MX388331B - Variantes de virus adenoasociado y métodos de uso de estas - Google Patents
Variantes de virus adenoasociado y métodos de uso de estasInfo
- Publication number
- MX388331B MX388331B MX2017012097A MX2017012097A MX388331B MX 388331 B MX388331 B MX 388331B MX 2017012097 A MX2017012097 A MX 2017012097A MX 2017012097 A MX2017012097 A MX 2017012097A MX 388331 B MX388331 B MX 388331B
- Authority
- MX
- Mexico
- Prior art keywords
- present disclosure
- capsid protein
- aav capsid
- raav
- methods
- Prior art date
Links
- 241000700605 Viruses Species 0.000 title 1
- 241000702421 Dependoparvovirus Species 0.000 abstract 6
- 108090000565 Capsid Proteins Proteins 0.000 abstract 5
- 102100023321 Ceruloplasmin Human genes 0.000 abstract 5
- 210000002845 virion Anatomy 0.000 abstract 3
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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- 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
- C12N15/861—Adenoviral vectors
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
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- 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
- C12N15/74—Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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- 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
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- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Physical Education & Sports Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La presente divulgación provee viriones de virus adenoasociado recombinante (rAAV) que comprenden una variante de proteína de cápside de AAV, por ejemplo, una proteína de cápside de AAV derivada de una secuencia de aminoácidos de proteína de cápside de AAV ancestral. Un virión de rAAV de la presente divulgación puede exhibir mayor capacidad de infección por una célula diana. La presente divulgación también provee métodos de administración de un producto génico a una célula diana en un individuo mediante la administración de un rAAV de la presente divulgación a un individuo. Además, la presente divulgación provee métodos para regenerar viriones de rAAV con una variante de proteína de cápside de AAV que deriva de una secuencia de aminoácidos de proteína de cápside de AAV ancestral.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562137580P | 2015-03-24 | 2015-03-24 | |
| PCT/US2016/023822 WO2016154344A1 (en) | 2015-03-24 | 2016-03-23 | Adeno-associated virus variants and methods of use thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2017012097A MX2017012097A (es) | 2018-06-15 |
| MX388331B true MX388331B (es) | 2025-03-19 |
Family
ID=56978687
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017012097A MX388331B (es) | 2015-03-24 | 2016-03-23 | Variantes de virus adenoasociado y métodos de uso de estas |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US10883117B2 (es) |
| EP (1) | EP3277819B1 (es) |
| JP (1) | JP6836999B2 (es) |
| CN (1) | CN107532177A (es) |
| AU (1) | AU2016235163B2 (es) |
| CA (1) | CA2979065A1 (es) |
| ES (1) | ES2860712T3 (es) |
| MX (1) | MX388331B (es) |
| WO (1) | WO2016154344A1 (es) |
Families Citing this family (83)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| EP3800191B1 (en) | 2014-03-17 | 2025-08-20 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| IL292999A (en) | 2014-11-14 | 2022-07-01 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| CN112410338A (zh) | 2014-11-14 | 2021-02-26 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016094783A1 (en) | 2014-12-12 | 2016-06-16 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| 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 |
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| US12188037B2 (en) | 2015-10-22 | 2025-01-07 | University Of Florida Research Foundation, Incorporated | Synthetic combinatorial AAV3 capsid library |
| WO2017096162A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809699XA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| KR20220108216A (ko) | 2016-05-18 | 2022-08-02 | 보이저 테라퓨틱스, 인크. | 헌팅톤 질환을 치료하기 위한 조성물 및 방법 |
| EP4700118A3 (en) | 2016-07-29 | 2026-04-22 | The Regents of the University of California | Adeno-associated virus virions with varint capsid and methods of use thereof |
