JP2020528734A5 - - Google Patents

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JP2020528734A5
JP2020528734A5 JP2019568621A JP2019568621A JP2020528734A5 JP 2020528734 A5 JP2020528734 A5 JP 2020528734A5 JP 2019568621 A JP2019568621 A JP 2019568621A JP 2019568621 A JP2019568621 A JP 2019568621A JP 2020528734 A5 JP2020528734 A5 JP 2020528734A5
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seq
amino acids
formula
capsid protein
aav
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JP2019568621A
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JP7744659B2 (ja
JP2020528734A (ja
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JP2019568621A 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 Active JP7744659B2 (ja)

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JP2023110850A JP7902481B2 (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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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

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JP2020528734A JP2020528734A (ja) 2020-10-01
JP2020528734A5 true JP2020528734A5 (enExample) 2021-07-29
JP7744659B2 JP7744659B2 (ja) 2025-09-26

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JP2023110850A Active JP7902481B2 (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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US (2) US20200121746A1 (enExample)
EP (1) EP3645052A4 (enExample)
JP (2) JP7744659B2 (enExample)
KR (2) KR20250023600A (enExample)
CN (1) CN110461368A (enExample)
AU (2) AU2018290954B2 (enExample)
BR (1) BR112019019015A2 (enExample)
CA (2) CA3314412A1 (enExample)
MX (1) MX2019010910A (enExample)
WO (1) WO2019006182A1 (enExample)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK3693025T3 (da) * 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US10577627B2 (en) 2014-06-09 2020-03-03 Voyager Therapeutics, Inc. Chimeric capsids
EP3606544B1 (en) 2017-04-05 2025-06-04 University of Massachusetts Minigene therapy
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.
AU2019220368B2 (en) * 2018-02-15 2024-06-13 Brave Bioscience Ab 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
US11149256B2 (en) 2018-09-26 2021-10-19 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
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
US12390539B2 (en) 2019-05-20 2025-08-19 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
WO2021243085A2 (en) * 2020-05-29 2021-12-02 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
EP4322976A4 (en) * 2021-04-13 2025-06-25 Capsida, Inc. AAV COMPOSITIONS WITH HIGH EXPRESSION LEVELS IN THE BRAIN
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
CN120187407A (zh) * 2021-12-15 2025-06-20 健达九州(北京)生物科技有限公司 重组aav载体及其用途
WO2023125481A1 (zh) * 2021-12-28 2023-07-06 成都弘基生物科技有限公司 经过修饰的aav衣壳蛋白及其用途
CA3255242A1 (en) * 2022-03-30 2023-10-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
TW202346600A (zh) * 2022-03-30 2023-12-01 高等教育聯邦系統 匹茲堡大學 用於將核酸遞送至視網膜神經節細胞和/或視網膜色素上皮細胞的腺相關病毒載體
US20250327092A1 (en) * 2022-03-30 2025-10-23 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells
JP2025531824A (ja) * 2022-09-09 2025-09-25 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア 受容体リガンドをタイリングするディスプレイされたペプチドを介したトロピズムのリプログラミング
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
JP2026509271A (ja) 2023-03-10 2026-03-17 ダイノ セラピューティクス,インコーポレーテッド カプシドポリペプチドおよびその使用方法
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
KR20260082361A (ko) 2023-08-31 2026-06-04 디노 테라퓨틱스, 인코포레이티드 캡시드 폴리펩타이드 및 이의 사용 방법
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
WO2026024045A1 (ko) * 2024-07-22 2026-01-29 한국생명공학연구원 변이체 캡시드를 포함하는 아데노-연관 바이러스 및 이의 용도
US20260042803A1 (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
WO2026122963A1 (en) 2024-12-06 2026-06-11 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
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
WO1997020925A1 (en) 1995-12-08 1997-06-12 Hybridon, Inc. Modified vegf antisense oligonucleotides for treatment of skin disorders
US6096548A (en) 1996-03-25 2000-08-01 Maxygen, Inc. Method for directing evolution of a virus
WO1997038723A1 (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
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
AU1449499A (en) 1997-10-31 1999-05-24 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
CN1159445C (zh) 1998-09-11 2004-07-28 加利福尼亚大学董事会 能在心脏中组织特异性表达的重组腺病毒
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill 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
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
US20030144221A1 (en) 2001-07-17 2003-07-31 Isis Pharmaceuticals Inc. Antisense modulation of BCL2-associated X protein expression
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
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
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
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
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
US7858367B2 (en) 2002-04-30 2010-12-28 Duke University Viral vectors and methods for producing and using the same
US7071172B2 (en) 2002-04-30 2006-07-04 The University Of North Carolina At Chapel Hill Secretion signal vectors
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
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
PL2657248T3 (pl) 2003-06-19 2017-09-29 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
AU2004276316A1 (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
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
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
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP2009502140A (ja) 2005-07-22 2009-01-29 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー 光活性化陽イオンチャネルおよびその使用
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
US8470790B2 (en) 2006-05-04 2013-06-25 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
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
RU2571931C2 (ru) 2007-04-13 2015-12-27 Каталист Биосайенсис, Инк. Полипептиды на основе модифицированного фактора vii и их применение
EP1985708B1 (en) 2007-04-27 2015-04-15 Universität Rostock Selective targeting of viruses to neural precursor cells
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
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker 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
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP3399024A1 (en) 2010-03-17 2018-11-07 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
AU2011256838B2 (en) 2010-05-17 2014-10-09 Sangamo Therapeutics, Inc. Novel DNA-binding proteins and uses thereof
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
US10738326B2 (en) 2010-10-27 2020-08-11 Jichi Medical University Adeno-associated virus vector for gene transfer to nervous system cells
US10568307B2 (en) 2010-11-05 2020-02-25 The Board Of Trustees Of The Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
EP3486253A1 (en) 2010-11-05 2019-05-22 The Board of Trustees of The Leland Stanford Junior University Light-activated chimeric opsins and methods of using the same
DK3693025T3 (da) 2011-04-22 2022-01-10 Univ California Adenoassocierede virus-virioner med variant capsid og fremgangsmåder til anvendelse heraf
EP3795581A3 (en) 2011-08-24 2021-06-09 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
JP6342886B2 (ja) 2012-04-18 2018-06-13 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia Aavカプシドバリアントを使用した高効率な遺伝子導入のための組成物及び方法
EP3470523A1 (en) 2012-05-09 2019-04-17 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)
EP2940131B1 (en) * 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
WO2014124282A1 (en) 2013-02-08 2014-08-14 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
US11136557B2 (en) 2013-05-31 2021-10-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US12374421B2 (en) 2013-06-10 2025-07-29 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
EP3049527A4 (en) 2013-09-26 2017-08-16 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
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
ES2822551T3 (es) 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2015191693A2 (en) 2014-06-10 2015-12-17 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
CA2977355A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
FI3265178T3 (fi) 2015-03-02 2026-08-03 Adverum Biotechnologies Inc Koostumuksia ja menetelmiä polynukleotidien viemiseksi intravitreaalisesti retinaalisiin tappisoluihin
US20180043034A1 (en) 2015-03-06 2018-02-15 Massachusetts Eye And 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
WO2017023724A1 (en) 2015-07-31 2017-02-09 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
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CN109640949B (zh) 2016-07-29 2022-11-25 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和其使用方法
MX2020001997A (es) 2017-08-28 2020-07-20 Univ California Variantes de capside de virus adenoasociado y metodos de uso de estas.

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