-
1
-
-
0026937479
-
A brief history of gene therapy
-
Friedmann T. A brief history of gene therapy. Nat Genet 1992;2:93-8.
-
(1992)
Nat Genet
, vol.2
, pp. 93-98
-
-
Friedmann, T.1
-
2
-
-
0142089747
-
Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer
-
DOI 10.1016/j.ymgme.2003.08.016
-
Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80:148-58. (Pubitemid 37272241)
-
(2003)
Molecular Genetics and Metabolism
, vol.80
, Issue.1-2
, pp. 148-158
-
-
Raper, S.E.1
Chirmule, N.2
Lee, F.S.3
Wivel, N.A.4
Bagg, A.5
Gao, G.-P.6
Wilson, J.M.7
Batshaw, M.L.8
-
3
-
-
0042738935
-
Occurrence of leukaemia following gene therapy of X-linked SCID
-
Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 2003;3:477-88. (Pubitemid 37328836)
-
(2003)
Nature Reviews Cancer
, vol.3
, Issue.7
, pp. 477-488
-
-
Kohn, D.B.1
Sadelain, M.2
Glorioso, J.C.3
-
4
-
-
58149484922
-
Ocular gene therapy: Current progress and future prospects
-
Colella P, Cotugno G, Auricchio A. Ocular gene therapy: current progress and future prospects. Trends Mol Med 2009;15:23-31.
-
(2009)
Trends Mol Med
, vol.15
, pp. 23-31
-
-
Colella, P.1
Cotugno, G.2
Auricchio, A.3
-
5
-
-
44049100684
-
AAV-mediated gene therapy for retinal disorders: From mouse to man
-
DOI 10.1038/gt.2008.66, PII GT200866, Special Issue: AAV Vectors for Clinical GeneTherapy
-
Buch PK, Bainbridge JW, Ali RR. AAV-mediated gene therapy for retinal disorders: from mouse to man. Gene Ther 2008;15:849-57. (Pubitemid 351712618)
-
(2008)
Gene Therapy
, vol.15
, Issue.11
, pp. 849-857
-
-
Buch, P.K.1
Bainbridge, J.W.2
Ali, R.R.3
-
6
-
-
9544226448
-
A humanized system for pharmacologic control of gene expression
-
DOI 10.1038/nm0996-1028
-
Rivera VM, Clackson T, Natesan S, et al. A humanized system for pharmacologic control of gene expression. Nat Med 1996;2:1028-32. (Pubitemid 26306843)
-
(1996)
Nature Medicine
, vol.2
, Issue.9
, pp. 1028-1032
-
-
Rivera, V.M.1
Clackson, T.2
Natesan, S.3
Pollock, R.4
Amara, J.F.5
Keenan, T.6
Magari, S.R.7
Phillips, T.8
Courage, N.L.9
Franklin, C.J.R.10
Holt, D.A.11
Gilman, M.12
-
7
-
-
35348985459
-
Oral administration of doxycycline allows tight control of transgene expression: A key step towards gene therapy of retinal diseases
-
DOI 10.1038/sj.gt.3303034, PII 3303034
-
Stieger K, Mendes-Madeira A, Meur GL, et al. Oral administration of doxycycline allows tight control of transgene expression: a key step towards gene therapy of retinal diseases. Gene Ther 2007;14:1668-73. (Pubitemid 350143192)
-
(2007)
Gene Therapy
, vol.14
, Issue.23
, pp. 1668-1673
-
-
Stieger, K.1
Mendes-Madeira, A.2
Meur, G.L.3
Weber, M.4
Deschamps, J.-Y.5
Nivard, D.6
Provost, N.7
Moullier, P.8
Rolling, F.9
-
8
-
-
1542437242
-
Sustained tetracycline-regulated transgene expression in vivo in rat retinal ganglion cells using a single type 2 adeno-associated viral vector
-
DOI 10.1002/jgm.367
-
Folliot S, Briot D, Conrath H, et al. Sustained tetracycline-regulated transgene expression in vivo in rat retinal ganglion cells using a single type 2 adeno-associated viral vector. J Gene Med 2003;5:493-501. (Pubitemid 40310392)
-
(2003)
Journal of Gene Medicine
, vol.5
, Issue.6
, pp. 493-501
-
-
Folliot, S.1
Briot, D.2
Conrath, H.3
Provost, N.4
Cherel, Y.5
Moullier, P.6
Rolling, F.7
-
9
-
-
40749138573
-
Robust hypoxia-selective regulation of a retinal pigment epithelium-specific adeno-associated virus vector
-
Dougherty CJ, Smith GW, Dorey CK, et al. Robust hypoxia-selective regulation of a retinal pigment epithelium-specific adeno-associated virus vector. Mol Vis 2008;14:471-80. (Pubitemid 351377319)
-
(2008)
Molecular Vision
, vol.14
, pp. 471-480
-
-
Dougherty, C.J.1
Smith, G.W.2
Dorey, C.K.3
Prentice, H.M.4
Webster, K.A.5
Blanks, J.C.6
-
10
-
-
9244257887
-
Biology of adenovirus and its use as a vector for gene therapy
-
DOI 10.1089/hum.2004.15.1022
-
McConnell MJ, Imperiale MJ. Biology of adenovirus and its use as a vector for gene therapy. Hum Gene Ther 2004;15:1022-33. (Pubitemid 39552284)
-
(2004)
Human Gene Therapy
, vol.15
, Issue.11
, pp. 1022-1033
-
-
McConnell, M.J.1
Imperiale, M.J.2
-
11
-
-
35148861116
-
Helper-dependent adenovirus for the gene therapy of proliferative retinopathies: Stable gene transfer, regulated gene expression and therapeutic efficacy
-
DOI 10.1002/jgm.1083
-
Lamartina S, Cimino M, Roscilli G, et al. Helper-dependent adenovirus for the gene therapy of proliferative retinopathies: stable gene transfer, regulated gene expression and therapeutic efficacy. J Gene Med 2007;9:862-74. (Pubitemid 47543253)
-
(2007)
Journal of Gene Medicine
, vol.9
, Issue.10
, pp. 862-874
-
-
Lamartina, S.1
Cimino, M.2
Roscilli, G.3
Dammassa, E.4
Lazzaro, D.5
Rota, R.6
Ciliberto, G.7
Toniatti, C.8
-
12
-
-
33645239884
-
Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors
-
Balaggan KS, Binley K, Esapa M, et al. Stable and efficient intraocular gene transfer using pseudotyped EIAV lentiviral vectors. J Gene Med 2006;8:275-85.
