Instituto de Medicina Molecular, Faculdade de Medicina da Universidade de Lisboa, Lisboa, Portugal.
J Neurochem. 2013 Nov;127(4):438-52. doi: 10.1111/jnc.12271. Epub 2013 May 8.
Several neurodegenerative diseases, such as Parkinson's disease (PD), Alzheimer's disease (AD), Huntington's disease (HD), amyotrophic lateral sclerosis (ALS), or prion diseases, are known for their intimate association with protein misfolding and aggregation. These disorders are characterized by the loss of specific neuronal populations in the brain and are highly associated with aging, suggesting a decline in proteostasis capacity may contribute to pathogenesis. Nevertheless, the precise molecular mechanisms that lead to the selective demise of neurons remain poorly understood. As a consequence, appropriate therapeutic approaches and effective treatments are largely lacking. The development of cellular and animal models that faithfully reproduce central aspects of neurodegeneration has been crucial for advancing our understanding of these diseases. Approaches involving the sequential use of different model systems, starting with simpler cellular models and ending with validation in more complex animal models, resulted in the discovery of promising therapeutic targets and small molecules with therapeutic potential. Within this framework, the simple and well-characterized eukaryote Saccharomyces cerevisiae, also known as budding yeast, is being increasingly used to study the molecular basis of several neurodegenerative disorders. Yeast provides an unprecedented toolbox for the dissection of complex biological processes and pathways. Here, we summarize how yeast models are adding to our current understanding of several neurodegenerative disorders.
几种神经退行性疾病,如帕金森病 (PD)、阿尔茨海默病 (AD)、亨廷顿病 (HD)、肌萎缩侧索硬化症 (ALS) 或朊病毒病,以其与蛋白质错误折叠和聚集的密切关联而闻名。这些疾病的特征是大脑中特定神经元群体的丧失,并且与衰老高度相关,这表明蛋白质稳态能力的下降可能导致发病机制。然而,导致神经元选择性死亡的确切分子机制仍知之甚少。因此,缺乏适当的治疗方法和有效的治疗方法。开发能够忠实地再现神经退行性变中心方面的细胞和动物模型对于我们深入了解这些疾病至关重要。涉及使用不同模型系统的顺序方法,从更简单的细胞模型开始,最后在更复杂的动物模型中进行验证,这导致发现了有希望的治疗靶点和具有治疗潜力的小分子。在这个框架内,简单且特征明确的真核生物酿酒酵母(也称为出芽酵母)越来越多地被用于研究几种神经退行性疾病的分子基础。酵母为剖析复杂的生物过程和途径提供了前所未有的工具包。在这里,我们总结了酵母模型如何帮助我们加深对几种神经退行性疾病的理解。