The Status of RPE65 Gene Therapy Trials: Safety and Efficacy

被引：112
|
作者：
Pierce, Eric A. [1 ,2 ]
Bennett, Jean [3 ]
机构：
[1] Massachusetts Eye & Ear Infirm, Ocular Genom Inst, Dept Ophthalmol, Boston, MA 02114 USA
[2] Harvard Univ, Sch Med, Boston, MA 02114 USA
[3] Univ Penn, Sch Med, FM Kirby Ctr Mol Ophthalmol, Dept Ophthalmol,Ctr Adv Retinal & Ophthalm Therap, Philadelphia, PA 19104 USA
来源：
COLD SPRING HARBOR PERSPECTIVES IN MEDICINE | 2015年 / 5卷 / 09期
关键词：
LEBER CONGENITAL AMAUROSIS; MOUSE MODEL; ADENOASSOCIATED VIRUS; RETINAL DEGENERATION; RESTORES VISION; VISUAL FUNCTION; CANINE MODEL; MURINE MODEL; CONE VISION; MUTATIONS;
D O I：
10.1101/cshperspect.a017285
中图分类号：
R-3 [医学研究方法]; R3 [基础医学];
学科分类号：
1001 ;
摘要：
Several groups have reported the results of clinical trials of gene augmentation therapy for Leber congenital amaurosis (LCA) because of mutations in the RPE65 gene. These studies have used subretinal injection of adeno-associated virus (AAV) vectors to deliver the human RPE65 cDNA to the retinal pigment epithelial (RPE) cells of the treated eyes. In all of the studies reported to date, this approach has been shown to be both safe and effective. The successful clinical trials of gene augmentation therapy for retinal degeneration caused by mutations in the RPE65 gene sets the stage for broad application of gene therapy to treat retinal degenerative disorders.
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页数：15
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