Mybpc3 gene therapy for neonatal cardiomyopathy enables long-term disease prevention in mice

Hereditary hypertrophic cardiomyopathy (HCM) is caused by mutations in cardiomyocyte genes, such as MYBPC3. Here, the authors use virus-mediated gene therapy to correct Mycbpc3 mutations in 1-day-old mice and, by administering just a single dose, prevent development of HCM over a period of 34 weeks.