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Volumn 109, Issue 20, 2012, Pages

Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE TRIPHOSPHATE; MITOCHONDRIAL DNA; PROTEIN VP2; REDUCED NICOTINAMIDE ADENINE DINUCLEOTIDE DEHYDROGENASE (UBIQUINONE);

EID: 84861203685     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1119577109     Document Type: Article
Times cited : (163)

References (43)
  • 1
    • 0033525773 scopus 로고    scopus 로고
    • Mitochondrial diseases in man and mouse
    • Wallace DC (1999) Mitochondrial diseases in man and mouse. Science 283:1482-1488.
    • (1999) Science , vol.283 , pp. 1482-1488
    • Wallace, D.C.1
  • 2
    • 23644433329 scopus 로고    scopus 로고
    • The mitochondrial genome in human adaptive radiation and disease: On the road to therapeutics and performance enhancement
    • Wallace DC (2005) The mitochondrial genome in human adaptive radiation and disease: On the road to therapeutics and performance enhancement. Gene 354:169-180.
    • (2005) Gene , vol.354 , pp. 169-180
    • Wallace, D.C.1
  • 3
    • 33645460107 scopus 로고    scopus 로고
    • Mitochondrial disease: Powerhouse of disease
    • Lane N (2006) Mitochondrial disease: Powerhouse of disease. Nature 440:600-602.
    • (2006) Nature , vol.440 , pp. 600-602
    • Lane, N.1
  • 4
    • 13944278132 scopus 로고    scopus 로고
    • Mitochondria, oxidants, and aging
    • DOI 10.1016/j.cell.2005.02.001
    • Balaban RS, Nemoto S, Finkel T (2005) Mitochondria, oxidants, and aging. Cell 120: 483-495. (Pubitemid 40269763)
    • (2005) Cell , vol.120 , Issue.4 , pp. 483-495
    • Balaban, R.S.1    Nemoto, S.2    Finkel, T.3
  • 5
    • 58149349815 scopus 로고    scopus 로고
    • Mitochondrial medicine: Entering the era of treatment
    • Koene S, Smeitink J (2009) Mitochondrial medicine: Entering the era of treatment. J Intern Med 265:193-209.
    • (2009) J Intern Med , vol.265 , pp. 193-209
    • Koene, S.1    Smeitink, J.2
  • 6
  • 7
    • 0033917385 scopus 로고    scopus 로고
    • Mutations in mtDNA: Are we scraping the bottom of the barrel?
    • DiMauro S, Andreu AL (2000) Mutations in mtDNA: Are we scraping the bottom of the barrel? Brain Pathol 10:431-441. (Pubitemid 30431296)
    • (2000) Brain Pathology , vol.10 , Issue.3 , pp. 431-441
    • DiMauro, S.1    Andreu, A.L.2
  • 8
    • 46449117734 scopus 로고    scopus 로고
    • Progress and prospects: Gene therapy for mitochondrial DNA disease
    • DOI 10.1038/gt.2008.91, PII GT200891
    • Kyriakouli DS, Boesch P, Taylor RW, Lightowlers RN (2008) Progress and prospects: Gene therapy for mitochondrial DNA disease. Gene Ther 15:1017-1023. (Pubitemid 351927692)
    • (2008) Gene Therapy , vol.15 , Issue.14 , pp. 1017-1023
    • Kyriakouli, D.S.1    Boesch, P.2    Taylor, R.W.3    Lightowlers, R.N.4
  • 9
    • 53049094677 scopus 로고    scopus 로고
    • How can we treat mitochondrial encephalomyopathies? Approaches to therapy
    • Horvath R, Gorman G, Chinnery PF (2008) How can we treat mitochondrial encephalomyopathies? Approaches to therapy. Neurotherapeutics 5:558-568.
    • (2008) Neurotherapeutics , vol.5 , pp. 558-568
    • Horvath, R.1    Gorman, G.2    Chinnery, P.F.3
  • 10
    • 68149099553 scopus 로고    scopus 로고
    • The pathophysiology of mitochondrial disease as modeled in the mouse
    • Wallace DC, Fan W (2009) The pathophysiology of mitochondrial disease as modeled in the mouse. Genes Dev 23:1714-1736.
