Ganzen, LoganKo, Mee JungZhang, MengruiXie, RuiChen, YongkaiZhang, LiyunJames, RebeccaMumm, Jeffvan Rijn, Richard M.Zhong, WenxuanPang, Chi PuiZhang, MingzhiTsujikawa, MotokazuLeung, Yuk Fai2023-01-032023-01-032021-06-01Ganzen L, Ko MJ, Zhang M, et al. Drug screening with zebrafish visual behavior identifies carvedilol as a potential treatment for an autosomal dominant form of retinitis pigmentosa. Sci Rep. 2021;11(1):11432. Published 2021 Jun 1. doi:10.1038/s41598-021-89482-zhttps://hdl.handle.net/1805/30823Retinitis Pigmentosa (RP) is a mostly incurable inherited retinal degeneration affecting approximately 1 in 4000 individuals globally. The goal of this work was to identify drugs that can help patients suffering from the disease. To accomplish this, we screened drugs on a zebrafish autosomal dominant RP model. This model expresses a truncated human rhodopsin transgene (Q344X) causing significant rod degeneration by 7 days post-fertilization (dpf). Consequently, the larvae displayed a deficit in visual motor response (VMR) under scotopic condition. The diminished VMR was leveraged to screen an ENZO SCREEN-WELL REDOX library since oxidative stress is postulated to play a role in RP progression. Our screening identified a beta-blocker, carvedilol, that ameliorated the deficient VMR of the RP larvae and increased their rod number. Carvedilol may directly on rods as it affected the adrenergic pathway in the photoreceptor-like human Y79 cell line. Since carvedilol is an FDA-approved drug, our findings suggest that carvedilol can potentially be repurposed to treat autosomal dominant RP patients.en-USAttribution 4.0 InternationalNeuroscienceVisual systemRetinaNeurodegenerationDrug discoveryDrug screeningPhenotypic screeningArticle