Stem Cells for the Treatment of Muscular Dystrophy

© 2014 Elsevier Inc. All rights reserved.. This chapter focuses on stem cell-based therapies for treating muscular dystrophy. It reviews previous attempts to use donor myoblasts in animal models and patients, explaining the likely reasons for poor clinical results. It also reports the identification of myogenic progenitors in the bone marrow, and the results of preclinical studies in mice to treat dystrophy by bone marrow transplantation. It then proceeds to describe recently identified stem/progenitor cells, either resident or cells that home dystrophic muscle and 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.

ISBN:9780124095038

Jaar van publicatie:2013