JP7291397B2 - 血管系を通過する遺伝子移入の方法および組成物 - Google Patents

血管系を通過する遺伝子移入の方法および組成物 Download PDF

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JP7291397B2
JP7291397B2 JP2019542561A JP2019542561A JP7291397B2 JP 7291397 B2 JP7291397 B2 JP 7291397B2 JP 2019542561 A JP2019542561 A JP 2019542561A JP 2019542561 A JP2019542561 A JP 2019542561A JP 7291397 B2 JP7291397 B2 JP 7291397B2
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JP2020505936A (ja
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アラヴィンド アソカン
ムルリダラン,ギリダル
ブレイク オルブライト
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University of North Carolina at Chapel Hill
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JP2019542561A 2017-02-15 2018-02-15 血管系を通過する遺伝子移入の方法および組成物 Active JP7291397B2 (ja)

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US (2) US11905312B2 (https=)
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CN (2) CN118978574A (https=)
AU (1) AU2018221738B2 (https=)
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Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
MX2020002148A (es) * 2017-08-25 2020-07-20 Ovid Therapeutics Inc Vectores adenoasociados recombinantes.
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
IL278598B2 (en) 2018-05-11 2025-11-01 Massachusetts Eye & Ear Infirmary Altering tissue tropism of adeno-associated viruses
CN112703198B (zh) 2018-07-11 2025-05-30 布里格姆妇女医院 用于跨血脑屏障递送试剂的方法和组合物
WO2020068990A1 (en) 2018-09-26 2020-04-02 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20210130158A (ko) * 2019-01-31 2021-10-29 오레곤 헬스 앤드 사이언스 유니버시티 Aav 캡시드의 전사 의존적 유도 진화를 사용하는 방법
BR112021015312A2 (pt) 2019-02-04 2021-11-09 Freeline Therapeutics Ltd Polinucleotídeos, partícula viral e composição
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
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
KR20220012231A (ko) * 2019-04-24 2022-02-03 리젠엑스바이오 인크. 완전-인간 번역 후 변형된 항체 치료제
JP7785352B2 (ja) * 2019-08-14 2025-12-15 ユニバーシティー オブ フロリダ リサーチ ファンデーション, インク. 遺伝子治療のためのaavカプシドバリアント
US12570998B2 (en) 2019-09-09 2026-03-10 Massachusetts Eye & Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (AAV) and the AAV receptor (AAVR) for altered bio-distribution of AAV
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
EP4051324A4 (en) * 2019-10-28 2023-11-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
PH12022551247A1 (en) * 2019-11-22 2023-11-13 Childrens Hospital Philadelphia Adeno-associated viral vector variants
AU2021206256A1 (en) 2020-01-10 2022-07-28 The Brigham And Women's Hospital, Inc. Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
WO2021163357A2 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease
MY209860A (en) 2020-02-13 2025-08-07 Tenaya Therapeutics Inc Gene therapy vectors for treating heart disease
TW202142554A (zh) * 2020-02-25 2021-11-16 澳洲兒童醫學研究所 腺相關病毒蛋白殼多肽及載體
AR122013A1 (es) * 2020-05-05 2022-08-03 Univ Duke Composiciones de virus adenoasociados compatibles entre especies y métodos para su uso
EP4200408A1 (en) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of rett syndrome
JP2023545384A (ja) * 2020-10-07 2023-10-30 リジェネックスバイオ インコーポレイテッド 中枢神経系または筋肉送達のための組換えアデノ随伴ウイルス
WO2022076750A2 (en) * 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
US20240060087A1 (en) * 2021-01-08 2024-02-22 Massachusetts Eye And Ear Infirmary Methods and compositions for modulating the interaction between adeno-associated virus (aav) and the aav receptor (aavr) for altered bio-distribution of aav
WO2022155358A1 (en) * 2021-01-13 2022-07-21 Duke University Compositions for and methods of improving fluid flux in the brain
