期刊
CURRENT OPINION IN NEUROLOGY
卷 25, 期 5, 页码 597-603出版社
LIPPINCOTT WILLIAMS & WILKINS
DOI: 10.1097/WCO.0b013e328357f288
关键词
cell therapy; diseases of skeletal muscle; muscular dystrophy; myogenic stem/progenitor cells
资金
- European Research Council
- Medical Research Council UK
- European Community (OptiStem)
- European Community (BioDesign)
- Duchenne Parent Project
- Telethon
- Medical Research Council [MR/J006785/1] Funding Source: researchfish
- MRC [MR/J006785/1] Funding Source: UKRI
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.
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