Retinitis Pigmentosa and Retinal Degenerations: Deciphering Pathways and Targets for Drug Discovery and Development

Inherited retinal diseases (IRDs) are a group of etinopatliies generally caused by genetic mutations. F.etinitis Pigmentosa (RP) rePresenotsone of the most studied IRDs. RP leads to intense vision loss or blindness resulting from the degeneration of photoreceptor cells. To date, RP is mainly treated with palliative suPPlementation of BitaminA a pharmacologicallyretinoids, gene therapies, or surgical interventions. Therefore based therapy is an urgent need requiring a medicinal chemistry approach, to validate molecular targets able to deal with retinal Rdegeneration. This Review aims at outlining the recent research drugefforts in identifying new targets for RP, especially focusing on the neuroprotective role of the Wnt/beta-catenin/GSK3 beta pathwayand apoptosis modulators (in particular FikRF-1) but also on growth factors such as VEGF and BDNF. FuiThermure, the ruie of spatiotempiirally expressed G protein-coupled receptors. (GpR124) in the retina and the emerging function of histone deacetylase inhibitors in promoting retinal neuroprotection will be discussed.