Promising and delivering gene therapies for vision loss

被引：34
作者：
Carvalho, Livia S. [1 ]
Vandenberghe, Luk H. [1 ]
机构：
[1] Harvard Univ, Schepens Eye Res Inst, Massachusetts Eye & Ear Infirm, Dept Ophthalmol,Med Sch, Boston, MA 02114 USA
来源：
VISION RESEARCH | 2015年 / 111卷
关键词：
Retina; Gene therapy; AAV; Lentiviral vector; LCA; Clinical trial; LEBER CONGENITAL AMAUROSIS; ADENOASSOCIATED VIRAL VECTORS; CILIARY NEUROTROPHIC FACTOR; LONG-TERM; RETINAL DEGENERATION; VIRUS VECTORS; SUBRETINAL INJECTION; VISUAL FUNCTION; MOUSE MODEL; RETINITIS-PIGMENTOSA;
D O I：
10.1016/j.visres.2014.07.013
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
The maturity in our understanding of the genetics and the pathogenesis of disease in degenerative retinal disorders has intersected in past years with a novel treatment paradigm in which a genetic intervention may lead to sustained therapeutic benefit, and in some cases even restoration of vision. Here, we review this prospect of retinal gene therapy, discuss the enabling technologies that have led to first-in-human demonstrations of efficacy and safety, and the road that led to this exciting point in time. (C) 2014 Elsevier Ltd. All rights reserved.
引用
收藏
页码：124 / 133
页数：10
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