ES2860712T3 - Variantes de virus adenoasociados y métodos de uso de las mismas - Google Patents
Variantes de virus adenoasociados y métodos de uso de las mismasInfo
- Publication number
- ES2860712T3 ES2860712T3 ES16769632T ES16769632T ES2860712T3 ES 2860712 T3 ES2860712 T3 ES 2860712T3 ES 16769632 T ES16769632 T ES 16769632T ES 16769632 T ES16769632 T ES 16769632T ES 2860712 T3 ES2860712 T3 ES 2860712T3
- Authority
- ES
- Spain
- Prior art keywords
- aav
- capsid protein
- cell
- virion
- raav
- 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.)
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- 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
- C12N15/861—Adenoviral vectors
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- 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
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
Landscapes
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- Peptides Or Proteins (AREA)
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| SG11201703148TA (en) | 2014-11-05 | 2017-05-30 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
| 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 |
| HK1245326A1 (zh) | 2014-12-12 | 2018-08-24 | Voyager Therapeutics, Inc. | 用於生产scaav的组合物和方法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| WO2017070476A2 (en) | 2015-10-22 | 2017-04-27 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav3 capsid library |
| WO2017096162A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| EP3448987A4 (en) | 2016-04-29 | 2020-05-27 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| CA3024449A1 (en) | 2016-05-18 | 2017-11-23 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CA3024448C (en) | 2016-05-18 | 2025-09-09 | Voyager Therapeutics, Inc. | MODULATING POLYNUCLEOTIDES |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| 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 |
| AU2018224044B2 (en) | 2017-02-21 | 2024-01-25 | The Uab Research Foundation | Modified AAV capsid proteins and uses thereof |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| 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 |
| WO2018211409A1 (en) | 2017-05-15 | 2018-11-22 | University Of Florida Research Foundation, Inc. | Core master regulators of glioblastoma stem cells |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| 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 |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| 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 |
| BR112020016699A2 (pt) * | 2018-02-28 | 2020-12-15 | The University Of North Carolina At Chapel Hill | Métodos e composições para vetores de vírus de evasão de anticorpos |
| 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 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| TW202005978A (zh) * | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| SG11202011296VA (en) | 2018-05-15 | 2020-12-30 | Voyager Therapeutics Inc | Compositions and methods for the treatment of parkinson's disease |
| CN112566923B (zh) * | 2018-06-12 | 2024-10-29 | 北卡罗来纳大学教堂山分校 | 合成嗜肝性腺相关病毒衣壳及其用途 |
| CA3111076A1 (en) | 2018-08-30 | 2020-03-05 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with myocardin and ascl1 |
| WO2020068990A1 (en) | 2018-09-26 | 2020-04-02 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| CN114450411A (zh) | 2019-04-01 | 2022-05-06 | 特纳亚治疗股份有限公司 | 具有工程化衣壳的腺相关病毒 |
| CN111909246B (zh) * | 2019-05-08 | 2024-01-19 | 中国科学院脑科学与智能技术卓越创新中心 | 高效感染支持细胞的aav突变体 |
| EP3980447A4 (en) * | 2019-06-10 | 2023-07-26 | Homology Medicines, Inc. | ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR ARSA GENE TRANSFER AND METHODS OF USE THEREOF |
