Abundant off-target edits from site-directed RNA editing can be reduced by nuclear localization of the editing enzyme.

ABSTRACT: Site-directed RNA editing (SDRE) is a general strategy for making targeted base changes in RNA molecules. Although the approach is relatively new, several groups, including our own, have been working on its development. The basic strategy has been to couple the catalytic domain of an adenosine (A) to inosine (I) RNA editing enzyme to a guide RNA that is used for targeting. Although highly efficient on-target editing has been reported, off-target events have not been rigorously quantified. In this report we target premature termination codons (PTCs) in messages encoding both a fluorescent reporter protein and the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein transiently transfected into human epithelial cells. We demonstrate that while on-target editing is efficient, of

SUBMITTER: Vallecillo-Viejo IC 

PROVIDER: S-EPMC5786015 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature