In vivo RNA editing of point mutations via RNA-guided adenosine deaminases

被引：149

作者：

Katrekar, Dhruva ^{[1
]}

Chen, Genghao ^{[1
]}

Meluzzi, Dario ^{[1
]}

Ganesh, Ashwin ^{[1
]}

Worlikar, Atharv ^{[1
]}

Shih, Yu-Ru ^{[1
,2
]}

Varghese, Shyni ^{[1
,2
]}

Mali, Prashant ^{[1
]}

机构：

[1] Univ Calif San Diego, Dept Bioengn, San Diego, CA 92103 USA

[2] Duke Univ, Dept Biomed Engn, Durham, NC 27706 USA

来源：

NATURE METHODS | 2019年 / 16卷 / 03期

基金：

美国国家卫生研究院;

关键词：

MOUSE MODEL; GENE; MUSCLE; ADAR1;

D O I：

10.1038/s41592-019-0323-0

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

We present in vivo sequence-specific RNA base editing via adenosine deaminases acting on RNA (ADAR) enzymes with associated ADAR guide RNAs (adRNAs). To achieve this, we systematically engineered adRNAs to harness ADARs, and comprehensively evaluated the specificity and activity of the toolsets in vitro and in vivo via two mouse models of human disease. We anticipate that this platform will enable tunable and reversible engineering of cellular RNAs for diverse applications.

引用

页码：239 / +

页数：6

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