-
1
-
-
44249120315
-
Effect of gene therapy on visual function in Leber's congenital amaurosis
-
Bainbridge, J.W., Smith, A.J., Barker, S.S., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med. 358, 2231-2239.
-
(2008)
N. Engl. J. Med.
, vol.358
, pp. 2231-2239
-
-
Bainbridge, J.W.1
Smith, A.J.2
Barker, S.S.3
-
2
-
-
77956985453
-
The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders
-
Bramall, A.N., Wright, A.F., Jacobson, S.G., and McInnes, R.R. (2010). The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders. Annu. Rev. Neurosci. 33, 441-472.
-
(2010)
Annu. Rev. Neurosci.
, vol.33
, pp. 441-472
-
-
Bramall, A.N.1
Wright, A.F.2
Jacobson, S.G.3
McInnes, R.R.4
-
3
-
-
54449085219
-
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
-
Cideciyan, A.V., Aleman, T.S., Boye, S.L., et al. (2008). 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. 105, 15112-15117.
-
(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
-
4
-
-
84873453664
-
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement
-
Cideciyan, A.V., Jacobson, S.G., Beltran, W.A., et al. (2013). Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc. Natl. Acad. Sci. U. S. A. 110, E517-525.
-
(2013)
Proc. Natl. Acad. Sci. U. S. A.
, vol.110
-
-
Cideciyan, A.V.1
Jacobson, S.G.2
Beltran, W.A.3
-
5
-
-
33750947173
-
Retinitis pigmentosa
-
Hartong, D.T., Berson, E.L., and Dryja, T.P. (2006). Retinitis pigmentosa. Lancet 368, 1795-1809.
-
(2006)
Lancet
, vol.368
, pp. 1795-1809
-
-
Hartong, D.T.1
Berson, E.L.2
Dryja, T.P.3
-
6
-
-
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
-
Hauswirth, W.W., Aleman, T.S., Kaushal, S., et al. (2008). 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, 979-990.
-
(2008)
Hum. Gene Ther.
, vol.19
, pp. 979-990
-
-
Hauswirth, W.W.1
Aleman, T.S.2
Kaushal, S.3
-
7
-
-
0027260657
-
Cellular interactions implicated in the mechanism of photoreceptor degeneration in transgenic mice expressing a mutant rhodopsin gene
-
Huang, P.C., Gaitan, A.E., Hao, Y., et al. (1993). Cellular interactions implicated in the mechanism of photoreceptor degeneration in transgenic mice expressing a mutant rhodopsin gene. Proc. Natl. Acad. Sci. U. S. A. 90, 8484-8488.
-
(1993)
Proc. Natl. Acad. Sci. U. S. A.
, vol.90
, pp. 8484-8488
-
-
Huang, P.C.1
Gaitan, A.E.2
Hao, Y.3
-
8
-
-
0032101737
-
Non-cell-autonomous photoreceptor degeneration in rds mutant mice mosaic for expression of a rescue transgene
-
Kedzierski, W., Bok, D., and Travis, G.H. (1998). Non-cell-autonomous photoreceptor degeneration in rds mutant mice mosaic for expression of a rescue transgene. J. Neurosci. 18, 4076-4082.
-
(1998)
J. Neurosci.
, vol.18
, pp. 4076-4082
-
-
Kedzierski, W.1
Bok, D.2
Travis, G.H.3
-
9
-
-
44249085878
-
Safety and efficacy of gene transfer for Leber's congenital amaurosis
-
Maguire, A.M., Simonelli, F., Pierce, E.A., et al. (2008). Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med. 358, 2240-2248.
-
(2008)
N. Engl. J. Med.
, vol.358
, pp. 2240-2248
-
-
Maguire, A.M.1
Simonelli, F.2
Pierce, E.A.3
-
10
-
-
70350620424
-
Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: A phase 1 dose-escalation trial
-
Maguire, A.M., High, K.A., Auricchio, A., et al. (2009). Agedependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374, 1597-1605.
-
(2009)
Lancet
, vol.374
, pp. 1597-1605
-
-
Maguire, A.M.1
High, K.A.2
Auricchio, A.3
-
11
-
-
84855832566
-
Loss of daylight vision in retinal degeneration: Are oxidative stress and metabolic dysregulation to blame? J
-
Punzo, C., Xiong, W., and Cepko, C.L. (2012). Loss of daylight vision in retinal degeneration: are oxidative stress and metabolic dysregulation to blame? J. Biol. Chem. 287, 1642-1648.
-
(2012)
Biol. Chem.
, vol.287
, pp. 1642-1648
-
-
Punzo, C.1
Xiong, W.2
Cepko, C.L.3
-
12
-
-
33644872951
-
Ciliary neurotrophic factor (CNTF) for human retinal degeneration: Phase i trial of Cntf delivered by encapsulated cell intraocular implants
-
Sieving, P.A., Caruso, R.C., Tao, W., et al. (2006). Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of Cntf delivered by encapsulated cell intraocular implants. Proc. Natl. Acad. Sci. U. S. A. 103, 3896-3901.
-
(2006)
Proc. Natl. Acad. Sci. U. S. A.
, vol.103
, pp. 3896-3901
-
-
Sieving, P.A.1
Caruso, R.C.2
Tao, W.3
-
13
-
-
84856950131
-
Novel adenoassociated viral vectors for retinal gene therapy
-
Vandenberghe, L.H., and Auricchio, A. (2012). Novel adenoassociated viral vectors for retinal gene therapy. Gene Ther. 19, 162-168.
-
(2012)
Gene Ther.
, vol.19
, pp. 162-168
-
-
Vandenberghe, L.H.1
Auricchio, A.2
|