-
(2006)
J Gene Med
, vol.8
, pp. 275-285
-
-
Balaggan, K.S.1
Binley, K.2
Esapa, M.3
-
13
-
-
4544296803
-
A synthetic rev-independent bovine immunodeficiency virus-based packaging construct
-
DOI 10.1089/hum.2004.15.865
-
Molina RP, Ye HQ, Brady J, et al. A synthetic Rev-independent bovine immunodeficiency virus-based packaging construct. Hum Gene Ther 2004;15:865-77. (Pubitemid 39244841)
-
(2004)
Human Gene Therapy
, vol.15
, Issue.9
, pp. 865-877
-
-
Molina, R.P.1
Ye, H.Q.2
Brady, J.3
Zhang, J.4
Zimmerman, H.5
Kaleko, M.6
Luo, T.7
-
14
-
-
0035103890
-
Construction and molecular analysis of gene transfer systems derived from bovine immunodeficiency virus
-
DOI 10.1128/JVI.75.7.3371-3382.2001
-
Berkowitz R, Ilves H, Lin WY, et al. Construction and molecular analysis of gene transfer systems derived from bovine immunodeficiency virus. J Virol 2001;75:3371-82. (Pubitemid 32225900)
-
(2001)
Journal of Virology
, vol.75
, Issue.7
, pp. 3371-3382
-
-
Berkowitz, R.1
Ilves, H.2
Wei, Y.L.3
Eckert, K.4
Coward, A.5
Tamaki, S.6
Veres, G.7
Plavec, I.8
-
15
-
-
33644822908
-
Effective gene therapy with nonintegrating lentiviral vectors
-
DOI 10.1038/nm1365, PII N1365
-
Yanez-Munoz RJ, Balaggan KS, MacNeil A, et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 2006;12:348-53. (Pubitemid 43355085)
-
(2006)
Nature Medicine
, vol.12
, Issue.3
, pp. 348-353
-
-
Yanez-Munoz, R.J.1
Balaggan, K.S.2
MacNeil, A.3
Howe, S.J.4
Schmidt, M.5
Smith, A.J.6
Buch, P.7
MacLaren, R.E.8
Anderson, P.N.9
Barker, S.E.10
Duran, Y.11
Bartholomae, C.12
Von Kalle, C.13
Heckenlively, J.R.14
Kinnon, C.15
Ali, R.R.16
Thrasher, A.J.17
-
16
-
-
53249103330
-
Nonviral ocular gene therapy: Assessment and future directions
-
Conley SM, Cai X, Naash MI. Nonviral ocular gene therapy: assessment and future directions. Curr Opin Mol Ther 2008;10:456-63.
-
(2008)
Curr Opin Mol Ther
, vol.10
, pp. 456-463
-
-
Conley, S.M.1
Cai, X.2
Naash, M.I.3
-
17
-
-
34248570405
-
Efficient non-viral ocular gene transfer with compacted DNA nanoparticles
-
Farjo R, Skaggs J, Quiambao AB, et al. Efficient non-viral ocular gene transfer with compacted DNA nanoparticles. PLoS One 2006;1:e38.
-
(2006)
PLoS One
, vol.1
-
-
Farjo, R.1
Skaggs, J.2
Quiambao, A.B.3
-
18
-
-
22144464502
-
phiC31 integrase confers genomic integration and long-term transgene expression in rat retina
-
Chalberg TW, Genise HL, Vollrath D, et al. phiC31 integrase confers genomic integration and long-term transgene expression in rat retina. Invest Ophthalmol Vis Sci 2005;46:2140-6.
-
(2005)
Invest Ophthalmol Vis Sci
, vol.46
, pp. 2140-2146
-
-
Chalberg, T.W.1
Genise, H.L.2
Vollrath, D.3
-
19
-
-
19944373097
-
Nonviral ocular gene transfer
-
DOI 10.1038/sj.gt.3302475
-
Kachi S, Oshima Y, Esumi N, et al. Nonviral ocular gene transfer. Gene Ther 2005;12:843-51. (Pubitemid 40753976)
-
(2005)
Gene Therapy
, vol.12
, Issue.10
, pp. 843-851
-
-
Kachi, S.1
Oshima, Y.2
Esumi, N.3
Kachi, M.4
Rogers, B.5
Zack, D.J.6
Campochiaro, P.A.7
-
20
-
-
85047699046
-
Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1
-
DOI 10.1038/sj/gt/3301680
-
Bainbridge JW, Mistry A, De AM, et al. Inhibition of retinal neovascularisation by gene transfer of soluble VEGF receptor sFlt-1. Gene Ther 2002;9:320-6. (Pubitemid 34285145)
-
(2002)
Gene Therapy
, vol.9
, Issue.5
, pp. 320-326
-
-
Bainbridge, J.W.B.1
Mistry, A.2
De Alwis, M.3
Paleolog, E.4
Baker, A.5
Thrasher, A.J.6
Ali, R.R.7
-
22
-
-
0036798747
-
Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents
-
DOI 10.1006/mthe.2002.0702
-
Auricchio A, Behling KC, Maguire AM, et al. Inhibition of retinal neovascularization by intraocular viral-mediated delivery of anti-angiogenic agents. Mol Ther 2002;6:490-4. (Pubitemid 35214870)
-
(2002)
Molecular Therapy
, vol.6
, Issue.4
, pp. 490-494
-
-
Auricchio, A.1
Behling, K.C.2
Maguire, A.M.3
O'Connor, E.E.4
Bennett, J.5
Wilson, J.M.6
Tolentino, M.J.7
-
23
-
-
33746164355
-
EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV
-
DOI 10.1038/sj.gt.3302769, PII 3302769
-
Balaggan KS, Binley K, Esapa M, et al. EIAV vector-mediated delivery of endostatin or angiostatin inhibits angiogenesis and vascular hyperpermeability in experimental CNV. Gene Ther 2006;13:1153-65. (Pubitemid 44084074)
-
(2006)
Gene Therapy
, vol.13
, Issue.15
, pp. 1153-1165
-
-
Balaggan, K.S.1
Binley, K.2
Esapa, M.3
MacLaren, R.E.4
Iqball, S.5
Duran, Y.6
Pearson, R.A.7
Kan, O.8
Barker, S.E.9
Smith, A.J.10
Bainbridge, J.W.B.11
Naylor, S.12
Ali, R.R.13
-
24
-
-
0037318972
-
Lentivirus-mediated expression of angiostatin efficiently inhibits neovascularization in a murine proliferative retinopathy model
-
DOI 10.1038/sj.gt.3301878
-
Igarashi T, Miyake K, Kato K, et al. Lentivirus-mediated expression of angiostatin efficiently inhibits neovascularization in a murine proliferative retinopathy model. Gene Ther 2003;10:219-26. (Pubitemid 36204410)
-
(2003)
Gene Therapy
, vol.10
, Issue.3
, pp. 219-226
-
-
Igarashi, T.1
Miyake, K.2
Kato, K.3
Watanabe, A.4
Ishizaki, M.5
Ohara, K.6
Shimada, T.7
-
25
-
-
0036271989
-
AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization
-
Mori K, Gehlbach P, Yamamoto S, et al. AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Invest Ophthalmol Vis Sci 2002;43:1994-2000. (Pubitemid 34587052)
-
(2002)
Investigative Ophthalmology and Visual Science
, vol.43
, Issue.6
, pp. 1994-2000
-
-
Mori, K.1
Gehlbach, P.2
Yamamoto, S.3
Duh, E.4
Zack, D.J.5
Li, Q.6
Berns, K.I.7
Raisler, B.J.8
Hauswirth, W.W.9
Campochiaro, P.A.10
-
26
-
-
0034945843
-
Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization
-
DOI 10.1002/jcp.1114
-
Mori K, Duh E, Gehlbach P, et al. Pigment epithelium-derived factor inhibits retinal and choroidal neovascularization. J Cell Physiol 2001;188:253-63. (Pubitemid 32622916)
-
(2001)
Journal of Cellular Physiology
, vol.188
, Issue.2
, pp. 253-263
-
-
Mori, K.1
Duh, E.2
Gehlbach, P.3
Ando, A.4
Takahashi, K.5
Pearlman, J.6
Mori, K.7
Yang, H.S.8
Zack, D.J.9
Ettyreddy, D.10
Brough, D.E.11
Wei, L.L.12
Campochiaro, P.A.13
-
27
-
-
20844447893
-
Periocular gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization in a human-sized eye
-
DOI 10.1089/hum.2005.16.473
-
Saishin Y, Silva RL, Saishin Y, et al. Periocular gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization in a human-sized eye. Hum Gene Ther 2005;16:473-8. (Pubitemid 40647144)
-
(2005)
Human Gene Therapy
, vol.16
, Issue.4
, pp. 473-478
-
-
Saishin, Y.1
Silva, R.L.2
Saishin, Y.3
Kachi, S.4
Aslam, S.5
Yuan, Y.G.6
Lai, H.7
Carrion, M.8
Harris, B.9
Hamilton, M.10
Wei, L.11
Campochiaro, P.A.12
-
28
-
-
0035514256
-
Clinical protocol. An open-label, phase I, singleadministration, dose-escalation study of ADGVPEDF.11D (ADPEDF) in neovascular age-related macular degeneration (AMD)
-
Rasmussen H, Chu KW, Campochiaro P, et al. Clinical protocol. An open-label, phase I, singleadministration, dose-escalation study of ADGVPEDF.11D (ADPEDF) in neovascular age-related macular degeneration (AMD). Hum Gene Ther 2001;12:2029-32.
