JP2021511803A - ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法 - Google Patents

ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法 Download PDF

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JP2021511803A
JP2021511803A JP2020541767A JP2020541767A JP2021511803A JP 2021511803 A JP2021511803 A JP 2021511803A JP 2020541767 A JP2020541767 A JP 2020541767A JP 2020541767 A JP2020541767 A JP 2020541767A JP 2021511803 A JP2021511803 A JP 2021511803A
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grna
aav
sequence
cas9
dmd
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エリック エヌ． オルソン
エリック エヌ． オルソン
チェンズ ロング
チェンズ ロング
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University of Texas System
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JP2020541767A 2018-01-31 2019-01-31 ヒト心筋細胞におけるジストロフィン変異を修正するための組成物および方法 Pending JP2021511803A (ja)

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US201862624748P 2018-01-31 2018-01-31
US62/624,748 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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JP2021511803A true JP2021511803A (ja) 2021-05-13
JP2021511803A5 JP2021511803A5 (https=) 2022-01-27

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

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WO2021064162A1 (en) * 2019-10-02 2021-04-08 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Treatment of diseases caused by frame shift mutations
EP4069314A4 (en) * 2019-12-03 2024-06-26 Duke University SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY
US20230272428A1 (en) * 2019-12-16 2023-08-31 President And Fellows Of Harvard College Methods and compositions for correction of dmd mutations
CN115011598A (zh) * 2020-09-02 2022-09-06 西湖大学 杜氏肌营养不良症相关的外显子剪接增强子、sgRNA、基因编辑工具及应用
US20240254509A1 (en) * 2021-03-04 2024-08-01 Research Institute At Nationwide Children's Hospital Products and methods for treatment of dystrophin-based myopathies using crispr-cas9 to correct dmd exon duplications
AU2022262420A1 (en) * 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
JP2024525608A (ja) 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2023039444A2 (en) * 2021-09-08 2023-03-16 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 51 for treatment of duchenne muscular dystrophy
WO2023172926A1 (en) * 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exons for treatment of duchenne muscular dystrophy
WO2023172927A1 (en) * 2022-03-08 2023-09-14 Vertex Pharmaceuticals Incorporated Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy
KR20230134098A (ko) * 2022-03-10 2023-09-20 주식회사 진코어 듀센 근이영양증 치료를 위한 유전자 편집 시스템 및 이를 이용한 질병 치료 방법
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