JP2021521782A5 - - Google Patents

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Publication number
JP2021521782A5
JP2021521782A5 JP2020557155A JP2020557155A JP2021521782A5 JP 2021521782 A5 JP2021521782 A5 JP 2021521782A5 JP 2020557155 A JP2020557155 A JP 2020557155A JP 2020557155 A JP2020557155 A JP 2020557155A JP 2021521782 A5 JP2021521782 A5 JP 2021521782A5
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JP
Japan
Prior art keywords
helix
triple
raav
dystrophin
mutant protein
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Application number
JP2020557155A
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English (en)
Japanese (ja)
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JP2021521782A (ja
JP7575273B2 (ja
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Priority claimed from PCT/US2019/027675 external-priority patent/WO2019204303A2/en
Publication of JP2021521782A publication Critical patent/JP2021521782A/ja
Publication of JP2021521782A5 publication Critical patent/JP2021521782A5/ja
Application granted granted Critical
Publication of JP7575273B2 publication Critical patent/JP7575273B2/ja
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JP2020557155A 2018-04-16 2019-04-16 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法 Active JP7575273B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862658464P 2018-04-16 2018-04-16
US62/658,464 2018-04-16
PCT/US2019/027675 WO2019204303A2 (en) 2018-04-16 2019-04-16 Compositions and methods for treating duchenne muscular dystrophy

Publications (3)

Publication Number Publication Date
JP2021521782A JP2021521782A (ja) 2021-08-30
JP2021521782A5 true JP2021521782A5 (enExample) 2022-04-19
JP7575273B2 JP7575273B2 (ja) 2024-10-29

Family

ID=68240632

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020557155A Active JP7575273B2 (ja) 2018-04-16 2019-04-16 デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法

Country Status (6)

Country Link
US (2) US12460226B2 (enExample)
EP (1) EP3780952A4 (enExample)
JP (1) JP7575273B2 (enExample)
CN (1) CN112601454B (enExample)
CA (1) CA3099958A1 (enExample)
WO (1) WO2019204303A2 (enExample)

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AU2020392243A1 (en) 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
WO2021144649A2 (en) * 2020-01-16 2021-07-22 Takeda Pharmaceutical Company Limited Adeno associated virus based gene therapy for phenylketonuria
CN113999861B (zh) * 2021-09-08 2024-03-15 三峡大学 腺相关病毒衣壳蛋白保守区多克隆抗体的制备方法及应用

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