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利用直接重编程策略生成患者特异性诱导神经元细胞。

Generation of patient-specific induced neuronal cells using a direct reprogramming strategy.

机构信息

1 Department of Neurosurgery, Fudan University Huashan Hospital , Shanghai, China .

出版信息

Stem Cells Dev. 2014 Jan 1;23(1):16-23. doi: 10.1089/scd.2013.0131. Epub 2013 Sep 17.

Abstract

Direct reprogramming of human fibroblasts into functional neurons in vitro by defined factors provides an invaluable resource for regenerative medicine. However, clinical applications must consider the risk of immune rejection, thus patient-specific induced neuronal cells (iNCs) may serve as an ideal source for autologous cell replacement. In this study, we report a robust process for functional neuronal cells from the patients' scalp by lentiviral gene delivery of Ascl1, Myt1l, and Sox2. These three-factor iNCs are similar to human neuronal cells in morphology, surface antigens, gene expression, and electrophysiological characteristics. Our findings might provide a source of patient-specific functional neurons for cell therapy.

摘要

通过定义因子将人类成纤维细胞直接重编程为功能性神经元在体外提供了宝贵的资源,用于再生医学。然而,临床应用必须考虑免疫排斥的风险,因此患者特异性诱导神经元细胞(iNCs)可能作为自体细胞替代的理想来源。在这项研究中,我们通过慢病毒基因传递 Ascl1、Myt1l 和 Sox2 报告了从患者头皮获得功能性神经元细胞的稳健过程。这三种因子的 iNCs 在形态、表面抗原、基因表达和电生理特征上与人神经元细胞相似。我们的发现可能为细胞治疗提供了患者特异性功能性神经元的来源。

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