Progress and prospects: Gene therapy for mitochondrial DNA disease

Gene Therapy

Volumn 15, Issue 14, 2008, Pages 1017-1023

  Kyriakouli, D S ^a   Boesch, P ^a   Taylor, R W ^a   Lightowlers, R N ^a  

^a NEWCASTLE UNIVERSITY   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE TRIPHOSPHATASE; ADENOSINE TRIPHOSPHATASE 6; ADENOSINE TRIPHOSPHATASE 8; ALTERNATIVE OXIDASE; CELL MEMBRANE CROSSING OLIGOMER; CIS ACTING ELEMENT; CYTOCHROME C OXIDASE 10; MITOCHONDRIAL DNA; NUCLEIC ACID; OLIGOMER; OXIDOREDUCTASE; PEPTIDE NUCLEIC ACID; PROTEIN NDI1; PROTEIN SOD2; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE DEHYDROGENASE; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE DEHYDROGENASE (UBIQUINONE); RESTRICTION ENDONUCLEASE; TRANSFER RNA; ZINC FINGER PROTEIN;

ALLOTYPE; ANTIOXIDANT ACTIVITY; DRUG DESIGN; ENDURANCE; FERTILIZATION IN VITRO; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENETIC ENGINEERING; GENETIC TRANSFECTION; GERM LINE GENE THERAPY; HETEROPLASMY; HUMAN; KINESIOTHERAPY; LIPOSOMAL GENE DELIVERY SYSTEM; MITOCHONDRIAL DNA DISORDER; MITOCHONDRIAL GENE; NONHUMAN; OXIDATIVE PHOSPHORYLATION; PARKINSON DISEASE; PRIORITY JOURNAL; REVIEW; SACCHAROMYCES CEREVISIAE; VIRAL GENE DELIVERY SYSTEM;

DNA, MITOCHONDRIAL; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; MITOCHONDRIAL DISEASES; MUTATION;

EID: 46449117734     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2008.91     Document Type: Review

References (21)

1. McFarland R, Taylor RW, Turnbull DM, Mitochondrial disease its impact, aetiology, and pathology. Curr Top Dev Biol 2007; 77: 113-155.
2. Nagley P, Farrell LB, Gearing DP, Nero D, Meltzer S, Devenish RJ. Assembly of functional proton-translocating ATTPase complex in yeast mitochondria with cytoplasmically syriflkesized subunit 8, a polypeptide normally encoded within the organelle. Proc Natl Acad Sci USA 1988; 85: 2091-2095.
3. Bokori-Brown M, Holt IJ. Expression of algal nuclear ATP synthase subunit 6 in human cells results in protein targeting to mitochondria but no assembly into ATP syrithase. Rejuvenation Res 2006; 9 455-469.
4. Kaltimbacher V, Bonnet C, Lecoeuvre G, Forster V, Sahel JA, Corral-Debrinski M. mRNA localization to the mitochondrial surface allows the efficient translocation inside the organelle of a nuclear recoded ATP6 protein. RNA 2006; 12: 1408-1417.
5. Bonnet C, Kaltimbacher V, Ellouze S, Augustin S, Benit P, Forster V et al. Allotopic mRNA localization to the mitochondrial surface rescues respiratory chain defects in fibroblasts harboring mitochondrial DNA mutations affecting complex I or V subunits. Rejuvenation Res 2007;, 10: 127-144.
6. Hakkaart GA, Dassa EP, Jacobs HT, Rustin P. Allotopic expression of a mitochondrial alternative oxidase confers cyanide resistance to human cell respiration. EMBO Rep 2006; 7: 341-345.
7. Seo BB, Nakamaru-Ogiso E, Flotte TR, Matsuno-Yagi A, Yagi T. In vivo complementation of complex I by the yeast Ndi1 enzyme. Possible applicadon for treatment of Parkinscn disease. J Biol Chem 2006; 281: 14250-14255.
8. Marella M, Seo BB, Nakamaru-Ogiso E, Greenamyre JT, Matsuno-Yagi A, Yagi T. Protection by the INDI1 gene against neurodegeneration in a rotenone rat model of Parkinson's disease. PLoS ONE 2008; 3: E1433.
9. Tarassov I, Kamenski P, Kolesnikova O, Karicheva O, Martin RP, Krasheninnikov IA et al. Import of nuclear DNA-encoded RNAs into mitochondria and mitochondrial translation. Cell Cycle 2007; 6 2473-2477.
10. Mukherjee S, Basu S, Home P, Dhar G, Adhya S. Necessary and sufficient factors for the import of transfer RNA into the kinetoplast mitochondrion. EMBO Rep 2007; 8: 589-595.
11. Mahata B, Mukherjee S, Mishra S, Bandyopadhyay A, Adhya S. Functional delivery of a cytosolic tRNA into mutant mitochondria of human cells. Science 2006; 314: 471-474.
12. Mukherjee S, Mahata B, Mahato B, Adhya S. Targeted mRNA degradation by complex-mediated delivery of antisense RNAs to intracellular human mitochondria. Hum Mol Genet 2008; 17: 1292-1298.
13. Koulintchenko M, Temperley RJ, Mason PA, Dietrich A, Lightowlers RN. Natural competence of mammalian mitochondria allows the molecular investigation of mitochondrial gene expression. Hum Mol Genet 2006; 15: 143-154.
14. Katrangi E, D'Souza G, Boddapati SV, Kulawiec M, Singh KK, Bigger B et al. Xenogenic transfer of isolated murine mitochondria into human rho(0) cells can improve respiratory function. Rejuvenation Res 2007; 10: 561-570.
15. Yamada Y, Akita H, Kamiya H, Kogure K, Yamamoto T, Shinohara Y et al MITO-Porter: A liposome-based carrier system for delivery of macro-molecules into mitochondria via membrane fusion. Biochim Biophys Acta 2008; 1778: 423-432.
16. Taivassalo T, Gardner JL, Taylor RW, Schaefer AM, Newman J, Barron MJ et al. Endurance training and detraining in mitochondrial myopathies due to single large-scale mtDNA deletions. Brain 2006; 129 (Part 12): 3391-3401.
17. Taivassalo T, Gardner JL, Taylor RW, Schaefer AM, Haller RG, Turnbull DM. Resistance exercise training in mitochondrial myopathy due to single, large-scale mtDNA deletions: Implications for therapy. Neuromuscul Disord 2007, 17: 829.
18. Bacman SR, Williams SL, Hernandez D, Moraes CT. Modulating mtDNA heteroplasmy by mitochondria-targeted restriction endonucleases in a 'differential multiple cleavage-site' model. Gene Ther 2007; 14 1309-1318.
19. Minczuk M, Papworth MA, Kolasinska P, Murphy MP, Klug A. Sequence-specific modification of mitochondrial DNA using a chimeric zinc finger methylase. Proc Natl Acad Sci USA 2006; 103: 19689-19694.
20. Brown DT, Herbert M, Lamb VK, Chinnery PF, Taylor RW, Lightowlers RN et al. Transmission of mitochondrial DNA disorders: Possibilities for the future. Lancet 2006; 368: 87-89.
21. Sato A, Kono T, Nakada K, Ishikawa K, Inoue S, Yonekawa H et al. Gene therapy for progeny of mito-mice carrying pathogenic mtDNA by nuclear transplantation. Proc Natl Acad Sci USA 102: 16765-16770.