神经退行性疾病细胞移植治疗的未来。

Stanley E Lazic, Roger A Barker
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引用次数: 27

摘要

帕金森病是一种常见的神经退行性疾病,终生发病率为2.5%,70岁以上人群的患病率至少为2%。针对多巴胺能黑质纹状体通路的药物可以有效治疗患者,但随着时间的推移,这些药物的疗效受到严重运动波动和运动障碍的发展的限制。这促使人们寻找替代疗法,包括使用细胞替代疗法。在过去的十年中,人类胎儿神经移植已经证明多巴胺能神经元可以存活,并为帕金森病患者提供临床益处。然而,有明显的伦理问题和限制这种组织的供应,以及最近对副作用的担忧。因此,组织的替代来源已经被研究,其中一个这样的来源是干细胞,它提供了一个有吸引力的可再生组织供应。在这篇综述中,我们将讨论当前的最新技术和帕金森病的特点,这些特点增加了其作为干细胞治疗目标的吸引力,而不是目前使用胎儿神经移植的临床试验的结果。然后我们将讨论干细胞的各种类型和来源,以及一些早期移植在帕金森病动物模型中的结果。最后,我们将讨论利用干细胞传递药物和神经营养因子参与帕金森病和其他神经退行性疾病的神经保护和神经修复策略的前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
The future of cell-based transplantation therapies for neurodegenerative disorders.

Parkinson's disease is a common neurodegenerative disease with a lifetime incidence of 2.5% and a prevalence of at least 2% in individuals over 70 years old. Patients can be effectively treated with drugs that target the dopaminergic nigro-striatal pathway, but over time the efficacy of these medications is limited by the development of profound motor fluctuations and dyskinesias. This has prompted the search for alternative treatments, including the use of cell replacement therapies. Over the last decade, human fetal nigral transplants have demonstrated that dopaminergic neurons can survive and provide clinical benefit for patients with Parkinson's disease. However, there are clearly ethical concerns and a limit to the supply of this tissue as well as more recently anxieties over side effects. As a result, alternative sources of tissue have been investigated, and one such source are stem cells, which provide an attractive renewable tissue supply. In this review, we will discuss the current state-of-the-art and the characteristics of Parkinson's disease that increase its attraction as a target of stem cell therapy against results of current clinical trials using fetal neural grafts. Then we will discuss the various types and sources of stem cells, and some early transplantation results in animal models of Parkinson's disease. Finally we will discuss the prospect of using stem cells to deliver drugs and neurotrophic factors involved in neuroprotective and neuroreparative strategies in Parkinson's disease and other neurodegenerative conditions.

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