-
1
-
-
80052920183
-
Leber's hereditary optic neuropathy-gene therapy: From benchtop to bedside
-
Koilkonda RD, Guy J. Leber's hereditary optic neuropathy-gene therapy: From benchtop to bedside. J Ophthalmol 2011; 2011:179412.
-
(2011)
J Ophthalmol
, vol.2011
, pp. 179412
-
-
Koilkonda, R.D.1
Guy, J.2
-
2
-
-
0024242545
-
Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy
-
[PMID: 3201231]
-
Wallace DC, Singh G, Lott MT, Hodge JA, Schurr TG, Lezza AM, Elsas LJ, Nikoskelainen EK. Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy. Science 1988; 242:1427-30. [PMID: 3201231]
-
(1988)
Science
, vol.242
, pp. 1427-1430
-
-
Wallace, D.C.1
Singh, G.2
Lott, M.T.3
Hodge, J.A.4
Schurr, T.G.5
Lezza, A.M.6
Elsas, L.J.7
Nikoskelainen, E.K.8
-
3
-
-
0033862962
-
The epidemiology of pathogenic mitochondrial DNA mutations
-
[PMID: 10939569]
-
Chinnery PF, Johnson MA, Wardell TM, Singh-Kler R, Hayes C, Brown DT, Taylor RW, Bindoff LA, Turnbull DM. The epidemiology of pathogenic mitochondrial DNA mutations. Ann Neurol 2000; 48:188-93. [PMID: 10939569]
-
(2000)
Ann Neurol
, vol.48
, pp. 188-193
-
-
Chinnery, P.F.1
Johnson, M.A.2
Wardell, T.M.3
Singh-Kler, R.4
Hayes, C.5
Brown, D.T.6
Taylor, R.W.7
Bindoff, L.A.8
Turnbull, D.M.9
-
4
-
-
0034691280
-
Resolution of the membrane domain of bovine complex I into subcomplexes: Implications for the structural organization of the enzyme
-
[PMID: 10852722]
-
Sazanov LA, Peak-Chew SY, Fearnley IM, Walker JE. Resolution of the membrane domain of bovine complex I into subcomplexes: implications for the structural organization of the enzyme. Biochemistry 2000; 39:7229-35. [PMID: 10852722]
-
(2000)
Biochemistry
, vol.39
, pp. 7229-7235
-
-
Sazanov, L.A.1
Peak-Chew, S.Y.2
Fearnley, I.M.3
Walker, J.E.4
-
5
-
-
0030806721
-
Leber hereditary optic neuropathy: Mitochondrial mutations and degeneration of the optic nerve
-
[PMID: 9425526]
-
Howell N. Leber hereditary optic neuropathy: mitochondrial mutations and degeneration of the optic nerve. Vision Res 1997; 37:3495-507. [PMID: 9425526]
-
(1997)
Vision Res
, vol.37
, pp. 3495-3507
-
-
Howell, N.1
-
6
-
-
0029965430
-
MITOMAP: A human mitochondrial genome database
-
[PMID: 8594574]
-
Kogelnik AM, Lott MT, Brown MD, Navathe SB, Wallace DC. MITOMAP: a human mitochondrial genome database. Nucleic Acids Res 1996; 24:177-9. [PMID: 8594574]
-
(1996)
Nucleic Acids Res
, vol.24
, pp. 177-179
-
-
Kogelnik, A.M.1
Lott, M.T.2
Brown, M.D.3
Navathe, S.B.4
Wallace, D.C.5
-
7
-
-
0037310673
-
Suppression of complex I gene expression induces optic neuropathy
-
[PMID: 12557286]
-
Qi X, Lewin AS, Hauswirth WW, Guy J. Suppression of complex I gene expression induces optic neuropathy. Ann Neurol 2003; 53:198-205. [PMID: 12557286]
-
(2003)
Ann Neurol
, vol.53
, pp. 198-205
-
-
Qi, X.1
Lewin, A.S.2
Hauswirth, W.W.3
Guy, J.4
-
8
-
-
33846905085
-
The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse
-
[PMID: 17197509]
-
