通过 RNA 引导的腺嘌呤脱氨酶对点突变进行体内 RNA 编辑。
In vivo RNA editing of point mutations via RNA-guided adenosine deaminases.
机构信息
Department of Bioengineering, University of California, San Diego, CA, USA.
Department of Biomedical Engineering, Duke University, Durham, NC, USA.
出版信息
Nat Methods. 2019 Mar;16(3):239-242. doi: 10.1038/s41592-019-0323-0. Epub 2019 Feb 8.
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.
我们通过 RNA 上的腺苷脱氨酶作用（ADAR）酶及其相关的 ADAR 向导 RNA（adRNA）展示了体内序列特异性 RNA 碱基编辑。为此，我们系统地设计了 adRNA 以利用 ADAR，通过两种人类疾病的小鼠模型在体外和体内全面评估了工具的特异性和活性。我们预计该平台将能够为各种应用实现细胞 RNA 的可调谐和可逆工程。
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