The longer, the better? Optimizing dystrophin constructs for cardiac gene therapy in Duchenne muscular dystrophy

Gene therapy for Duchenne muscular dystrophy (DMD) has so far focused mainly on improving skeletal muscle function; however, effective treatment of the disease will also require protection of the heart. In a recently published study in Molecular Therapy Advances, Keegan et al.1 addressed this challenge using a patient-in-a-dish model based on induced pluripotent stem cell-derived cardiomyocytes (iPSC-CMs), as discussed in the following text.