Qi X, Sun L, Lewin AS, Hauswirth WW, Guy J. The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse. Invest Ophthalmol Vis Sci 2007; 48:1-10. [PMID: 17197509]
-
(2007)
Invest Ophthalmol Vis Sci
, vol.48
, pp. 1-10
-
-
Qi, X.1
Sun, L.2
Lewin, A.S.3
Hauswirth, W.W.4
Guy, J.5
-
9
-
-
0024311525
-
Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression
-
[PMID: 2547998]
-
Samulski RJ, Chang LS, Shenk T. Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 1989; 63:3822-8. [PMID: 2547998]
-
(1989)
J Virol
, vol.63
, pp. 3822-3828
-
-
Samulski, R.J.1
Chang, L.S.2
Shenk, T.3
-
10
-
-
2642565909
-
Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N-terminus
-
[PMID: 15163751]
-
Warrington KH Jr, Gorbatyuk OS, Harrison JK, Opie SR, Zolotukhin S, Muzyczka N. Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N-terminus. J Virol 2004; 78:6595-609. [PMID: 15163751]
-
(2004)
J Virol
, vol.78
, pp. 6595-6609
-
-
Warrington Jr., K.H.1
Gorbatyuk, O.S.2
Harrison, J.K.3
Opie, S.R.4
Zolotukhin, S.5
Muzyczka, N.6
-
11
-
-
0034091924
-
Production and purification of recombinant adeno-associated virus
-
[PMID: 10800712]
-
Hauswirth WW, Lewin AS, Zolotukhin S, Muzyczka N. Production and purification of recombinant adeno-associated virus. Methods Enzymol 2000; 316:743-61. [PMID: 10800712]
-
(2000)
Methods Enzymol
, vol.316
, pp. 743-761
-
-
Hauswirth, W.W.1
Lewin, A.S.2
Zolotukhin, S.3
Muzyczka, N.4
-
12
-
-
70349238784
-
Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system
-
[PMID: 19387075]
-
Guy J, Qi X, Koilkonda RD, Arguello T, Chou TH, Ruggeri M, Porciatti V, Lewin AS, Hauswirth WW. Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Invest Ophthalmol Vis Sci 2009; 50:4205-14. [PMID: 19387075]
-
(2009)
Invest Ophthalmol Vis Sci
, vol.50
, pp. 4205-4214
-
-
Guy, J.1
Qi, X.2
Koilkonda, R.D.3
Arguello, T.4
Chou, T.H.5
Ruggeri, M.6
Porciatti, V.7
Lewin, A.S.8
Hauswirth, W.W.9
-
13
-
-
0036830565
-
Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy
-
[PMID: 12402249]
-
Guy J, Qi X, Pallotti F, Schon EA, Manfredi G, Carelli V, Martinuzzi A, Hauswirth WW, Lewin AS. Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Ann Neurol 2002; 52:534-42. [PMID: 12402249]
-
(2002)
Ann Neurol
, vol.52
, pp. 534-542
-
-
Guy, J.1
Qi, X.2
Pallotti, F.3
Schon, E.A.4
Manfredi, G.5
Carelli, V.6
Martinuzzi, A.7
Hauswirth, W.W.8
Lewin, A.S.9
-
14
-
-
0842281697
-
Mitochondrial dysfunction as a cause of optic neuropathies
-
[PMID: 14766317]
-
Carelli V, Ross-Cisneros FN, Sadun AA. Mitochondrial dysfunction as a cause of optic neuropathies. Prog Retin Eye Res 2004; 23:53-89. [PMID: 14766317]
-
(2004)
Prog Retin Eye Res
, vol.23
, pp. 53-89
-
-
Carelli, V.1
Ross-Cisneros, F.N.2
Sadun, A.A.3
-
15
-
-
0034520010
-
Leber's hereditary optic neuropathy differentially affects smaller axons in the optic nerve
-
[PMID: 11190025]
-
Sadun AA, Win PH, Ross-Cisneros FN, Walker SO, Carelli V. Leber's hereditary optic neuropathy differentially affects smaller axons in the optic nerve. Trans Am Ophthalmol Soc 2000; 98:223-32. [PMID: 11190025]
