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Targeting protein aggregation in neurodegeneration--lessons from polyglutamine disorders.

机译：在神经退行性疾病中靶向蛋白质聚集-来自聚谷氨酰胺疾病的经验教训。

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摘要

Polyglutamine diseases, such as Huntington's disease, are among the most common inherited neurodegenerative disorders. They share salient clinical and pathological features with major sporadic neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease and amyotropic lateral sclerosis. Over the last decade, protein aggregation has emerged as a common pathological hallmark in neurodegenerative diseases and has, therefore, attracted considerable attention as a likely shared therapeutic target. Because of their clearly defined molecular genetic basis, polyglutamine diseases have allowed researchers to dissect the relationship between neurodegeneration and protein aggregation. In this review, the authors discuss recent progress in understanding polyglutamine-mediated neurotoxicity, and discuss the most promising therapeutic strategies being developed in the polyglutamine diseases and related neurodegenerative disorders.
机译：聚谷氨酰胺病，例如亨廷顿氏病，是最常见的遗传性神经退行性疾病。它们与主要的偶发性神经退行性疾病（例如阿尔茨海默氏病，帕金森氏病和肌萎缩性侧索硬化症）具有明显的临床和病理特征。在过去的十年中，蛋白质聚集已成为神经退行性疾病中常见的病理学标志，因此作为一种可能的共有治疗靶点引起了广泛的关注。由于其明确的分子遗传基础，聚谷氨酰胺疾病使研究人员能够剖析神经退行性病变与蛋白质聚集之间的关系。在这篇综述中，作者讨论了在理解聚谷氨酰胺介导的神经毒性方面的最新进展，并讨论了在聚谷氨酰胺疾病和相关神经退行性疾病中开发的最有希望的治疗策略。

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