Mitochondria as a therapeutic target for common pathologies.


Murphy, Michael P; Hartley, Richard C

Publication Year 1905
Journal Nature Reviews. Drug Discovery
Pages 865-886
Volume 17
Issue 12
Issn 1474-1776
PMID 30393373.0
DOI 10.1038/nrd.2018.174

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.