-
(2001)
Hum Gene Ther
, vol.12
, pp. 2029-2032
-
-
Rasmussen, H.1
Chu, K.W.2
Campochiaro, P.3
-
29
-
-
0033947697
-
Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy
-
DOI 10.1038/77068
-
Ali RR, Sarra GM, Stephens C, et al. Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy. Nat Genet 2000;25:306-10. (Pubitemid 30437318)
-
(2000)
Nature Genetics
, vol.25
, Issue.3
, pp. 306-310
-
-
Ali, R.R.1
Sarra, G.-M.2
Stephens, C.3
De Alwis, M.4
Bainbridge, J.W.B.5
Munro, P.M.6
Fauser, S.7
Reichell, M.B.8
Kinnon, C.9
Hunt, D.M.10
Bhattacharya, S.S.11
Thrasher, A.J.12
-
30
-
-
1242271326
-
Rd2/Rd2 mice following gene therapy
-
DOI 10.1016/j.mcn.2003.09.020
-
Schlichtenbrede FC, Smith AJ, Bainbridge JW, et al. Improvement of neuronal visual responses in the superior colliculus in Prph2(Rd2/Rd2) mice following gene therapy. Mol Cell Neurosci 2004;25:103-10. (Pubitemid 38220868)
-
(2004)
Molecular and Cellular Neuroscience
, vol.25
, Issue.1
, pp. 103-110
-
-
Schlichtenbrede, F.C.1
Smith, A.J.2
Bainbridge, J.W.B.3
Thrasher, A.J.4
Salt, T.E.5
Ali, R.R.6
-
31
-
-
0035504663
-
Gene replacement therapy in the retinal degeneration slow (rds) mouse: The effect on retinal degeneration following partial transduction of the retina
-
Sarra GM, Stephens C, De AM, et al. Gene replacement therapy in the retinal degeneration slow (rds) mouse: the effect on retinal degeneration following partial transduction of the retina. Hum Mol Genet 2001;10:2353-61.
-
(2001)
Hum Mol Genet
, vol.10
, pp. 2353-2361
-
-
Sarra, G.M.1
Stephens, C.2
De, A.M.3
-
32
-
-
0038335879
-
Rd2/Rd2 mice following AAV-mediated gene replacement therapy
-
DOI 10.1002/jgm.401
-
Schlichtenbrede FC, da CL, Stephens C, et al. Long-term evaluation of retinal function in Prph2Rd2/Rd2 mice following AAV-mediated gene replacement therapy. J Gene Med 2003;5:757-64. (Pubitemid 40309713)
-
(2003)
Journal of Gene Medicine
, vol.5
, Issue.9
, pp. 757-764
-
-
Schlichtenbrede, F.C.1
Da, C.L.2
Stephens, C.3
Smith, A.J.4
Georgiadis, A.5
Thrasher, A.J.6
Bainbridge, J.W.B.7
Seeliger, M.W.8
Ali, R.R.9
-
33
-
-
63949087144
-
Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy
-
Chadderton N, Millington-Ward S, Palfi A, et al. Improved retinal function in a mouse model of dominant retinitis pigmentosa following AAV-delivered gene therapy. Mol Ther 2009;17:593-9.
-
(2009)
Mol Ther
, vol.17
, pp. 593-599
-
-
Chadderton, N.1
Millington-Ward, S.2
Palfi, A.3
-
34
-
-
0030759730
-
Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration
-
Jomary C, Vincent KA, Grist J, et al. Rescue of photoreceptor function by AAV-mediated gene transfer in a mouse model of inherited retinal degeneration. Gene Ther 1997;4:683-90. (Pubitemid 27343130)
-
(1997)
Gene Therapy
, vol.4
, Issue.7
, pp. 683-690
-
-
Jomary, C.1
Vincent, K.A.2
Grist, J.3
Neal, M.J.4
Jones, S.E.5
-
35
-
-
0032816002
-
Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer
-
Takahashi M, Miyoshi H, Verma IM, et al. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 1999;73:7812-16. (Pubitemid 29383266)
-
(1999)
Journal of Virology
, vol.73
, Issue.9
, pp. 7812-7816
-
-
Takahashi, M.1
Miyoshi, H.2
Verma, I.M.3
Gage, F.H.4
-
36
-
-
0031713983
-
Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse
-
Bennett J, Zeng Y, Bajwa R, et al. Adenovirus-mediated delivery of rhodopsin-promoted bcl-2 results in a delay in photoreceptor cell death in the rd/rd mouse. Gene Ther 1998;5:1156-64. (Pubitemid 28448422)
-
(1998)
Gene Therapy
, vol.5
, Issue.9
, pp. 1156-1164
-
-
Bennett, J.1
Zeng, Y.2
Bajwa, R.3
Klatt, L.4
Li, Y.5
Maguire, A.M.6
-
37
-
-
0042025129
-
Simian lentiviral vector-mediated retinal gene transfer of pigment epithelium-derived factor protects retinal degeneration and electrical defect in Royal College of Surgeons rats
-
DOI 10.1038/sj.gt.3302028
-
Miyazaki M, Ikeda Y, Yonemitsu Y, et al. Simian lentiviral vector-mediated retinal gene transfer of pigment epithelium-derived factor protects retinal degeneration and electrical defect in Royal College of Surgeons rats. Gene Ther 2003;10:1503-11. (Pubitemid 36987144)
-
(2003)
Gene Therapy
, vol.10
, Issue.17
, pp. 1503-1511
-
-
Miyazaki, M.1
Ikeda, Y.2
Yonemitsu, Y.3
Goto, Y.4
Sakamoto, T.5
Tabata, T.6
Ueda, Y.7
Hasegawa, M.8
Tobimatsu, S.9
Ishibashi, T.10
Sueishi, K.11
-
38
-
-
0033774933
-
Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2
-
Lau D, McGee LH, Zhou S, et al. Retinal degeneration is slowed in transgenic rats by AAV-mediated delivery of FGF-2. Invest Ophthalmol Vis Sci 2000;41:3622-33.
-
(2000)
Invest Ophthalmol Vis Sci
, vol.41
, pp. 3622-3633
-
-
Lau, D.1
McGee, L.H.2
Zhou, S.3
-
39
-
-
58949084050
-
Synergistic neuroprotective effect via simian lentiviral vector-mediated simultaneous gene transfer of human pigment epithelium-derived factor and human fibroblast growth factor-2 in rodent models of retinitis pigmentosa
-
Miyazaki M, Ikeda Y, Yonemitsu Y, et al. Synergistic neuroprotective effect via simian lentiviral vector-mediated simultaneous gene transfer of human pigment epithelium-derived factor and human fibroblast growth factor-2 in rodent models of retinitis pigmentosa. J Gene Med 2008;10:1273-81.
