FDA-Approved Drug May Prevent Vision Loss

Newswise — Denver, Colo. – An FDA-approved oral medication, 4-phenylbutyrate (PBA), has been found to prevent vision loss in mice with Leber’s congenital amaurosis (LCA). The research is being presented at the 2015 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) this week in Denver, Colo.

PBA was found to help fold a mutant version of the enzyme RPE65 that is responsible for vision loss from LCA. With proper folding encouraged by PBA, RPE65 function was rescued and vision loss was prevented.

LCA is caused by mutations in RPE65 that result in the misfolding and rapid degradation of the enzyme.

Abstract Title: Attempts to increase protein level, membrane-association, and isomerase activity of R91W RPE65 in mouse model Presentation Start/End Time: Thursday, May 7, 8:30 – 10:15am Location: Exhibit HallAbstract Number: 5454 – B0013

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The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include nearly 12,000 eye and vision researchers from over 75 countries. ARVO advances research worldwide into understanding the visual system and preventing, treating and curing its disorders.

All abstracts accepted for presentation at the ARVO Annual Meeting represent previously unpublished data and conclusions. This research may be proprietary or may have been submitted for journal publication. Embargo policy: Journalists must seek approval from the presenter(s) before reporting data from paper or poster presentations. Press releases or stories on information presented at the ARVO Annual Meeting may not be released or published until the conclusion of the presentation.