title: Translational Medicine. A target for pharmacological intervention in an untreatable human disease.
authors.affiliation: The Albert Einstein College of Medicine, 1301 Morris Park Avenue, Room 469, Bronx, NY 10462, USA. simon.johnson@einstein.yu.edu.
authors.forename: Simon C
authors.initials: SC
authors.lastname: Johnson
mesh_terms: D000818:Animals; D031204:Caloric Restriction; D004195:Disease Models, Animal; D004305:Dose-Response Relationship, Drug; D042967:Electron Transport Complex I / Q000235:genetics; D006801:Humans; D007888:Leigh Disease / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D051379:Mice; D058990:Molecular Targeted Therapy; D012441:Saccharomyces cerevisiae / Q000235:genetics; D020123:Sirolimus / Q000008:administration & dosage*; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors* / Q000378:metabolism
chemical_list: C534449:Ndufs4 protein, mouse; D058570:TOR Serine-Threonine Kinases; D042967:Electron Transport Complex I; D020123:Sirolimus