Cas9 targeting of toxic foci of RNA repeats

被引：0
|
作者：
Denis Furling
机构：
[1] Sorbonne Université,
[2] Inserm,undefined
[3] Institut de Myologie,undefined
[4] Centre de Recherche en Myologie,undefined
来源：
Nature Biomedical Engineering | 2021年 / 5卷
关键词：
D O I：
暂无
中图分类号：
学科分类号：
摘要：
The sustained expression of RNA-targeting Cas9 delivered intramuscularly or systemically by adeno-associated viral vectors eliminates pathogenic foci of expanded-repeat transcripts and reverses muscle-disease phenotypes in mouse models of myotonic dystrophy type 1.
引用
收藏
页码：130 / 131
页数：1
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