Skeletal muscle generated from induced pluripotent stem cells - induction and application.
Skeletal muscle generated from induced pluripotent stem cells - induction and application.
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DOI：
10.4252/wjsc.v9.i6.89
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发表时间：
2017-06-26
影响因子：
4.1
通讯作者：
Takeda S
中科院分区：
文献类型：
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作者：
Miyagoe-Suzuki Y;Takeda S
人诱导多能干细胞（hiPS细胞或hiPSC）可以来源于患有严重肌肉疾病的患者的细胞。如果从患者-iPSC诱导的骨骼肌显示疾病特异性表型，则其可用于研究疾病发病机制和药物开发。另一方面，来自健康供体或遗传性肌肉疾病的人iPSC-其基因组被编辑以表达正常蛋白质的iPSC有望成为细胞治疗的细胞来源。已经报道了从人iPSC衍生骨骼肌的几种方案，以允许开发针对破坏性肌肉疾病的有效治疗。2017年，研究重点正在转向另一个阶段：（1）建立适合研究肌肉疾病发病机制的成熟肌纤维;（2）建立高通量药物筛选系统;（3）大量制备用于细胞移植的高度再生的非致癌细胞等。
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.