-
1
-
-
84871388247
-
Clinical applications of retinal gene therapy
-
1 Lipinski, D.M., Thake, M., MacLaren, R.E., Clinical applications of retinal gene therapy. Prog. Retin. Eye Res. 32 (2013), 22–47.
-
(2013)
Prog. Retin. Eye Res.
, vol.32
, pp. 22-47
-
-
Lipinski, D.M.1
Thake, M.2
MacLaren, R.E.3
-
2
-
-
74049085498
-
Effect of genome size on AAV vector packaging
-
2 Wu, Z., Yang, H., Colosi, P., Effect of genome size on AAV vector packaging. Mol. Ther. 18 (2010), 80–86.
-
(2010)
Mol. Ther.
, vol.18
, pp. 80-86
-
-
Wu, Z.1
Yang, H.2
Colosi, P.3
-
3
-
-
84883719513
-
Immunology of AAV-mediated gene transfer in the eye
-
3 Willett, K., Bennett, J., Immunology of AAV-mediated gene transfer in the eye. Front. Immunol., 4, 2013, 261.
-
(2013)
Front. Immunol.
, vol.4
, pp. 261
-
-
Willett, K.1
Bennett, J.2
-
4
-
-
0029914677
-
Gene transfer into the mouse retina mediated by an adeno-associated viral vector
-
4 Ali, R.R., Reichel, M.B., Thrasher, A.J., Levinsky, R.J., Kinnon, C., Kanuga, N., Hunt, D.M., Bhattacharya, S.S., Gene transfer into the mouse retina mediated by an adeno-associated viral vector. Hum. Mol. Genet. 5 (1996), 591–594.
-
(1996)
Hum. Mol. Genet.
, vol.5
, pp. 591-594
-
-
Ali, R.R.1
Reichel, M.B.2
Thrasher, A.J.3
Levinsky, R.J.4
Kinnon, C.5
Kanuga, N.6
Hunt, D.M.7
Bhattacharya, S.S.8
-
5
-
-
0035894739
-
Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model
-
5 Auricchio, A., Kobinger, G., Anand, V., Hildinger, M., O'Connor, E., Maguire, A.M., Wilson, J.M., Bennett, J., Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model. Hum. Mol. Genet. 10 (2001), 3075–3081.
-
(2001)
Hum. Mol. Genet.
, vol.10
, pp. 3075-3081
-
-
Auricchio, A.1
Kobinger, G.2
Anand, V.3
Hildinger, M.4
O'Connor, E.5
Maguire, A.M.6
Wilson, J.M.7
Bennett, J.8
-
6
-
-
0036310297
-
Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size
-
6 Yang, G.S., Schmidt, M., Yan, Z., Lindbloom, J.D., Harding, T.C., Donahue, B.A., Engelhardt, J.F., Kotin, R., Davidson, B.L., Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size. J. Virol. 76 (2002), 7651–7660.
-
(2002)
J. Virol.
, vol.76
, pp. 7651-7660
-
-
Yang, G.S.1
Schmidt, M.2
Yan, Z.3
Lindbloom, J.D.4
Harding, T.C.5
Donahue, B.A.6
Engelhardt, J.F.7
Kotin, R.8
Davidson, B.L.9
-
7
-
-
84876013531
-
Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina
-
7 Charbel Issa, P., De Silva, S.R., Lipinski, D.M., Singh, M.S., Mouravlev, A., You, Q., Barnard, A.R., Hankins, M.W., During, M.J., Maclaren, R.E., Assessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina. PLoS ONE, 8, 2013, e60361.
-
(2013)
PLoS ONE
, vol.8
, pp. e60361
-
-
Charbel Issa, P.1
De Silva, S.R.2
Lipinski, D.M.3
Singh, M.S.4
Mouravlev, A.5
You, Q.6
Barnard, A.R.7
Hankins, M.W.8
During, M.J.9
Maclaren, R.E.10
-
8
-
-
84875220318
-
A comprehensive review of retinal gene therapy
-
8 Boye, S.E., Boye, S.L., Lewin, A.S., Hauswirth, W.W., A comprehensive review of retinal gene therapy. Mol. Ther. 21 (2013), 509–519.