| WO2021007515A1 (en) | 2019-07-11 | 2021-01-14 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with micrornas and other factors |
| CN110295151A (zh) * | 2019-07-17 | 2019-10-01 | 博鑫仪器(天津)有限公司 | 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法 |
| EP4010357A2 (en) * | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
| WO2021041489A1 (en) * | 2019-08-29 | 2021-03-04 | The Board Of Trustees Of The Leland Stanford Junior University | Adeno-associated viral vectors for crossing the human blood brain barrier |
| WO2021050974A1 (en) * | 2019-09-12 | 2021-03-18 | The Broad Institute, Inc. | Engineered adeno-associated virus capsids |
| US20220403414A1 (en) * | 2019-10-16 | 2022-12-22 | Wuxi Apptec (Shanghai) Co., Ltd. | Novel aav variant |
| AU2020368539A1 (en) | 2019-10-16 | 2022-04-28 | Massachusetts Institute Of Technology | Engineered muscle targeting compositions |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| CN110950934B (zh) * | 2019-12-31 | 2022-11-04 | 复旦大学 | 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用 |
| WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| MY209860A (en) | 2020-02-13 | 2025-08-07 | Tenaya Therapeutics Inc | Gene therapy vectors for treating heart disease |
| US20230103731A1 (en) | 2020-03-02 | 2023-04-06 | Tenaya Therapeutics, Inc. | Gene vector control by cardiomyocyte-expressed micrornas |
| AU2021263563B2 (en) | 2020-04-27 | 2023-02-02 | 4D Molecular Therapeutics Inc. | Codon optimized GLA genes and uses thereof |
| KR20230005941A (ko) * | 2020-05-01 | 2023-01-10 | 트러스티스 오브 보스톤 칼리지 | 향상된 유전자 요법을 위한 아데노-관련 바이러스(aav)의 제어된 변형 |
| CN111560399B (zh) * | 2020-05-22 | 2021-07-06 | 苏州君盟生物医药科技有限公司 | 细胞大规模瞬时转染方法 |
| JP2023528801A (ja) | 2020-05-29 | 2023-07-06 | 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 | バリアントキャプシドを有するアデノ随伴ウイルスビリオン及びその使用方法 |
| EP4591708A3 (en) | 2020-07-08 | 2026-01-14 | Baylor College of Medicine | Gene therapy for stxbp1 encephalopathy |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| CN112226461B (zh) * | 2020-08-21 | 2022-04-22 | 华侨大学 | Cd4阳性细胞特异性基因传递载体及其应用 |
| CN112080521B (zh) * | 2020-09-07 | 2022-05-31 | 山东农业大学 | 一种表达外源蛋白的重组伪狂犬病毒载体构建及重组伪狂犬病毒制备方法 |
| CN112194706B (zh) * | 2020-09-30 | 2022-03-08 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| IL301675A (en) | 2020-10-09 | 2023-05-01 | Tenaya Therapeutics Inc | Methods and preparations for placophilin-2 gene therapy |
| US20240269322A1 (en) * | 2020-10-21 | 2024-08-15 | Chan Zuckerberg Biohub, Inc. | Adeno-associated virus virions and methods of use thereof |
| EP4244360A4 (en) * | 2020-11-16 | 2025-05-07 | Beijing SoloBio Genetechnology Company Ltd. | Recombinant adeno-associated viruses with enhanced liver tropism and uses thereof |
| US12285444B2 (en) * | 2020-12-08 | 2025-04-29 | Duke University | Multicistronic miRNA delivery for cardiac reprogramming |
| CN113121653B (zh) * | 2021-04-19 | 2021-11-19 | 上海信致医药科技有限公司 | 肌肉和视网膜特异性的新型腺相关病毒衣壳蛋白 |
| CN113121654B (zh) * | 2021-04-19 | 2023-11-17 | 信念医药科技(上海)有限公司 | 腺相关病毒衣壳蛋白及包含其的腺相关病毒载体 |
| WO2023106256A1 (ja) * | 2021-12-06 | 2023-06-15 | 学校法人順天堂 | 改変型アデノ随伴ウイルスベクター |
| WO2023108507A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Recombinant aav vectors and use thereof |
| WO2024054864A1 (en) | 2022-09-06 | 2024-03-14 | Tenaya Therapeutics, Inc. | Cardioprotective heart disease therapies |
| CN116789738B (zh) * | 2022-12-08 | 2023-12-19 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| WO2024215653A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| AU2024271436A1 (en) * | 2023-05-15 | 2025-11-13 | Sangamo Therapeutics, Inc. | Engineered blood brain barrier penetrant aav capsids |
