Human induced pluripotent stem cells (hiPS cells or hiPSCs) can be derived from cells of patients with severe muscle disease. If skeletal muscle induced from patient-iPSCs shows disease-specific phenotypes, it can be useful for studying the disease pathogenesis and for drug development. On the other hand, human iPSCs from healthy donors or hereditary muscle disease-iPSCs whose genomes are edited to express normal protein are expected to be a cell source for cell therapy. Several protocols for the derivation of skeletal muscle from human iPSCs have been reported to allow the development of efficient treatments for devastating muscle diseases. In 2017, the focus of research is shifting to another stage: (1) the establishment of mature myofibers that are suitable for study of the pathogenesis of muscle disease; (2) setting up a high-throughput drug screening system; and (3) the preparation of highly regenerative, non-oncogenic cells in large quantities for cell transplantation, etc.

中文翻译：

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诱导多能干细胞产生的骨骼肌-诱导和应用。

人诱导的多能干细胞（hiPS细胞或hiPSC）可以源自患有严重肌肉疾病的患者的细胞。如果从患者iPSC诱导的骨骼肌显示出疾病特定的表型，则对于研究疾病的发病机理和药物开发可能是有用的。另一方面，来自健康供体的人类iPSC或遗传基因疾病被编辑为表达正常蛋白质的遗传性肌肉疾病-iPSC，有望成为细胞疗法的细胞来源。据报道，从人iPSC衍生骨骼肌的几种方案可以开发出用于破坏性肌肉疾病的有效治疗方法。2017年，研究重点转移到另一个阶段：（1）建立适合研究肌肉疾病发病机理的成熟肌纤维; （二）建立高通量毒品筛查制度；（3）大量制备高度再生的非致癌细胞用于细胞移植，等。