Derivation of Skeletal Myogenic Precursors from Human Pluripotent Stem Cells Using Conditional Expression of PAX7
Derivation of Skeletal Myogenic Precursors from Human Pluripotent Stem Cells Using Conditional Expression of PAX7
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DOI：
10.1007/7651_2014_134
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发表时间：
2016-01-01
期刊：
影响因子：
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通讯作者：
Perlingeiro, Rita C. R.
中科院分区：
文献类型：
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作者：
Darabi, Radbod;Perlingeiro, Rita C. R.
细胞疗法被认为是治疗退行性疾病（包括肌肉疾病和营养不良）最有前途的方法之一。体细胞重编程成诱导多能干细胞（iPS）方法的进展使得利用患者自身的多能细胞生成用于自体移植的特异性组织成为可能。此外，患者特异性组织衍生物已被证明是疾病建模和药物发现的宝贵材料。然而，多能干细胞定向分化为特定谱系并不是一项微不足道的任务，特别是在骨骼肌发生的情况下，在多能干细胞的体外分化过程中，骨骼肌发生通常很难重现。在这里，我们描述了一种实用而有效的方法，通过控制PAX7的表达，从分化人类多能干细胞中衍生出骨骼肌前体细胞。流式细胞术（FACS）纯化的肌源性前体可以在体外呈指数级扩增并分化成肌管，使研究人员能够将这些细胞用于疾病建模和治疗目的。
Cell-based therapies are considered as one of the most promising approaches for the treatment of degenerating pathologies including muscle disorders and dystrophies. Advances in the approach of reprogramming somatic cells into induced pluripotent stem (iPS) cells allow for the possibility of using the patient's own pluripotent cells to generate specific tissues for autologous transplantation. In addition, patient-specific tissue derivatives have been shown to represent valuable material for disease modeling and drug discovery. Nevertheless, directed differentiation of pluripotent stem cells into a specific lineage is not a trivial task especially in the case of skeletal myogenesis, which is generally poorly recapitulated during the in vitro differentiation of pluripotent stem cells.Here, we describe a practical and efficient method for the derivation of skeletal myogenic precursors from differentiating human pluripotent stem cells using controlled expression of PAX7. Flow cytometry (FACS) purified myogenic precursors can be expanded exponentially and differentiated in vitro into myotubes, enabling researchers to use these cells for disease modeling as well as therapeutic purposes.