JPWO2020069461A5 - - Google Patents

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Publication number
JPWO2020069461A5
JPWO2020069461A5 JP2021517461A JP2021517461A JPWO2020069461A5 JP WO2020069461 A5 JPWO2020069461 A5 JP WO2020069461A5 JP 2021517461 A JP2021517461 A JP 2021517461A JP 2021517461 A JP2021517461 A JP 2021517461A JP WO2020069461 A5 JPWO2020069461 A5 JP WO2020069461A5
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JP
Japan
Prior art keywords
vector genome
aav vector
sequence
seq
aav
Prior art date
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
Application number
JP2021517461A
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English (en)
Japanese (ja)
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JP2022500066A (ja
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Application filed filed Critical
Priority claimed from PCT/US2019/053681 external-priority patent/WO2020069461A1/fr
Publication of JP2022500066A publication Critical patent/JP2022500066A/ja
Publication of JPWO2020069461A5 publication Critical patent/JPWO2020069461A5/ja
Pending legal-status Critical Current

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JP2021517461A 2018-09-28 2019-09-27 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 Pending JP2022500066A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862738519P 2018-09-28 2018-09-28
US62/738,519 2018-09-28
US201962901769P 2019-09-17 2019-09-17
US62/901,769 2019-09-17
PCT/US2019/053681 WO2020069461A1 (fr) 2018-09-28 2019-09-27 Constructions d'expression de frataxine comprenant des promoteurs modifiés et leurs méthodes d'utilisation

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024021997A Division JP2024073447A (ja) 2018-09-28 2024-02-16 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法

Publications (2)

Publication Number Publication Date
JP2022500066A JP2022500066A (ja) 2022-01-04
JPWO2020069461A5 true JPWO2020069461A5 (fr) 2022-10-04

Family

ID=68426807

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021517461A Pending JP2022500066A (ja) 2018-09-28 2019-09-27 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法

Country Status (9)

Country Link
US (1) US20210395776A1 (fr)
EP (1) EP3856762A1 (fr)
JP (1) JP2022500066A (fr)
KR (1) KR20210068068A (fr)
CN (1) CN113383010A (fr)
AU (1) AU2019346655A1 (fr)
CA (1) CA3114621A1 (fr)
SG (1) SG11202103151RA (fr)
WO (1) WO2020069461A1 (fr)

Families Citing this family (10)

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JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
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CN115927398A (zh) * 2020-07-29 2023-04-07 舒泰神(北京)生物制药股份有限公司 一组肝靶向新型腺相关病毒的获得及其应用
US11926842B2 (en) 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
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CN114276419B (zh) * 2021-12-30 2023-11-17 上海勉亦生物科技有限公司 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用
WO2023150563A1 (fr) * 2022-02-01 2023-08-10 Cornell University Méthodes et compositions pharmaceutiques pour le traitement et la prévention d'une cardiomyopathie associée à l'ataxie de friedreich
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
CN116041443B (zh) * 2022-12-30 2023-09-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
CN117624311B (zh) * 2023-11-29 2024-05-28 广州译码基因科技有限公司 可提高aav病毒神经靶向性的衣壳蛋白突变体及其应用

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