Noah Weisleder, PhD
Targeting Membrane Repair to Treat Pediatric Dilated Cardiomyopathy
Ohio State University, Columbus, OH
2016 Amount Awarded – $50,000
Dilated cardiomyopathy (DCM) is the most common form of pediatric cardiomyopathy, which results from the death of heart cells in a child. When these cells die, the heart loses some of its function and continues to lose more cells as the affected child ages. In many forms of DCM, and particularly in DCM associated with muscular dystrophy, the membrane that surrounds the heart cells break and cause the heart cell to die. This study will determine if a new method for increasing the ability of heart cells to repair themselves can act as a treatment for child onset DCM. The study will focus on increasing the ability of these heart cells to repair its damaged membranes and determine if this can improve the disease associated with DCM. A new therapeutic protein drug or a new gene replacement therapy will be tested to minimize the symptoms of DCM in a mouse model of this disease. These therapies may prove to be effective for many types of DCM since one common aspect of the pathology associated with different types of DCM is heart cell death. Increasing the repair capacity of heart cells could have broader therapeutic application to other DCM types that have compromised integrity of their membranes.