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.