| CN110650673B (zh) | 2016-08-30 | 2024-04-09 | 加利福尼亚大学董事会 | 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统 |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| CA3114549A1 (en) | 2017-02-21 | 2018-08-30 | University Of Florida Research Foundation, Incorporated | Modified aav capsid proteins and uses thereof |
| SG11201909870SA (en) | 2017-05-05 | 2019-11-28 | Voyager Therapeutics Inc | Compositions and methods of treating 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 |
| EP3645052A4 (en) | 2017-06-30 | 2021-07-28 | Regents of the University of California | ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE |
| 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 |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| PE20201264A1 (es) | 2017-09-20 | 2020-11-19 | 4D Molecular Therapeutics Inc | Variantes de capsides de virus adenoasociados y metodos de uso de estas |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| CA3092107A1 (en) * | 2018-02-28 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| WO2019195423A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
| WO2019195444A1 (en) | 2018-04-03 | 2019-10-10 | 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 |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| US12410441B2 (en) * | 2018-06-12 | 2025-09-09 | The University Of North Carolina At Chapel Hill | Synthetic liver-tropic adeno-associated virus capsids and uses thereof |
| WO2020047467A2 (en) | 2018-08-30 | 2020-03-05 | Tenaya Therapeutics, Inc. | Cardiac cell reprogramming with myocardin and ascl1 |
| SG11202103102WA (en) | 2018-09-26 | 2021-04-29 | California Inst Of Techn | Adeno-associated virus compositions for targeted gene therapy |
| CN113383010B (zh) | 2018-09-28 | 2025-09-09 | 沃雅戈治疗公司 | 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法 |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| WO2020205889A1 (en) | 2019-04-01 | 2020-10-08 | Tenaya Therapeutics, Inc. | Adeno-associated virus with engineered capsid |
| 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 |
| JP2022536338A (ja) * | 2019-06-10 | 2022-08-15 | ホモロジー・メディシンズ・インコーポレイテッド | Ａｒｓａ遺伝子移入のためのアデノ随伴ウイルス組成物およびその使用方法 |
| 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 |
| WO2021030225A2 (en) * | 2019-08-09 | 2021-02-18 | 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 |
| JP2022547570A (ja) * | 2019-09-12 | 2022-11-14 | ザ・ブロード・インスティテュート・インコーポレイテッド | 操作済みのアデノ随伴ウイルスキャプシド |
| US20220403414A1 (en) * | 2019-10-16 | 2022-12-22 | Wuxi Apptec (Shanghai) Co., Ltd. | Novel aav variant |
| CA3153902A1 (en) | 2019-10-16 | 2021-04-22 | Pardis SABETI | Engineered muscle targeting compositions |
| AU2020367532A1 (en) | 2019-10-17 | 2022-05-12 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick disease type C |
| KR20220083708A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | Aav 전달 카세트 |
| 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 |
| MX2022009883A (es) | 2020-02-13 | 2022-10-03 | Tenaya Therapeutics Inc | Vectores de genoterapia para tratar enfermedades cardíacas. |
| WO2021163357A2 (en) | 2020-02-13 | 2021-08-19 | Tenaya Therapeutics, Inc. | Gene therapy vectors for treating heart disease |
| CA3173567A1 (en) | 2020-03-02 | 2021-09-10 | Tenaya Therapeutics, Inc. | Gene vector control by cardiomyocyte-expressed micrornas |
| CN115515613A (zh) | 2020-04-27 | 2022-12-23 | 4D分子治疗有限公司 | 密码子优化的gla基因及其用途 |
| CN115551878A (zh) * | 2020-05-01 | 2022-12-30 | 波士顿学院董事会 | 用于增强的基因疗法的腺相关病毒(aav)的受控修饰 |
| 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 |
| CA3179597A1 (en) | 2020-05-29 | 2021-12-02 | John G. Flannery | Adeno-associated virus virions with variant capsids and methods of use thereof |
| US20230265453A1 (en) | 2020-07-08 | 2023-08-24 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
| US20230159949A1 (en) | 2020-07-22 | 2023-05-25 | The Broad Institute, Inc. | Engineered muscle targeting compositions |
| MX2023002016A (es) | 2020-08-19 | 2023-06-26 | Sarepta Therapeutics Inc | Vectores de virus adenoasociados para el tratamiento del sindrome de rett. |
| 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 |
| CN113754727B (zh) * | 2020-09-30 | 2022-07-12 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| JP7415042B2 (ja) | 2020-10-09 | 2024-01-16 | テナヤ セラピューティクス， インコーポレイテッド | プラコフィリン２遺伝子治療の方法および組成物 |
| US11781156B2 (en) | 2020-10-09 | 2023-10-10 | Tenaya Therapeutics, Inc. | Plakophillin-2 gene therapy methods and compositions |
