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

页码：130 / 131

页数：1

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