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

TitleIn vivo RNA editing of point mutations via RNA-guided adenosine deaminases.
Publication TypeJournal Article
Year of Publication2019
AuthorsD Katrekar, G Chen, D Meluzzi, A Ganesh, A Worlikar, Y-R Shih, S Varghese, and P Mali
JournalNat Methods
Volume16
Start Page239
Issue3
Pagination239 - 242
Date Published03/2019
Abstract

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.

DOI10.1038/s41592-019-0323-0
Short TitleNat Methods