In Vivo Genome Engineering for the Treatment of Muscular Dystrophies

被引：0
作者：
Monika Kustermann
Matthew J. Rok
Ronald D. Cohn
Evgueni A. Ivakine
机构：
[1] Peter Gilgan Centre for Research and Learning,Program in Genetics and Genome Biology
[2] The Hospital for Sick Children Research Institute,Department of Molecular Genetics
[3] University of Toronto,Department of Pediatrics
[4] The Hospital for Sick Children,Institute of Medical Science
[5] University of Toronto,Department of Physiology
[6] University of Toronto,undefined
来源：
Current Stem Cell Reports | 2020年 / 6卷
关键词：
Muscular dystrophy; Duchenne muscular dystrophy; Genome editing; CRISPR/Cas9; Adeno-associated viruses; Nanoparticles;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
引用
收藏
页码：52 / 66
页数：14
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[61]  
Kirschner J(2019)A mutation-independent approach for muscular dystrophy via upregulation of a modifier gene Nature. 572 125-1784
[62]  
Flanigan KM(2017)Impeding transcription of expanded microsatellite repeats by deactivated Cas9 Mol Cell 68 479-517
[63]  
Straub V(2019)Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in the muscle of DM1 mice Mol Ther 27 1372-R1R7
[64]  
Bushby K(2016)Exon snipping in Duchenne muscular dystrophy Trends Mol Med 22 187-150
[65]  
Verschuuren J(2019)CRISPR correction of Duchenne muscular dystrophy Annu Rev Med 70 239-1675
[66]  
Aartsma-Rus A(2017)Revealing hidden complexities of genomic rearrangements generated with Cas9 Sci Rep 7 12867-364
[67]  
Béroud C(2015)Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9 Cell Stem Cell 17 213-1656
[68]  
Lochmüller H(1828)Restoration of dystrophin protein expression by exon skipping utilizing CRISPR-Cas9 in myoblasts derived from DMD patient iPS cells Methods Mol Biol 2018 191-855
[69]  
Wong TWY(2017)CRISPR-Cpf1 correction of muscular dystrophy mutations in human cardiomyocytes and mice Sci Adv 3 e1602814-150
[70]  
Cohn RD(2016)212th ENMC international workshop: animal models of congenital muscular dystrophies, Naarden, the Netherlands, 29-31 May 2015 Neuromuscul Disord 26 252-1463
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