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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

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)‐deri...

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Dettagli Bibliografici
Pubblicato in:Stem Cells Transl Med
Autori principali: Kase, Naoya, Terashima, Madoka, Ohta, Akira, Niwa, Akira, Honda‐Ozaki, Fumiko, Kawasaki, Yuri, Nakahata, Tatsutoshi, Kanazawa, Nobuo, Saito, Megumu K.
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley & Sons, Inc. 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7900583/
https://ncbi.nlm.nih.gov/pubmed/33280267
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/sctm.20-0198
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