Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration.

Generation of human myogenic progenitors from pluripotent stem cells for in vivo regeneration.
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DOI:
10.1007/s00018-022-04434-8
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发表时间:
2022-07-08
期刊:
Cellular and molecular life sciences : CMLS
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其他
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肌营养不良包括大量异质性遗传疾病,其特征在于进行性和破坏性肌肉萎缩。旨在促进骨骼肌再生的基于细胞的替代策略代表了治疗肌营养不良症的候选治疗方法。由于从肌肉活检获得大量干细胞以及在体外扩增这些干细胞的困难,多能干细胞(PSC)代表了用于产生肌原性祖细胞的有吸引力的细胞来源,因为PSC可以重复产生大量谱系特异性组织,代表了用于治疗的无限细胞来源。在这篇综述中,我们重点介绍了迄今为止在不同方法上的进展,用于产生人PSC衍生的肌源性祖细胞,其移植后的再生能力,其同种异体和自体移植的潜力,以及未来治疗应用中需要考虑的具体挑战。
Muscular dystrophy encompasses a large number of heterogeneous genetic disorders characterized by progressive and devastating muscle wasting. Cell-based replacement strategies aimed at promoting skeletal muscle regeneration represent a candidate therapeutic approach to treat muscular dystrophies. Due to the difficulties of obtaining large numbers of stem cells from a muscle biopsy as well as expanding these in vitro, pluripotent stem cells (PSCs) represent an attractive cell source for the generation of myogenic progenitors, given that PSCs can repeatedly produce large amounts of lineage-specific tissue, representing an unlimited source of cells for therapy. In this review, we focus on the progress to date on different methods for the generation of human PSC-derived myogenic progenitor cells, their regenerative capabilities upon transplantation, their potential for allogeneic and autologous transplantation, as well as the specific challenges to be considered for future therapeutic applications.
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