Small molecules targeting glycogen synthase kinase 3 as potential drug candidates for the treatment of retinitis pigmentosa |
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Authors: | Miguel Marchena Beatriz Villarejo-Zori Josefa Zaldivar-Diez Valle Palomo Carmen Gil Catalina Hernández-Sánchez |
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Institution: | 1. Department of Cellular and Molecular Medicine, Centro de Investigaciones Biológicas (CSIC), Madrid, Spain;2. Department of Chemical and Physical Biology, Centro de Investigaciones Biológicas (CSIC), Madrid, Spain |
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Abstract: | Retinitis pigmentosa (RP) is an inherited retinal dystrophy that courses with progressive degeneration of retinal tissue and loss of vision. Currently, RP is an unpreventable, incurable condition. We propose glycogen synthase kinase 3 (GSK-3) inhibitors as potential leads for retinal cell neuroprotection, since the retina is also a part of the central nervous system and GSK-3 inhibitors are potent neuroprotectant agents. Using a chemical genetic approach, diverse small molecules with different potency and binding mode to GSK-3 have been used to validate and confirm GSK-3 as a pharmacological target for RP. Moreover, this medicinal chemistry approach has provided new leads for the future disease-modifying treatment of RP. |
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Keywords: | GSK-3 inhibitors retinal diseases retinitis pigmentosa glaucoma |
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