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

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Published in 2020 at "Stem Cells Translational Medicine"

DOI: 10.1002/sctm.20-0198

Abstract: 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… read more here.

Keywords: nishimura syndrome; nakajo nishimura; stem cell; cudc 907 ... See more keywords