Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation.
作者信息
Ameri Hossein
机构信息
USC Roski Eye Institute, Keck School of Medicine, University of Southern California, 1450 San Pablo Street, Los Angeles, CA, 90033, USA.
出版信息
J Curr Ophthalmol. 2018 Feb 16;30(1):1-2. doi: 10.1016/j.joco.2018.01.006. eCollection 2018 Mar.
Abstract
摘要
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本文引用的文献
1
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14.
2
Intravitreous injection of AAV2-sFLT01 in patients with advanced neovascular age-related macular degeneration: a phase 1, open-label trial.
Lancet. 2017 Jul 1;390(10089):50-61. doi: 10.1016/S0140-6736(17)30979-0. Epub 2017 May 17.
3
Improvement and decline in vision with gene therapy in childhood blindness.
N Engl J Med. 2015 May 14;372(20):1920-6. doi: 10.1056/NEJMoa1412965. Epub 2015 May 3.
4
Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.
Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25. doi: 10.1073/pnas.1218933110. Epub 2013 Jan 22.
5
Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Lancet. 2009 Nov 7;374(9701):1597-605. doi: 10.1016/S0140-6736(09)61836-5. Epub 2009 Oct 23.
6
Gene therapy restores vision in a canine model of childhood blindness.
Nat Genet. 2001 May;28(1):92-5. doi: 10.1038/ng0501-92.
7
Mutations in the RPE65 gene in patients with autosomal recessive retinitis pigmentosa or leber congenital amaurosis.
Proc Natl Acad Sci U S A. 1998 Mar 17;95(6):3088-93. doi: 10.1073/pnas.95.6.3088.
8
Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy.
Nat Genet. 1997 Oct;17(2):194-7. doi: 10.1038/ng1097-194.
9
Mutations in RPE65 cause Leber's congenital amaurosis.
Nat Genet. 1997 Oct;17(2):139-41. doi: 10.1038/ng1097-139.
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