Progress in gene therapy and future prospects

Hypertrophic cardiomyopathy (HCM) is the most common genetic cardiomyopathy worldwide, affecting around 1 in 500 people. Current therapeutic interventions include lifestyle optimization, medications, septal reduction therapies and, rarely, heart transplantation. Advances in the understanding of disease-causing genetic variants in HCM and their molecular mechanisms have opened up the potential for targeted therapies and the implementation of precision and personalized medicine. Results from preclinical research are promising and raise the question of whether a cure for some HCM subtypes may be possible in the future.