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阿尔茨海默病:表观遗传学治疗的潜力和当前的临床候选药物。

Alzheimer's disease: the potential of epigenetic treatments and current clinical candidates.

机构信息

Leonard Wolfson Experimental Neurology Centre, Institute of Neurology, University College London, London WC1N 3BG, UK.

School of Biomedical Sciences, Faculty of Biological Sciences, University of Leeds, Leeds LS2 9JT, UK.

出版信息

Neurodegener Dis Manag. 2020 Jun;10(3):543-558. doi: 10.2217/nmt-2019-0034. Epub 2020 Jun 17.

DOI:10.2217/nmt-2019-0034
PMID:32552286
Abstract

Alzheimer's disease is a progressive and fatal neurodegenerative disease affecting 50 million people worldwide, characterized by memory loss and neuronal degeneration. Current treatments have limited efficacy and there is no cure. Alzheimer's is likely caused by a combination of factors, providing several potential therapeutic targets. One area of interest is the epigenetic regulation of gene expression within the brain. Epigenetic marks, including DNA methylation and histone modifications, show consistent changes with age and in those with Alzheimer's. Some epigenetic regulation has been linked to disease pathology and progression and are the focus of current research. Epigenetic regulators might make promising therapeutic targets yet challenges need to be overcome to generate an efficacious drug lacking deleterious side effects.

摘要

阿尔茨海默病是一种影响全球 5000 万人的进行性和致命性神经退行性疾病,其特征是记忆丧失和神经元退化。目前的治疗方法疗效有限,尚无治愈方法。阿尔茨海默病可能是由多种因素共同作用引起的,这为治疗提供了几个潜在的靶点。一个研究热点是大脑内基因表达的表观遗传调控。表观遗传标记,包括 DNA 甲基化和组蛋白修饰,随着年龄的增长和阿尔茨海默病患者的变化而发生一致的变化。一些表观遗传调控与疾病病理和进展有关,是当前研究的重点。表观遗传调节剂可能成为有前途的治疗靶点,但仍需要克服挑战,以生成一种有效且无有害副作用的药物。

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