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
收藏
页码：513 / 514
页数：2
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