Matthew J Burke

Advancing adeno-associated virus (AAV) gene therapy for Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a lethal degenerative muscle disease. DMD results from mutations preventing the production of the subsarcolemmal protein dystrophin, a protein critical for muscle stability. In the absence of dystrophin, muscle fibers are damaged and degenerate, leading to an inflamed and fibrotic m...