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Zhuo-Hua Pan; Optogenetic approaches to restoring vision. Invest. Ophthalmol. Vis. Sci. 2019;60(9):5.
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Presentation Description :
Severe loss of photoreceptor cells in inherited or acquired retinal degenerative diseases, such as retinitis pigmentosa (RP) and age-related macular degeneration (AMD), can result in partial or complete blindness. The optogenetic strategy to treating blindness involves ectopic expression of genetically encoded light sensors in surviving retinal neurons, thereby imparting light sensitivity to retinas after the loss of photoreceptor cells. Since our first demonstration of the feasibility of this strategy by expressing channelrhodopsin-2 (ChR2) in retinal ganglion cells in rd mice, optogenetic vision restoration has been reported by using a variety of optogenetic tools and different retinal cell targeting strategies. Optogenetic therapy for RP by targeting ChRs to retinal ganglion cells is currently in clinical trials. In this presentation, I will review the current state of the field and discuss our recent and ongoing studies that aim at developing better ChR tools, determining optimal retinal cell targeting strategies, and investigating the impact of the severity and stage of retinal degeneration on optogenetic vision restoration.
This abstract was presented at the 2019 ARVO Annual Meeting, held in Vancouver, Canada, April 28 - May 2, 2019.
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