-
(2008)
J Gene Med
, vol.10
, pp. 1273-1281
-
-
Miyazaki, M.1
Ikeda, Y.2
Yonemitsu, Y.3
-
40
-
-
34848863345
-
Rescue of sight by gene therapy - Closer than it may appear
-
DOI 10.1080/13816810701503707, PII 782465485
-
Rex TS. Rescue of sight by gene therapy - closer than it may appear. Ophthalmic Genet 2007;28:127-33. (Pubitemid 47493765)
-
(2007)
Ophthalmic Genetics
, vol.28
, Issue.3
, pp. 127-133
-
-
Rex, T.S.1
-
41
-
-
0032533962
-
Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse
-
Cayouette M, Behn D, Sendtner M, et al. Intraocular gene transfer of ciliary neurotrophic factor prevents death and increases responsiveness of rod photoreceptors in the retinal degeneration slow mouse. J Neurosci 1998;18:9282-93. (Pubitemid 28512459)
-
(1998)
Journal of Neuroscience
, vol.18
, Issue.22
, pp. 9282-9293
-
-
Cayouette, M.1
Behn, D.2
Sendtner, M.3
Lachapelle, P.4
Gravel, C.5
-
42
-
-
0030997980
-
Adenovirus-mediated gene transfer of ciliary neurotrophic factor can prevent photoreceptor degeneration in the retinal degeneration (rd) mouse
-
Cayouette M, Gravel C. Adenovirus-mediated gene transfer of ciliary neurotrophic factor can prevent photoreceptor degeneration in the retinal degeneration (rd) mouse. Hum Gene Ther 1997;8:423-30. (Pubitemid 27140545)
-
(1997)
Human Gene Therapy
, vol.8
, Issue.4
, pp. 423-430
-
-
Cayouette, M.1
Gravel, C.2
-
43
-
-
0034980737
-
AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse
-
DOI 10.1006/mthe.2000.0252
-
Liang FQ, Dejneka NS, Cohen DR, et al. AAV-mediated delivery of ciliary neurotrophic factor prolongs photoreceptor survival in the rhodopsin knockout mouse. Mol Ther 2001;3:241-8. (Pubitemid 32509827)
-
(2001)
Molecular Therapy
, vol.3
, Issue.2
, pp. 241-248
-
-
Liang, F.-Q.1
Dejneka, N.S.2
Cohen, D.R.3
Krasnoperova, N.V.4
Lem, J.5
Maguire, A.M.6
Dudus, L.7
Fisher, K.J.8
Bennett, J.9
-
44
-
-
0037352472
-
Rd2/Rd2 model of retinal degeneration
-
DOI 10.1038/sj.gt.3301929
-
Schlichtenbrede FC, MacNeil A, Bainbridge JW, et al. Intraocular gene delivery of ciliary neurotrophic factor results in significant loss of retinal function in normal mice and in the Prph2Rd2/Rd2 model of retinal degeneration. Gene Ther 2003;10:523-7. (Pubitemid 36395269)
-
(2003)
Gene Therapy
, vol.10
, Issue.6
, pp. 523-527
-
-
Schlichtenbrede, F.C.1
MacNeil, A.2
Bainbridge, J.W.B.3
Tschernutter, M.4
Thrasher, A.J.5
Smith, A.J.6
Ali, R.R.7
-
45
-
-
0035940504
-
Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk
-
DOI 10.1073/pnas.221364198
-
Vollrath D, Feng W, Duncan JL, et al. Correction of the retinal dystrophy phenotype of the RCS rat by viral gene transfer of Mertk. Proc Natl Acad Sci U S A 2001;98:12584-9. (Pubitemid 33019989)
-
(2001)
Proceedings of the National Academy of Sciences of the United States of America
, vol.98
, Issue.22
, pp. 12584-12589
-
-
Vollrath, D.1
Feng, W.2
Duncan, J.L.3
Yasumura, D.4
D'Cruz, P.M.5
Chappelow, A.6
Matthes, M.T.7
Kay, M.A.8
Lavail, M.M.9
-
46
-
-
0041854336
-
AAV-mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa
-
DOI 10.1016/S1525-0016(03)00144-8
-
Smith AJ, Schlichtenbrede FC, Tschernutter M, et al. AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa. Mol Ther 2003;8:188-95. (Pubitemid 37062829)
-
(2003)
Molecular Therapy
, vol.8
, Issue.2
, pp. 188-195
-
-
Smith, A.J.1
Schlichtenbrede, F.C.2
Tschernutter, M.3
Bainbridge, J.W.4
Thrasher, A.J.5
Ali, R.R.6
-
47
-
-
17644415316
-
Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy
-
DOI 10.1038/sj.gt.3302460
-
Tschernutter M, Schlichtenbrede FC, Howe S, et al. Long-term preservation of retinal function in the RCS rat model of retinitis pigmentosa following lentivirus-mediated gene therapy. Gene Ther 2005;12:694-701. (Pubitemid 40561508)
-
(2005)
Gene Therapy
, vol.12
, Issue.8
, pp. 694-701
-
-
Tschernutter, M.1
Schlichtenbrede, F.C.2
Howe, S.3
Balaggan, K.S.4
Munro, P.M.5
Bainbridge, J.W.B.6
Thrasher, A.J.7
Smith, A.J.8
Ali, R.R.9
-
48
-
-
33745608443
-
Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness
-
Williams ML, Coleman JE, Haire SE, et al. Lentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindness. PLoS Med 2006;3:e201.
-
(2006)
PLoS Med
, vol.3
-
-
Williams, M.L.1
Coleman, J.E.2
Haire, S.E.3
-
49
-
-
74349104948
-
Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
-
Sun X, Pawlyk B, Xu X, et al. Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations. Gene Ther 2010;17:117-31.
-
(2010)
Gene Ther
, vol.17
, pp. 117-131
-
-
Sun, X.1
Pawlyk, B.2
Xu, X.3
-
50
-
-
27244446791
-
Gene replacement therapy rescues photoreceptor degeneration in a murine model of leber congenital amaurosis lacking RPGRIP
-
DOI 10.1167/iovs.05-0371
-
Pawlyk BS, Smith AJ, Buch PK, et al. Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP. Invest Ophthalmol Vis Sci 2005;46:3039-45. (Pubitemid 44264286)
-
(2005)
Investigative Ophthalmology and Visual Science
, vol.46
, Issue.9
, pp. 3039-3045
-
-
Pawlyk, B.S.1
Smith, A.J.2
Buch, P.K.3
Adamian, M.4
Hong, D.-H.5
Sandberg, M.A.6
Ali, R.R.7
Li, T.8
-
51
-
-
32944473999
-
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 leber congenital amaurosis
-
DOI 10.1016/j.ymthe.2005.09.001, PII S1525001605015881
-
Pang JJ, Chang B, Kumar A, et al. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Mol Ther 2006;13:565-72. (Pubitemid 43257624)
-
(2006)
Molecular Therapy
, vol.13
, Issue.3
, pp. 565-572
-
-
Pang, J.-J.1
Chang, B.2
Kumar, A.3
Nusinowitz, S.4
Noorwez, S.M.5
Li, J.6
Rani, A.7
Foster, T.C.8
Chiodo, V.A.9
Doyle, T.10
Li, H.11
Malhotra, R.12
Teusner, J.T.13
McDowell, J.H.14
Min, S.-H.15
Li, Q.16
Kaushal, S.17
Hauswirth, W.W.18
-
52
-
-
33750855742
-
Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis
-
Bemelmans AP, Kostic C, Crippa SV, et al. Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis. PLoS Med 2006;3:e347.
