Key Laboratory of Molecular Biophysics of the Ministry of Education, College of Life Science and Technology, Huazhong University of Science and Technology, Wuhan 430074, China.
Cells. 2024 Jan 3;13(1):99. doi: 10.3390/cells13010099.
Amyotrophic Lateral Sclerosis (ALS) is a debilitating neurodegenerative condition characterized by the progressive degeneration of motor neurons. Despite extensive research in various model animals, the cellular signal mechanisms of ALS remain elusive, impeding the development of efficacious treatments. Among these models, a well-characterized and diminutive organism, (), has emerged as a potent tool for investigating the molecular and cellular dimensions of ALS pathogenesis. This review summarizes the contributions of models to our comprehension of ALS, emphasizing pivotal findings pertaining to genetics, protein aggregation, cellular pathways, and potential therapeutic strategies. We analyze both the merits and constraints of the system in the realm of ALS research and point towards future investigations that could bridge the chasm between foundational discoveries and clinical applications.
肌萎缩侧索硬化症(ALS)是一种使人虚弱的神经退行性疾病,其特征是运动神经元的进行性退化。尽管在各种模式动物中进行了广泛的研究,但 ALS 的细胞信号机制仍难以捉摸,阻碍了有效治疗方法的发展。在这些模型中,一个特征明确且体型微小的生物体(秀丽隐杆线虫)已成为研究 ALS 发病机制的分子和细胞层面的有力工具。
本篇综述总结了秀丽隐杆线虫模型对我们理解 ALS 的贡献,重点介绍了与遗传学、蛋白质聚集、细胞途径和潜在治疗策略相关的关键发现。我们分析了秀丽隐杆线虫系统在 ALS 研究中的优缺点,并指出了未来的研究方向,这些研究可能会弥合基础发现与临床应用之间的差距。