A Novel Engineered U7 Small Nuclear RNA Scaffold Greatly Increases in vitro and in vivo ADAR-Mediated Programmable RNA Base Editing
Susan M. Byrne, Stephen M. Burleigh, Robert Fragoza, Yue Jiang, Yiannis A. Savva, Ricky Pabon, Evan E. Kania, Joseph Rainaldi, Andrew J. Portell, Prashant Mali, Adrian W. Briggs
This paper develops a novel U7 snRNA scaffold that significantly enhances ADAR-mediated RNA base editing in vitro and in vivo, achieving up to 76% editing efficiency in mouse models.
Nature Communications · May 26, 2025Peer-reviewedBioengineering