Stem Cells for the Treatment of Muscular Dystrophy

This chapter focuses on stem cell-based therapies to treat muscular dystrophy. It reviews previous attempts of cell therapy in animal models and patients by the use of donor myoblasts, explaining the likely reasons for poor clinical results. It also reports the identification of myogenic progenitors in the bone marrow and the related trials, this time in mice only, to treat dystrophy by bone marrow transplantation. It then proceeds to describe recently identified stem/progenitor cells, either resident or able to home in dystrophic muscle and to differentiate into skeletal muscle cells. Different features of the various stem cells are compared in this perspective and the few available examples of their use in preclinical models of muscular dystrophy reported. Finally, current clinical trials are discussed. On the basis of current knowledge and waiting for a rapid advance in stem cell biology, a prediction of the initial outcome of currently running trials and further development in the field will be presented.