P236Evaluation of safety and feasibility of Mybpc3 gene therapy in a mouse model of hypertrophic cardiomyopathy

Purpose: Hypertrophic cardiomyopathy (HCM) is characterized by left ventricular hypertrophy (LVH) and diastolic dysfunction, and is frequently caused by MYBPC3 mutations, which mainly result in haploinsufficiency of cardiac myosin-binding protein C (cMyBP-C). We recently provided proof-of-concept studies that adeno-associated virus (AAV)-mediated RNA-based therapies (exon skipping and trans-splicing) remove the mutation in a homozygous...