{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Singh R"],"funding":["NEI NIH HHS","NICHD NIH HHS"],"pagination":["1700-1711"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC4817951"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["23(11)"],"pubmed_abstract":["Degradation of photoreceptor outer segments (POS) by retinal pigment epithelium (RPE) is essential for vision, and studies have implicated altered POS processing in the pathogenesis of some retinal degenerative diseases. Consistent with this concept, a recently established hiPSC-RPE model of inherited macular degeneration, Best disease (BD), displayed reduced rates of POS breakdown. Herein we utilized this model to determine (i) if disturbances in protein degradation pathways are associated with delayed POS digestion and (ii) whether such defect(s) can be pharmacologically targeted. We found that BD hiPSC-RPE cultures possessed increased protein oxidation, decreased free-ubiquitin levels, and altered rates of exosome secretion, consistent with altered POS processing. Application of valproi"],"journal":["Molecular therapy : the journal of the American Society of Gene Therapy"],"pubmed_title":["Pharmacological Modulation of Photoreceptor Outer Segment Degradation in a Human iPS Cell Model of Inherited Macular Degeneration."],"pmcid":["PMC4817951"],"funding_grant_id":["P30 EY001583","P30 HD03352","R01 EY006855","R01 EY021218","P30 HD003352"],"pubmed_authors":["Aguirre GD","Smith M","Lu J","Guziewicz KE","Meyer J","Verhoeven A","Singh R","Wilson M","Gamm DM","Kuai D","Clark E"],"additional_accession":[]},"is_claimable":false,"name":"Pharmacological Modulation of Photoreceptor Outer Segment Degradation in a Human iPS Cell Model of Inherited Macular Degeneration.","description":"Degradation of photoreceptor outer segments (POS) by retinal pigment epithelium (RPE) is essential for vision, and studies have implicated altered POS processing in the pathogenesis of some retinal degenerative diseases. Consistent with this concept, a recently established hiPSC-RPE model of inherited macular degeneration, Best disease (BD), displayed reduced rates of POS breakdown. Herein we utilized this model to determine (i) if disturbances in protein degradation pathways are associated with delayed POS digestion and (ii) whether such defect(s) can be pharmacologically targeted. We found that BD hiPSC-RPE cultures possessed increased protein oxidation, decreased free-ubiquitin levels, and altered rates of exosome secretion, consistent with altered POS processing. Application of valproi","dates":{"release":"2015-01-01T00:00:00Z","publication":"2015 Nov","modification":"2025-04-22T19:09:51.362Z","creation":"2019-03-27T03:11:07Z"},"accession":"S-EPMC4817951","cross_references":{"pubmed":["26300224"],"doi":["10.1038/mt.2015.141"]}}