-
(2006)
PLoS Med
, vol.3
-
-
Bemelmans, A.P.1
Kostic, C.2
Crippa, S.V.3
-
53
-
-
28444442243
-
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness
-
DOI 10.1016/j.ymthe.2005.08.008, PII S1525001605015613
-
Acland GM, Aguirre GD, Bennett J, et al. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Mol Ther 2005;12:1072-82. (Pubitemid 41723041)
-
(2005)
Molecular Therapy
, vol.12
, Issue.6
, pp. 1072-1082
-
-
Acland, G.M.1
Aguirre, G.D.2
Bennett, J.3
Aleman, T.S.4
Cideciyan, A.V.5
Bennicelli, J.6
Dejneka, N.S.7
Pearce-Kelling, S.E.8
Maguire, A.M.9
Palczewski, K.10
Hauswirth, W.W.11
Jacobson, S.G.12
-
54
-
-
33846933945
-
Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium
-
DOI 10.1038/sj.gt.3302861, PII 3302861
-
Le MG, Stieger K, Smith AJ, et al. 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. (Pubitemid 46231923)
-
(2007)
Gene Therapy
, vol.14
, Issue.4
, pp. 292-303
-
-
Le, M.G.1
Stieger, K.2
Smith, A.J.3
Weber, M.4
Deschamps, J.Y.5
Nivard, D.6
Mendes-Madeira, A.7
Provost, N.8
Pereon, Y.9
Cherel, Y.10
Ali, R.R.11
Hamel, C.12
Moullier, P.13
Rolling, F.14
-
55
-
-
33750591238
-
Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis
-
DOI 10.1089/hum.2006.17.845
-
Jacobson SG, Boye SL, Aleman TS, et al. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Hum Gene Ther 2006;17:845-58. (Pubitemid 44681454)
-
(2006)
Human Gene Therapy
, vol.17
, Issue.8
, pp. 845-858
-
-
Jacobson, S.G.1
Boye, S.L.2
Aleman, T.S.3
Conlon, T.J.4
Zeiss, C.J.5
Roman, A.J.6
Cideciyan, A.V.7
Schwartz, S.B.8
Komaromy, A.M.9
Doobrajh, M.10
Cheung, A.Y.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Aguirre, G.D.14
Kaushal, S.15
Maguire, A.M.16
Flotte, T.R.17
Hauswirth, W.W.18
-
56
-
-
20944447776
-
Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success
-
DOI 10.1073/pnas.0500646102
-
Jacobson SG, Aleman TS, Cideciyan AV, et al. Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success. Proc Natl Acad Sci U S A 2005;102:6177-82. (Pubitemid 40594274)
-
(2005)
Proceedings of the National Academy of Sciences of the United States of America
, vol.102
, Issue.17
, pp. 6177-6182
-
-
Jacobson, S.G.1
Aleman, T.S.2
Cideciyan, A.V.3
Sumaroka, A.4
Schwartz, S.B.5
Windsor, E.A.M.6
Traboulsi, E.I.7
Heon, E.8
Pittler, S.J.9
Milam, A.H.10
Maguire, A.M.11
Palczewski, K.12
Stone, E.M.13
Bennett, J.14
-
57
-
-
34249987381
-
Restoration of cone vision in a mouse model of achromatopsia
-
DOI 10.1038/nm1596, PII NM1596
-
Alexander JJ, Umino Y, Everhart D, et al. Restoration of cone vision in a mouse model of achromatopsia. Nat Med 2007;13:685-7. (Pubitemid 46889758)
-
(2007)
Nature Medicine
, vol.13
, Issue.6
, pp. 685-687
-
-
Alexander, J.J.1
Umino, Y.2
Everhart, D.3
Chang, B.4
Min, S.H.5
Li, Q.6
Timmers, A.M.7
Hawes, N.L.8
Pang, J.-J.9
Barlow, R.B.10
Hauswirth, W.W.11
-
58
-
-
43049119982
-
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice
-
DOI 10.1172/JCI34316
-
Allocca M, Doria M, Petrillo M, et al. Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 2008;118:1955-64. (Pubitemid 351632397)
-
(2008)
Journal of Clinical Investigation
, vol.118
, Issue.5
, pp. 1955-1964
-
-
Allocca, M.1
Doria, M.2
Petrillo, M.3
Colella, P.4
Garcia-Hoyos, M.5
Gibbs, D.6
Kim, S.R.7
Maguire, A.8
Rex, T.S.9
Di, V.U.10
Cutillo, L.11
Sparrow, J.R.12
Williams, D.S.13
Bennett, J.14
Auricchio, A.15
-
59
-
-
52049107645
-
Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy
-
Kong J, Kim SR, Binley K, et al. Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy. Gene Ther 2008;15:1311-20.
-
(2008)
Gene Ther
, vol.15
, pp. 1311-1320
-
-
Kong, J.1
Kim, S.R.2
Binley, K.3
-
60
-
-
34648823212
-
Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: Long-term rescue from retinal degeneration
-
DOI 10.1167/iovs.07-0203
-
Kjellstrom S, Bush RA, Zeng Y, et al. Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: long-term rescue from retinal degeneration. Invest Ophthalmol Vis Sci 2007;48:3837-45. (Pubitemid 351261125)
-
(2007)
Investigative Ophthalmology and Visual Science
, vol.48
, Issue.8
, pp. 3837-3845
-
-
Kjellstrom, S.1
Bush, R.A.2
Zeng, Y.3
Takada, Y.4
Sieving, P.A.5
-
61
-
-
49049118498
-
Synaptic pathology in retinoschisis knockout (Rs1-/y) mouse retina and modification by rAAV-Rs1 gene delivery
-
Takada Y, Vijayasarathy C, Zeng Y, et al. Synaptic pathology in retinoschisis knockout (Rs1-/y) mouse retina and modification by rAAV-Rs1 gene delivery. Invest Ophthalmol Vis Sci 2008;49:3677-86.
-
(2008)
Invest Ophthalmol Vis Sci
, vol.49
, pp. 3677-3686
-
-
Takada, Y.1
Vijayasarathy, C.2
Zeng, Y.3
-
62
-
-
67749111389
-
Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse
-
Park TK, Wu Z, Kjellstrom S, et al. Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse. Gene Ther 2009;16:916-26.
