Gene Editing for the Heart: Correcting Dystrophin Mutations.

Abstract

Gene editing is moving rapidly from a highly useful laboratory-based tool towards human clinical application. <i>In vivo</i> 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 <i>in vivo</i> application of cardiac gene correction.