In Vivo <i>Ryr</i> 2 Editing Corrects Catecholaminergic Polymorphic Ventricular Tachycardia

Abstract

AAV serotype 9-based delivery of the SaCas9 system can efficiently disrupt a disease-causing allele in cardiomyocytes in vivo. This work highlights the potential of somatic genome editing approaches for the treatment of lethal autosomal-dominant inherited cardiac disorders, such as catecholaminergic polymorphic ventricular tachycardia.