Duchenne muscular dystrophy: CRISPR/Cas9 treatment

被引：41

作者：

Mendell, Jerry R. ^{[1
,2
,3
]}

Rodino-Klapac, Louise R. ^{[1
,2
,3
]}

机构：

[1] Nationwide Childrens Hosp, Dept Pediat, Columbus, OH 43205 USA

[2] Nationwide Childrens Hosp, Ctr Gene Therapy, Columbus, OH 43205 USA

[3] Ohio State Univ, Columbus, OH 43205 USA

来源：

CELL RESEARCH | 2016年 / 26卷 / 05期

关键词：

MOUSE MODEL; MUSCLE;

D O I：

10.1038/cr.2016.28

中图分类号：

Q2 [细胞生物学];

学科分类号：

071009 ; 090102 ;

摘要：

A novel approach to gene correction by genome editing shows great promise as a treatment for Duchenne muscular dystrophy (DMD). CRISPR/Cas9 delivered by adeno-associated virus to a mouse model for DMD demonstrated improvement in function and histology.

引用

页码：513 / 514

页数：2

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