Medical Research Council (MRC) Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
WestCHEM School of Chemistry, University of Glasgow, Glasgow, UK.
Nat Rev Drug Discov. 2018 Dec;17(12):865-886. doi: 10.1038/nrd.2018.174. Epub 2018 Nov 5.
Although the development of mitochondrial therapies has largely focused on diseases caused by mutations in mitochondrial DNA or in nuclear genes encoding mitochondrial proteins, it has been found that mitochondrial dysfunction also contributes to the pathology of many common disorders, including neurodegeneration, metabolic disease, heart failure, ischaemia-reperfusion injury and protozoal infections. Mitochondria therefore represent an important drug target for these highly prevalent diseases. Several strategies aimed at therapeutically restoring mitochondrial function are emerging, and a small number of agents have entered clinical trials. This Review discusses the opportunities and challenges faced for the further development of mitochondrial pharmacology for common pathologies.
虽然线粒体治疗的发展主要集中在由线粒体 DNA 突变或编码线粒体蛋白的核基因突变引起的疾病上，但人们发现线粒体功能障碍也与许多常见疾病的病理学有关，包括神经退行性疾病、代谢疾病、心力衰竭、缺血再灌注损伤和原生动物感染。因此，线粒体是这些高度流行疾病的重要药物靶点。目前正在出现几种旨在治疗性恢复线粒体功能的策略，并且少数药物已经进入临床试验。这篇综述讨论了进一步开发线粒体药理学治疗常见疾病所面临的机遇和挑战。
