Article
Prime editing for functional repair in patient-derived disease models.
Nature communications - 23 Oct 2020
Schene Imre F, Joore Indi P, Oka Rurika, Mokry Michal, van Vugt Anke H M, van Boxtel Ruben, van der Doef Hubert P J, van der Laan Luc J W, Verstegen Monique M A, van Hasselt Peter M, Nieuwenhuis Edward E S, Fuchs Sabine A
Abstract excerpt
Prime editing is a recent genome editing technology using fusion proteins of Cas9-nickase and reverse transcriptase, that holds promise to correct the vast majority of genetic defects. Here, we develop prime editing for primary adult stem cells grown in organoid culture models. First, we generate precise in-frame deletions in the gene encoding β-catenin (CTNNB1) that result in proliferation independent of...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.