-
(2013)
Mol. Ther.
, vol.21
, pp. 509-519
-
-
Boye, S.E.1
Boye, S.L.2
Lewin, A.S.3
Hauswirth, W.W.4
-
9
-
-
33744495152
-
Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection
-
9 Jacobson, S.G., Acland, G.M., Aguirre, G.D., Aleman, T.S., Schwartz, S.B., Cideciyan, A.V., Zeiss, C.J., Komaromy, A.M., Kaushal, S., Roman, A.J., et al. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Mol. Ther. 13 (2006), 1074–1084.
-
(2006)
Mol. Ther.
, vol.13
, 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
-
10
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
10 Bainbridge, J.W., Smith, A.J., Barker, S.S., Robbie, S., Henderson, R., Balaggan, K., Viswanathan, A., Holder, G.E., Stockman, A., Tyler, N., et al. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358 (2008), 2231–2239.
-
(2008)
N. Engl. J. Med.
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.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
-
11
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
11 Maguire, A.M., Simonelli, F., Pierce, E.A., Pugh, E.N. Jr., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K.A., Testa, F., Surace, E.M., et al. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358 (2008), 2240–2248.
-
(2008)
N. Engl. J. Med.
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
Pugh, E.N.4
Mingozzi, F.5
Bennicelli, J.6
Banfi, S.7
Marshall, K.A.8
Testa, F.9
Surace, E.M.10
-
12
-
-
54949104686
-
Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial
-
12 Hauswirth, W.W., Aleman, T.S., Kaushal, S., Cideciyan, A.V., Schwartz, S.B., Wang, L., Conlon, T.J., Boye, S.L., Flotte, T.R., Byrne, B.J., Jacobson, S.G., Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum. Gene Ther. 19 (2008), 979–990.
-
(2008)
Hum. Gene Ther.
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
Cideciyan, A.V.4
Schwartz, S.B.5
Wang, L.6
Conlon, T.J.7
Boye, S.L.8
Flotte, T.R.9
Byrne, B.J.10
Jacobson, S.G.11
-
13
-
-
84897051037
-
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial
-
13 MacLaren, R.E., Groppe, M., Barnard, A.R., Cottriall, C.L., Tolmachova, T., Seymour, L., Clark, K.R., During, M.J., Cremers, F.P., Black, G.C., et al. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. Lancet 383 (2014), 1129–1137.
-
(2014)
Lancet
, vol.383
, pp. 1129-1137
-
-
MacLaren, R.E.1
Groppe, M.2
Barnard, A.R.3
Cottriall, C.L.4
Tolmachova, T.5
Seymour, L.6
Clark, K.R.7
During, M.J.8
Cremers, F.P.9
Black, G.C.10
-
14
-
-
84973472718
-
Visual acuity after retinal gene therapy for choroideremia
-
14 Edwards, T.L., Jolly, J.K., Groppe, M., Barnard, A.R., Cottriall, C.L., Tolmachova, T., Black, G.C., Webster, A.R., Lotery, A.J., Holder, G.E., et al. Visual acuity after retinal gene therapy for choroideremia. N. Engl. J. Med. 374 (2016), 1996–1998.
-
(2016)
N. Engl. J. Med.
, vol.374
, pp. 1996-1998
-
-
Edwards, T.L.1
Jolly, J.K.2
Groppe, M.3
Barnard, A.R.4
Cottriall, C.L.5
Tolmachova, T.6
Black, G.C.7
Webster, A.R.8
Lotery, A.J.9
Holder, G.E.10
-
15
-
-
84958777137
-
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
-
15 Ghazi, N.G., Abboud, E.B., Nowilaty, S.R., Alkuraya, H., Alhommadi, A., Cai, H., Hou, R., Deng, W.T., Boye, S.L., Almaghamsi, A., et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Hum. Genet. 135 (2016), 327–343.
-
(2016)
Hum. Genet.
