JPWO2020069461A5 - - Google Patents

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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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vector genome
aav vector
sequence
seq
aav
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JP2021517461A
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JP7528066B2 (ja
JP2022500066A (ja
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Priority to JP2024021997A priority Critical patent/JP7761686B2/ja
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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 Active JP7528066B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2024021997A JP7761686B2 (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
JP2025126955A JP2025179046A (ja) 2018-09-28 2025-07-30 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

Applications Claiming Priority (5)

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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 (en) 2018-09-28 2019-09-27 Frataxin expression constructs having engineered promoters and methods of use thereof

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JP2024021997A Division JP7761686B2 (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

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JP2022500066A JP2022500066A (ja) 2022-01-04
JPWO2020069461A5 true JPWO2020069461A5 (enExample) 2022-10-04
JP7528066B2 JP7528066B2 (ja) 2024-08-05

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JP2021517461A Active JP7528066B2 (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
JP2024021997A Active JP7761686B2 (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
JP2025126955A Pending JP2025179046A (ja) 2018-09-28 2025-07-30 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

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JP2024021997A Active JP7761686B2 (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
JP2025126955A Pending JP2025179046A (ja) 2018-09-28 2025-07-30 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)

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US (1) US12281321B2 (enExample)
EP (1) EP3856762A1 (enExample)
JP (3) JP7528066B2 (enExample)
KR (1) KR20210068068A (enExample)
CN (2) CN113383010B (enExample)
AU (1) AU2019346655B2 (enExample)
CA (1) CA3114621A1 (enExample)
SG (1) SG11202103151RA (enExample)
WO (1) WO2020069461A1 (enExample)

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JP7528066B2 (ja) 2018-09-28 2024-08-05 ボイジャー セラピューティクス インコーポレイテッド 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
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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
JP2025504081A (ja) * 2022-02-01 2025-02-06 コーネル・ユニバーシティー フリードライヒ運動失調症に関連する心筋症の治療及び予防のための方法及び医薬組成物
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
CN120035671A (zh) * 2023-05-11 2025-05-23 四川至善唯新生物科技有限公司 工程化的肝脏特异性核心启动子及其应用
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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
WO2025122531A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to frataxin deficiency
CN118421807B (zh) * 2024-07-01 2024-09-20 西南民族大学 牦牛多胸腰椎性状筛查的引物对、试剂盒及其用途和筛查方法

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