Stem cell therapy is an attractive method to treat muscular dystrophy because only a small number of cells, together with a stimulatory signal for expansion, are required to obtain a therapeutic effect. Recently, it was published the isolation of human MSC-like cells from adult skeletal muscle. The aim of this review is to describe the recent progress in understanding MSC biology and their therapeutic applications. Given that these cells can be obtained with non- or minimally invasive-biopsy procedures, there is growing evidence that skeletal muscle may be an important clinical source of MSCs for use in therapeutic applications. Various in vitro and in vivo studies have evaluated the safety, feasibility and efficacy of transplanting MSCs for clinical trials. Currently, there are many registered clinical trial sites for evaluating MSC therapy throughout the world.