R. Mark Payne
Department of Pediatrics (Cardiology) Department of Medical and Molecular Genetics Wells Center for Pediatric Research
Developing a Cure for Friedreich's Ataxia
My laboratory focuses on understanding the role of mitochondrial biogenesis and function in mediating heart disease in children. Specifically, we are interested in: 1) Understanding the process of protein targeting to mammalian mitochondria, and how this may be disrupted in cardiomyopathies or ischemic heart disease. 2) Developing novel gene therapies to target therapeutic proteins to cardiac mitochondria as treatment for disease. We are currently developing protein replacement approaches for a mitochondrial disorder, Friedreich's Ataxia, in which mitochondrial dysfunction destroys cardiac and neural function over time. These approaches are based on the use of cell penetrant peptides to deliver proteins to mitochondria in tissues and cells. A wide variety of molecular biology, protein chemistry, and imaging techniques are used to test hypotheses in genetically altered mice. These goals will further our understanding and treatment of heart failure in children and young adults.