, vol.135
, pp. 327-343
-
-
Ghazi, N.G.1
Abboud, E.B.2
Nowilaty, S.R.3
Alkuraya, H.4
Alhommadi, A.5
Cai, H.6
Hou, R.7
Deng, W.T.8
Boye, S.L.9
Almaghamsi, A.10
-
16
-
-
84952639507
-
Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial
-
16 Rakoczy, E.P., Lai, C.-M., Magno, A.L., Wikstrom, M.E., French, M.A., Pierce, C.M., Schwartz, S.D., Blumenkranz, M.S., Chalberg, T.W., Degli-Esposti, M.A., Constable, I.J., Gene therapy with recombinant adeno-associated vectors for neovascular age-related macular degeneration: 1 year follow-up of a phase 1 randomised clinical trial. Lancet 386 (2015), 2395–2403.
-
(2015)
Lancet
, vol.386
, pp. 2395-2403
-
-
Rakoczy, E.P.1
Lai, C.-M.2
Magno, A.L.3
Wikstrom, M.E.4
French, M.A.5
Pierce, C.M.6
Schwartz, S.D.7
Blumenkranz, M.S.8
Chalberg, T.W.9
Degli-Esposti, M.A.10
Constable, I.J.11
-
17
-
-
84929353143
-
Long-term effect of gene therapy on Leber's congenital amaurosis
-
17 Bainbridge, J.W.B., Mehat, M.S., Sundaram, V., Robbie, S.J., Barker, S.E., Ripamonti, C., Georgiadis, A., Mowat, F.M., Beattie, S.G., Gardner, P.J., et al. Long-term effect of gene therapy on Leber's congenital amaurosis. N. Engl. J. Med. 372 (2015), 1887–1897.
-
(2015)
N. Engl. J. Med.
, vol.372
, pp. 1887-1897
-
-
Bainbridge, J.W.B.1
Mehat, M.S.2
Sundaram, V.3
Robbie, S.J.4
Barker, S.E.5
Ripamonti, C.6
Georgiadis, A.7
Mowat, F.M.8
Beattie, S.G.9
Gardner, P.J.10
-
18
-
-
84988661136
-
Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65
-
18 Georgiadis, A., Duran, Y., Ribeiro, J., Abelleira-Hervas, L., Robbie, S.J., Sünkel-Laing, B., Fourali, S., Gonzalez-Cordero, A., Cristante, E., Michaelides, M., et al. Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65. Gene Ther. 23 (2016), 857–862.
-
(2016)
Gene Ther.
, vol.23
, pp. 857-862
-
-
Georgiadis, A.1
Duran, Y.2
Ribeiro, J.3
Abelleira-Hervas, L.4
Robbie, S.J.5
Sünkel-Laing, B.6
Fourali, S.7
Gonzalez-Cordero, A.8
Cristante, E.9
Michaelides, M.10
-
19
-
-
0033588890
-
Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy
-
19 Loeb, J.E., Cordier, W.S., Harris, M.E., Weitzman, M.D., Hope, T.J., Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy. Hum. Gene Ther. 10 (1999), 2295–2305.
-
(1999)
Hum. Gene Ther.
, vol.10
, pp. 2295-2305
-
-
Loeb, J.E.1
Cordier, W.S.2
Harris, M.E.3
Weitzman, M.D.4
Hope, T.J.5
-
20
-
-
0032978712
-
Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors
-
20 Zufferey, R., Donello, J.E., Trono, D., Hope, T.J., Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73 (1999), 2886–2892.
-
(1999)
J. Virol.
, vol.73
, pp. 2886-2892
-
-
Zufferey, R.1
Donello, J.E.2
Trono, D.3
Hope, T.J.4
-
21
-
-
0033870226
-
Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain
-
21 Paterna, J.-C., Moccetti, T., Mura, A., Feldon, J., Büeler, H., Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain. Gene Ther. 7 (2000), 1304–1311.
-
(2000)
Gene Ther.
