Laboratory of Integrative and Systems Physiology, École Polytechnique Fédérale de Lausanne, CH-1015 Lausanne, Switzerland.
Nat Rev Drug Discov. 2013 Jun;12(6):465-83. doi: 10.1038/nrd4023. Epub 2013 May 13.
Mitochondrial dysfunction is not only a hallmark of rare inherited mitochondrial disorders but also implicated in age-related diseases, including those that affect the metabolic and nervous system, such as type 2 diabetes and Parkinson's disease. Numerous pathways maintain and/or restore proper mitochondrial function, including mitochondrial biogenesis, mitochondrial dynamics, mitophagy and the mitochondrial unfolded protein response. New and powerful phenotypic assays in cell-based models as well as multicellular organisms have been developed to explore these different aspects of mitochondrial function. Modulating mitochondrial function has therefore emerged as an attractive therapeutic strategy for several diseases, which has spurred active drug discovery efforts in this area.
线粒体功能障碍不仅是罕见遗传性线粒体疾病的标志,而且与年龄相关的疾病有关,包括影响代谢和神经系统的疾病,如 2 型糖尿病和帕金森病。许多途径可维持和/或恢复适当的线粒体功能,包括线粒体生物发生、线粒体动力学、线粒体自噬和线粒体未折叠蛋白反应。已经开发出基于细胞模型以及多细胞生物的新型强大表型测定法来探索线粒体功能的这些不同方面。因此,调节线粒体功能已成为几种疾病的有吸引力的治疗策略,这促使该领域积极开展药物发现工作。