AU2022307365A1 (en) 2021-07-08 2024-02-01 Tenaya Therapeutics, Inc. Optimized expression cassettes for gene therapy
US20250281639A1 (en) * 2021-07-14 2025-09-11 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for treating chronic pain and for retrograde transduction of neurons
CN113563430B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 用于治疗眼部疾病的基因递送系统及其应用
CN113480615B (zh) * 2021-07-30 2022-05-31 上海信致医药科技有限公司 高视网膜亲和性的新型腺相关病毒衣壳蛋白及其应用
IL310949A (en) * 2021-08-20 2024-04-01 Biocad Joint Stock Co A method for preparing an adapted adeno-associated virus envelope
AR126839A1 (es) * 2021-08-20 2023-11-22 Llc «Anabion» Proteína de la cápside vp1 modificada aislada del virus adeno-asociado de serotipo 9 (aav9), cápside y vector basado en esta
US20240425549A1 (en) * 2021-08-25 2024-12-26 Children's Medical Research Institute Modified aav capsid polypeptides and vectors
MX2024002379A (es) * 2021-08-25 2024-04-29 Childrens Medical Res Institute Cápsides y vectores de aav modificados.
WO2023060272A2 (en) * 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
CN114516901B (zh) * 2022-03-11 2023-01-17 上海勉亦生物科技有限公司 一种神经系统高亲和性的aav载体及其应用
WO2023201277A1 (en) * 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
WO2024054864A1 (en) 2022-09-06 2024-03-14 Tenaya Therapeutics, Inc. Cardioprotective heart disease therapies
KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
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
AU2024294136A1 (en) * 2023-07-20 2026-02-05 Children's Medical Research Institute Modified cardiotropic aav capsid polypeptides and vectors
KR102868933B1 (ko) * 2023-11-08 2025-10-14 주식회사 글루진테라퓨틱스 성상세포 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
KR102844372B1 (ko) * 2023-12-06 2025-08-11 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
WO2025147741A1 (en) * 2024-01-11 2025-07-17 Children's Medical Research Institute Aav capsids and vectors for transduction of cells
CN119405806A (zh) * 2024-10-25 2025-02-11 南通大学 小肌营养蛋白域含2抑制剂在制备促进神经生长药物中的应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2340230T3 (es) 1998-11-10 2010-05-31 University Of North Carolina At Chapel Hill Vectores viricos y sus procedimientos de preparacion y administracion.
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
WO2004027019A2 (en) * 2002-05-01 2004-04-01 University Of Florida Research Foundation, Inc. Improved raav expression systems for genetic modification of specific capsid proteins
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
JP6348064B2 (ja) * 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
US9636370B2 (en) * 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
WO2017015102A1 (en) * 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids

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EP3583220A1 (en) 2019-12-25
JP2020505936A (ja) 2020-02-27
IL312788A (en) 2024-07-01
EA201991911A1 (ru) 2020-02-20
JP7574508B2 (ja) 2024-10-29
KR20190117571A (ko) 2019-10-16
BR112019016769A2 (pt) 2020-05-26
US11905312B2 (en) 2024-02-20
IL268534A (en) 2019-09-26
CA3052829A1 (en) 2018-08-23
JP2023052541A (ja) 2023-04-11
EA038695B1 (ru) 2021-10-06
AU2018221738B2 (en) 2024-06-27
SG11201907298WA (en) 2019-09-27
AU2018221738A1 (en) 2019-08-22
WO2018152333A1 (en) 2018-08-23
MX2019009720A (es) 2019-10-07
CN110520536A (zh) 2019-11-29
CN118978574A (zh) 2024-11-19
IL268534B1 (en) 2024-06-01
US20240209031A1 (en) 2024-06-27
IL268534B2 (en) 2024-10-01
US20190367562A1 (en) 2019-12-05
EP3583220A4 (en) 2021-07-07
CN110520536B (zh) 2024-07-09

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