-
(2009)
Gene Ther
, vol.16
, pp. 916-926
-
-
Park, T.K.1
Wu, Z.2
Kjellstrom, S.3
-
63
-
-
44349124665
-
Effect of Late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse
-
DOI 10.1038/mt.2008.57, PII MT200857
-
Janssen A, Min SH, Molday LL, et al. Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse. Mol Ther 2008;16:1010-17. (Pubitemid 351737064)
-
(2008)
Molecular Therapy
, vol.16
, Issue.6
, pp. 1010-1017
-
-
Janssen, A.1
Min, S.H.2
Molday, L.L.3
Tanimoto, N.4
Seeliger, M.W.5
Hauswirth, W.W.6
Molday, R.S.7
Weber, B.H.F.8
-
64
-
-
0141430092
-
Gene therapy with brain-derived neurotrophic factor as a protection: Retinal ganglion cells in a rat glaucoma model
-
DOI 10.1167/iovs.02-1332
-
Martin KR, Quigley HA, Zack DJ, et al. Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model. Invest Ophthalmol Vis Sci 2003;44:4357-65. (Pubitemid 37186270)
-
(2003)
Investigative Ophthalmology and Visual Science
, vol.44
, Issue.10
, pp. 4357-4365
-
-
Martin, K.R.G.1
Quigley, H.A.2
Zack, D.J.3
Levkovitch-Verbin, H.4
Kielczewski, J.5
Valenta, D.6
Baumrind, L.7
Pease, M.E.8
Klein, R.L.9
Hauswirth, W.W.10
-
65
-
-
0037258145
-
Delivery of ciliary neurotrophic factor via lentiviral-mediated transfer protects axotomized retinal ganglion cells for an extended period of time
-
DOI 10.1089/104303403321070801
-
van Adel BA, Kostic C, Deglon N, et al. Delivery of ciliary neurotrophic factor via lentiviral-mediated transfer protects axotomized retinal ganglion cells for an extended period of time. Hum Gene Ther 2003;14:103-15. (Pubitemid 36418815)
-
(2003)
Human Gene Therapy
, vol.14
, Issue.2
, pp. 103-115
-
-
Van Adel, B.A.1
Kostic, C.2
Deglon, N.3
Ball, A.K.4
Arsenijevic, Y.5
-
66
-
-
14644423897
-
Effect of GDNF gene transfer into axotomized retinal ganglion cells using in vivo electroporation with a contact lens-type electrode
-
DOI 10.1038/sj.gt.3302277
-
Ishikawa H, Takano M, Matsumoto N, et al. Effect of GDNF gene transfer into axotomized retinal ganglion cells using in vivo electroporation with a contact lens-type electrode. Gene Ther 2005;12:289-98. (Pubitemid 40310131)
-
(2005)
Gene Therapy
, vol.12
, Issue.4
, pp. 289-298
-
-
Ishikawa, H.1
Takano, M.2
Matsumoto, N.3
Sawada, H.4
Ide, C.5
Mimura, O.6
Dezawa, M.7
-
67
-
-
0036406868
-
Inhibition of experimental autoimmune uveoretinitis by systemic and subconjunctival adenovirus-mediated transfer of the viral IL-10 gene
-
De KY, Thillaye-Goldenberg B, Naud MC, et al. Inhibition of experimental autoimmune uveoretinitis by systemic and subconjunctival adenovirus-mediated transfer of the viral IL-10 gene. Clin Exp Immunol 2002;130:212-23.
-
(2002)
Clin Exp Immunol
, vol.130
, pp. 212-223
-
-
De, K.Y.1
Thillaye-Goldenberg, B.2
Naud, M.C.3
-
68
-
-
22644445751
-
Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis
-
DOI 10.1016/j.ymthe.2005.03.018, PII S1525001605001401
-
Broderick CA, Smith AJ, Balaggan KS, et al. Local administration of an adeno-associated viral vector expressing IL-10 reduces monocyte infiltration and subsequent photoreceptor damage during experimental autoimmune uveitis. Mol Ther 2005;12:369-73. (Pubitemid 41025109)
-
(2005)
Molecular Therapy
, vol.12
, Issue.2
, pp. 369-373
-
-
Broderick, C.A.1
Smith, A.J.2
Balaggan, K.S.3
Georgarias, A.4
Buch, P.K.5
Trittibach, P.C.6
Barker, S.E.7
Sarra, G.-M.8
Thrasher, A.J.9
Dick, A.D.10
Ali, R.R.11
-
69
-
-
24944449409
-
Tetracycline-inducible viral interleukin-10 intraocular gene transfer, using adeno-associated virus in experimental autoimmune uveoretinitis
-
DOI 10.1089/hum.2005.16.1037
-
Smith JR, Verwaerde C, Rolling F, et al. Tetracycline-inducible viral interleukin-10 intraocular gene transfer, using adeno-associated virus in experimental autoimmune uveoretinitis. Hum Gene Ther 2005;16:1037-46. (Pubitemid 41324563)
-
(2005)
Human Gene Therapy
, vol.16
, Issue.9
, pp. 1037-1046
-
-
Smith, J.R.1
Verwaerde, C.2
Rolling, F.3
Naud, M.-C.4
Delanoye, A.5
Thillaye-Goldenberg, B.6
Apparailly, F.7
De Kozak, Y.8
-
70
-
-
68749105905
-
Suppression of experimental uveitis by a recombinant adeno-associated virus vector encoding interleukin-1 receptor antagonist
-
Tsai ML, Horng CT, Chen SL, et al. Suppression of experimental uveitis by a recombinant adeno-associated virus vector encoding interleukin-1 receptor antagonist. Mol Vis 2009;15:1542-52.
-
(2009)
Mol Vis
, vol.15
, pp. 1542-1552
-
-
Tsai, M.L.1
Horng, C.T.2
Chen, S.L.3
-
71
-
-
65249152968
-
AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models
-
Shih CS, Laurie N, Holzmacher J, et al. AAV-mediated local delivery of interferon-beta for the treatment of retinoblastoma in preclinical models. Neuromolecular Med 2009;11:43-52.
-
(2009)
Neuromolecular Med
, vol.11
, pp. 43-52
-
-
Shih, C.S.1
Laurie, N.2
Holzmacher, J.3
-
72
-
-
0033540660
-
Suicide gene therapy for treatment of retinoblastoma in a murine model
-
DOI 10.1089/10430349950018887
-
Hurwitz MY, Marcus KT, Chevez-Barrios P, et al. Suicide gene therapy for treatment of retinoblastoma in a murine model. Hum Gene Ther 1999;10:441-8. (Pubitemid 29097322)
-
(1999)
Human Gene Therapy
, vol.10
, Issue.3
, pp. 441-448
-
-
Hurwitz, M.Y.1
Marcus, K.T.2
Chevez-Barrios, P.3
Louie, K.4
Aguilar-Cordova, E.5
Hurwitz, R.L.6
-
73
-
-
70349971731
-
Gene therapy for red-green colour blindness in adult primates
-
Mancuso K, Hauswirth WW, Li Q, et al. Gene therapy for red-green colour blindness in adult primates. Nature 2009;461:784-7.
-
(2009)
Nature
, vol.461
, pp. 784-787
-
-
Mancuso, K.1
Hauswirth, W.W.2
Li, Q.3
-
74
-
-
69249213481
-
Subconjunctival gene delivery of the transcription factor GA-binding protein delays corneal neovascularization in a mouse model
-
Yoon KC, Bae JA, Park HJ, et al. Subconjunctival gene delivery of the transcription factor GA-binding protein delays corneal neovascularization in a mouse model. Gene Ther 2009;16:973-81.
-
(2009)
Gene Ther
, vol.16
, pp. 973-981
-
-
Yoon, K.C.1
Bae, J.A.2
Park, H.J.3
-
75
-
-
0033895534
-
Retinal and choroidal neovascularization
-
DOI 10.1002/1097-4652(200009)184:3<301::AID-JCP3>3.0.CO;2-H
-
Campochiaro PA. Retinal and choroidal neovascularization. J Cell Physiol 2000;184:301-10. (Pubitemid 30625492)
-
(2000)
Journal of Cellular Physiology
, vol.184
, Issue.3
, pp. 301-310
-
-
Campochiaro, P.A.1
-
77
-
-
0038724167
-
Gene therapy for ocular angiogenesis
-
DOI 10.1042/CS20020314
-
Bainbridge JW, Mistry AR, Thrasher AJ, et al. Gene therapy for ocular angiogenesis. Clin Sci (Lond) 2003;104:561-75. (Pubitemid 36713846)
-
(2003)
Clinical Science
, vol.104
, Issue.6
, pp. 561-575
-
-
Bainbridge, J.W.B.1
Mistry, A.R.2
Thrasher, A.J.3
Ali, R.R.4
-
78
-
-
34447253623
-
Prevention of diabetic retinopathy by intraocular soluble flt-1 gene transfer in a spontaneously diabetic rat model
-
Ideno J, Mizukami H, Kakehashi A, et al. Prevention of diabetic retinopathy by intraocular soluble flt-1 gene transfer in a spontaneously diabetic rat model. Int J Mol Med 2007;19:75-9.
