Gene Editing for the Heart

Abstract

Gene editing is moving rapidly from a highly useful laboratory-based tool towards human clinical application. In vivo gene editing has been documented in mouse models of Duchenne Muscular Dystrophy, where skeletal and cardiac muscle editing produces internally-truncated dystrophin. A recent report now demonstrates that editing the dystrophin gene in the heart improves cardiac function, paving the path to in vivo application of cardiac gene correction.