CN111836893A - 用于校正人心肌细胞中肌养蛋白突变的组合物和方法 - Google Patents

用于校正人心肌细胞中肌养蛋白突变的组合物和方法 Download PDF

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CN111836893A
CN111836893A CN201980011157.5A CN201980011157A CN111836893A CN 111836893 A CN111836893 A CN 111836893A CN 201980011157 A CN201980011157 A CN 201980011157A CN 111836893 A CN111836893 A CN 111836893A
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grna
aav
vector
cas9
dmd
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埃里克·N·奥尔松
龙城祖
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University of Texas System
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CN201980011157.5A 2018-01-31 2019-01-31 用于校正人心肌细胞中肌养蛋白突变的组合物和方法 Pending CN111836893A (zh)

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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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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=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
EP3740580A4 (en) 2018-01-19 2021-10-20 Duke University GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CA3108282A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
AR118668A1 (es) * 2019-04-14 2021-10-20 Univ Duke Composiciones para editar genoma a base de crispr / cas para restaurar la función de distrofina
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
WO2023240157A2 (en) * 2022-06-08 2023-12-14 Scribe Therapeutics Inc. Compositions and methods for the targeting of dmd

Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010048586A1 (en) * 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
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
WO2014144978A2 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
WO2016161380A1 (en) * 2015-04-01 2016-10-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
WO2017049407A1 (en) * 2015-09-23 2017-03-30 UNIVERSITé LAVAL Modification of the dystrophin gene and uses thereof
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
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
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript

Family Cites Families (6)

* Cited by examiner, † Cited by third party
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
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
JP7075597B2 (ja) * 2016-05-05 2022-05-26 デューク ユニバーシティ デュシェンヌ型筋ジストロフィーを治療するためのｃｒｉｓｐｒ／ｃａｓ関連の方法および組成物
JP7490211B2 (ja) * 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
US10687520B2 (en) * 2017-03-07 2020-06-23 The Board Of Regents Of The University Of Texas System Generation and correction of a humanized mouse model with a deletion of dystrophin exon 44

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2010048586A1 (en) * 2008-10-24 2010-04-29 Avi Biopharma, Inc. Multiple exon skipping compositions for dmd
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
WO2014144978A2 (en) * 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
WO2016161380A1 (en) * 2015-04-01 2016-10-06 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating duchenne muscular dystrophy and becker muscular dystrophy
WO2017049407A1 (en) * 2015-09-23 2017-03-30 UNIVERSITé LAVAL Modification of the dystrophin gene and uses thereof
WO2017062835A2 (en) * 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
WO2018014042A1 (en) * 2016-07-15 2018-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dystrophin transcript
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LEONELA AMOASII 等: "Single-cut genome editing restores dystrophin expression in a new mouse model of muscular dystrophy", 《SCIENCE TRANSLATIONAL MEDICINE》, vol. 9, 24 January 2018 (2018-01-24), pages 1 - 11 *

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