Institut de Myologie, CNRS FRE3617, UPMC Univ Paris 06, UM76, INSERM U974, Sorbonne Universités, 47 bd de l'Hôpital, Paris, 75013, France.
Neuropathol Appl Neurobiol. 2015 Apr;41(3):270-87. doi: 10.1111/nan.12198.
Despite considerable progress to increase our understanding of muscle genetics, pathophysiology, molecular and cellular partners involved in muscular dystrophies and muscle ageing, there is still a crucial need for effective treatments to counteract muscle degeneration and muscle wasting in such conditions. This review focuses on cell-based therapy for muscle diseases. We give an overview of the different parameters that have to be taken into account in such a therapeutic strategy, including the influence of muscle ageing, cell proliferation and migration capacities, as well as the translation of preclinical results in rodent into human clinical approaches. We describe recent advances in different types of human myogenic stem cells, with a particular emphasis on myoblasts but also on other candidate cells described so far [CD133+ cells, aldehyde dehydrogenase-positive cells (ALDH+), muscle-derived stem cells (MuStem), embryonic stem cells (ES) and induced pluripotent stem cells (iPS)]. Finally, we provide an update of ongoing clinical trials using cell therapy strategies.
尽管在提高对肌肉遗传学、病理生理学、涉及肌肉疾病和肌肉老化的分子和细胞伙伴的理解方面取得了相当大的进展，但仍然迫切需要有效的治疗方法来对抗这些情况下的肌肉退化和肌肉萎缩。本综述重点介绍了肌肉疾病的基于细胞的治疗方法。我们概述了这种治疗策略中必须考虑的不同参数，包括肌肉老化、细胞增殖和迁移能力的影响，以及将啮齿动物的临床前结果转化为人类临床方法的影响。我们描述了不同类型的人类成肌细胞的最新进展，特别强调了成肌细胞，但也描述了迄今为止描述的其他候选细胞[CD133+细胞、醛脱氢酶阳性细胞(ALDH+)、肌肉源性干细胞(MuStem)、胚胎干细胞(ES)和诱导多能干细胞(iPS)]。最后，我们提供了正在进行的使用细胞治疗策略的临床试验的最新情况。
