Grant: Doctoral Award - Frederick Banting and Charles Best Canada Graduate Scholarships

Duchenne muscular dystrophy (DMD) is a devastating childhood disorder marked by severely debilitating muscle weakness that worsens with age. DMD arises as a consequence of muscular degeneration caused by a genetic mutation that primarily affects boys, with a worldwide incidence of 1 in every 3500 male births. Defects caused by the mutant gene create abnormalities in muscle fibers and muscle stem cells. Because of these abnormalities, the muscles of people with DMD are highly prone to damage. Normally, muscle stem cells are able to easily heal injured muscle; however, the muscle stem cells residing in people with DMD are defective and can't keep up with the muscle damage. Even with current treatments, boys with the disease are wheelchair bound between the ages of 9-12, paralysed below the neck by 21, and usually die in their 20's or 30's due to heart or breathing failure. Previous studies in our lab have shown that treatment with a specific protein (Wnt7a) improves the symptoms of DMD. Wnt7a improves DMD symptoms by stimulating the defective muscle stem cells to multiply and heal damaged muscle more effectively. However, the cellular mechanism behind Wnt7a's ability to improve muscle stem cell function remains unknown. My project aims to identify precisely how Wnt7a improves muscle regeneration by studying the molecular events responsible for making a muscle stem cell divide and multiply. Studying the molecular events within muscle stem cells will allow us to use Wnt7a more effectively for improving muscle regeneration, and potentially reveal new therapeutic targets for treating people suffering from DMD. The ultimate goal of this research is to set the stage for Wnt7a's use as a clinical therapeutic that improves the quality of life and lifespan of people suffering from DMD. Furthermore, it is predicted that this treatment will be used to regenerate muscle in combination with future gene therapies that attempt to fix the underlying genetic mutation that causes DMD.