CN108779466B - 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法 - Google Patents

用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法 Download PDF

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CN108779466B
CN108779466B CN201680080439.7A CN201680080439A CN108779466B CN 108779466 B CN108779466 B CN 108779466B CN 201680080439 A CN201680080439 A CN 201680080439A CN 108779466 B CN108779466 B CN 108779466B
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molecule
grna
nucleotide sequence
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CN108779466A (zh
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C.A.格尔斯巴赫
J.N.罗宾逊哈姆
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Duke University
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CN201680080439.7A 2015-11-30 2016-11-30 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法 Active CN108779466B (zh)

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CN202410269446.8A CN118147141A (zh) 2015-11-30 2016-11-30 用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法

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US201562260712P 2015-11-30 2015-11-30
US62/260712 2015-11-30
US201662330336P 2016-05-02 2016-05-02
US62/330336 2016-05-02
PCT/US2016/064285 WO2017095967A2 (en) 2015-11-30 2016-11-30 Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

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WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2016130600A2 (en) 2015-02-09 2016-08-18 Duke University Compositions and methods for epigenome editing
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11369692B2 (en) 2015-10-28 2022-06-28 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of Duchenne Muscular Dystrophy
EP3384055B1 (en) * 2015-11-30 2025-07-16 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
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
EP3487523B1 (en) 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
JOP20190166A1 (ar) * 2017-01-05 2019-07-02 Univ Texas استراتيجية مثلى من أجل تعديلات تخطي إكسون باستخدام crispr/cas9 مع متواليات توجيه ثلاثي
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
WO2019036599A1 (en) * 2017-08-18 2019-02-21 The Board Of Regents Of The University Of Texas System EXON DELETION CORRECTION OF MUTATIONS OF DUCHENNE MUSCLE DYSTROPHY IN ACTINE DYSTROPHINE BINDING DOMAIN 1 Using a GENOME CRISPR EDITION
WO2019092507A2 (en) * 2017-11-09 2019-05-16 Crispr Therapeutics Ag Crispr/cas systems for treatment of dmd
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