EA202091828A1 - Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека - Google Patents

Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека

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EA202091828A1
EA202091828A1 EA202091828A EA202091828A EA202091828A1 EA 202091828 A1 EA202091828 A1 EA 202091828A1 EA 202091828 A EA202091828 A EA 202091828A EA 202091828 A EA202091828 A EA 202091828A EA 202091828 A1 EA202091828 A1 EA 202091828A1
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subject
compositions
methods
grna
human cardiomyocytes
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EA202091828A
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Эрик Н. Олсон
Чэнцзу Лун
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Дзе Борд Оф Риджентс Оф Дзе Юниверсити Оф Техас Систем
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Publication of EA202091828A1 publication Critical patent/EA202091828A1/ru

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Abstract

Изобретение относится к способу лечения или профилактики мышечной дистрофии Дюшенна (DMD) у нуждающегося в этом субъекта, включающему введение субъекту нуклеазы Cas9 или последовательности, кодирующей нуклеазу Cas9, и гРНК или последовательности, кодирующей гРНК, причем гРНК нацелена на донорный сайт сплайсинга или акцепторный сайт сплайсинга гена дистрофина. Введение обеспечивает восстановление экспрессии дистрофина, по меньшей мере, в подгруппе кардиомиоцитов субъекта и позволяет, по меньшей мере, частично или полностью восстановить сердечную сократимость.
EA202091828A 2018-01-31 2019-01-31 Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека EA202091828A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862624748P 2018-01-31 2018-01-31
PCT/US2019/015988 WO2019152609A1 (en) 2018-01-31 2019-01-31 Compositions and methods for correcting dystrophin mutations in human cardiomyocytes

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EA202091828A1 true EA202091828A1 (ru) 2021-05-24

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US (1) US20200370042A1 (ru)
EP (1) EP3746557A1 (ru)
JP (1) JP2021511803A (ru)
KR (1) KR20200116933A (ru)
CN (1) CN111836893A (ru)
AU (1) AU2019216321A1 (ru)
BR (1) BR112020015617A2 (ru)
CA (1) CA3088547A1 (ru)
EA (1) EA202091828A1 (ru)
IL (1) IL276139A (ru)
WO (1) WO2019152609A1 (ru)

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WO2023172116A1 (ko) * 2022-03-10 2023-09-14 주식회사 진코어 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법
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EP3746557A1 (en) 2020-12-09
US20200370042A1 (en) 2020-11-26
BR112020015617A2 (pt) 2021-01-05
WO2019152609A1 (en) 2019-08-08
CA3088547A1 (en) 2019-08-08
AU2019216321A1 (en) 2020-07-30
CN111836893A (zh) 2020-10-27
JP2021511803A (ja) 2021-05-13
KR20200116933A (ko) 2020-10-13

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