    • (2009) Genes Dev , vol.23 , pp. 1714-1736
    • Wallace, D.C.1    Fan, W.2
  • 11
    • 64349099993 scopus 로고    scopus 로고
    • The role of abnormal mitochondrial dynamics in the pathogenesis of Alzheimer's disease
    • Wang X, et al. (2009) The role of abnormal mitochondrial dynamics in the pathogenesis of Alzheimer's disease. J Neurochem 109(Suppl 1):153-159.
    • (2009) J Neurochem , vol.109 , Issue.SUPPL. 1 , pp. 153-159
    • Wang, X.1
  • 13
    • 0030806721 scopus 로고    scopus 로고
    • Leber hereditary optic neuropathy: Mitochondrial mutations and degeneration of the optic nerve
    • Howell N (1997) Leber hereditary optic neuropathy: Mitochondrial mutations and degeneration of the optic nerve. Vision Res 37:3495-3507.
    • (1997) Vision Res , vol.37 , pp. 3495-3507
    • Howell, N.1
  • 15
    • 0034691280 scopus 로고    scopus 로고
    • Resolution of the membrane domain of bovine complex I into subcomplexes: Implications for the structural organization of the enzyme
    • DOI 10.1021/bi000335t
    • Sazanov LA, Peak-Chew SY, Fearnley IM, Walker JE (2000) Resolution of the membrane domain of bovine complex I into subcomplexes: Implications for the structural organization of the enzyme. Biochemistry 39:7229-7235. (Pubitemid 30413113)
    • (2000) Biochemistry , vol.39 , Issue.24 , pp. 7229-7235
    • Sazanov, L.A.1    Peak-Chew, S.Y.2    Fearnley, I.M.3    Walker, J.E.4
  • 16
    • 0024536726 scopus 로고
    • DNA-protein conjugates can enter mitochondria via the protein import pathway
    • DOI 10.1038/338170a0
    • Vestweber D, Schatz G (1989) DNA-protein conjugates can enter mitochondria via the protein import pathway. Nature 338:170-172. (Pubitemid 19074109)
    • (1989) Nature , vol.338 , Issue.6211 , pp. 170-172
    • Vestweber, D.1    Schatz, G.2
  • 17
    • 0031054053 scopus 로고    scopus 로고
    • Introduction of plasmid DNA into isolated mitochondria by electroporation: A novel approach toward gene correction for mitochondrial disorders
    • DOI 10.1074/jbc.272.8.5342
    • Collombet JM, Wheeler VC, Vogel F, Coutelle C (1997) Introduction of plasmid DNA into isolated mitochondria by electroporation. A novel approach toward gene correction for mitochondrial disorders. J Biol Chem 272:5342-5347. (Pubitemid 27090099)
    • (1997) Journal of Biological Chemistry , vol.272 , Issue.8 , pp. 5342-5347
    • Collombet, J.-M.1    Wheeler, V.C.2    Vogel, F.3    Coutelle, C.4
  • 18
    • 0037335660 scopus 로고    scopus 로고
    • Efficient cloning and engineering of entire mitochondrial genomes in Escherichia coli and transfer into transcriptionally active mitochondria
    • DOI 10.1093/nar/gkg228
    • Yoon YG, Koob MD (2003) Efficient cloning and engineering of entire mitochondrial genomes in Escherichia coli and transfer into transcriptionally active mitochondria. Nucleic Acids Res 31:1407-1415. (Pubitemid 36304902)
    • (2003) Nucleic Acids Research , vol.31 , Issue.5 , pp. 1407-1415
    • Yoon, Y.G.1    Koob, M.D.2
  • 19
    • 10244252759 scopus 로고    scopus 로고
    • Development of mitochondrial gene replacement therapy
    • DOI 10.1023/B:JOBB.0000041773.20072.9e
    • Khan SM, Bennett JP, Jr. (2004) Development of mitochondrial gene replacement therapy. J Bioenerg Biomembr 36:387-393. (Pubitemid 39619846)
    • (2004) Journal of Bioenergetics and Biomembranes , vol.36 , Issue.4 SPEC.ISS. , pp. 387-393
    • Khan, S.M.1    Bennett Jr., J.P.2
  • 20
    • 84856230357 scopus 로고    scopus 로고
    • DNA delivery to mitochondria: Sequence specificity and energy enhancement
    • Ibrahim N, et al. (2011) DNA delivery to mitochondria: Sequence specificity and energy enhancement. Pharm Res 28:2871-2882.
