GeneticsNature CommunicationsPeer-reviewed
Striated muscle-specific base editing enables correction of mutations causing dilated cardiomyopathy
Markus Grosch, Laura Schraft, Adrian Chan…·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.