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通过小分子靶向新的候选基因治疗神经退行性疾病

Targeting New Candidate Genes by Small Molecules Approaching Neurodegenerative Diseases.

作者信息

Fan Hueng-Chuen, Chi Ching-Shiang, Cheng Shin-Nan, Lee Hsiu-Fen, Tsai Jeng-Dau, Lin Shinn-Zong, Harn Horng-Jyh

机构信息

Department of Pediatrics, Tung's Taichung Metroharbor Hospital, Wuchi, Taichung 435, Taiwan.

Department of Nursing, Jen-Teh Junior College of Medicine, Nursing and Management, Miaoli 356, Taiwan.

出版信息

Int J Mol Sci. 2015 Dec 25;17(1):26. doi: 10.3390/ijms17010026.

Abstract

Neurodegenerative diseases (NDs) are among the most feared of the disorders that afflict humankind for the lack of specific diagnostic tests and effective treatments. Understanding the molecular, cellular, biochemical changes of NDs may hold therapeutic promise against debilitating central nerve system (CNS) disorders. In the present review, we summarized the clinical presentations and biology backgrounds of NDs, including Parkinson's disease (PD), Huntington's disease (HD), and Alzheimer's disease (AD) and explored the role of molecular mechanisms, including dys-regulation of epigenetic control mechanisms, Ataxia-telangiectasia-mutated protein kinase (ATM), and neuroinflammation in the pathogenesis of NDs. Targeting these mechanisms may hold therapeutic promise against these devastating diseases.

摘要

神经退行性疾病(NDs)是困扰人类的最可怕的疾病之一,因为缺乏特异性诊断测试和有效治疗方法。了解NDs的分子、细胞和生化变化可能为治疗使人衰弱的中枢神经系统(CNS)疾病带来希望。在本综述中,我们总结了NDs的临床表现和生物学背景,包括帕金森病(PD)、亨廷顿舞蹈症(HD)和阿尔茨海默病(AD),并探讨了分子机制的作用,包括表观遗传控制机制失调、共济失调毛细血管扩张突变蛋白激酶(ATM)和神经炎症在NDs发病机制中的作用。针对这些机制可能为治疗这些毁灭性疾病带来希望。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/79ac/4730273/f105f9c7356e/ijms-17-00026-g001.jpg

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