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利用基因编程的人胚胎干细胞衍生神经祖细胞进行帕金森病细胞治疗的潜力。

Potential for cell therapy in Parkinson's disease using genetically programmed human embryonic stem cell-derived neural progenitor cells.

作者信息

Ambasudhan Rajesh, Dolatabadi Nima, Nutter Anthony, Masliah Eliezer, Mckercher Scott R, Lipton Stuart A

机构信息

Del E. Webb Center for Neuroscience, Aging, and Stem Cell Research, Sanford-Burnham Medical Research Institute, 10901 North Torrey Pines Road, La Jolla, California, 92037.

出版信息

J Comp Neurol. 2014 Aug 15;522(12):2845-56. doi: 10.1002/cne.23617. Epub 2014 May 7.

Abstract

Neural transplantation is a promising strategy for restoring dopaminergic dysfunction and modifying disease progression in Parkinson's disease (PD). Human embryonic stem cells (hESCs) are a potential resource in this regard because of their ability to provide a virtually limitless supply of homogenous dopaminergic progenitors and neurons of appropriate lineage. The recent advances in developing robust cell culture protocols for directed differentiation of hESCs to near pure populations of ventral mesencephalic (A9-type) dopaminergic neurons has heightened the prospects for PD cell therapy. Here, we focus our review on current state-of-the-art techniques for harnessing hESC-based strategies toward development of a stem cell therapeutic for PD. Importantly, we also briefly describe a novel genetic-programming approach that may address many of the key challenges that remain in the field and that may hasten clinical translation.

摘要

神经移植是恢复帕金森病(PD)中多巴胺能功能障碍和改变疾病进展的一种有前景的策略。人类胚胎干细胞(hESCs)在这方面是一种潜在资源,因为它们有能力提供几乎无限量的同源多巴胺能祖细胞和具有适当谱系的神经元。在开发强大的细胞培养方案以将hESCs定向分化为近纯的腹侧中脑(A9型)多巴胺能神经元群体方面的最新进展提高了PD细胞治疗的前景。在这里,我们的综述重点关注利用基于hESC的策略开发PD干细胞疗法的当前最先进技术。重要的是,我们还简要描述了一种新的基因编程方法,该方法可能解决该领域中仍然存在的许多关键挑战,并可能加速临床转化。

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