Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy
Markus Grosch, Laura Schraft, Adrian Chan, Leonie Küchenhoff, Kleopatra Rapti, Anne-Maud Ferreira, Julia Kornienko, Shengdi Li, Michael H. Radke, Chiara Krämer, Sandra Clauder‐Münster, Emerald Perlas, Johannes Backs, Michael Gotthardt, Christoph Dieterich, , Lars Michael Steinmetz
This study uses base editors and AAVMYO to correct Rbm20 mutations in cardiomyocytes, improving splicing and cardiac function in mouse models of dilated cardiomyopathy.
Nature Communications · Jun 22, 2023Peer-reviewedGenetics