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半胱胺和半胱氨酸在治疗神经退行性疾病方面的潜力。

Potential of cystamine and cysteamine in the treatment of neurodegenerative diseases.

机构信息

Centre de Recherche du CHUL (CHUQ), Axe Neurosciences, 2705 Boulevard Laurier, Québec, QC, Canada, G1V 4G2.

出版信息

Prog Neuropsychopharmacol Biol Psychiatry. 2011 Mar 30;35(2):380-9. doi: 10.1016/j.pnpbp.2010.11.023. Epub 2010 Nov 24.

Abstract

Neurodegenerative disorders are a subset of disabling pathologies characterized, in part, by a progressive and specific loss of certain brain cell populations. Current therapeutic approaches for the treatment of these disorders are mainly designed towards symptom management and do not manifestly block their typified neuronal loss. However, research conducted over the past decade has reflected the increasing interest and need to find disease-modifying molecules. Among the several neuroprotective agents emerging from experimental animal work, cystamine, as well as its reduced form cysteamine, have been identified as potential candidate drugs. Given the significant benefits observed in a Huntington's disease (HD) model, cysteamine has recently leaped to clinical trial. Here, we review the beneficial properties of these compounds as reported in animal studies, their mechanistic underpinnings, and their potential implications for the future treatment of patients suffering from neurodegenerative diseases, and more specifically for HD and Parkinson's disease (PD).

摘要

神经退行性疾病是一组致残性疾病,其部分特征是某些脑细胞群体的进行性和特异性丧失。目前治疗这些疾病的方法主要是针对症状管理,并没有明显阻止其典型的神经元丧失。然而,过去十年的研究反映出人们越来越有兴趣和需要寻找能够改变疾病进程的分子。在从实验动物研究中涌现出的几种神经保护剂中,半胱胺及其还原形式半胱氨酸已被确定为潜在的候选药物。鉴于在亨廷顿病 (HD) 模型中观察到的显著益处,半胱胺最近已跃入临床试验。在这里,我们综述了这些化合物在动物研究中的有益特性、其作用机制以及它们对未来治疗神经退行性疾病患者的潜在影响,特别是对 HD 和帕金森病 (PD) 的影响。

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