Chen L.Cui Y.Jiang D.Ma C.Y.Tse H.-F.WUH-LIANG HWULian Q.2020-12-162020-12-1620180009-9163https://www.scopus.com/inward/record.uri?eid=2-s2.0-85041744347&doi=10.1111%2fcge.13139&partnerID=40&md5=fb3bb7add040b8a8090457dc542759a0https://scholars.lib.ntu.edu.tw/handle/123456789/525821Leigh syndrome (LS) is an inherited mitochondrial encephalopathy associated with gene mutations of oxidative phosphorylation pathway that result in early disability and death in affected young children. Currently, LS is incurable and unresponsive to many treatments, although some case reports indicate that supplements can improve the condition. Many novel therapies are being continuously tested in pre-clinical studies. In this review, we summarize the genetic basis of LS, current treatment, pre-clinical studies in animal models and the management of other mitochondrial diseases. Future therapeutical strategies and challenges are also discussed. ? 2017 John Wiley & Sons A/S. Published by John Wiley & Sons Ltdgenetics; Leigh syndrome; neurology; therapy and pre-clinical research[SDGs]SDG3dichloroacetic acid; pyruvic acid; rapamycin; ubidecarenone; vitamin; Adeno associated virus; exercise; heredity; human; hypoxia; ketogenic diet; Leigh disease; metabolic regulation; mitochondrial biogenesis; nonhuman; preclinical study; priority journal; Review; viral gene therapy; disorders of mitochondrial functions; genetic predisposition; genetics; Leigh disease; medical research; metabolism; mitochondrion; Biomedical Research; Genetic Predisposition to Disease; Humans; Leigh Disease; Mitochondria; Mitochondrial Diseasesreview10.1111/cge.13139289053872-s2.0-85041744347