Unraveling protein misfolding diseases using model systems.
Unraveling protein misfolding diseases using model systems.
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DOI：
10.4155/fso.15.41
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发表时间：
2015-09
影响因子：
2.5
通讯作者：
Morano KA
中科院分区：
文献类型：
--
作者：
Peffer S;Cope K;Morano KA
长期以来，实验模型系统一直被用于探索导致人类疾病的病理学的原因、后果和机制。理想情况下，可以利用这些信息来制定治疗策略或治疗方法，以对抗疾病进展。在蛋白质错误折叠疾病的情况下，已经开发了广泛的模型系统来研究包括亨廷顿病、帕金森病、阿尔茨海默病以及肌萎缩性侧索硬化症在内的疾病的不同方面。这些系统的实用性与进化的复杂性广泛相关：小动物模型，如啮齿动物和果蝇是适当的药理学建模和认知/行为评估，蛔虫秀丽隐杆线虫允许组织特异性疾病特征的分析，和单细胞生物，如酵母酿酒酵母和细菌大肠杆菌是理想的分子研究。在这一章中，我们强调我们的理解模型系统提供的蛋白质错误折叠/展开疾病的关键进展。
Experimental model systems have long been used to probe the causes, consequences and mechanisms of pathology leading to human disease. Ideally, such information can be exploited to inform the development of therapeutic strategies or treatments to combat disease progression. In the case of protein misfolding diseases, a wide range of model systems have been developed to investigate different aspects of disorders including Huntington's disease, Parkinson's disease, Alzheimer's disease as well as amyotrophic lateral sclerosis. Utility of these systems broadly correlates with evolutionary complexity: small animal models such as rodents and the fruit fly are appropriate for pharmacological modeling and cognitive/behavioral assessment, the roundworm Caenorhabditis elegans allows analysis of tissue-specific disease features, and unicellular organisms such as the yeast Saccharomyces cerevisiae and the bacterium Escherichia coli are ideal for molecular studies. In this chapter, we highlight key advances in our understanding of protein misfolding/unfolding disease provided by model systems.