Family Cites Families (98)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| WO1992001070A1 (en) | 1990-07-09 | 1992-01-23 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | High efficiency packaging of mutant adeno-associated virus using amber suppressions |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| JP3534749B2 (ja) | 1991-08-20 | 2004-06-07 | アメリカ合衆国 | アデノウイルスが介在する胃腸管への遺伝子の輸送 |
| GB9206016D0 (en) | 1992-03-19 | 1992-04-29 | Sandoz Ltd | Improvements in or relating to organic compounds |
| AU7676894A (en) | 1993-08-27 | 1995-03-21 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Convection-enhanced drug delivery |
| US6440425B1 (en) | 1995-05-01 | 2002-08-27 | Aventis Pasteur Limited | High molecular weight major outer membrane protein of moraxella |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US6001650A (en) | 1995-08-03 | 1999-12-14 | Avigen, Inc. | High-efficiency wild-type-free AAV helper functions |
| US6096548A (en) | 1996-03-25 | 2000-08-01 | Maxygen, Inc. | Method for directing evolution of a virus |
| CA2251738A1 (en) | 1996-04-16 | 1997-10-23 | Immusol Incorporated | Targeted viral vectors |
| US6710036B2 (en) | 1997-07-25 | 2004-03-23 | Avigen, Inc. | Induction of immune response to antigens expressed by recombinant adeno-associated virus |
| CA2303768C (en) | 1997-09-19 | 2009-11-24 | The Trustees Of The University Of Pennsylvania | Methods and vector constructs useful for production of recombinant aav |
| WO1999023107A1 (en) | 1997-10-31 | 1999-05-14 | Maxygen, Incorporated | Modification of virus tropism and host range by viral genome shuffling |
| US6410300B1 (en) | 1998-01-12 | 2002-06-25 | The University Of North Carolina At Chapel Hill | Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV |
| US6551795B1 (en) | 1998-02-18 | 2003-04-22 | Genome Therapeutics Corporation | Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics |
| DE19827457C1 (de) | 1998-06-19 | 2000-03-02 | Medigene Ag | Strukturprotein von AAV, seine Herstellung und Verwendung |
| WO2000015821A1 (en) | 1998-09-11 | 2000-03-23 | The Regents Of The University Of California | Recombinant adenovirus for tissue specific expression in heart |
| ES2340230T3 (es) | 1998-11-10 | 2010-05-31 | University Of North Carolina At Chapel Hill | Vectores viricos y sus procedimientos de preparacion y administracion. |
| 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 |
| US6498244B1 (en) | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
| US7314912B1 (en) | 1999-06-21 | 2008-01-01 | Medigene Aktiengesellschaft | AAv scleroprotein, production and use thereof |
| DE19933288A1 (de) | 1999-07-15 | 2001-01-18 | Medigene Ag | Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung |
| DE19933719A1 (de) | 1999-07-19 | 2001-01-25 | Medigene Ag | Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung |
| US8232081B2 (en) | 1999-09-21 | 2012-07-31 | Basf Se | Methods and microorganisms for production of panto-compounds |
| WO2002081628A2 (en) | 2001-04-05 | 2002-10-17 | Ribozyme Pharmaceuticals, Incorporated | Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| IL140110A0 (en) | 2000-12-05 | 2002-02-10 | Applied Research Systems | Improvement of homogeneity and secretion of recombinant proteins in mammalian systems |
| US7749492B2 (en) | 2001-01-05 | 2010-07-06 | Nationwide Children's Hospital, Inc. | AAV vectors and methods |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US7647184B2 (en) | 2001-08-27 | 2010-01-12 | Hanall Pharmaceuticals, Co. Ltd | High throughput directed evolution by rational mutagenesis |
| US20030129203A1 (en) | 2001-08-27 | 2003-07-10 | Nautilus Biotech S.A. | Mutant recombinant adeno-associated viruses |
