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

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 pluripotent stem cell (...

Full description

Bibliographic Details
Main Authors: Naoya Kase, Madoka Terashima, Akira Ohta, Akira Niwa, Fumiko Honda‐Ozaki, Yuri Kawasaki, Tatsutoshi Nakahata, Nobuo Kanazawa, Megumu K. Saito
Format: Article
Language:English
Published: Wiley 2021-03-01
Series:Stem Cells Translational Medicine
Subjects:
Online Access:https://doi.org/10.1002/sctm.20-0198