Journal of IiME Volume 1 Issue 2 www.investinme.org Gene therapy for mitochondrial dysfunctions using optimized mRNA transport to the mitochondrial surface (continued) 14. Acland G, Aguirre G, Ray J, et al. Gene therapy restores vision in a canine model of childhood blindness. Nat. Genet. 2001;28:92-95. 15. Narfstrom K, Vaegan M, Katz M, Bragadottir R, Rakoczy E, Seeliger M. Assessment of structure and function over a 3-year period after gene transfer in RPE65-/- dogs. Doc. Ophthalmol. 2005;111:39-48. 16. LeMeur G KS, AJ Smith, M Weber, JY Deschamps, D Nivard, A Mendes-Madeira, N Provost, Y Pereon, Y Cherel, RR Ali, C Hamel, P Moullier, F Rolling. Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium. Gene Ther. 2007;14:292-303. 17. Yen M-Y, Wang A-G, Wei Y-H. Leber's hereditary optici neuropathy: A multifactorial disease. Prog. in Retina Eye Res. 2006;25:381-396. 18. Yu Wai Man CY CP, Griffiths PG. Optic neuropathies-importance of spatial distribution of mitochondria as well as function. Med Hypotheses 2005;65(6):1038-1042. 19. Carelli V LMC, Iommarini L, Carroccia R, Mattiazzi M, Sangiorgi S, Farne' S, Maresca A, Foscarini B, Lanzi L, Amadori M, Bellan M, Valentino ML. Mitochondrial optic neuropathies: how two genomes may kill the same cell type? Biosci. Rep. 2007;27:173-184. * Tel.: +33 1 40 01 13 66 fax: +33 1 49 28 66 63. 20. DiMauro S MM. Mitochondrial diseases: therapeutic approaches. Biosci. Rep. 2007;27:125-137. 21. Manfredi G., Fu J., Ojaimi J., et al. Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene to the nucleus. Nature Genet. 2002;30:394-399. 22. Oca-Cossio J., KenyonL., Hao H., T MC. Limitations of allotopic expression of mitochondrial genes in mammalian cells. Genetics 2003;165:707-720. 23. Bokori-Brown M, Holt IJ. Expression of Alga1 nuclear ATP synthase subunit 6 in human cells results in protein targeting to mitochondria but no assembly into ATP synthase. Rejuvenation Res. 2006;9(4):455-469. 24. Smith PM, Ross GF, Taylor RW, Turnbull DM, Lightowlers RN. Strategies for treating disorders of the mitochondrial genome. Biochem. Biophys. Acta 2004;1659:232-239. 25. Sylvestre J., Vialette S., Corral-Debrinski M., C. J. Long mRNAs coding for yeast mitochondrial proteins of prokaryotic origin localize to the vicinity of mitochondria. Genome Biology 2003;4(7):R44.1-R44.9. (continued on page 29) E-mail address: corral@st-antoine.inserm.fr Additional articles from Marisol’s team can be found at the following sites: - ScienceDirect RNA Journal http://www.sciencedirect.com/science/journal/01674889 http://www.rnajournal.org/cgi/content/full/12/7/1408 Rejuvenation research http://www.liebertonline.com/doi/abs/10.1089/rej.2006.0526 Invest in ME Charity Nr 1114035 Page 28/72

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