| WO2003042361A2 (en) | 2001-11-09 | 2003-05-22 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| NZ532635A (en) | 2001-11-13 | 2007-05-31 | Univ Pennsylvania | A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method |
| US20050106558A1 (en) | 2001-12-21 | 2005-05-19 | Luca Perabo | Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism |
| AU2003221733A1 (en) | 2002-04-17 | 2003-11-03 | University Of Florida Research Foundation, Inc. | Improved raav vectors |
| AU2003223775A1 (en) | 2002-04-30 | 2003-11-17 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| AU2003237159A1 (en) | 2002-04-30 | 2003-11-17 | University Of Florida | Treatment for phenylketonuria |
| US7254489B2 (en) * | 2002-05-31 | 2007-08-07 | Microsoft Corporation | Systems, methods and apparatus for reconstructing phylogentic trees |
| AU2003265855A1 (en) | 2002-08-28 | 2004-03-19 | University Of Florida | Modified aav |
| WO2004083441A2 (en) | 2003-03-19 | 2004-09-30 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Random peptide library displayed on aav vectors |
| WO2004108922A2 (en) | 2003-04-25 | 2004-12-16 | The Trustees Of The University Of Pennsylvania | Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| SI3235827T1 (sl) | 2003-06-19 | 2021-05-31 | Genzyme Corporation | AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen |
| KR20060070491A (ko) | 2003-06-23 | 2006-06-23 | 에이 앤드 지 파마슈티컬즈, 인코포레이티드 | 항종양 요법에 대한 종양 세포의 감수성 회복 및 세포 사멸유도를 위한 조성물 및 방법 |
| GB0314856D0 (en) | 2003-06-25 | 2003-07-30 | Unitargeting Res As | Protein expression system |
| US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
| US8106180B2 (en) | 2003-08-07 | 2012-01-31 | Whitehead Institute For Biomedical Research | Methods and products for expression of micro RNAs |
| US20050142581A1 (en) | 2003-09-04 | 2005-06-30 | Griffey Richard H. | Microrna as ligands and target molecules |
| WO2005030932A2 (en) | 2003-09-24 | 2005-04-07 | The Board Of Trustees Of The Leland Stanford Junior University | Igf-1 instructs multipotent adult cns neural stem cells to an oligodendroglial lineage |
| CN1856576B (zh) * | 2003-09-30 | 2011-05-04 | 宾夕法尼亚州立大学托管会 | 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用 |
| US20050266552A1 (en) | 2003-12-05 | 2005-12-01 | Doench John G | Reagents and methods for identification of RNAi pathway genes and chemical modulators of RNAi |
| KR100614827B1 (ko) | 2004-05-06 | 2006-08-25 | 재단법인서울대학교산학협력재단 | 양방향 은닉 마코프 모델을 이용한 완숙한마이크로알엔에이 위치예측방법 및 이를 구현하기 위한컴퓨터 프로그램을 기록한 저장매체 |
| US7427396B2 (en) | 2004-06-03 | 2008-09-23 | Genzyme Corporation | AAV vectors for gene delivery to the lung |
| EP1828390B1 (en) | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| US20100172871A1 (en) | 2005-02-17 | 2010-07-08 | Flannery John G | Muller Cell Specific Gene Therapy |
| WO2006110689A2 (en) | 2005-04-07 | 2006-10-19 | The Trustees Of The University Of Pennsylvania | Method of increasing the function of an aav vector |
| WO2006128063A2 (en) | 2005-05-25 | 2006-11-30 | Irm Llc | Methods and compositions for inhibiting glioma growth |
| US8119766B2 (en) | 2005-06-02 | 2012-02-21 | University Of Iowa Research Foundation | Increasing functional glycosylation of alpha-dystroglycan in the treatment of muscle degeneration |
| US7867484B2 (en) | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| WO2007127803A2 (en) | 2006-04-25 | 2007-11-08 | The Regents Of The University Of California | Administration of growth factors for the treatment of cns disorders |
| EP2019588A4 (en) | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| ES2385679T3 (es) | 2006-08-24 | 2012-07-30 | Virovek, Inc. | Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos |
| CN1966082B (zh) | 2006-11-03 | 2010-06-30 | 许瑞安 | 一种防治结直肠癌的基因药物及其制备方法和用途 |
