Project 442961

Duchenne muscular dystrophy (DMD) is a devastating and debilitating muscle degenerative disease affecting 1 in every 3,500 male births worldwide. DMD symptoms manifest in early childhood, as boys between 2-5 years of age exhibit motor development delays. Accumulated and progressive weakening of the muscle tissue leads to an inability to walk and patients are wheelchair-bound by the age of 12. DMD is fatal and patients succumb to death from respiratory and cardiac failure in their 20's and 30's. Despite research efforts to understand DMD, there still remains no effective cure. Historically, DMD has been viewed as a disease affecting the structural integrity of the muscle tissue, which leads to weakening and damage of the muscle fibers. However, recent studies have shown that muscle stem cells, which are adult stem cells that reside within muscle, are also affected in DMD. DMD muscle stem cells do not function as normal healthy stem cells and their dysfunction plays a role in disease progression. Importantly, current therapeutic strategies for muscular dystrophy do not address these deficiencies in muscle stem cell function. Our research aims to understand the cellular processes that are altered in DMD muscle stem cells and devise strategies to restore muscle stem cell function to improve muscle repair.