-
(2000)
Trans Am Ophthalmol Soc
, vol.98
, pp. 223-232
-
-
Sadun, A.A.1
Win, P.H.2
Ross-Cisneros, F.N.3
Walker, S.O.4
Carelli, V.5
-
16
-
-
29644437055
-
Natural competence of mammalian mitochondria allows the molecular investigation of mitochondrial gene expression
-
[PMID: 16321989]
-
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; 15:143-54. [PMID: 16321989]
-
(2006)
Hum Mol Genet
, vol.15
, pp. 143-154
-
-
Koulintchenko, M.1
Temperley, R.J.2
Mason, P.A.3
Dietrich, A.4
Lightowlers, R.N.5
-
17
-
-
0344522705
-
Plant mitochondria actively import DNA via the permeability transition pore complex
-
[PMID: 12628917]
-
Koulintchenko M, Konstantinov Y, Dietrich A. Plant mitochondria actively import DNA via the permeability transition pore complex. EMBO J 2003; 22:1245-54. [PMID: 12628917]
-
(2003)
EMBO J
, vol.22
, pp. 1245-1254
-
-
Koulintchenko, M.1
Konstantinov, Y.2
Dietrich, A.3
-
18
-
-
33847618832
-
Mitochondrial drug delivery and mitochondrial disease therapy-an approach to liposome-based delivery targeted to mitochondria
-
[PMID: 17296332]
-
Yamada Y, Akita H, Kogure K, Kamiya H, Harashima H. Mitochondrial drug delivery and mitochondrial disease therapy-an approach to liposome-based delivery targeted to mitochondria. Mitochondrion 2007; 7:63-71. [PMID: 17296332]
-
(2007)
Mitochondrion
, vol.7
, pp. 63-71
-
-
Yamada, Y.1
Akita, H.2
Kogure, K.3
Kamiya, H.4
Harashima, H.5
-
19
-
-
73449136874
-
Targeted nucleic acid delivery to mitochondria
-
[PMID: 20021331]
-
Vaidya B, Mishra N, Dube D, Tiwari S, Vyas SP. Targeted nucleic acid delivery to mitochondria. Curr Gene Ther 2009; 9:475-86. [PMID: 20021331]
-
(2009)
Curr Gene Ther
, vol.9
, pp. 475-486
-
-
Vaidya, B.1
Mishra, N.2
Dube, D.3
Tiwari, S.4
Vyas, S.P.5
-
20
-
-
0038365420
-
Targeted delivery of DNA to the mitochondrial compartment via import sequence-conjugated peptide nucleic acid
-
[PMID: 12727119]
-
Flierl A, Jackson C, Cottrell B, Murdock D, Seibel P, Wallace DC. Targeted delivery of DNA to the mitochondrial compartment via import sequence-conjugated peptide nucleic acid. Mol Ther 2003; 7:550-7. [PMID: 12727119]
-
(2003)
Mol Ther
, vol.7
, pp. 550-557
-
-
Flierl, A.1
Jackson, C.2
Cottrell, B.3
Murdock, D.4
Seibel, P.5
Wallace, D.C.6
-
21
-
-
0032718476
-
Two distinct mechanisms drive protein translocation across the mitochondrial outer membrane in the late step of the cytochrome b(2) import pathway
-
[PMID: 10518525]
-
Esaki M, Kanamori T, Nishikawa S, Endo T. Two distinct mechanisms drive protein translocation across the mitochondrial outer membrane in the late step of the cytochrome b(2) import pathway. Proc Natl Acad Sci USA 1999; 96:11770-5. [PMID: 10518525]
-
(1999)
Proc Natl Acad Sci USA
, vol.96
, pp. 11770-11775
-
-
Esaki, M.1
Kanamori, T.2
Nishikawa, S.3
Endo, T.4
-
22
-
-
46449117734
-
Progress and prospects: Gene therapy for mitochondrial DNA disease
-
[PMID: 18496570]
-