    • (2011) Pharm Res , vol.28 , pp. 2871-2882
    • Ibrahim, N.1
  • 21
    • 80053118301 scopus 로고    scopus 로고
    • Mitochondrial transfection for studying organellar DNA repair, genome maintenance and aging
    • Mileshina D, et al. (2011) Mitochondrial transfection for studying organellar DNA repair, genome maintenance and aging. Mech Ageing Dev 132:412-423.
    • (2011) Mech Ageing Dev , vol.132 , pp. 412-423
    • Mileshina, D.1
  • 22
    • 67949099046 scopus 로고    scopus 로고
    • Intra- and inter-molecular recombination of mitochondrial DNA after in vivo induction of multiple double-strand breaks
    • Bacman SR, Williams SL, Moraes CT (2009) Intra- and inter-molecular recombination of mitochondrial DNA after in vivo induction of multiple double-strand breaks. Nucleic Acids Res 37:4218-4226.
    • (2009) Nucleic Acids Res , vol.37 , pp. 4218-4226
    • Bacman, S.R.1    Williams, S.L.2    Moraes, C.T.3
  • 23
    • 29644437055 scopus 로고    scopus 로고
    • Natural competence of mammalian mitochondria allows the molecular investigation of mitochondrial gene expression
    • DOI 10.1093/hmg/ddi435
    • Koulintchenko M, Temperley RJ, Mason PA, Dietrich A, Lightowlers RN (2006) Natural competence of mammalian mitochondria allows the molecular investigation of mitochondrial gene expression. Hum Mol Genet 15:143-154. (Pubitemid 43020112)
    • (2006) Human Molecular Genetics , vol.15 , Issue.1 , pp. 143-154
    • Koulintchenko, M.1    Temperley, R.J.2    Mason, P.A.3    Dietrich, A.4    Lightowlers, R.N.5
  • 24
    • 0344522705 scopus 로고    scopus 로고
    • Plant mitochondria actively import DNA via the permeability transition pore complex
    • DOI 10.1093/emboj/cdg128
    • Koulintchenko M, Konstantinov Y, Dietrich A (2003) Plant mitochondria actively import DNA via the permeability transition pore complex. EMBO J 22:1245-1254. (Pubitemid 36362688)
    • (2003) EMBO Journal , vol.22 , Issue.6 , pp. 1245-1254
    • Koulintchenko, M.1    Konstantinov, Y.2    Dietrich, A.3
  • 25
    • 33847618832 scopus 로고    scopus 로고
    • Mitochondrial drug delivery and mitochondrial disease therapy - An approach to liposome-based delivery targeted to mitochondria
    • DOI 10.1016/j.mito.2006.12.003, PII S1567724906002443, Mitochondria and Life
    • Yamada Y, Akita H, Kogure K, Kamiya H, Harashima H (2007) Mitochondrial drug delivery and mitochondrial disease therapy-An approach to liposome-based delivery targeted to mitochondria. Mitochondrion 7:63-71. (Pubitemid 46351977)
    • (2007) Mitochondrion , vol.7 , Issue.1-2 , pp. 63-71
    • Yamada, Y.1    Akita, H.2    Kogure, K.3    Kamiya, H.4    Harashima, H.5
  • 27
    • 0024311525 scopus 로고
    • Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression
    • Samulski RJ, Chang LS, Shenk T (1989) Helper-free stocks of recombinant adenoassociated viruses: Normal integration does not require viral gene expression. J Virol 63:3822-3828. (Pubitemid 19205454)
    • (1989) Journal of Virology , vol.63 , Issue.9 , pp. 3822-3828
    • Samulski, R.J.1    Chang, L.-S.2    Shenk, T.3
  • 28
    • 2642565909 scopus 로고    scopus 로고
    • Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus
    • Warrington KH, Jr., et al. (2004) Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus. J Virol 78: 6595-6609.
    • (2004) J Virol , vol.78 , pp. 6595-6609
    • Warrington Jr., K.H.1
  • 30
    • 0036544631 scopus 로고    scopus 로고
    • Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus
    • Manfredi G, et al. (2002) Rescue of a deficiency in ATP synthesis by transfer of MTATP6, a mitochondrial DNA-encoded gene, to the nucleus. Nat Genet 30:394-399.
