🫀 Gene Therapy, Hidden Hazard, Cardiac Caution: A Gene Therapy Wake-Up Call?

🧬🫀 Can successful gene therapy still leave the heart vulnerable? A fascinating NEJM correspondence describes a patient with Duchenne muscular dystrophy who developed early and delayed cardiotoxicity after microdystrophin gene therapy. Despite higher cardiac vector copy numbers, myocardial microdystrophin expression remained low and heterogeneous, raising important questions about efficacy, safety, and long-term surveillance. An important reminder that motor improvement may not mirror cardiac benefit. Join me as I unpack the clinical implications on Dr RR Baliga's Podkast for the Kurious Doc—Where Curiosity Meets Clarity! 🎙️🫀