BainbridgeJ.W., SmithA.J., BarkerS.S.et al.2008. Effect of gene therapy on visual function in Leber's congenital amaurosis. N. Engl. J. Med., 358:2231–2239.
2.
BramallA.N., WrightA.F., JacobsonS.G., McInnesR.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.
3.
CideciyanA.V., AlemanT.S., BoyeS.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.
4.
CideciyanA.V., JacobsonS.G., BeltranW.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.
HauswirthW.W., AlemanT.S., KaushalS.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.
7.
HuangP.C., GaitanA.E., HaoY.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.
8.
KedzierskiW., BokD., TravisG.H.1998. Non-cell-autonomous photoreceptor degeneration in rds mutant mice mosaic for expression of a rescue transgene. J. Neurosci., 18:4076–4082.
9.
MaguireA.M., SimonelliF., PierceE.A.et al.2008. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N. Engl. J. Med., 358:2240–2248.
10.
MaguireA.M., HighK.A., AuricchioA.et al.2009. Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet, 374:1597–1605.
11.
PunzoC., XiongW., CepkoC.L.2012. Loss of daylight vision in retinal degeneration: are oxidative stress and metabolic dysregulation to blame?J. Biol. Chem., 287:1642–1648.
12.
SievingP.A., CarusoR.C., TaoW.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.