CRISPR/Cas9-Mediated Genome Editing Corrects Dystrophin Mutation in Skeletal Muscle Stem Cells in a Mouse Model of Muscle Dystrophy

被引：58

作者：

Zhu, Pei ^{[1
,2
]}

Wu, Furen ^{[1
,2
]}

Mosenson, Jeffrey ^{[1
,2
]}

Zhang, Hongmei ^{[3
]}

He, Tong-Chuan ^{[3
]}

Wu, Wen-Shu ^{[1
,2
]}

机构：

[1] Univ Illinois, Dept Med, Div Hematol Oncol, Chicago, IL 60612 USA

[2] Univ Illinois, Ctr Canc, Chicago, IL 60612 USA

[3] Univ Chicago, Med Ctr, Dept Orthopaed Surg & Rehabil Med, Chicago, IL 60637 USA

来源：

MOLECULAR THERAPY-NUCLEIC ACIDS | 2017年 / 7卷

关键词：

MUSCULAR-DYSTROPHY; IN-VIVO; SATELLITE CELLS; SELF-RENEWAL; REPLICATIVE SENESCENCE; MDX MICE; GENE; EXPRESSION; CRISPR-CAS9; EXPANSION;

D O I：

10.1016/j.omtn.2017.02.007

中图分类号：

R-3 [医学研究方法]; R3 [基础医学];

学科分类号：

1001 ;

摘要：

Muscle stem cells (MuSCs) hold great therapeutic potential for muscle genetic disorders, such as Duchenne muscular dystrophy (DMD). The CRISP/Cas9-based genome editing is a promising technology for correcting genetic alterations in mutant genes. In this study, we used fibrin-gel culture system to selectively expand MuSCs from crude skeletal muscle cells of mdx mice, a mouse model of DMD. By CRISP/Cas9-based genome editing, we corrected the dystrophin mutation in expanded MuSCs and restored the skeletal muscle dystrophin expression upon transplantation in mdx mice. Our studies established a reliable and feasible platform for gene correction in MuSCs by genome editing, thus greatly advancing tissue stem cell-based therapies for DMD and other muscle disorders.

引用

页码：31 / 41

页数：11

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