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帕金森病的细胞替代疗法。

Cell replacement therapy for Parkinson's disease.

作者信息

Wijeyekoon Ruwani, Barker Roger A

机构信息

Cambridge Centre for Brain Repair, Forvie Site, Cambridge, UK.

出版信息

Biochim Biophys Acta. 2009 Jul;1792(7):688-702. doi: 10.1016/j.bbadis.2008.10.007. Epub 2008 Oct 25.

Abstract

Parkinson's disease (PD) is a progressive neurodegenerative disorder in which the degeneration of dopaminergic neurons projecting from the substantia nigra to the striatum is a key pathological feature of the disease. Although pharmacological dopamine replacement is generally very effective in early disease, it is only a symptomatic therapy and can have significant side effects with long term use. One of the key strategies in a more restorative approach to PD therapy involves replacement of this degenerating nigro-striatal dopaminergic network with cells and several possible cell sources are being explored. While much experience and some success have been gained with fetal ventral mesencephalic (FVM) tissue transplants, the rapidly advancing stem cell field is providing attractive alternative options which circumvent many of the ethical and practical problems inherent in trials with FVM tissue. Of these embryonic stem cells and induced pluripotent stem cells seem the most promising. However further development and optimisation of the safety and efficacy of the techniques involved in generating and manipulating these, as well as other, cell sources will be essential before any further clinical trials are carried out.

摘要

帕金森病(PD)是一种进行性神经退行性疾病,其中从黑质投射到纹状体的多巴胺能神经元变性是该疾病的关键病理特征。虽然药理学多巴胺替代疗法在疾病早期通常非常有效,但它只是一种对症治疗,长期使用可能会有显著的副作用。对帕金森病治疗采取更具恢复性方法的关键策略之一是用细胞替代这种退化的黑质 - 纹状体多巴胺能网络,目前正在探索几种可能的细胞来源。虽然胎儿腹侧中脑(FVM)组织移植已经积累了很多经验并取得了一些成功,但快速发展的干细胞领域正在提供有吸引力的替代选择,这些选择规避了FVM组织试验中固有的许多伦理和实际问题。其中,胚胎干细胞和诱导多能干细胞似乎最有前景。然而,在进行任何进一步的临床试验之前,对涉及生成和操纵这些细胞以及其他细胞来源的技术的安全性和有效性进行进一步的开发和优化至关重要。

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