Healing Hearts: Advancing cardiomyopathy research, one beat at a time

Inherited heart muscle diseases or cardiomyopathies affect 1 in 250 people worldwide and run in families. Often the first time a family learns that they have this condition is when a family member dies unexpectedly. Hear how we aim to cure this devastating condition using cutting edge gene editing technology.

Titin is the largest protein in the body, and it serves a crucial purpose providing structure to our heart cells. Genetic mutations that run in families can reduce Titin levels in the heart and cause dilated cardiomyopathy, a common and potentially devastating disease of the heart muscle that currently has no cure. In this talk you will learn about the obstacles to creating gene therapies targeting this giant protein, and current research being undertaken to get closer to a cure.

We are up to the challenge of penetrating plasma membranes, crossing the endothelium of blood vessels, and resisting extracellular nuclease degradation until we reach your heart. Let's escape the endosome together and hear our plans to deliver therapeutic compounds to cardiomyocytes and modify the expression of genes causing cardiomyopathies.