Therapeutic Approaches for Inhibition of Protein Aggregation in Huntington's Disease.
Therapeutic Approaches for Inhibition of Protein Aggregation in Huntington's Disease.
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DOI：
10.5607/en.2014.23.1.36
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发表时间：
2014-03
影响因子：
2.4
通讯作者：
Kim KT
中科院分区：
文献类型：
--
作者：
Kim S;Kim KT
亨廷顿病（Huntington 'sdisease，HD）是一种迟发性、进行性神经退行性疾病，由含有扩展的多聚谷氨酰胺的突变亨廷顿蛋白聚集引起。分子伴侣在HD的早期阶段调节聚集，并且在HD的动物模型中被认为是最有效的神经变性保护剂。在过去的几十年中，许多研究表明分子伴侣通过polyQ介导的毒性减轻致病症状。此外，伴侣诱导药物和抗聚集药物对疾病症状具有有益作用。本文就分子伴侣在HD动物模型中的作用、分子伴侣的表达调控及抗聚集药物的研究进展作一综述。
Huntington's disease (HD) is a late-onset and progressive neurodegenerative disorder that is caused by aggregation of mutant huntingtin protein which contains expanded-polyglutamine. The molecular chaperones modulate the aggregation in early stage and known for the most potent protector of neurodegeneration in animal models of HD. Over the past decades, a number of studies have demonstrated molecular chaperones alleviate the pathogenic symptoms by polyQ-mediated toxicity. Moreover, chaperone-inducible drugs and anti-aggregation drugs have beneficial effects on symptoms of disease. Here, we focus on the function of molecular chaperone in animal models of HD, and review the recent therapeutic approaches to modulate expression and turn-over of molecular chaperone and to develop anti-aggregation drugs.