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源自胚胎干细胞的多巴胺能神经元在帕金森病动物模型中发挥作用。

Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease.

作者信息

Kim Jong-Hoon, Auerbach Jonathan M, Rodríguez-Gómez José A, Velasco Iván, Gavin Denise, Lumelsky Nadya, Lee Sang-Hun, Nguyen John, Sánchez-Pernaute Rosario, Bankiewicz Krys, McKay Ron

机构信息

Laboratory of Molecular Biology, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland 20892, USA.

出版信息

Nature. 2002 Jul 4;418(6893):50-6. doi: 10.1038/nature00900. Epub 2002 Jun 20.

Abstract

Parkinson's disease is a widespread condition caused by the loss of midbrain neurons that synthesize the neurotransmitter dopamine. Cells derived from the fetal midbrain can modify the course of the disease, but they are an inadequate source of dopamine-synthesizing neurons because their ability to generate these neurons is unstable. In contrast, embryonic stem (ES) cells proliferate extensively and can generate dopamine neurons. If ES cells are to become the basis for cell therapies, we must develop methods of enriching for the cell of interest and demonstrate that these cells show functions that will assist in treating the disease. Here we show that a highly enriched population of midbrain neural stem cells can be derived from mouse ES cells. The dopamine neurons generated by these stem cells show electrophysiological and behavioural properties expected of neurons from the midbrain. Our results encourage the use of ES cells in cell-replacement therapy for Parkinson's disease.

摘要

帕金森病是一种常见病症,由合成神经递质多巴胺的中脑神经元丧失所致。源自胎儿中脑的细胞可改变疾病进程,但它们作为多巴胺合成神经元的来源并不充足,因为其生成这些神经元的能力不稳定。相比之下,胚胎干细胞(ES细胞)能大量增殖并可生成多巴胺神经元。若ES细胞要成为细胞疗法的基础,我们必须开发富集目标细胞的方法,并证明这些细胞具有有助于治疗该疾病的功能。在此我们表明,可从小鼠ES细胞中获得高度富集的中脑神经干细胞群体。这些干细胞生成的多巴胺神经元表现出中脑神经元预期的电生理和行为特性。我们的结果为在帕金森病的细胞替代疗法中使用ES细胞提供了支持。

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