JP2023126919A5 - - Google Patents

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Publication number
JP2023126919A5
JP2023126919A5 JP2023110850A JP2023110850A JP2023126919A5 JP 2023126919 A5 JP2023126919 A5 JP 2023126919A5 JP 2023110850 A JP2023110850 A JP 2023110850A JP 2023110850 A JP2023110850 A JP 2023110850A JP 2023126919 A5 JP2023126919 A5 JP 2023126919A5
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JP
Japan
Prior art keywords
capsid protein
virion
aav2
amino acids
pharmaceutical composition
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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.)
Pending
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JP2023110850A
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English (en)
Japanese (ja)
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JP2023126919A (ja
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Priority claimed from PCT/US2018/040115 external-priority patent/WO2019006182A1/en
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Publication of JP2023126919A publication Critical patent/JP2023126919A/ja
Publication of JP2023126919A5 publication Critical patent/JP2023126919A5/ja
Pending legal-status Critical Current

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JP2023110850A 2017-06-30 2023-07-05 バリアントカ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 Pending JP2023126919A (ja)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762527871P 2017-06-30 2017-06-30
US62/527,871 2017-06-30
US201762535042P 2017-07-20 2017-07-20
US62/535,042 2017-07-20
PCT/US2018/040115 WO2019006182A1 (en) 2017-06-30 2018-06-28 VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
JP2019568621A JP7744659B2 (ja) 2017-06-30 2018-06-28 バリアントカ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

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JP2019568621A Division JP7744659B2 (ja) 2017-06-30 2018-06-28 バリアントカ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

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JP2023126919A JP2023126919A (ja) 2023-09-12
JP2023126919A5 true JP2023126919A5 (https=) 2025-11-26

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JP2019568621A Active JP7744659B2 (ja) 2017-06-30 2018-06-28 バリアントカ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
JP2023110850A Pending JP2023126919A (ja) 2017-06-30 2023-07-05 バリアントカ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

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JP2019568621A Active JP7744659B2 (ja) 2017-06-30 2018-06-28 バリアントカ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

Country Status (10)

Country Link
US (2) US20200121746A1 (https=)
EP (1) EP3645052A4 (https=)
JP (2) JP7744659B2 (https=)
KR (2) KR102766238B1 (https=)
CN (1) CN110461368A (https=)
AU (2) AU2018290954B2 (https=)
BR (1) BR112019019015A2 (https=)
CA (1) CA3053154A1 (https=)
MX (1) MX2019010910A (https=)
WO (1) WO2019006182A1 (https=)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
CN110709095B (zh) 2017-04-05 2024-05-03 马萨诸塞大学 小基因治疗
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
SG11202009854XA (en) * 2018-02-15 2020-11-27 Bjoerklund Tomas Modified viral capsids
US11821009B2 (en) 2018-05-15 2023-11-21 Cornell University Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
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
TW202102526A (zh) * 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
WO2020236815A1 (en) 2019-05-20 2020-11-26 University Of Massachusetts Minigene therapy
CN114729009A (zh) * 2019-10-31 2022-07-08 伯尔尼大学 用于眼部基因递送的aav载体变体
CN113025618B (zh) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
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
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
JP2024513949A (ja) * 2021-04-13 2024-03-27 カプシダ, インコーポレイテッド 脳内発現レベルが高いaav組成物
US20240299586A1 (en) * 2021-05-06 2024-09-12 The Regents Of The University Of California Novel aav serotypes derived from a library screen
KR20240095539A (ko) 2021-10-08 2024-06-25 디노 테라퓨틱스, 인코포레이티드 캡시드 변이체 및 이의 사용 방법
CN114195859B (zh) * 2021-12-10 2022-11-18 和元生物技术(上海)股份有限公司 一种适用于特异感染u251细胞的腺相关病毒突变体
WO2023108507A1 (en) * 2021-12-15 2023-06-22 National Institute Of Biological Sciences, Beijing Recombinant aav vectors and use thereof
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
US20250213722A1 (en) * 2022-03-30 2025-07-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells
IL316014A (en) * 2022-03-30 2024-11-01 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated virus vectors for delivering nucleic acids to retinal cells
EP4499847A4 (en) * 2022-03-30 2026-03-25 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
WO2024055020A2 (en) * 2022-09-09 2024-03-14 The Regents Of The University Of California Reprogramming tropism via displayed peptides tiling receptor-ligands
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
WO2024191778A1 (en) 2023-03-10 2024-09-19 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
CN121712809A (zh) * 2023-07-03 2026-03-20 Ucb生物制药有限责任公司 工程化的aav衣壳蛋白
AU2024332181A1 (en) 2023-08-31 2026-02-12 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof
TW202545971A (zh) 2024-02-08 2025-12-01 美商戴諾治療公司 殼體多肽及其使用方法
KR20260014488A (ko) * 2024-07-22 2026-01-30 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도
WO2026033140A1 (en) * 2024-08-09 2026-02-12 Revvity Gene Delivery Gmbh Variant aav capsid polypeptides targeting the eye
WO2026064442A2 (en) 2024-09-18 2026-03-26 Dyno Therapeutics, Inc. Capsid polypeptides and methods of use thereof