, vol.7
, pp. 1304-1311
-
-
Paterna, J.-C.1
Moccetti, T.2
Mura, A.3
Feldon, J.4
Büeler, H.5
-
22
-
-
17944378442
-
Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes
-
22 Xu, R., Janson, C.G., Mastakov, M., Lawlor, P., Young, D., Mouravlev, A., Fitzsimons, H., Choi, K.L., Ma, H., Dragunow, M., et al. Quantitative comparison of expression with adeno-associated virus (AAV-2) brain-specific gene cassettes. Gene Ther. 8 (2001), 1323–1332.
-
(2001)
Gene Ther.
, vol.8
, pp. 1323-1332
-
-
Xu, R.1
Janson, C.G.2
Mastakov, M.3
Lawlor, P.4
Young, D.5
Mouravlev, A.6
Fitzsimons, H.7
Choi, K.L.8
Ma, H.9
Dragunow, M.10
-
23
-
-
0036315645
-
Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain
-
23 Klein, R.L., Hamby, M.E., Gong, Y., Hirko, A.C., Wang, S., Hughes, J.A., King, M.A., Meyer, E.M., Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain. Exp. Neurol. 176 (2002), 66–74.
-
(2002)
Exp. Neurol.
, vol.176
, pp. 66-74
-
-
Klein, R.L.1
Hamby, M.E.2
Gong, Y.3
Hirko, A.C.4
Wang, S.5
Hughes, J.A.6
King, M.A.7
Meyer, E.M.8
-
24
-
-
3042790053
-
Increased in vitro and in vivo transgene expression levels mediated through cis-acting elements
-
24 Johansen, J., Tornøe, J., Møller, A., Johansen, T.E., Increased in vitro and in vivo transgene expression levels mediated through cis-acting elements. J. Gene Med. 5 (2003), 1080–1089.
-
(2003)
J. Gene Med.
, vol.5
, pp. 1080-1089
-
-
Johansen, J.1
Tornøe, J.2
Møller, A.3
Johansen, T.E.4
-
25
-
-
84866899464
-
Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union
-
25 Ylä-Herttuala, S., Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union. Mol. Ther. 20 (2012), 1831–1832.
-
(2012)
Mol. Ther.
, vol.20
, pp. 1831-1832
-
-
Ylä-Herttuala, S.1
-
26
-
-
34249298695
-
Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors
-
26 Schambach, A., Galla, M., Maetzig, T., Loew, R., Baum, C., Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol. Ther. 15 (2007), 1167–1173.
-
(2007)
Mol. Ther.
, vol.15
, pp. 1167-1173
-
-
Schambach, A.1
Galla, M.2
Maetzig, T.3
Loew, R.4
Baum, C.5
-
27
-
-
34548125356
-
The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors
-
27 Higashimoto, T., Urbinati, F., Perumbeti, A., Jiang, G., Zarzuela, A., Chang, L.-J., Kohn, D.B., Malik, P., The woodchuck hepatitis virus post-transcriptional regulatory element reduces readthrough transcription from retroviral vectors. Gene Ther. 14 (2007), 1298–1304.
-
(2007)
Gene Ther.
, vol.14
, pp. 1298-1304
-
-
Higashimoto, T.1
Urbinati, F.2
Perumbeti, A.3
Jiang, G.4
Zarzuela, A.5
Chang, L.-J.6
Kohn, D.B.7
Malik, P.8
-
28
-
-
84879842921
-
Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo
-
28 Tolmachova, T., Tolmachov, O.E., Barnard, A.R., de Silva, S.R., Lipinski, D.M., Walker, N.J., Maclaren, R.E., Seabra, M.C., Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo. J. Mol. Med. (Berl.) 91 (2013), 825–837.
-
(2013)
J. Mol. Med. (Berl.)
, vol.91
, pp. 825-837
-
-
Tolmachova, T.1
Tolmachov, O.E.2
Barnard, A.R.3
de Silva, S.R.4
Lipinski, D.M.5
Walker, N.J.6
Maclaren, R.E.7
Seabra, M.C.8
-
29
-
-
84859913048
-
Optimization of in vivo confocal autofluorescence imaging of the ocular fundus in mice and its application to models of human retinal degeneration
-
29 Charbel Issa, P., Singh, M.S., Lipinski, D.M., Chong, N.V., Delori, F.C., Barnard, A.R., MacLaren, R.E., Optimization of in vivo confocal autofluorescence imaging of the ocular fundus in mice and its application to models of human retinal degeneration. Invest. Ophthalmol. Vis. Sci. 53 (2012), 1066–1075.
