Unknown

Dataset Information

0

Drug screening with zebrafish visual behavior identifies carvedilol as a potential treatment for an autosomal dominant form of retinitis pigmentosa.


ABSTRACT: Retinitis 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.

SUBMITTER: Ganzen L 

PROVIDER: S-EPMC8169685 | biostudies-literature |

REPOSITORIES: biostudies-literature

Similar Datasets

| S-EPMC4602186 | biostudies-literature
| S-EPMC7859630 | biostudies-literature
| S-EPMC7545001 | biostudies-literature
| S-EPMC4363838 | biostudies-literature
| S-EPMC8478325 | biostudies-literature
| S-EPMC5122955 | biostudies-literature
| S-EPMC2585827 | biostudies-literature
| S-EPMC2585828 | biostudies-literature
| S-EPMC5752119 | biostudies-literature
| S-EPMC4812529 | biostudies-literature