Family Cites Families (113)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
US6306829B1 (en) 1995-12-08 2001-10-23 Hybridon, Inc. Modified VEGF oligonucleotides for treatment of skin disorders
US5639872A (en) 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
US6440425B1 (en) 1995-05-01 2002-08-27 Aventis Pasteur Limited High molecular weight major outer membrane protein of moraxella
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
DE19961180C2 (de) 1999-12-18 2002-02-28 Daimler Chrysler Ag Dünnschichtwiderstand mit hohem Temperaturkoeffizienten als passives Halbleiterbauelement für integrierte Schaltungen und Herstellungsverfahren
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
US20030149235A1 (en) 2000-11-17 2003-08-07 Baker Andrew Howard Targeting peptides
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
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
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
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
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
US6846666B2 (en) 2002-03-21 2005-01-25 Council Of Scientific And Industrial Research Bacterial strain MTCC 5098 and a method of reducing total dissolved solids (TDS) from pulp and paper wastewater effluents using the said strain
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
AU2003223766A1 (en) 2002-04-30 2003-11-17 University Of North Carolina At Chapel Hill Secretion signal vectors
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
WO2003093479A1 (en) 2002-05-01 2003-11-13 University Of Florida Research Foundation, Inc. Improved raav expression systems and methods for enhancing transduction of mammalian neural cells
US7254489B2 (en) 2002-05-31 2007-08-07 Microsoft Corporation Systems, methods and apparatus for reconstructing phylogentic trees
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
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
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
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
US20050019927A1 (en) 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
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
ES2423060T3 (es) 2004-03-12 2013-09-17 Alnylam Pharmaceuticals, Inc. Agentes iRNA que tienen como diana al VEGF
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
EP1919497B1 (en) 2005-07-22 2020-02-12 The Board of Trustees of the Leland Stanford Junior University Light-activated cation channel and uses thereof
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
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
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
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
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
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
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
EP2547762B1 (en) 2010-03-17 2018-04-25 The Board of Trustees of the Leland Stanford Junior University Light-sensitive ion-passing molecules
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US8263396B2 (en) 2010-04-01 2012-09-11 Weidong Xiao Methods and compositions for the production of recombinant virus vectors
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
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
EP2635111B1 (en) 2010-11-05 2018-05-23 The Board of Trustees of the Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
WO2012061679A2 (en) 2010-11-05 2012-05-10 The Board Of Trustees Of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
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
NZ701693A (en) 2012-04-18 2017-02-24 The Children’S Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using aav capsid variants
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)
US9938541B2 (en) * 2012-12-25 2018-04-10 Takara Bio Inc. AAV variant
US9567376B2 (en) * 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
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
WO2014200910A2 (en) 2013-06-10 2014-12-18 Iogenetics, Llc Bioinformatic processes for determination of peptide binding
WO2014207190A1 (en) 2013-06-28 2014-12-31 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for expressing a polynucleotide of interest in the retina of a subject
WO2015012501A1 (ko) 2013-07-25 2015-01-29 주식회사 스마트캐스터 비공기압 바퀴
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
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能CRISPR−Cas系による配列操作のための系、方法および組成物
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
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
DE102014217790A1 (de) 2014-09-05 2016-03-10 Evonik Degussa Gmbh Verfahren zur Herstellung von hydrosilylierbaren Eugenol-Polyethern und Eugenol-Polyethersiloxanen sowie deren Verwendung
AU2016219789B2 (en) 2015-02-20 2021-03-25 Fondazione Telethon Methods and compositions for treating genetic eye diseases
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
JP6836999B2 (ja) 2015-03-24 2021-03-03 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The Regents Of The University Of California アデノ随伴ウイルス変異体及びその使用方法
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
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
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
JP2020534788A (ja) 2017-08-28 2020-12-03 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 アデノ随伴ウイルスカプシド変異体及びその使用方法

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