JP2020505936A5 - - Google Patents

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JP2020505936A5
JP2020505936A5 JP2019542561A JP2019542561A JP2020505936A5 JP 2020505936 A5 JP2020505936 A5 JP 2020505936A5 JP 2019542561 A JP2019542561 A JP 2019542561A JP 2019542561 A JP2019542561 A JP 2019542561A JP 2020505936 A5 JP2020505936 A5 JP 2020505936A5
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Japan
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amino acid
seq
acid sequence
capsid protein
aav capsid
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JP2019542561A
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Japanese (ja)
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JP7291397B2 (ja
JP2020505936A (ja
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Priority claimed from PCT/US2018/018381 external-priority patent/WO2018152333A1/en
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Priority to JP2023007075A priority Critical patent/JP7574508B2/ja
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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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023007075A JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762459286P 2017-02-15 2017-02-15
US62/459,286 2017-02-15
PCT/US2018/018381 WO2018152333A1 (en) 2017-02-15 2018-02-15 Methods and compositions for gene transfer across the vasculature

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JP2023007075A Division JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

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JP2020505936A JP2020505936A (ja) 2020-02-27
JP2020505936A5 true JP2020505936A5 (enExample) 2021-03-04
JP7291397B2 JP7291397B2 (ja) 2023-06-15

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JP2019542561A Active JP7291397B2 (ja) 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
JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

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JP2023007075A Active JP7574508B2 (ja) 2017-02-15 2023-01-20 血管系を通過する遺伝子移入の方法および組成物

Country Status (13)

Country Link
US (2) US11905312B2 (enExample)
EP (1) EP3583220A4 (enExample)
JP (2) JP7291397B2 (enExample)
KR (1) KR20190117571A (enExample)
CN (2) CN118978574A (enExample)
AU (1) AU2018221738B2 (enExample)
BR (1) BR112019016769A2 (enExample)
CA (1) CA3052829A1 (enExample)
EA (1) EA038695B1 (enExample)
IL (2) IL268534B2 (enExample)
MX (1) MX2019009720A (enExample)
SG (1) SG11201907298WA (enExample)
WO (1) WO2018152333A1 (enExample)

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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
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
WO2021087007A1 (en) * 2019-10-28 2021-05-06 University Of Florida Research Foundation, Incorporated Gene therapy vectors
KR20220116459A (ko) * 2019-11-22 2022-08-23 더 칠드런스 호스피탈 오브 필라델피아 아데노(adeno)-관련 바이러스 벡터 변이체
CN115279400B (zh) 2020-01-10 2026-04-28 布里格姆妇女医院 用于跨血脑屏障递送免疫治疗剂以治疗脑癌的方法和组合物
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
BR112022016965A2 (pt) * 2020-02-25 2022-12-06 Childrens Medical Res Institute Polipeptídeos e vetores de capsídeo do vírus adenoassociado
CA3177869A1 (en) * 2020-05-05 2021-11-11 Duke University Cross-species compatible adeno-associated virus compositions and methods of use thereof
AU2021328475A1 (en) 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
WO2022076750A2 (en) * 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
KR20230082613A (ko) * 2020-10-07 2023-06-08 리젠엑스바이오 인크. Cns 또는 근육 전달을 위한 재조합 아데노-연관 바이러스
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EP4277524A4 (en) * 2021-01-13 2025-06-04 Duke University Compositions for and methods of improving fluid flux in the brain
KR20240032971A (ko) 2021-07-08 2024-03-12 테나야 테라퓨틱스, 인코포레이티드 유전자 요법을 위한 최적화된 발현 카세트
WO2023288184A2 (en) * 2021-07-14 2023-01-19 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
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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
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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
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KR102855674B1 (ko) * 2022-10-06 2025-09-08 주식회사 글루진테라퓨틱스 폐 기관지 특이적 유전자 전달이 가능한 아데노부속바이러스 벡터
WO2024215655A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
WO2024215653A1 (en) 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2025015388A1 (en) * 2023-07-20 2025-01-23 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
WO2025121957A1 (ko) * 2023-12-06 2025-06-12 주식회사 글루진테라퓨틱스 뇌 성상교세포 특이적 유전자 전달이 가능한 아데노부속바이러스 변이체와 이의 응용
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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

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