RNA编辑
清脆的
核糖核酸
基因组编辑
生物
计算生物学
阿达尔
核酸
核糖核酸酶
遗传学
基因
作者
David Benjamin Turitz Cox,Jonathan S. Gootenberg,Omar O. Abudayyeh,Brian Franklin,Max J. Kellner,Julia Joung,Feng Zhang
出处
期刊:Science
[American Association for the Advancement of Science (AAAS)]
日期:2017-11-24
卷期号:358 (6366): 1019-1027
被引量:1330
标识
DOI:10.1126/science.aaq0180
摘要
Precise transcriptome engineering Efficient and precise RNA editing to correct disease-relevant transcripts holds great promise for treating genetic disease. Cox et al. took advantage of the ability of Cas13b, an effector from a type VI CRISPR-Cas system, to target specific RNAs directly (see the Perspective by Yang and Chen). They fused Cas13b with the ADAR2 adenosine deaminase domain and used rational protein engineering to improve the resultant enzyme. These approaches yielded an RNA knockdown and editing platform that allowed efficient and specific RNA depletion and correction in mammalian cells. Science , this issue p. 1019 ; see also p. 996
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