Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease.
Direct reprogramming of urine-derived cells with inducible MyoD for modeling human muscle disease.
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DOI：
10.1186/s13395-016-0103-9
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发表时间：
2016
期刊：
影响因子：
4.9
通讯作者：
Wyatt EJ
中科院分区：
文献类型：
--
作者：
Kim EY;Page P;Dellefave-Castillo LM;McNally EM;Wyatt EJ
肌肉疾病的细胞模型变得越来越重要，因为大量基因和突变与引起肌病有关，并且需要测试个性化疗法。开发细胞模型依赖于容易获得的细胞来源，如果细胞不是来自肌肉本身，则需要强大的重编程过程。成纤维细胞是一种非常适合生成诱导多能干细胞的人类细胞来源，然后诱导多能干细胞可以分化为心肌细胞谱系，以及效率较低的骨骼肌样谱系。或者，用转录因子 MyoD 直接重编程已用于从培养的人成纤维细胞中产生肌管。尽管有用，但成纤维细胞需要进行皮肤活检才能获得，这可能会限制它们的获取，特别是对于儿科人群。我们现在证明，尿液来源的细胞的直接重编程是一种高效且可重复的过程，可用于建立人类肌原细胞。我们证明这种方法可以应用于来自正常个体以及肌肉疾病患者的尿细胞。此外，我们还发现尿液来源的细胞可以使用 CRISPR/Cas9 技术进行编辑。随着对人类肌肉疾病分子病因学的了解取得进展，拥有一种容易获得的、非侵入性的细胞来源来产生肌肉样细胞是非常有用的。本文的在线版本 (doi:10.1186/s13395-016-0103-9) 包含补充材料，可供授权用户使用。
Cellular models of muscle disease are taking on increasing importance with the large number of genes and mutations implicated in causing myopathies and the concomitant need to test personalized therapies. Developing cell models relies on having an easily obtained source of cells, and if the cells are not derived from muscle itself, a robust reprogramming process is needed. Fibroblasts are a human cell source that works well for the generation of induced pluripotent stem cells, which can then be differentiated into cardiomyocyte lineages, and with less efficiency, skeletal muscle-like lineages. Alternatively, direct reprogramming with the transcription factor MyoD has been used to generate myotubes from cultured human fibroblasts. Although useful, fibroblasts require a skin biopsy to obtain and this can limit their access, especially from pediatric populations. We now demonstrate that direct reprogramming of urine-derived cells is a highly efficient and reproducible process that can be used to establish human myogenic cells. We show that this method can be applied to urine cells derived from normal individuals as well as those with muscle diseases. Furthermore, we show that urine-derived cells can be edited using CRISPR/Cas9 technology. With progress in understanding the molecular etiology of human muscle diseases, having a readily available, noninvasive source of cells from which to generate muscle-like cells is highly useful. The online version of this article (doi:10.1186/s13395-016-0103-9) contains supplementary material, which is available to authorized users.
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发表时间：
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