-
(2007)
Int J Mol Med
, vol.19
, pp. 75-79
-
-
Ideno, J.1
Mizukami, H.2
Kakehashi, A.3
-
79
-
-
32944481724
-
Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: Results of a phase I clinical trial
-
DOI 10.1089/hum.2006.17.167
-
Campochiaro PA, Nguyen QD, Shah SM, et al. Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial. Hum Gene Ther 2006;17:167-76. (Pubitemid 43262032)
-
(2006)
Human Gene Therapy
, vol.17
, Issue.2
, pp. 167-176
-
-
Campochiaro, P.A.1
Nguyen, Q.D.2
Shah, S.M.3
Klein, M.L.4
Holz, E.5
Frank, R.N.6
Saperstein, D.A.7
Gupta, A.8
Stout, J.T.9
Macko, J.10
DiBartolomeo, R.11
Wei, L.L.12
-
80
-
-
0033870580
-
Spectrum of retGC1 mutations in Leber's congenital amaurosis
-
DOI 10.1038/sj.ejhg.5200503
-
Perrault I, Rozet JM, Gerber S, et al. Spectrum of retGC1 mutations in Leber's congenital amaurosis. Eur J Hum Genet 2000;8:578-82. (Pubitemid 30639363)
-
(2000)
European Journal of Human Genetics
, vol.8
, Issue.8
, pp. 578-582
-
-
Perrault, I.1
Rozet, J.-M.2
Gerber, S.3
Ghazi, I.4
Ducroq, D.5
Souied, E.6
Leowski, C.7
Bonnemaison, M.8
Dufier, J.-L.9
Munnich, A.10
Kaplan, J.11
-
81
-
-
24644507141
-
RPE65 is the isomerohydrolase in the retinoid visual cycle
-
DOI 10.1073/pnas.0503460102
-
Moiseyev G, Chen Y, Takahashi Y, et al. RPE65 is the isomerohydrolase in the retinoid visual cycle. Proc Natl Acad Sci U S A 2005;102:12413-18. (Pubitemid 41266373)
-
(2005)
Proceedings of the National Academy of Sciences of the United States of America
, vol.102
, Issue.35
, pp. 12413-12418
-
-
Moiseyev, G.1
Chen, Y.2
Takahashi, Y.3
Wu, B.X.4
Ma, J.-X.5
-
82
-
-
48449085738
-
Leber congenital amaurosis: Genes, proteins and disease mechanisms
-
den Hollander AI, Roepman R, Koenekoop RK, et al. Leber congenital amaurosis: genes, proteins and disease mechanisms. Prog Retin Eye Res 2008;27:391-419.
-
(2008)
Prog Retin Eye Res
, vol.27
, pp. 391-419
-
-
Den Hollander, A.I.1
Roepman, R.2
Koenekoop, R.K.3
-
83
-
-
33744495152
-
Safety of Recombinant Adeno-Associated Virus Type 2-RPE65 Vector Delivered by Ocular Subretinal Injection
-
DOI 10.1016/j.ymthe.2006.03.005, PII S1525001606000815
-
Jacobson SG, Acland GM, Aguirre GD, et al. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol Ther 2006;13:1074-84. (Pubitemid 43796158)
-
(2006)
Molecular Therapy
, vol.13
, Issue.6
, pp. 1074-1084
-
-
Jacobson, S.G.1
Acland, G.M.2
Aguirre, G.D.3
Aleman, T.S.4
Schwartz, S.B.5
Cideciyan, A.V.6
Zeiss, C.J.7
Komaromy, A.M.8
Kaushal, S.9
Roman, A.J.10
Windsor, E.A.M.11
Sumaroka, A.12
Pearce-Kelling, S.E.13
Conlon, T.J.14
Chiodo, V.A.15
Boye, S.L.16
Flotte, T.R.17
Maguire, A.M.18
Bennett, J.19
Hauswirth, W.W.20
more..
-
84
-
-
33748537392
-
Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function
-
DOI 10.1016/j.visres.2006.05.010, PII S004269890600246X
-
Nusinowitz S, Ridder WH III, Pang JJ, et al. Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function. Vision Res 2006;46:3926-34. (Pubitemid 44374620)
-
(2006)
Vision Research
, vol.46
, Issue.22
, pp. 3926-3934
-
-
Nusinowitz, S.1
Ridder III, W.H.2
Pang, J.J.3
Chang, B.4
Noorwez, S.M.5
Kaushal, S.6
Hauswirth, W.W.7
Heckenlively, J.R.8
-
85
-
-
0027521914
-
Apoptosis: Final common pathway of photoreceptor death in rd, rds, and rhodopsin mutant mice
-
DOI 10.1016/0896-6273(93)90072-Y
-
Chang GQ, Hao Y, Wong F. Apoptosis: final common pathway of photoreceptor death in rd, rds, and rhodopsin mutant mice. Neuron 1993;11:595-605. (Pubitemid 23321770)
-
(1993)
Neuron
, vol.11
, Issue.4
, pp. 595-605
-
-
Chang, G.-Q.1
Hao, Y.2
Wong, F.3
-
86
-
-
7044228129
-
Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration
-
DOI 10.1016/j.ymthe.2004.07.027, PII S1525001604013619
-
Rex TS, Allocca M, Domenici L, et al. Systemic but not intraocular Epo gene transfer protects the retina from light-and genetic-induced degeneration. Mol Ther 2004;10:855-61. (Pubitemid 39419912)
-
(2004)
Molecular Therapy
, vol.10
, Issue.5
, pp. 855-861
-
-
Rex, T.S.1
Allocca, M.2
Domenici, L.3
Surace, E.M.4
Maguire, A.M.5
Lyubarsky, A.6
Cellerino, A.7
Bennett, J.8
Auricchio, A.9
-
87
-
-
0027522462
-
Intravitreal injections of neurotrophic factors support the survival of axotomized retinal ganglion cells in adult rats in vivo
-
DOI 10.1016/0006-8993(93)90695-J
-
Mey J, Thanos S. Intravitreal injections of neurotrophic factors support the survival of axotomized retinal ganglion cells in adult rats in vivo. Brain Res 1993;602:304-17. (Pubitemid 23053202)
-
(1993)
Brain Research
, vol.602
, Issue.2
, pp. 304-317
-
-
Mey, J.1
Thanos, S.2
-
88
-
-
62549088292
-
Prolonged transgene expression with lentiviral vectors in the aqueous humor outflow pathway of nonhuman primates
-
Barraza RA, Rasmussen CA, Loewen N, et al. Prolonged transgene expression with lentiviral vectors in the aqueous humor outflow pathway of nonhuman primates. Hum Gene Ther 2009;20:191-200.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 191-200
-
-
Barraza, R.A.1
Rasmussen, C.A.2
Loewen, N.3
-
89
-
-
66949169925
-
Gene therapy targeting glaucoma: Where are we?
-
Liu X, Rasmussen CA, Gabelt BT, et al. Gene therapy targeting glaucoma: where are we? Surv Ophthalmol 2009;54:472-86.
-
(2009)
Surv Ophthalmol
, vol.54
, pp. 472-486
-
-
Liu, X.1
Rasmussen, C.A.2
Gabelt, B.T.3
-
90
-
-
76749093047
-
The anti-glioma effect of suicide gene therapy using BMSC expressing HSV/TK combined with overexpression of Cx43 in glioma cells
-
Huang Q, Liu XZ, Kang CS, et al. The anti-glioma effect of suicide gene therapy using BMSC expressing HSV/TK combined with overexpression of Cx43 in glioma cells. Cancer Gene Ther 2010;17:192-202.
