Alpha protein kinase 3 gene therapy restores heart function in mouse and human models of cardiomyopathy
It is demonstrated that adeno-associated virus-mediated delivery of full-length human ALPK3 prevents cardiomyopathy in neonatal mouse models and reverses established disease in adult mice, restoring cardiac structure, function and proteostasis, as well as contractile function in human cardiac organoids carrying ALPK3-...