Grant: Project Grant

Muscular Dystrophy is a collection of genetic disease that lead to progressive skeletal muscle degeneration and eventual death. Duchenne Muscular Dystrophy (DMD) is cause by a mutation in the dystrophin gene (mdx) that normally helps anchor muscle cells to the muscle sheath. This leads to chronic muscle damage and a gradual replacement of health muscle tissue with fat and scar tissue as the body makes a failed attempt at tissue repair. Recently we have identified a new rare population of immune cells as the drivers of tissue fibrosis in some disease models. Intriguingly, we find that perturbing these cells in mice carrying the mdx gene can dramatically alter the disease progression. Accordingly, we propose to use a variety of novel techniques we have developed to skew the behaviour of these cells as a potential therapy for slowing the progression DMD. Because DMD is relatively frequently leads and to death in late adolescence, the ability to slow the progression of muscle degeneration and fibrosis could have a profound effect on the quality of life for huge number of Canadians.