MX388331B - Variantes de virus adenoasociado y métodos de uso de estas - Google Patents

Variantes de virus adenoasociado y métodos de uso de estas

Info

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
Application number
MX2017012097A
Other languages
English (en)
Other versions
MX2017012097A (es
Inventor
David S Ojala
David V Schaffer
Ian H Holmes
John Weinstein
Jorge Santiago-Ortiz
Oscar Westesson
Original Assignee
Univ California
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ California filed Critical Univ California
Publication of MX2017012097A publication Critical patent/MX2017012097A/es
Publication of MX388331B publication Critical patent/MX388331B/es

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/74Vectors or expression systems specially adapted for prokaryotic hosts other than E. coli, e.g. Lactobacillus, Micromonospora
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (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.
MX2017012097A 2015-03-24 2016-03-23 Variantes de virus adenoasociado y métodos de uso de estas MX388331B (es)

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 (82)

* Cited by examiner, † Cited by third party
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
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
RU2716991C2 (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
WO2016077687A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
KR20230145206A (ko) 2014-11-14 2023-10-17 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
US12188037B2 (en) 2015-10-22 2025-01-07 University Of Florida Research Foundation, Incorporated Synthetic combinatorial AAV3 capsid library
EP3384035A4 (en) 2015-12-02 2019-08-07 Voyager Therapeutics, Inc. ASSAYS FOR THE DETECTION OF AAV-NEUTRALIZING ANTIBODIES
US11299751B2 (en) 2016-04-29 2022-04-12 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
KR102427379B1 (ko) 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
KR20240056729A (ko) 2016-05-18 2024-04-30 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
AU2017302013B2 (en) 2016-07-29 2022-05-26 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
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
JP2020518258A (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. 筋萎縮性側索硬化症(als)治療組成物および方法
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
EP3625344A1 (en) 2017-05-15 2020-03-25 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
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
CA3070087A1 (en) 2017-07-17 2019-01-24 Voyager Therapeutics, Inc. Trajectory array guide system
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
RU2770922C2 (ru) 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения
AU2018338728B2 (en) 2017-09-29 2025-01-02 Centre National De La Recherche Scientifique (Cnrs) Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery
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
EP3697908A1 (en) 2017-10-16 2020-08-26 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
EP3759218A4 (en) * 2018-02-28 2021-12-08 The University of North Carolina at Chapel Hill PROCEDURES AND COMPOSITIONS FOR ANTIBODY ELIMINATING VIRUS VECTORS
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, 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
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
AU2019287468B2 (en) * 2018-06-12 2024-10-10 The University Of North Carolina At Chapel Hill Synthetic liver-tropic adeno-associated virus capsids and uses thereof
JP7450945B2 (ja) 2018-08-30 2024-03-18 テナヤ セラピューティクス, インコーポレイテッド ミオカルディンおよびascl1を用いた心細胞リプログラミング
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
EP3856762A1 (en) 2018-09-28 2021-08-04 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
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
EP3947700A4 (en) 2019-04-01 2023-01-04 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
WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
EP3997226A1 (en) 2019-07-11 2022-05-18 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
CA3146702A1 (en) * 2019-08-09 2021-02-18 Sergei Zolotukhin Aav capsid variants for targeting human glioblastoma cells
CN115087738A (zh) * 2019-08-29 2022-09-20 小利兰·斯坦福大学托管委员会 用于穿越人血脑屏障的腺相关病毒载体
EP4028064A1 (en) * 2019-09-12 2022-07-20 The Broad Institute, Inc. Engineered adeno-associated virus capsids
CN118221785A (zh) * 2019-10-16 2024-06-21 上海药明康德新药开发有限公司 新的aav变体
AU2020368539A1 (en) 2019-10-16 2022-04-28 Massachusetts Institute Of Technology Engineered muscle targeting compositions
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ,インコーポレイテッド ニーマン・ピック病c型の治療のためのアデノ随伴ウイルスベクター
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
EP4103214B1 (en) 2020-02-13 2026-04-29 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
EP4103211A4 (en) 2020-04-27 2023-08-23 4D Molecular Therapeutics Inc. CODON-OPTIMISED GLA GENES AND THEIR USES
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
US12031147B2 (en) 2020-05-29 2024-07-09 The Regents Of The University Of California 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
AU2021328475A1 (en) 2020-08-19 2023-03-16 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
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
US11781156B2 (en) 2020-10-09 2023-10-10 Tenaya Therapeutics, Inc. Plakophillin-2 gene therapy methods and compositions
BR112023006305A2 (pt) 2020-10-09 2023-05-09 Tenaya Therapeutics Inc Métodos e composições de terapia gênica com placofilina-2
WO2022087104A1 (en) * 2020-10-21 2022-04-28 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
CN116789739B (zh) * 2022-12-08 2024-01-26 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)

