In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
In vivo RNA editing of point mutations via RNA-guided adenosine deaminases
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DOI：
10.1038/s41592-019-0323-0
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发表时间：
2019-03-01
期刊：
影响因子：
48
通讯作者：
Mali, Prashant
中科院分区：
文献类型：
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作者：
Katrekar, Dhruva;Chen, Genghao;Mali, Prashant
我们介绍了通过作用于RNA酶(ADAR)的腺苷脱氨酶及其相关的ADAR引导RNA(AdRNAs)进行的体内序列特异性RNA碱基编辑。为了实现这一目标，我们系统地设计了adRNAs来利用ADAR，并通过两种人类疾病的小鼠模型在体外和体内综合评估了这些工具集的特异性和活性。我们预计，该平台将为不同的应用实现可调和可逆的细胞RNA工程。
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.