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

Journal Article (Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Katrekar, D; Chen, G; Meluzzi, D; Ganesh, A; Worlikar, A; Shih, Y-R; Varghese, S; Mali, P

Published Date

  • March 2019

Published In

Volume / Issue

  • 16 / 3

Start / End Page

  • 239 - 242

PubMed ID

  • 30737497

Pubmed Central ID

  • PMC6395520

Electronic International Standard Serial Number (EISSN)

  • 1548-7105

Digital Object Identifier (DOI)

  • 10.1038/s41592-019-0323-0


  • eng

Conference Location

  • United States