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Article

Pluripotent stem cell-based screening identifies CUDC-907 as an effective compound for restoring the in vitro phenotype of Nakajo-Nishimura syndrome.

Stem cells translational medicine - 1 Mar 2021

Kase Naoya, Terashima Madoka, Ohta Akira, Niwa Akira, Honda-Ozaki Fumiko, Kawasaki Yuri, Nakahata Tatsutoshi, Kanazawa Nobuo, Saito Megumu K

Abstract excerpt

Nakajo-Nishimura syndrome (NNS) is an autoinflammatory disorder caused by a homozygous mutations in the PSMB8 gene. 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...

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Identifiers

PMID: 33280267
DOI: 10.1002/sctm.20-0198
PMCID: PMC7900583

Genes

PSMB8

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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