Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward

被引：80

作者：

Bennett, Jean ^{[1
,2
]}

机构：

[1] Univ Penn, Perelman Sch Med, CAROT, Philadelphia, PA 19104 USA

[2] Univ Penn, FM Kirby Ctr Mol Ophthalmol, Perelman Sch Med, Scheie Eye Inst, Philadelphia, PA 19104 USA

来源：

MOLECULAR THERAPY | 2017年 / 25卷 / 05期

关键词：

LEBER CONGENITAL AMAUROSIS; OCULAR SUBRETINAL INJECTION; KNOCKOUT MOUSE MODEL; RETINITIS-PIGMENTOSA; ADENOASSOCIATED VIRUS; MACULAR DEGENERATION; BETA-SUBUNIT; IN-VIVO; RPE65; MUTATIONS; RHODOPSIN GENE;

D O I：

10.1016/j.ymthe.2017.03.008

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：

Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one earlyonset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.

引用

页码：1076 / 1094

页数：19

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