Perspectives of stem cell therapy in Duchenne musculardystrophy

摘要

Muscular dystrophies are heritable and heterogeneous neuromuscular disorderscharacterized by the primary wasting of skeletal muscle, usuallycaused by mutations in the proteins forming the link between the cytoskeletonand the basal lamina. As a result of mutations in the dystrophin gene,Duchenne muscular dystrophy patients suffer from progressive muscleatrophy and an exhaustion of muscular regenerative capacity. No efficienttherapies are available. The evidence that adult stem cells were capable ofparticipating in the regeneration of more than their resident organ led tothe development of potential stem cell treatments for degenerative disorder.In the present review, we describe the different types of myogenic stem cellsand their possible use for the progression of cell therapy in Duchenne musculardystrophy.