Gene therapy for inherited retinal degenerations: initial successes and future challenges

被引：22

作者：

Gupta, Priya R. ^{[1
]}

Huckfeldt, Rachel M. ^{[1
]}

机构：

[1] Harvard Med Sch, Massachusetts Eye & Ear, Dept Ophthalmol, Boston, MA USA

来源：

JOURNAL OF NEURAL ENGINEERING | 2017年 / 14卷 / 05期

关键词：

inherited retinal degeneration; gene therapy; genome editing; LEBER CONGENITAL AMAUROSIS; HIGH-EFFICIENCY TRANSDUCTION; COMPACTED DNA NANOPARTICLES; LINKED RETINITIS-PIGMENTOSA; KNOCKOUT MOUSE MODELS; ADENOASSOCIATED VIRUS; IN-VIVO; RPE65; MUTATIONS; STEM-CELLS; LONG-TERM;

D O I：

10.1088/1741-2552/aa7a27

中图分类号：

R318 [生物医学工程];

学科分类号：

0831 ;

摘要：

Inherited retinal degenerations are a clinically and genetically heterogeneous group of conditions that have historically shared an untreatable course. In recent years, however, a wide range of therapeutic strategies have demonstrated efficacy in preclinical studies and entered clinical trials with a common goal of improving visual function for patients affected with these conditions. Gene therapy offers a particularly elegant and precise opportunity to target the causative genetic mutations underlying these monogenic diseases. The present review will provide an overview of gene therapy with particular emphasis on key clinical results to date and challenges for the future.

引用

页数：19

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