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The administration of systemic corticosteroids is partially effective, but continuous treatment causes severe side effects. We previously established a pluripotent stem cell (PSC)-derived NNS disease model that reproduces several inflammatory phenotypes, including the overproduction of monocyte chemoattractant protein-1 (MCP-1) and interferon gamma-induced protein-10 (IP-10). Here we performed high-throughput compound screening (HTS) using this PSC-derived NNS model to find potential therapeutic candidates and identified CUDC-907 as an effective inhibitor of the release of MCP-1 and IP-10. Short-term treatment of CUDC-907 did not induce cell death within therapeutic concentrations and was also effective on primary patient cells. Further analysis indicated that the inhibitory effect was post-transcriptional. These findings suggest that HTS with PSC-derived disease models is useful for finding drug candidates for autoinflammatory diseases.<\/jats:p>","DOI":"10.1002\/sctm.20-0198","type":"journal-article","created":{"date-parts":[[2020,10,14]],"date-time":"2020-10-14T09:14:18Z","timestamp":1602666858000},"page":"455-464","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Pluripotent stem cell-based screening identifies CUDC-907 as an effective compound for restoring the in vitro phenotype of Nakajo-Nishimura syndrome"],"prefix":"10.1093","volume":"10","author":[{"given":"Naoya","family":"Kase","sequence":"first","affiliation":[{"name":"Department of Clinical Application Center for iPS Cell Research and Application (CiRA), Kyoto University, Kyoto, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Madoka","family":"Terashima","sequence":"additional","affiliation":[{"name":"Department of Clinical 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