-
(2010)
Cancer Gene Ther
, vol.17
, pp. 192-202
-
-
Huang, Q.1
Liu, X.Z.2
Kang, C.S.3
-
91
-
-
70349595944
-
Adenovirus-mediated suicide gene therapy under the control of Cox-2 promoter for colorectal cancer
-
Wang ZX, Bian HB, Yang JS, et al. Adenovirus-mediated suicide gene therapy under the control of Cox-2 promoter for colorectal cancer. Cancer Biol Ther 2009;8:1480-8.
-
(2009)
Cancer Biol Ther
, vol.8
, pp. 1480-1488
-
-
Wang, Z.X.1
Bian, H.B.2
Yang, J.S.3
-
92
-
-
68949197421
-
Phase I and biodistribution study of recombinant adenovirus vector-mediated herpes simplex virus thymidine kinase gene and ganciclovir administration in patients with head and neck cancer and other malignant tumors
-
Xu F, Li S, Li XL, et al. Phase I and biodistribution study of recombinant adenovirus vector-mediated herpes simplex virus thymidine kinase gene and ganciclovir administration in patients with head and neck cancer and other malignant tumors. Cancer Gene Ther 2009;16:723-30.
-
(2009)
Cancer Gene Ther
, vol.16
, pp. 723-730
-
-
Xu, F.1
Li, S.2
Li, X.L.3
-
93
-
-
0346957021
-
Prospects for gene therapy in corneal disease
-
DOI 10.1038/sj.eye.6700565
-
Jun AS, Larkin DF. Prospects for gene therapy in corneal disease. Eye (Lond) 2003;17:906-11. (Pubitemid 37541043)
-
(2003)
Eye
, vol.17
, Issue.8
, pp. 906-911
-
-
Jun, A.S.1
Larkin, D.F.P.2
-
94
-
-
0035346066
-
Adenovirus-mediated gene transfer in canine eyes: A preclinical study for gene therapy of human uveal melanoma
-
DOI 10.1002/1521-2254(200105/06)3:3<228::AID-JGM186>3.0.CO;2-A
-
Andrawiss M, Maron A, Beltran W, et al. Adenovirus-mediated gene transfer in canine eyes: a preclinical study for gene therapy of human uveal melanoma. J Gene Med 2001;3:228-39. (Pubitemid 33684771)
-
(2001)
Journal of Gene Medicine
, vol.3
, Issue.3
, pp. 228-239
-
-
Andrawiss, M.1
Maron, A.2
Beltran, W.3
Opolon, P.4
Connault, E.5
Griscelli, F.6
Yeh, P.7
Perricaudet, M.8
Devauchelle, P.9
-
95
-
-
9944226710
-
Gene therapy for optic nerve disease
-
DOI 10.1038/sj.eye.6701579
-
Martin KR, Quigley HA. Gene therapy for optic nerve disease. Eye (Lond) 2004;18:1049-55. (Pubitemid 39591786)
-
(2004)
Eye
, vol.18
, Issue.11
, pp. 1049-1055
-
-
Martin, K.R.G.1
Quigley, H.A.2
-
96
-
-
58149478117
-
Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization
-
Pechan P, Rubin H, Lukason M, et al. Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization. Gene Ther 2009;16:10-16.
-
(2009)
Gene Ther
, vol.16
, pp. 10-16
-
-
Pechan, P.1
Rubin, H.2
Lukason, M.3
-
97
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
DOI 10.1056/NEJMoa0802268
-
Bainbridge JW, Smith AJ, Barker SS, et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 2008;358:2231-9. (Pubitemid 351724452)
-
(2008)
New England Journal of Medicine
, vol.358
, Issue.21
, pp. 2231-2239
-
-
Bainbridge, J.W.B.1
Smith, A.J.2
Barker, S.S.3
Robbie, S.4
Henderson, R.5
Balaggan, K.6
Viswanathan, A.7
Holder, G.E.8
Stockman, A.9
Tyler, N.10
Petersen-Jones, S.11
Bhattacharya, S.S.12
Thrasher, A.J.13
Fitzke, F.W.14
Carter, B.J.15
Rubin, G.S.16
Moore, A.T.17
Ali, R.R.18
-
98
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan AV, Aleman TS, Boye SL, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 2008;105:15112-17.
-
(2008)
Proc Natl Acad Sci U S A
, vol.105
, pp. 15112-15117
-
-
Cideciyan, A.V.1
Aleman, T.S.2
Boye, S.L.3
-
99
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
DOI 10.1056/NEJMoa0802315
-
Maguire AM, Simonelli F, Pierce EA, et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 2008;358:2240-8. (Pubitemid 351724453)
-
(2008)
New England Journal of Medicine
, vol.358
, Issue.21
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh Jr., E.N.4
Mingozzi, F.5
Bennicelli, J.6
Banfi, S.7
Marshall, K.A.8
Testa, F.9
Surace, E.M.10
Rossi, S.11
Lyubarsky, A.12
Arruda, V.R.13
Konkle, B.14
Stone, E.15
Sun, J.16
Jacobs, J.17
Dell'Osso, L.18
Hertle, R.19
Ma, J.-X.20
Redmond, T.M.21
Zhu, X.22
Hauck, B.23
Zelenaia, O.24
Shindler, K.S.25
Maguire, M.G.26
Wright, J.F.27
Volpe, N.J.28
McDonnell, J.W.29
Auricchio, A.30
High, K.A.31
Bennett, J.32
more..
-
100
-
-
70349105559
-
Human RPE65 gene therapy for Leber congenital amaurosis: Persistence of early visual improvements and safety at 1 year
-
Cideciyan AV, Hauswirth WW, Aleman TS, et al. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther 2009;20:999-1004.
-
(2009)
Hum Gene Ther
, vol.20
, pp. 999-1004
-
-
Cideciyan, A.V.1
Hauswirth, W.W.2
Aleman, T.S.3
-
101
-
-
77649242176
-
Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration
-
Simonelli F, Maguire AM, Testa F, et al. Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 2010;18:643-50.
-
(2010)
Mol Ther
, vol.18
, pp. 643-650
-
-
Simonelli, F.1
Maguire, A.M.2
Testa, F.3
-
102
-
-
70350620424
-
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial
-
Maguire AM, High KA, Auricchio A, et al. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009;374:1597-605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
-
103
-
-
31644447744
-
Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir
-
DOI 10.1200/JCO.2004.00.1883
-
Chévez-Barrios P, Chintagumpala M, Mieler W, et al. Response of retinoblastoma with vitreous tumor seeding to adenovirus-mediated delivery of thymidine kinase followed by ganciclovir. J Clin Oncol 2005;23:7927-35. (Pubitemid 46657392)
-
(2005)
Journal of Clinical Oncology
, vol.23
, Issue.31
, pp. 7927-7935
-
-
Chevez-Barrios, P.1
Chintagumpala, M.2
Mieler, W.3
Paysse, E.4
Boniuk, M.5
Kozinetz, C.6
Hurwitz, M.Y.7
Hurwitz, R.L.8
-
104
-
-
67650409973
-
Oncolytic adenovirus delivering herpes simplex virus thymidine kinase suicide gene reduces the growth of human retinoblastoma in an in vivo mouse model
-
Ji X, Zhang J, Cheng L, et al. Oncolytic adenovirus delivering herpes simplex virus thymidine kinase suicide gene reduces the growth of human retinoblastoma in an in vivo mouse model. Exp Eye Res 2009;89:193-9.
-
(2009)
Exp Eye Res
, vol.89
, pp. 193-199
-
-
Ji, X.1
Zhang, J.2
Cheng, L.3
|