Project 3. Therapeutic potential of myosin Regulatory Light Chain (RLC) phosphorylation in treatment of heart disease

Genetic cardiomyopathies are the most prevalent inherited heart diseases and no therapies are currently available to cure or delay progression from hypertrophy to heart failure, and/or to prevent sudden cardiac death. Thus, efforts must be undertaken to develop new and effective therapeutic strategies. In this research project we aim to explore the therapeutic potential of the pseudo-phosphorylated RLC construct, in which Aspartic acid is replaced for phosphorylatable Serine-15 (S15D) using the adeno-associated virus (AAV) delivery system. The central hypothesis is that RLC phosphorylation plays a universal rescue role in the disease and the pseudo-phosphorylated RLC construct can act as a therapeutic agent to mitigate detrimental HCM and HF phenotypes.