-
(2012)
Invest. Ophthalmol. Vis. Sci.
, vol.53
, pp. 1066-1075
-
-
Charbel Issa, P.1
Singh, M.S.2
Lipinski, D.M.3
Chong, N.V.4
Delori, F.C.5
Barnard, A.R.6
MacLaren, R.E.7
-
30
-
-
84882787378
-
Fundus autofluorescence in the Abca4(-/-) mouse model of Stargardt disease-correlation with accumulation of A2E, retinal function, and histology
-
30 Charbel Issa, P., Barnard, A.R., Singh, M.S., Carter, E., Jiang, Z., Radu, R.A., Schraermeyer, U., MacLaren, R.E., Fundus autofluorescence in the Abca4(-/-) mouse model of Stargardt disease-correlation with accumulation of A2E, retinal function, and histology. Invest. Ophthalmol. Vis. Sci. 54 (2013), 5602–5612.
-
(2013)
Invest. Ophthalmol. Vis. Sci.
, vol.54
, pp. 5602-5612
-
-
Charbel Issa, P.1
Barnard, A.R.2
Singh, M.S.3
Carter, E.4
Jiang, Z.5
Radu, R.A.6
Schraermeyer, U.7
MacLaren, R.E.8
-
31
-
-
84923914151
-
Gene therapy for choroideremia using an adeno-associated viral (AAV) vector
-
31 Barnard, A.R., Groppe, M., MacLaren, R.E., Gene therapy for choroideremia using an adeno-associated viral (AAV) vector. Cold Spring Harb. Perspect. Med., 5, 2014, a017293.
-
(2014)
Cold Spring Harb. Perspect. Med.
, vol.5
, pp. a017293
-
-
Barnard, A.R.1
Groppe, M.2
MacLaren, R.E.3
-
32
-
-
70349850388
-
Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates
-
32 Lawlor, P.A., Bland, R.J., Mouravlev, A., Young, D., During, M.J., Efficient gene delivery and selective transduction of glial cells in the mammalian brain by AAV serotypes isolated from nonhuman primates. Mol. Ther. 17 (2009), 1692–1702.
-
(2009)
Mol. Ther.
, vol.17
, pp. 1692-1702
-
-
Lawlor, P.A.1
Bland, R.J.2
Mouravlev, A.3
Young, D.4
During, M.J.5
-
33
-
-
49049092393
-
Development and characterization of an adult retinal explant organotypic tissue culture system as an in vitro intraocular stem cell transplantation model
-
33 Johnson, T.V., Martin, K.R., Development and characterization of an adult retinal explant organotypic tissue culture system as an in vitro intraocular stem cell transplantation model. Invest. Ophthalmol. Vis. Sci. 49 (2008), 3503–3512.
-
(2008)
Invest. Ophthalmol. Vis. Sci.
, vol.49
, pp. 3503-3512
-
-
Johnson, T.V.1
Martin, K.R.2
-
34
-
-
84942292881
-
Gene therapy for retinal disease: what lies ahead
-
34 MacLaren, R.E., Gene therapy for retinal disease: what lies ahead. Ophthalmologica 234 (2015), 1–5.
-
(2015)
Ophthalmologica
, vol.234
, pp. 1-5
-
-
MacLaren, R.E.1
-
35
-
-
0037064165
-
Subthalamic GAD gene therapy in a Parkinson's disease rat model
-
35 Luo, J., Kaplitt, M.G., Fitzsimons, H.L., Zuzga, D.S., Liu, Y., Oshinsky, M.L., During, M.J., Subthalamic GAD gene therapy in a Parkinson's disease rat model. Science 298 (2002), 425–429.
