Jun 27, 2020

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

ACS Chemical Neuroscience
Gabriele CarulloGiuseppe Campiani

Abstract

Inherited retinal diseases (IRDs) are a group of retinopathies generally caused by genetic mutations. Retinitis pigmentosa (RP) represents one of the most studied IRD, leading 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, apoptosis modulators (in particular PARP-1) but also on growth factors such as VEGF and BDNF. Furthermore, the role of spatiotemporally expressed GPCRs (GPR124) in the retina, and the emerging function of HDAC inhibitors in promoting retinal neuroprotection, will be discussed.

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Mentioned in this Paper

Histone deacetylase inhibitor
Brain-Derived Neurotrophic Factor
Growth Factor
Retina
Low Vision
Retinitis Pigmentosa
Drug Discovery
CTNND2
Beta catenin
GPR124

Related Feeds

Apoptosis

Apoptosis is a specific process that leads to programmed cell death through the activation of an evolutionary conserved intracellular pathway leading to pathognomic cellular changes distinct from cellular necrosis

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