| WO2022087104A1 (en) * | 2020-10-21 | 2022-04-28 | Chan Zuckerberg Biohub, Inc. | Adeno-associated virus virions and methods of use thereof |
| KR20230089571A (ko) * | 2020-11-16 | 2023-06-20 | 베이징 솔로바이오 진테크놀로지 컴퍼니 리미티드 | 간 친화성이 강화된 재조합 아데노-관련 바이러스 및 이의 용도 |
| 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 |
| CN118510905A (zh) * | 2021-12-06 | 2024-08-16 | 学校法人顺天堂 | 改变型腺伴随病毒载体 |
| WO2023108507A1 (en) * | 2021-12-15 | 2023-06-22 | National Institute Of Biological Sciences, Beijing | Recombinant aav vectors and use thereof |
| TW202421787A (zh) | 2022-09-06 | 2024-06-01 | 美商特納亞治療股份有限公司 | 保護心臟之心臟病療法 |
| 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 |
| WO2024238684A1 (en) * | 2023-05-15 | 2024-11-21 | 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 |
| AU8200191A (en) | 1990-07-09 | 1992-02-04 | United States of America, as represented by the Secretary, U.S. Department of Commerce, The | 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 |
| WO1993003769A1 (en) | 1991-08-20 | 1993-03-04 | THE UNITED STATES OF AMERICA, represented by THE SECRETARY, DEPARTEMENT OF HEALTH AND HUMAN SERVICES | Adenovirus mediated transfer of genes to the gastrointestinal tract |
| GB9206016D0 (en) | 1992-03-19 | 1992-04-29 | Sandoz Ltd | Improvements in or relating to organic compounds |
| WO1995005864A1 (en) | 1993-08-27 | 1995-03-02 | Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services | 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 |
| AU2678097A (en) | 1996-04-16 | 1997-11-07 | 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 |
| EP1030861A4 (en) | 1997-10-31 | 2001-09-05 | Maxygen Inc | MODIFICATION OF VIRUSTROPISMIUS AND THE ECONOMIC SPECTRUM BY VIRAL GENOM MIXING |
| 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 |
| JP2002525064A (ja) | 1998-09-11 | 2002-08-13 | ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア | 心臓で組織特異的に発現する新規組換えアデノウイルス |
| ATE454445T1 (de) | 1998-11-10 | 2010-01-15 | Univ North Carolina | Virusvektoren und verfahren für ihre herstellung und verabreichung. |
| 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 |
| US20070026394A1 (en) | 2000-02-11 | 2007-02-01 | Lawrence Blatt | 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 |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| US20030129203A1 (en) | 2001-08-27 | 2003-07-10 | Nautilus Biotech S.A. | Mutant recombinant adeno-associated viruses |
| US7647184B2 (en) | 2001-08-27 | 2010-01-12 | Hanall Pharmaceuticals, Co. Ltd | High throughput directed evolution by rational mutagenesis |
| AU2002360355B2 (en) | 2001-11-09 | 2005-07-07 | Government Of The United States Of America, Department Of Health And Human Services | Production of adeno-associated virus in insect cells |
| SG10202108118RA (en) | 2001-11-13 | 2021-08-30 | Univ Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| ES2467156T3 (es) | 2001-12-21 | 2014-06-12 | Medigene Ag | Una biblioteca de genes estructurales modificados o de partículas modificadas de cápside útiles para la identificación de clones virales con tropismo celular deseado |
| US20060292117A1 (en) | 2002-04-17 | 2006-12-28 | Loiler Scott A | 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 |
| US20040142416A1 (en) | 2002-04-30 | 2004-07-22 | Laipis Philip J. | Treatment for phenylketonuria |
| US7254489B2 (en) | 2002-05-31 | 2007-08-07 | Microsoft Corporation | Systems, methods and apparatus for reconstructing phylogentic trees |
| US7220577B2 (en) | 2002-08-28 | 2007-05-22 | University Of Florida Research Foundation, Inc. | Modified AAV |
| US20070172460A1 (en) | 2003-03-19 | 2007-07-26 | Jurgen Kleinschmidt | 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 |
| PL2357189T3 (pl) | 2003-06-19 | 2017-08-31 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie |
| CA2530582C (en) | 2003-06-23 | 2014-09-09 | A&G Pharmaceutical, Inc. | Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis |
| 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 |
| US20050148069A1 (en) | 2003-09-24 | 2005-07-07 | Gage Fred H. | IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage |
| HUE054805T2 (hu) * | 2003-09-30 | 2021-09-28 | Univ Pennsylvania | Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik |
| WO2005056761A2 (en) | 2003-12-05 | 2005-06-23 | Massachusetts Institute Of Technology | 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 |