    • (2002) Nat Genet , vol.30 , pp. 394-399
    • Manfredi, G.1
  • 32
    • 54849162100 scopus 로고    scopus 로고
    • Self-complementary AAV vectors; advances and applications
    • McCarty DM (2008) Self-complementary AAV vectors; advances and applications. Mol Ther 16:1648-1656.
    • (2008) Mol Ther , vol.16 , pp. 1648-1656
    • McCarty, D.M.1
  • 33
    • 24644461049 scopus 로고    scopus 로고
    • Caspase-independent death of Leber's hereditary optic neuropathy cybrids is driven by energetic failure and mediated by AIF and Endonuclease G
    • DOI 10.1007/s10495-005-0742-5
    • Zanna C, et al. (2005) Caspase-independent death of Leber's hereditary optic neuropathy cybrids is driven by energetic failure and mediated by AIF and Endonuclease G. Apoptosis 10:997-1007. (Pubitemid 41285578)
    • (2005) Apoptosis , vol.10 , Issue.5 , pp. 997-1007
    • Zanna, C.1    Ghelli, A.2    Porcelli, A.M.3    Martinuzzi, A.4    Carelli, V.5    Rugolo, M.6
  • 34
    • 70349238784 scopus 로고    scopus 로고
    • Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system
    • Guy J, et al. (2009) Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Invest Ophthalmol Vis Sci 50: 4205-4214.
    • (2009) Invest Ophthalmol Vis Sci , vol.50 , pp. 4205-4214
    • Guy, J.1
  • 36
    • 50949130569 scopus 로고    scopus 로고
    • Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction
    • Ellouze S, et al. (2008) Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction. Am J Hum Genet 83:373-387.
    • (2008) Am J Hum Genet , vol.83 , pp. 373-387
    • Ellouze, S.1
  • 37
    • 0038365420 scopus 로고    scopus 로고
    • Targeted delivery of DNA to the mitochondrial compartment via import sequence-conjugated peptide nucleic acid
    • DOI 10.1016/S1525-0016(03)00037-6
    • Flierl A, et al. (2003) Targeted delivery of DNA to the mitochondrial compartment via import sequence-conjugated peptide nucleic acid. Mol Ther 7:550-557. (Pubitemid 36553425)
    • (2003) Molecular Therapy , vol.7 , Issue.4 , pp. 550-557
    • Flierl, A.1    Jackson, C.2    Cottrell, B.3    Murdock, D.4    Seibel, P.5    Wallace, D.C.6
  • 39
    • 77955480347 scopus 로고    scopus 로고
    • Mitochondrial matrix delivery using MITO-Porter, a liposome-based carrier that specifies fusion with mitochondrial membranes
    • Yasuzaki Y, Yamada Y, Harashima H (2010) Mitochondrial matrix delivery using MITO-Porter, a liposome-based carrier that specifies fusion with mitochondrial membranes. Biochem Biophys Res Commun 397:181-186.
    • (2010) Biochem Biophys Res Commun , vol.397 , pp. 181-186
    • Yasuzaki, Y.1    Yamada, Y.2    Harashima, H.3
  • 40
    • 70349284435 scopus 로고    scopus 로고
    • Mitochondrial gene replacement in primate offspring and embryonic stem cells
    • Tachibana M, et al. (2009) Mitochondrial gene replacement in primate offspring and embryonic stem cells. Nature 461:367-372.
    • (2009) Nature , vol.461 , pp. 367-372
    • Tachibana, M.1
  • 42
    • 0034091924 scopus 로고    scopus 로고
    • Production and purification of recombinant AAV vectors
    • ed Palczewski K (Academic, New York)
    • HauswirthWW, et al. (2000) Production and purification of recombinant AAV vectors. Vertebrate Phototransduction and the Visual Cycle, ed Palczewski K (Academic, New York), Vol. 316, pp 743-761.
    • (2000) Vertebrate Phototransduction and the Visual Cycle , vol.316 , pp. 743-761
    • Hauswirth, W.W.1
  • 43
    • 56249142321 scopus 로고    scopus 로고
    • Electrophoresis techniques to investigate defects in oxidative phosphorylation
    • Calvaruso MA, Smeitink J, Nijtmans L (2008) Electrophoresis techniques to investigate defects in oxidative phosphorylation. Methods 46:281-287.
    • (2008) Methods , vol.46 , pp. 281-287
    • Calvaruso, M.A.1    Smeitink, J.2    Nijtmans, L.3


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