| 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 |
| BRPI0810172A2 (pt) | 2007-04-13 | 2014-10-14 | Catalyst Biosciences Inc | Polipeptídeos de fator vii modificado e seus usos |
| EP1985708B1 (en) | 2007-04-27 | 2015-04-15 | Universität Rostock | Selective targeting of viruses to neural precursor cells |
| US7951785B2 (en) | 2007-09-21 | 2011-05-31 | California Institute Of Technology | NFIA in glial fate determination, glioma therapy and astrocytoma treatment |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| EP2910637A1 (en) | 2008-05-20 | 2015-08-26 | University of Florida Research Foundation, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| WO2009151539A1 (en) | 2008-05-24 | 2009-12-17 | Sirnaomics, Inc. | COMPOSITIONS AND METHODS USING siRNA MOLECULES FOR TREATMENT OF GLIOMAS |
| WO2009154452A1 (en) | 2008-06-17 | 2009-12-23 | Amsterdam Molecular Therapeutics B.V. | Parvoviral capsid with incorporated Gly-Ala repeat region |
| 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 |
| CN101532024A (zh) | 2009-04-30 | 2009-09-16 | 许瑞安 | 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| US20110059114A1 (en) | 2009-08-05 | 2011-03-10 | Duke University | Compositions and Methods for the Treatment of Radioresistant Glioma Stem Cells |
| WO2011117258A2 (en) | 2010-03-22 | 2011-09-29 | Association Institut De Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| WO2011134023A1 (en) | 2010-04-30 | 2011-11-03 | The University Of Queensland | Inhibition of glioma |
| US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| ES2857773T5 (es) | 2011-08-24 | 2024-06-04 | Univ Leland Stanford Junior | Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos |
| WO2013155330A1 (en) | 2012-04-11 | 2013-10-17 | University Of Massachusetts | Compositions and methods for characterizing and treating muscular dystrophy |
| WO2013170078A1 (en) | 2012-05-09 | 2013-11-14 | Oregon Health & Science University | Adeno associated virus plasmids and vectors |
| TWI775096B (zh) | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| EP2940131B1 (en) | 2012-12-25 | 2019-02-20 | Takara Bio Inc. | Aav variant |
| US11685935B2 (en) | 2013-05-29 | 2023-06-27 | Cellectis | Compact scaffold of Cas9 in the type II CRISPR system |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| WO2015048534A1 (en) | 2013-09-26 | 2015-04-02 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| HUE052676T2 (hu) * | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| IL248102B (en) | 2014-05-02 | 2022-07-01 | Genzyme Corp | aav vectors for gene therapy of the central nervous system and retina |
| WO2015191693A2 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| MY187898A (en) | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| PL3265568T3 (pl) | 2015-03-06 | 2020-11-30 | Massachusetts Eye & Ear Infirmary | Terapie augmentacji genów w przypadku wrodzonego zwyrodnienia siatkówki spowodowanego mutacjami genu prpf31 |
| US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| FI3445773T3 (fi) | 2016-05-13 | 2023-03-30 | 4D Molecular Therapeutics Inc | Adenoassosioituneen viruksen kapsidimuunnoksia ja niiden käyttömenetelmiä |
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| EP3277819B1 (en) | 2021-03-03 |
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| CA2979065A1 (en) | 2016-09-29 |
| US10883117B2 (en) | 2021-01-05 |
| AU2016235163A1 (en) | 2017-10-05 |
| CN107532177A (zh) | 2018-01-02 |
| AU2016235163B2 (en) | 2022-03-24 |
| HK1246355A1 (zh) | 2018-09-07 |
| US20210147876A1 (en) | 2021-05-20 |
| JP2018514190A (ja) | 2018-06-07 |
| EP3277819A4 (en) | 2018-09-05 |
| MX2017012097A (es) | 2018-06-15 |
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