On Retinal Gene Therapy

被引：9
作者：
Fischer, M. Dominik [1 ,2 ]
机构：
[1] Univ Tubingen, Univ Eye Hosp, Ctr Ophthalmol, Tubingen, Germany
[2] Univ Oxford, Dept Clin Neurosci, Nuffield Lab Ophthalmol, Oxford, England
来源：
OPHTHALMOLOGICA | 2016年 / 236卷 / 01期
关键词：
Gene therapy; Retinal dystrophy; Vitreo-retinal surgery; LEBER CONGENITAL AMAUROSIS; LINKED RETINITIS-PIGMENTOSA; OCULAR SUBRETINAL INJECTION; ADENOASSOCIATED VIRUS; CHILDHOOD BLINDNESS; RPE65; MUTATIONS; VIRAL VECTORS; CANINE MODEL; CONE VISION; CHOROIDEREMIA;
D O I：
10.1159/000445782
中图分类号：
R77 [眼科学];
学科分类号：
100212 ;
摘要：
Mutations in a large number of genes cause retinal degeneration and blindness with no cure currently available. Retinal gene therapy has evolved over the last decades to become a promising new treatment paradigm for these rare disorders. This article reflects on the ideas and concepts arising from basic science towards the translation of retinal gene therapy into the clinical realm. It describes the advances and present thinking on the efficacy of current clinical trials and discusses potential roadblocks and solutions for the future of retinal gene therapy. (C) 2016 S. Karger AG, Basel
引用
收藏
页码：1 / 7
页数：7
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