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清除突变蛋白作为神经退行性疾病的治疗靶点。

Clearance of mutant proteins as a therapeutic target in neurodegenerative diseases.

作者信息

Krainc Dimitri

机构信息

Department of Neurology, MassGeneral Institute for Neurodegenerative Disease, Massachusetts General Hospital, Harvard Medical School, 114 16th St, Room 2007, Charlestown, MA 02129, USA.

出版信息

Arch Neurol. 2010 Apr;67(4):388-92. doi: 10.1001/archneurol.2010.40.

DOI:10.1001/archneurol.2010.40
PMID:20385902
Abstract

Accumulation and aggregation of disease-causing proteins is a hallmark of several neurodegenerative disorders such as Parkinson, Alzheimer, and Huntington diseases. One of the main goals of research in neurodegenerative disorders has been to improve clearance of these accumulated proteins. Using the example of Huntington disease, I discuss strategies to selectively activate cellular degradation machinery to improve clearance of the mutant protein and to identify therapeutic targets for the treatment of Huntington disease and related neurodegenerative disorders.

摘要

致病蛋白的积累和聚集是帕金森病、阿尔茨海默病和亨廷顿病等几种神经退行性疾病的一个标志。神经退行性疾病研究的主要目标之一是改善这些积累蛋白的清除。以亨廷顿病为例,我讨论了选择性激活细胞降解机制以改善突变蛋白清除的策略,以及确定治疗亨廷顿病和相关神经退行性疾病的治疗靶点。

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