Laura Schraft

The paper describes an ex vivo study using normothermic machine-perfused human hearts to evaluate AAV-mediated gene delivery for cardiac gene therapy.

This study examines the effects of RBM20-R636Q mutation on atrial electrophysiology and evaluates SGLT inhibitors as potential therapeutics for atrial remodeling and arrhythmias.

This study identifies CAMK2D overactivation as the cause of heart failure in RBM20 cardiomyopathy and shows pharmacological inhibition can reverse cardiac dysfunction.

This study uses base editors and AAVMYO to correct Rbm20 mutations in cardiomyocytes, improving splicing and cardiac function in mouse models of dilated cardiomyopathy.