Full-length dystrophin deficiency leads to contractile and calcium transient defects in human engineered heart tissues

Cardiomyopathy is currently the leading cause of death for patients with Duchenne muscular dystrophy (DMD), a severe neuromuscular disorder affecting young boys. Animal models have provided insight into mechanisms by which dystrophin protein deficiency causes cardiomyopathy, but there remains need to develop human DMD validate pathogenic and identify therapeutic targets. Here, we developed engineered heart tissues (EHTs) from genetically edited, induced pluripotent stem cell-derived cardiomyocytes (hiPSC-CMs) expressing truncated protein.