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胚胎和诱导多能干细胞生成骨骼肌细胞作为体外模型及用于治疗肌肉疾病。

Generation of skeletal muscle cells from embryonic and induced pluripotent stem cells as an in vitro model and for therapy of muscular dystrophies.

机构信息

Department of Neurological Sciences, Dino Ferrari Centre, University of Milan, IRCCS Fondazione Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.

出版信息

J Cell Mol Med. 2012 Jul;16(7):1353-64. doi: 10.1111/j.1582-4934.2011.01498.x.

Abstract

Muscular dystrophies (MDs) are a heterogeneous group of inherited disorders characterized by progressive muscle wasting and weakness likely associated with exhaustion of muscle regeneration potential. At present, no cures or efficacious treatments are available for these diseases, but cell transplantation could be a potential therapeutic strategy. Transplantation of myoblasts using satellite cells or other myogenic cell populations has been attempted to promote muscle regeneration, based on the hypothesis that the donor cells repopulate the muscle and contribute to its regeneration. Embryonic stem cells (ESCs) and more recently induced pluripotent stem cells (iPSCs) could generate an unlimited source of differentiated cell types, including myogenic cells. Here we review the literature regarding the generation of myogenic cells considering the main techniques employed to date to elicit efficient differentiation of human and murine ESCs or iPSCs into skeletal muscle. We also critically analyse the possibility of using these cellular populations as an alternative source of myogenic cells for cell therapy of MDs.

摘要

肌肉萎缩症(MDs)是一组异质性的遗传性疾病,其特征为肌肉进行性萎缩和无力,可能与肌肉再生潜力衰竭有关。目前,这些疾病尚无治愈或有效的治疗方法,但细胞移植可能是一种潜在的治疗策略。基于供体细胞重新填充肌肉并有助于其再生的假设,已尝试使用卫星细胞或其他成肌细胞群移植成肌细胞以促进肌肉再生。胚胎干细胞(ESCs)和最近的诱导多能干细胞(iPSCs)可以产生无限来源的分化细胞类型,包括成肌细胞。在这里,我们回顾了有关生成成肌细胞的文献,考虑了迄今为止用于有效诱导人类和鼠类 ESCs 或 iPSCs 分化为骨骼肌的主要技术。我们还批判性地分析了使用这些细胞群体作为肌肉萎缩症细胞治疗的成肌细胞替代来源的可能性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/9cc5/3823206/8d69fb37c2d6/jcmm0016-1353-f1.jpg

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