| AU2005316476A1 (en) | 2004-12-15 | 2006-06-22 | University Of Florida Research Foundation, Inc. | Chimeric vectors |
| WO2006101634A1 (en) | 2005-02-17 | 2006-09-28 | The Regents Of The University Of California | Müller cell specific gene therapy |
| EP3085389A1 (en) | 2005-04-07 | 2016-10-26 | 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 |
| WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
| PL2019683T5 (pl) | 2006-04-25 | 2022-12-05 | The Regents Of The University Of California | Podawanie czynników wzrostu do leczenia zaburzeń OUN |
| WO2007131180A2 (en) | 2006-05-04 | 2007-11-15 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| EP2061891B1 (en) | 2006-08-24 | 2012-04-11 | Virovek, Inc. | Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor |
| 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 |
| EP2481797A1 (en) | 2007-04-13 | 2012-08-01 | Catalyst Biosciences, Inc. | Modified factor VII polypeptides and uses thereof |
| 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 |
| WO2009137006A2 (en) | 2008-04-30 | 2009-11-12 | The University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| EP2315833B8 (en) | 2008-05-20 | 2015-05-27 | Eos Neuroscience, Inc. | Vectors for delivery of light-sensitive proteins and methods of use |
| US8541568B2 (en) | 2008-05-24 | 2013-09-24 | Hai Yan | Compositions and methods using siRNA molecules for treatment of gliomas |
| EP2297185A1 (en) | 2008-06-17 | 2011-03-23 | Amsterdam Molecular Therapeutics (AMT) B.V. | Parvoviral capsid with incorporated gly-ala repeat region |
| US8889641B2 (en) | 2009-02-11 | 2014-11-18 | 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 |
| CN107012171B (zh) * | 2011-04-22 | 2021-07-13 | 加利福尼亚大学董事会 | 具有变异衣壳的腺相关病毒病毒体及其使用方法 |
| EP3147295B2 (en) | 2011-08-24 | 2023-11-22 | The Board of Trustees of the Leland Stanford Junior University | New avv capsid proteins for nucleic acid transfer |
| WO2013155330A1 (en) | 2012-04-11 | 2013-10-17 | University Of Massachusetts | Compositions and methods for characterizing and treating muscular dystrophy |
| EP3470523A1 (en) | 2012-05-09 | 2019-04-17 | 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) |
| LT3401400T (lt) | 2012-05-25 | 2019-06-10 | The Regents Of The University Of California | Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui |
| EP2940131B1 (en) | 2012-12-25 | 2019-02-20 | Takara Bio Inc. | Aav variant |
| EP3004339B1 (en) | 2013-05-29 | 2021-07-07 | Cellectis | New compact scaffold of cas9 in the type ii crispr system |
| CN105247044B (zh) | 2013-05-31 | 2021-05-07 | 加利福尼亚大学董事会 | 腺相关病毒变体及其使用方法 |
| AU2014324717B2 (en) | 2013-09-26 | 2020-10-08 | University Of Florida Research Foundation, Inc. | Synthetic combinatorial AAV capsid library for targeted gene therapy |
| NZ718926A (en) * | 2013-10-11 | 2021-12-24 | Massachusetts Eye & Ear Infirmary | Methods of predicting ancestral virus sequences and uses thereof |
| EP3137497B2 (en) | 2014-05-02 | 2025-03-05 | Genzyme Corporation | Aav vectors for retinal and cns gene therapy |
| WO2015191693A2 (en) | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| JP7095994B2 (ja) | 2015-03-02 | 2022-07-05 | アドヴェラム バイオテクノロジーズ， インコーポレイテッド | ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 |
| PT3265568T (pt) | 2015-03-06 | 2020-08-20 | Massachusetts Eye & Ear Infirmary | Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31 |
| US20190000940A1 (en) | 2015-07-31 | 2019-01-03 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of aadc deficiency |
| WO2017197355A2 (en) * | 2016-05-13 | 2017-11-16 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
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| US10883117B2 (en) | 2021-01-05 |
| AU2016235163B2 (en) | 2022-03-24 |
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| US20180066285A1 (en) | 2018-03-08 |
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| CN107532177A (zh) | 2018-01-02 |
| US20210147876A1 (en) | 2021-05-20 |
| EP3277819B1 (en) | 2021-03-03 |
| HK1246355A1 (zh) | 2018-09-07 |
| EP3277819A1 (en) | 2018-02-07 |
| WO2016154344A1 (en) | 2016-09-29 |
| JP2018514190A (ja) | 2018-06-07 |
| AU2016235163A1 (en) | 2017-10-05 |
| ES2860712T3 (es) | 2021-10-05 |
| EP3277819A4 (en) | 2018-09-05 |
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