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表观遗传学修饰作为亨廷顿病的新治疗靶点。

Epigenetic modifications as novel therapeutic targets for Huntington's disease.

机构信息

State Key Laboratory of Biomembrane & Membrane Biotechnology, Institute of Zoology, Chinese Academy of Sciences, Beijing 100101, China.

出版信息

Epigenomics. 2014 Jun;6(3):287-97. doi: 10.2217/epi.14.19.

Abstract

Huntington's disease is a late-onset, autosomal dominant neurodegenerative disorder characterized by motor, cognitive and psychiatric symptomatology. The earliest stage of Huntington's disease is marked by alterations in gene expression, which partially results from dysregulated epigenetic modifications. In past decades, altered epigenetic markers including histone modifications (acetylation, methylation, ubiquitylation and phosphorylation) and DNA modifications (cytosine methylation and hydroxymethylation) have been reported as important epigenetic features in patients and multiple animal models of Huntington's disease. Drugs aimed to correct some of those alterations have shown promise in treating Huntington's disease. This article discusses the field of epigenetics for potential Huntington's disease interventions and presents the most recent findings in this area.

摘要

亨廷顿病是一种迟发性、常染色体显性神经退行性疾病,其特征为运动、认知和精神症状。亨廷顿病的最早阶段表现为基因表达的改变,这部分是由于表观遗传修饰的失调所致。在过去的几十年中,改变的表观遗传标记物,包括组蛋白修饰(乙酰化、甲基化、泛素化和磷酸化)和 DNA 修饰(胞嘧啶甲基化和羟甲基化),已被报道为亨廷顿病患者和多种动物模型中的重要表观遗传特征。旨在纠正其中一些改变的药物在治疗亨廷顿病方面显示出了希望。本文讨论了针对亨廷顿病干预的表观遗传学领域,并介绍了该领域的最新发现。

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