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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Prospect of retinal gene therapy following commercialization of voretigene neparvovec-rzyl for retinal dystrophy mediated by RPE65 mutation.维替泊芬基因治疗药物voretigene neparvovec-rzyl用于治疗由RPE65基因突变介导的视网膜营养不良商业化后的视网膜基因治疗前景。
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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.治疗 RPE65 介导的遗传性视网膜营养不良患者的 voretigene neparvovec（AAV2-hRPE65v2）的疗效和安全性：一项随机、对照、开放标签、3 期临床试验。
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.玻璃体内注射 AAV2-sFLT01 治疗晚期新生血管性年龄相关性黄斑变性患者：一项 1 期、开放标签试验。
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.儿童失明基因治疗后的视力改善与下降
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.尽管人类视网膜基因治疗先天性黑蒙 10 型的视力得到了持久改善，但仍显示出进行性视网膜变性。
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.RPE65基因疗法治疗莱伯先天性黑蒙的年龄依赖性效应：一项1期剂量递增试验。
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.基因疗法恢复了儿童失明犬模型的视力。
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.常染色体隐性遗传性视网膜色素变性或莱伯先天性黑矇患者的RPE65基因突变。
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.RPE65基因的突变会导致常染色体隐性遗传的儿童期严重视网膜营养不良。
Nat Genet. 1997 Oct;17(2):194-7. doi: 10.1038/ng1097-194.
9
Mutations in RPE65 cause Leber's congenital amaurosis.RPE65基因的突变会导致莱伯先天性黑蒙。
Nat Genet. 1997 Oct;17(2):139-41. doi: 10.1038/ng1097-139.
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