BS-513-01 SUPPRESSION-REPLACEMENT GENE THERAPY FOR SCN5A-MEDIATED TYPE 3 LONG QT SYNDROME

Abstract

Congenital long QT syndrome (LQTS) is an autosomal dominant disorder characterized by delayed repolarization of the myocardium with a prolonged QT interval on electrocardiogram that may manifest as syncope, seizure, or sudden cardiac arrest/death. Long QT syndrome type 3 (LQT3) is caused by gain-of-function mutations in the SCN5A-encoded Nav1.5 sodium channel. No current therapies target the molecular cause of LQT3.