文章基本信息

标题：Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy
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作者：Samantha R. De Silva ; Alun R. Barnard ; Steven Hughes 等
期刊名称：Proceedings of the National Academy of Sciences
印刷版ISSN：0027-8424
电子版ISSN：1091-6490
出版年度：2017
卷号：114
期号：42
页码：11211-11216
DOI：10.1073/pnas.1701589114
语种：English
出版社：The National Academy of Sciences of the United States of America
摘要：Optogenetic strategies to restore vision in patients who are blind from end-stage retinal degenerations aim to render remaining retinal cells light sensitive once photoreceptors are lost. Here, we assessed long-term functional outcomes following subretinal delivery of the human melanopsin gene (OPN4) in the rd1 mouse model of retinal degeneration using an adeno-associated viral vector. Ectopic expression of OPN4 using a ubiquitous promoter resulted in cellular depolarization and ganglion cell action potential firing. Restoration of the pupil light reflex, behavioral light avoidance, and the ability to perform a task requiring basic image recognition were restored up to 13 mo following injection. These data suggest that melanopsin gene therapy via a subretinal route may be a viable and stable therapeutic option for the treatment of end-stage retinal degeneration in humans.
关键词：human melanopsin ; gene therapy ; optogenetics