title: Cell-permeable protein therapy for complex I dysfunction.
abstract: Complex I deficiency is difficult to treat because of the size and complexity of the multi-subunit enzyme complex. Mutations or deletions in the mitochondrial genome are not amenable to gene therapy. However, animal studies have shown that yeast-derived internal NADH quinone oxidoreductase (Ndi1) can be delivered as a cell-permeable recombinant protein (Tat-Ndi1) that can functionally replace complex I damaged by ischemia/reperfusion. Current and future treatment of disorders affecting complex I are discussed, including the use of Tat-Ndi1.
authors.affiliation: Heart Research, Murdoch Children's Research Institute, The Royal Children's Hospital, Melbourne, Australia.
authors.forename: Salvatore
authors.initials: S
authors.lastname: Pepe
authors.forename: Robert M
authors.initials: RM
authors.lastname: Mentzer
authors.forename: Roberta A
authors.initials: RA
authors.lastname: Gottlieb
mesh_terms: D000818:Animals; D057846:Cell-Penetrating Peptides / Q000235:genetics / Q000627:therapeutic use*; D042967:Electron Transport Complex I / Q000235:genetics / Q000627:therapeutic use*; D006801:Humans; D028361:Mitochondrial Diseases / Q000188:drug therapy* / Q000201:enzymology / Q000235:genetics; D012441:Saccharomyces cerevisiae / Q000201:enzymology* / Q000235:genetics; D029701:Saccharomyces cerevisiae Proteins / Q000235:genetics / Q000627:therapeutic use*
chemical_list: D057846:Cell-Penetrating Peptides; C493237:Ndi1 protein, S cerevisiae; D029701:Saccharomyces cerevisiae Proteins; D042967:Electron Transport Complex I