Department of Cell and Developmental Biology and Centre for Stem Cells and Regenerative Medicine, University College London, London, UK.
Curr Opin Neurol. 2012 Oct;25(5):597-603. doi: 10.1097/WCO.0b013e328357f288.
This review focuses on stem cell-based therapies to treat skeletal muscle disorders, with a special emphasis on muscular dystrophies.
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.
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.
本综述重点关注基于干细胞的治疗骨骼肌疾病的方法，特别关注肌肉营养不良症。
我们简要回顾了以前使用供体成肌细胞进行细胞治疗的尝试，解释了临床疗效不佳的可能原因；然后描述了目前在不同骨骼肌疾病的局部治疗中使用相同细胞的有前途的试验。此外，我们还讨论了肌肉干细胞/祖细胞生物学的重要新发现及其对未来临床转化的前景。还描述了新型成肌干细胞/祖细胞的临床前和临床应用。
我们总结了几种不同肌肉疾病的正在进行的临床试验，以及对这些试验中使用的成肌祖细胞的生物学的理解的进展。根据目前的信息，提出了对该领域发展的预测。