-
(2002)
Science
, vol.298
, pp. 425-429
-
-
Luo, J.1
Kaplitt, M.G.2
Fitzsimons, H.L.3
Zuzga, D.S.4
Liu, Y.5
Oshinsky, M.L.6
During, M.J.7
-
36
-
-
34250683023
-
Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial
-
36 Kaplitt, M.G., Feigin, A., Tang, C., Fitzsimons, H.L., Mattis, P., Lawlor, P.A., Bland, R.J., Young, D., Strybing, K., Eidelberg, D., During, M.J., Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial. Lancet 369 (2007), 2097–2105.
-
(2007)
Lancet
, vol.369
, pp. 2097-2105
-
-
Kaplitt, M.G.1
Feigin, A.2
Tang, C.3
Fitzsimons, H.L.4
Mattis, P.5
Lawlor, P.A.6
Bland, R.J.7
Young, D.8
Strybing, K.9
Eidelberg, D.10
During, M.J.11
-
37
-
-
84877148806
-
AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models
-
37 Vasireddy, V., Mills, J.A., Gaddameedi, R., Basner-Tschakarjan, E., Kohnke, M., Black, A.D., Alexandrov, K., Zhou, S., Maguire, A.M., Chung, D.C., et al. AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models. PLoS ONE, 8, 2013, e61396.
-
(2013)
PLoS ONE
, vol.8
, pp. e61396
-
-
Vasireddy, V.1
Mills, J.A.2
Gaddameedi, R.3
Basner-Tschakarjan, E.4
Kohnke, M.5
Black, A.D.6
Alexandrov, K.7
Zhou, S.8
Maguire, A.M.9
Chung, D.C.10
-
38
-
-
84904540483
-
Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models
-
38 Black, A., Vasireddy, V., Chung, D.C., Maguire, A.M., Gaddameedi, R., Tolmachova, T., Seabra, M., Bennett, J., Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models. J. Gene Med. 16 (2014), 122–130.
-
(2014)
J. Gene Med.
, vol.16
, pp. 122-130
-
-
Black, A.1
Vasireddy, V.2
Chung, D.C.3
Maguire, A.M.4
Gaddameedi, R.5
Tolmachova, T.6
Seabra, M.7
Bennett, J.8
-
39
-
-
84981508189
-
Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4(-/-) mouse and bipolar cells in the rd1 mouse and human retina ex vivo
-
39 De Silva, S.R., Charbel Issa, P., Singh, M.S., Lipinski, D.M., Barnea-Cramer, A.O., Walker, N.J., Barnard, A.R., Hankins, M.W., MacLaren, R.E., Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4(-/-) mouse and bipolar cells in the rd1 mouse and human retina ex vivo. Gene Ther. 23 (2016), 767–774.
-
(2016)
Gene Ther.
, vol.23
, pp. 767-774
-
-
De Silva, S.R.1
Charbel Issa, P.2
Singh, M.S.3
Lipinski, D.M.4
Barnea-Cramer, A.O.5
Walker, N.J.6
Barnard, A.R.7
Hankins, M.W.8
MacLaren, R.E.9
-
40
-
-
0029995531
-
A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells
-
40 Zolotukhin, S., Potter, M., Hauswirth, W.W., Guy, J., Muzyczka, N., A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells. J. Virol. 70 (1996), 4646–4654.
-
(1996)
J. Virol.
, vol.70
, pp. 4646-4654
-
-
Zolotukhin, S.1
Potter, M.2
Hauswirth, W.W.3
Guy, J.4
Muzyczka, N.5
-
41
-
-
0033022953
-
Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield
-
41 Zolotukhin, S., Byrne, B.J., Mason, E., Zolotukhin, I., Potter, M., Chesnut, K., Summerford, C., Samulski, R.J., Muzyczka, N., Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield. Gene Ther. 6 (1999), 973–985.
-
(1999)
Gene Ther.
, vol.6
, pp. 973-985
-
-
Zolotukhin, S.1
Byrne, B.J.2
Mason, E.3
Zolotukhin, I.4
Potter, M.5
Chesnut, K.6
Summerford, C.7
Samulski, R.J.8
Muzyczka, N.9
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