* Cited by examiner, † Cited by third party
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
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
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
EP0927044A4 (en) 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET VIRAL VECTORS
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The 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
AU5819599A (en) 1998-09-11 2000-04-03 Regents Of The University Of California, The Recombinant adenovirus for tissue specific expression in heart
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
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
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
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
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
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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
EP1456383B1 (en) 2001-12-21 2014-03-12 Medigene AG A 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
US20040142416A1 (en) 2002-04-30 2004-07-22 Laipis Philip J. Treatment for phenylketonuria
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
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
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
SI1633772T1 (sl) 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US7368428B2 (en) 2003-06-23 2008-05-06 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
CA2539947A1 (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
EP2345731B1 (en) * 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
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
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
ES2525067T3 (es) 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
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
DK2019683T4 (da) 2006-04-25 2022-08-29 Univ California Indgivelse af vækstfaktorer til behandling af CNS-lidelser
CN101484005A (zh) 2006-05-04 2009-07-15 韦恩州立大学 通过向体内递送视紫红质核酸恢复视觉响应
WO2008024998A2 (en) 2006-08-24 2008-02-28 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
CA2683443A1 (en) 2007-04-13 2008-10-23 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
WO2010011404A2 (en) 2008-05-20 2010-01-28 Eos Neuroscience, 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
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts 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
US9546112B2 (en) 2010-03-22 2017-01-17 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
CN105755044A (zh) 2011-04-22 2016-07-13 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
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
TWI698240B (zh) 2012-05-15 2020-07-11 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)
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US9938541B2 (en) 2012-12-25 2018-04-10 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
EP3003391B1 (en) 2013-05-31 2021-09-22 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
CN106232618A (zh) 2013-10-11 2016-12-14 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
EP3155116A4 (en) 2014-06-10 2017-12-27 Massachusetts Institute Of Technology Method for gene editing
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3748007B1 (en) * 2015-03-06 2024-06-26 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
US20190000940A1 (en) 2015-07-31 2019-01-03 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
US11364308B2 (en) 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof

Also Published As

Publication number Publication date
JP6836999B2 (ja) 2021-03-03
MX2017012097A (es) 2018-06-15
JP2018514190A (ja) 2018-06-07
WO2016154344A1 (en) 2016-09-29
EP3277819A1 (en) 2018-02-07
CA2979065A1 (en) 2016-09-29
ES2860712T3 (es) 2021-10-05
US20180066285A1 (en) 2018-03-08
US20210147876A1 (en) 2021-05-20
AU2016235163A1 (en) 2017-10-05
EP3277819A4 (en) 2018-09-05
HK1246355A1 (zh) 2018-09-07
EP3277819B1 (en) 2021-03-03
US10883117B2 (en) 2021-01-05
CN107532177A (zh) 2018-01-02
AU2016235163B2 (en) 2022-03-24

Similar Documents

Publication Publication Date Title
MX388331B (es) Variantes de virus adenoasociado y métodos de uso de estas
MX2020003176A (es) Variantes de capsides de virus adenoasociados y metodos de uso de estas.
MX2023009627A (es) Capsides variantes de virus adenoasociados y metodos de uso de las mismas.
MX2020005451A (es) Capsides variantes de virus adenoasociados y uso para inhibir la angiogenesis.
EA202091712A1 (ru) МОДИФИЦИРОВАННЫЙ КАПСИДНЫЙ БЕЛОК rAAV ДЛЯ ГЕННОЙ ТЕРАПИИ
MX2019001938A (es) Metodos para detectar aav.
WO2020028751A3 (en) Aav variants with enhanced tropism
CL2023000264A1 (es) Nuevos vectores virales adeno-asociados dirigidos al hígado
WO2017066764A3 (en) Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
MX2016010649A (es) Vector de virus adeno-asociado.
WO2017058892A3 (en) Methods and compositions for antibody-evading virus vectors
MX2022006652A (es) Proteinas de capside modificadas para un suministro mejorado de vectores de parvovirus.
MX2018006840A (es) Focalizacion de peptidos para dirigir virus adenoasociados.
MX2022013963A (es) Composiciones de virus adenoasociados compatibles entre especies y metodos de uso de las mismas.
BR112019002904A2 (pt) métodos e composições para transferência gênica direcionada
EA201891415A1 (ru) Антигены вируса иммунодефицита человека, векторы, композиции и способы их применения
PE20150163A1 (es) Composicion y metodos para una transferencia de genes altamente eficiente usando variantes capside aav
EA201201025A1 (ru) Молекулы нуклеиновых кислот гриппа и вакцины, изготовленные из них
MX390966B (es) Vacunas contra el dengue
MX2019007924A (es) Vacunas contra la influenza.
MX2018000023A (es) Particulas semejantes a virus recombinantes que utilizan la proteina gag del virus de inmunodeficiencia bovina.
EA201990955A1 (ru) Разработка капсидов aav
MY185444A (en) Enterovirus 71 antiviral peptides
MY186651A (en) Adeno-associated virus variant capsids and methods of use thereof
EA202191862A2 (ru) Антигены вируса иммунодефицита человека, векторы, композиции и способы их применения