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

Autor: Katrekar D; Department of Bioengineering, University of California, San Diego, CA, USA., Chen G; Department of Bioengineering, University of California, San Diego, CA, USA., Meluzzi D; Department of Bioengineering, University of California, San Diego, CA, USA., Ganesh A; Department of Bioengineering, University of California, San Diego, CA, USA., Worlikar A; Department of Bioengineering, University of California, San Diego, CA, USA., Shih YR; Department of Bioengineering, University of California, San Diego, CA, USA.; Department of Biomedical Engineering, Duke University, Durham, NC, USA., Varghese S; Department of Bioengineering, University of California, San Diego, CA, USA.; Department of Biomedical Engineering, Duke University, Durham, NC, USA., Mali P; Department of Bioengineering, University of California, San Diego, CA, USA. pmali@ucsd.edu.
Jazyk: angličtina
Zdroj: Nature methods [Nat Methods] 2019 Mar; Vol. 16 (3), pp. 239-242. Date of Electronic Publication: 2019 Feb 08.
DOI: 10.1038/s41592-019-0323-0
Abstrakt: 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.
Databáze: MEDLINE
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