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Retinitis pigmentosa and retinal degenerations: deciphering pathways and targets for drug discovery and development

Title: Retinitis pigmentosa and retinal degenerations: deciphering pathways and targets for drug discovery and development
Authors: Carullo G.; Federico S.; Relitti N.; Gemma S.; Butini S.; Campiani G.
Contributors: Carullo, G.; Federico, S.; Relitti, N.; Gemma, S.; Butini, S.; Campiani, G.
Publication Year: 2020
Collection: Università degli Studi di Siena: USiena air
Subject Terms: apoptosi; GPCR; growth factor; HDAC; Retinitis pigmentosa; Wnt/β-catenin/GSK3β
Description: 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.
Document Type: article in journal/newspaper
File Description: STAMPA
Language: English
Relation: info:eu-repo/semantics/altIdentifier/pmid/32589402; info:eu-repo/semantics/altIdentifier/wos/WOS:000558790100008; volume:11; issue:15; firstpage:2173; lastpage:2191; numberofpages:19; journal:ACS CHEMICAL NEUROSCIENCE; https://hdl.handle.net/11365/1115556
DOI: 10.1021/acschemneuro.0c00358
Availability: https://hdl.handle.net/11365/1115556; https://doi.org/10.1021/acschemneuro.0c00358; https://pubs.acs.org/doi/10.1021/acschemneuro.0c00358
Rights: info:eu-repo/semantics/closedAccess
Accession Number: edsbas.C52568A5
Database: BASE