Inherited diseases of photoreceptors and prospects for gene therapy

被引：7
|
作者：
Montana, Cynthia L. [1 ]
Corbo, Joseph C. [1 ]
机构：
[1] Washington Univ, Sch Med, Dept Pathol & Immunol, St Louis, MO 63110 USA
来源：
PHARMACOGENOMICS | 2008年 / 9卷 / 03期
关键词：
adeno-associated virus; cis-regulatory element; gene therapy; photoreceptors; retina;
D O I：
10.2217/14622416.9.3.335
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
The photoreceptor cells of the retina are subject to a wide range of genetic diseases. This review summarizes current knowledge regarding an important group of retinal diseases caused by mutations in photoreceptor-enriched genes. In addition, progress toward treatment of a variety of these diseases in animal models via adeno-associated virus gene therapy is described. Although no human trials have yet been initiated to treat diseases caused by mutations in photoreceptor-enriched genes, there is a great deal of optimism regarding the prospects of treating these diseases using adeno-associated virus gene therapy.
引用
收藏
页码：335 / 347
页数：13
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