO2004027019A3 (en) | 2004-08-05 |
| HK1071518A1 (en) | 2005-07-22 |
| US8802080B2 (en) | 2014-08-12 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK1496944T3 (da) | Forbedrede rAAV-ekspressionssystemer til genetisk modificering af specifikke sapsid-proteiner | |
| CY1124586T1 (el) | Ομαδες, αλληλουχιες, φορεις αδενο-σχετιζομενου ιου (aav) που περιεχουν αυτον, και οι χρησεις τους | |
| MY199445A (en) | Adeno-associated virus variant capsids and methods of use thereof | |
| ATE353365T1 (de) | Neue b7-4 moleküle und deren verwendungen | |
| WO2005077333A3 (en) | Gel-based delivery of recombinant adeno-associated virus vectors | |
| ATE366814T1 (de) | Gl50 moleküle, sowie verwendungen derselben | |
| WO2003042362A8 (en) | PGC-1β, A NOVEL PGC-1 HOMOLOGUE AND USES THEREFOR | |
| WO2002063025A3 (en) | Muscle-directed gene therapy with aav-1 and aav-6 vectors | |
| DE60031418D1 (de) | 15571, ein neues gpcr-ähnliches molekül der secretin-ähnlichen familie und dessen verwendungen | |
| ES2226597T1 (es) | Vectores recombinantes virales y no virales que contienen el gen humano de urokinasa del activador del plasminogeno y su utilizacion en el tratamiento de varios tipos de fibrosis hepatica, renal, pulmonar, pancreatica y cardiaca y cicatrices hipertroficas. | |
| DE60237613D1 (de) | Neue pgc-1-isoformen und deren verwendungen | |
| WO2002002148A3 (en) | Dual recombinant gene therapy compositions and methods of use | |
| DK0781344T3 (da) | Hidtil ukendt implantat og hidtil ukendt vektor til behandling af erhvervede sygdomme | |
| WO2004087886A3 (en) | Modified adenoviral e1a constructs and methods of use thereof | |
| WO2001083781A3 (en) | 14094, a novel human trypsin family member and uses thereof | |
| WO2001064894A3 (en) | Human phospholipid scramblase-like molecules and uses thereof | |
| ATE476194T1 (de) | Ein mitglied der menschlichen proteinkinasefamilie, und verwendungen dafür | |
| DE60234047D1 (de) | 15603, ein mitglied der humanen ionenkanal-familie | |
| WO2003035840A3 (en) | 69583 and 85924, novel human protein kinase family members and uses therefor | |
| DE60136832D1 (de) | Atcr-1, eine humane acyltransferase und verwendungen davon | |
| AU2001288284A1 (en) | 14189, a human kinase and uses thereof | |
| WO2002048330A3 (en) | 21908 and 21911, human guanylate kinase family members and uses thereof | |
| WO2001064908A3 (en) | 14790, a protein kinase and uses therefor | |
| WO2003038040A3 (en) | 32235, a human aminotransferase family member and uses therefor | |
| ATE331033T1 (de) | Proteinkinase-molekül 14911 und dessen anwendungen |