Gene Therapy for Catecholaminergic Polymorphic Ventricular Tachycardia by Inhibition of Ca ²⁺ /Calmodulin-Dependent Kinase II

Abstract

This proof-of-concept study showed that AAV-mediated delivery of a CaMKII peptide inhibitor to the heart was effective in suppressing arrhythmias in a murine model of CPVT. CaMKII inhibition also reversed the arrhythmia phenotype in human CPVT induced pluripotent stem cell-derived cardiomyocyte models with different pathogenic mutations.