Kyriakouli DS, Boesch P, Taylor RW, Lightowlers RN. Progress and prospects: gene therapy for mitochondrial DNA disease. Gene Ther 2008; 15:1017-23. [PMID: 18496570]
-
(2008)
Gene Ther
, vol.15
, pp. 1017-1023
-
-
Kyriakouli, D.S.1
Boesch, P.2
Taylor, R.W.3
Lightowlers, R.N.4
-
23
-
-
77955480347
-
Mitochondrial matrix delivery using MITO-Porter, a liposome-based carrier that specifies fusion with mitochondrial membranes
-
[PMID: 20580633]
-
Yasuzaki Y, Yamada Y, Harashima H. Mitochondrial matrix delivery using MITO-Porter, a liposome-based carrier that specifies fusion with mitochondrial membranes. Biochem Biophys Res Commun 2010; 397:181-6. [PMID: 20580633]
-
(2010)
Biochem Biophys Res Commun
, vol.397
, pp. 181-186
-
-
Yasuzaki, Y.1
Yamada, Y.2
Harashima, H.3
-
24
-
-
70349284435
-
Mitochondrial gene replacement in primate offspring and embryonic stem cells
-
[PMID: 19710649]
-
Tachibana M, Sparman M, Sritanaudomchai H, Ma H, Clepper L, Woodward J, Li Y, Ramsey C, Kolotushkina O, Mitalipov S. Mitochondrial gene replacement in primate offspring and embryonic stem cells. Nature 2009; 461:367-72. [PMID: 19710649]
-
(2009)
Nature
, vol.461
, pp. 367-372
-
-
Tachibana, M.1
Sparman, M.2
Sritanaudomchai, H.3
Ma, H.4
Clepper, L.5
Woodward, J.6
Li, Y.7
Ramsey, C.8
Kolotushkina, O.9
Mitalipov, S.10
-
26
-
-
41449089840
-
Mice with mitochondrial complex I deficiency develop a fatal encephalomyopathy
-
[PMID: 18396137]
-
Kruse SE, Watt WC, Marcinek DJ, Kapur RP, Schenkman KA, Palmiter RD. Mice with mitochondrial complex I deficiency develop a fatal encephalomyopathy. Cell Metab 2008; 7:312-20. [PMID: 18396137]
-
(2008)
Cell Metab
, vol.7
, pp. 312-320
-
-
Kruse, S.E.1
Watt, W.C.2
Marcinek, D.J.3
Kapur, R.P.4
Schenkman, K.A.5
Palmiter, R.D.6
-
27
-
-
64549117348
-
NDUFS4: Creation of a mouse model mimicking a Complex I disorder
-
[PMID: 19460290]
-
Ingraham CA, Burwell LS, Skalska J, Brookes PS, Howell RL, Sheu SS, Pinkert CA. NDUFS4: creation of a mouse model mimicking a Complex I disorder. Mitochondrion 2009; 9:204-10. [PMID: 19460290]
-
(2009)
Mitochondrion
, vol.9
, pp. 204-210
-
-
Ingraham, C.A.1
Burwell, L.S.2
Skalska, J.3
Brookes, P.S.4
Howell, R.L.5
Sheu, S.S.6
Pinkert, C.A.7
-
28
-
-
50949130569
-
Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction
-
[PMID: 18771762]
-
Ellouze S, Augustin S, Bouaita A, Bonnet C, Simonutti M, Forster V, Picaud S, Sahel JA, Corral-Debrinski M. Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction. Am J Hum Genet 2008; 83:373-87. [PMID: 18771762]
-
(2008)
Am J Hum Genet
, vol.83
, pp. 373-387
-
-
Ellouze, S.1
Augustin, S.2
Bouaita, A.3
Bonnet, C.4
Simonutti, M.5
Forster, V.6
Picaud, S.7
Sahel, J.A.8
Corral-Debrinski, M.9
-
29
-
-
48249156188
-
Mitochondrial disorders in the nervous system
-
[PMID: 18333761]
-
DiMauro S, Schon EA. Mitochondrial disorders in the nervous system. Annu Rev Neurosci 2008; 31:91-123. [PMID: 18333761]
-
(2008)
Annu Rev Neurosci
, vol.31
, pp. 91-123
-
-
Di Mauro, S.1
Schon, E.A.2
|