EA202091828A1 - Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека - Google Patents
Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человекаInfo
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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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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.
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
EA202091828A1 true EA202091828A1 (ru) | 2021-05-24 |
Family
ID=65657512
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA202091828A EA202091828A1 (ru) | 2018-01-31 | 2019-01-31 | Композиции и способы коррекции мутаций дистрофина в кардиомиоцитах человека |
Country Status (11)
Country | Link |
---|---|
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) |
Families Citing this family (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
JP2021532831A (ja) | 2018-08-02 | 2021-12-02 | ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. | 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それらの使用 |
US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
AU2020357668A1 (en) * | 2019-10-02 | 2022-03-10 | Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) | Treatment of diseases caused by frame shift mutations |
US20230272428A1 (en) * | 2019-12-16 | 2023-08-31 | President And Fellows Of Harvard College | Methods and compositions for correction of dmd mutations |
CA3212108A1 (en) * | 2021-03-04 | 2022-09-09 | 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 |
US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
WO2023039444A2 (en) * | 2021-09-08 | 2023-03-16 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 51 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 |
WO2023172926A1 (en) * | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exons 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 |
WO2023240157A2 (en) * | 2022-06-08 | 2023-12-14 | Scribe Therapeutics Inc. | Compositions and methods for the targeting of dmd |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP0273085A1 (en) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | A method for internalizing nucleic acids into eukaryotic cells |
CN105779453A (zh) * | 2008-10-24 | 2016-07-20 | 萨雷普塔治疗公司 | 用于dmd的多外显子跳跃组合物 |
CN102625840A (zh) * | 2009-04-10 | 2012-08-01 | 肌肉学研究协会 | 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法 |
TR201806812T4 (tr) | 2012-05-25 | 2018-06-21 | Charpentier Emmanuelle | Rna-yönlendirmeli hedef dna modifikasyonu için ve rna-yönlendirmeli transkripsiyon modifikasyonu için yöntemler ve bileşimler. |
KR20150133768A (ko) * | 2013-03-15 | 2015-11-30 | 사렙타 쎄러퓨틱스 인코퍼레이티드 | 근육 이영양증의 치료를 위한 개선된 조성물 |
CA2959130A1 (en) * | 2014-08-11 | 2016-02-18 | The Board Of Regents Of The University Of Texas System | Prevention of muscular dystrophy by crispr/cas9-mediated gene editing |
EP3277816B1 (en) * | 2015-04-01 | 2020-06-17 | Editas Medicine, Inc. | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy |
US20180265859A1 (en) * | 2015-09-23 | 2018-09-20 | UNIVERSITé LAVAL | Modification of the dystrophin gene and uses thereof |
MA45819A (fr) * | 2015-10-09 | 2018-08-15 | Sarepta Therapeutics Inc | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
US20190134221A1 (en) * | 2016-05-05 | 2019-05-09 | Duke University | Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy |
US20190330626A1 (en) * | 2016-07-15 | 2019-10-31 | Ionis Pharmaceuticals, Inc. | Compounds and methods for use in dystrophin transcript |
EP3487523B1 (en) * | 2016-07-19 | 2023-09-06 | Duke University | Therapeutic applications of cpf1-based genome editing |
RU2625003C1 (ru) * | 2016-10-04 | 2017-07-11 | Федеральное государственное бюджетное научное учреждение "Медико-генетический научный центр" | Набор последовательностей нуклеотидов для медицинской технологии детекции наиболее частых в россии делеций гена dmd методом мультиплексного пцр/пдаф анализа |
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2019
- 2019-01-31 EP EP19708910.5A patent/EP3746557A1/en not_active Withdrawn
- 2019-01-31 KR KR1020207022943A patent/KR20200116933A/ko not_active Application Discontinuation
- 2019-01-31 AU AU2019216321A patent/AU2019216321A1/en active Pending
- 2019-01-31 US US16/966,274 patent/US20200370042A1/en not_active Abandoned
- 2019-01-31 BR BR112020015617-5A patent/BR112020015617A2/pt not_active Application Discontinuation
- 2019-01-31 EA EA202091828A patent/EA202091828A1/ru unknown
- 2019-01-31 CA CA3088547A patent/CA3088547A1/en active Pending
- 2019-01-31 JP JP2020541767A patent/JP2021511803A/ja active Pending
- 2019-01-31 CN CN201980011157.5A patent/CN111836893A/zh active Pending
- 2019-01-31 WO PCT/US2019/015988 patent/WO2019152609A1/en unknown
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2020
- 2020-07-19 IL IL276139A patent/IL276139A/en unknown
Also Published As
Publication number | Publication date |
---|---|
IL276139A (en) | 2020-09-30 |
KR20200116933A (ko) | 2020-10-13 |
WO2019152609A1 (en) | 2019-08-08 |
CA3088547A1 (en) | 2019-08-08 |
JP2021511803A (ja) | 2021-05-13 |
CN111836893A (zh) | 2020-10-27 |
BR112020015617A2 (pt) | 2021-01-05 |
EP3746557A1 (en) | 2020-12-09 |
US20200370042A1 (en) | 2020-11-26 |
AU2019216321A1 (en) | 2020-07-30 |
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