A Comprehensive Review of Retinal Gene Therapy

被引：214

作者：

Boye, Shannon E. ^{[1
,2
]}

Boye, Sanford L. ^{[1
,2
]}

Lewin, Alfred S. ^{[2
,3
]}

Hauswirth, William W. ^{[1
,2
]}

机构：

[1] Univ Florida, Dept Ophthalmol, Gainesville, FL 32610 USA

[2] Univ Florida, Powell Gene Therapy Ctr, Gainesville, FL 32610 USA

[3] Univ Florida, Dept Mol Genet & Microbiol, Gainesville, FL 32610 USA

来源：

MOLECULAR THERAPY | 2013年 / 21卷 / 03期

关键词：

LEBER CONGENITAL AMAUROSIS; LONG-TERM PRESERVATION; X-LINKED RETINOSCHISIS; MEMBRANE GUANYLYL CYCLASE; KNOCKOUT MOUSE MODELS; RAB ESCORT PROTEIN-1; USHER 1B SYNDROME; MYOSIN VIIA; RETINITIS-PIGMENTOSA; IN-VIVO;

D O I：

10.1038/mt.2012.280

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

Blindness, although not life threatening, is a debilitating disorder for which few, if any treatments exist. Ocular gene therapies have the potential to profoundly improve the quality of life in patients with inherited retinal disease. As such, tremendous focus has been given to develop such therapies. Several factors make the eye an ideal organ for gene-replacement therapy including its accessibility, immune privilege, small size, compartmentalization, and the existence of a contralateral control. This review will provide a comprehensive summary of (i) existing gene therapy clinical trials for several genetic forms of blindness and (ii) preclinical efficacy and safety studies in a variety of animal models of retinal disease which demonstrate strong potential for clinical application. To be as comprehensive as possible, we include additional proof of concept studies using gene replacement, neurotrophic/neuroprotective, optogenetic, antiangiogenic, or antioxidative stress strategies as well as a description of the current challenges and future directions in the ocular gene therapy field to this review as a supplement.

引用

页码：509 / 519

页数：11

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