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Stem cell therapies for muscle disorders

Tedesco, Francesco S.a,b; Cossu, Giulioa,b

doi: 10.1097/WCO.0b013e328357f288

Purpose of review This review focuses on stem cell-based therapies to treat skeletal muscle disorders, with a special emphasis on muscular dystrophies.

Recent findings We briefly review previous attempts at cell therapy by the use of donor myoblasts, explaining the likely reasons for the poor clinical results; we then describe the use of the same cells in current promising trials for localized treatments of different diseases of skeletal muscle. Moreover, we discuss important novel findings on muscle stem/progenitor cell biology and their promise for future clinical translation. Preclinical and clinical applications of novel myogenic stem/progenitor cells are also described.

Summary We summarize several ongoing clinical trials for different muscle disorders and the advances in the understanding of the biology of the myogenic progenitors used in such trials. On the basis of the currently available information, a prediction of developments in the field is proposed.

aDepartment of Cell and Developmental Biology and Centre for Stem Cells and Regenerative Medicine, University College London, London, UK

bDivision of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Scientific Institute, Milan, Italy

Correspondence to Giulio Cossu, Department of Cell and Developmental Biology, University College London, 21 University Street, WC1E 6DE London, UK. Tel: +44 0 2076796947; fax: +44 0 20767 97349; e-mail:

© 2012 Lippincott Williams & Wilkins, Inc.