Muscular dystrophy is a group of inherited neuromuscular diseases that attacks the muscles and heart. The most common form of the disease is Duchenne’s Muscular Dystrophy (DMD), which primarily affects boys and is caused by a mutation in the dystrophin gene. This mutation prevents the body from producing dystrophin protein, which functions as a stabilizer for the muscles and keeps them intact during regular wear and tear. Without dystrophin, muscle cells cannot regenerate properly and they become progressively more inflamed and waste away over time. Few patients with DMD survive into their twenties or thirties. There are no therapies to treat the disease and most treatments are aimed at controlling symptoms.
Currently there are no Health Canada or FDA approved stem cell treatments available for muscular dystrophy. Researchers have found a type of stem cell in muscle that helps build and maintain the tissue, however, the intensity of the disease causes these stem cells to tire out and eventually lose their ability to replace and repair cells. The two main approaches currently being studied involved the use of stem cells to regenerate the damaged muscle or as vehicles to deliver gene therapies that replace or repair the defective gene.