Inherited retinal diseases (IRDs) are a group of retinopathies generally caused by genetic mutations. Retinitis pigmentosa (RP) represents one 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 vitamin A and retinoids, gene therapies, or surgical interventions. Therefore, a pharmacologically based therapy is an urgent need requiring a medicinal chemistry approach, to validate molecular targets able to deal with retinal degeneration. This Review aims at outlining the recent research efforts in identifying new drug targets for RP, especially focusing on the neuroprotective role of the Wnt/β-catenin/GSK3β pathway and apoptosis modulators (in particular PARP-1) but also on growth factors such as VEGF and BDNF. Furthermore, the role of spatiotemporally expressed G protein-coupled receptors (GPR124) in the retina and the emerging function of histone deacetylase inhibitors in promoting retinal neuroprotection will be discussed.

中文翻译：

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色素性视网膜炎和视网膜变性：为药物发现和开发提供途径和目标。

遗传性视网膜疾病（IRD）是通常由遗传突变引起的一组视网膜病变。色素性视网膜炎（RP）代表研究最多的IRD之一。RP会导致由于感光细胞变性而导致的严重视力丧失或失明。迄今为止，RP主要通过姑息补充维生素A和类维生素A，基因疗法或外科手术治疗。因此，基于药理学的疗法是迫切需要药物化学方法，以验证能够处理视网膜变性的分子靶标。这篇综述的目的是概述最近在确定RP的新药物靶标方面的研究工作，尤其着重于Wnt /β-catenin/GSK3β途径和凋亡调节剂（特别是PARP-1）的神经保护作用，以及诸如VEGF和BDNF。