PMID- 33280267
OWN - NLM
STAT- MEDLINE
DCOM- 20220322
LR  - 20221224
IS  - 2157-6580 (Electronic)
IS  - 2157-6564 (Print)
IS  - 2157-6564 (Linking)
VI  - 10
IP  - 3
DP  - 2021 Mar
TI  - Pluripotent stem cell-based screening identifies CUDC-907 as an effective 
      compound for restoring the in vitro phenotype of Nakajo-Nishimura syndrome.
PG  - 455-464
LID - 10.1002/sctm.20-0198 [doi]
AB  - 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 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.
CI  - (c) 2020 The Authors. STEM CELLS TRANSLATIONAL MEDICINE published by Wiley 
      Periodicals LLC on behalf of AlphaMed Press.
FAU - Kase, Naoya
AU  - Kase N
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
FAU - Terashima, Madoka
AU  - Terashima M
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
FAU - Ohta, Akira
AU  - Ohta A
AD  - Department of Fundamental Cell Technology, Center for iPS Cell Research and 
      Application (CiRA), Kyoto University, Kyoto, Japan.
FAU - Niwa, Akira
AU  - Niwa A
AUID- ORCID: 0000-0002-6412-2686
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
FAU - Honda-Ozaki, Fumiko
AU  - Honda-Ozaki F
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
AD  - Department of Pediatrics and Developmental Biology, Graduate School of Medical 
      and Dental Sciences, Tokyo Medical and Dental University, Tokyo, Japan.
FAU - Kawasaki, Yuri
AU  - Kawasaki Y
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
FAU - Nakahata, Tatsutoshi
AU  - Nakahata T
AD  - Department of Fundamental Cell Technology, Center for iPS Cell Research and 
      Application (CiRA), Kyoto University, Kyoto, Japan.
FAU - Kanazawa, Nobuo
AU  - Kanazawa N
AD  - Department of Dermatology, Wakayama Medical University, Wakayama, Japan.
FAU - Saito, Megumu K
AU  - Saito MK
AUID- ORCID: 0000-0001-8813-3614
AD  - Department of Clinical Application, Center for iPS Cell Research and Application 
      (CiRA), Kyoto University, Kyoto, Japan.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20201014
PL  - England
TA  - Stem Cells Transl Med
JT  - Stem cells translational medicine
JID - 101578022
RN  - 0 (CCL2 protein, human)
RN  - 0 (CUDC-907)
RN  - 0 (Chemokine CCL2)
RN  - 0 (Chemokine CXCL10)
RN  - 0 (Morpholines)
RN  - 0 (Pyrimidines)
RN  - Nakajo syndrome
SB  - IM
EIN - Stem Cells Transl Med. 2023 Mar 3;12(2):123. PMID: 36565066
MH  - Chemokine CCL2/genetics
MH  - *Chemokine CXCL10/genetics
MH  - Erythema Nodosum/*drug therapy
MH  - Fingers/*abnormalities
MH  - Humans
MH  - Morpholines/*pharmacology
MH  - Phenotype
MH  - *Pluripotent Stem Cells
MH  - Pyrimidines/*pharmacology
PMC - PMC7900583
OTO - NOTNLM
OT  - LMP7 protein
OT  - chemokines
OT  - hereditary autoinflammatory diseases
OT  - high-throughput screening assays
OT  - histone deacetylase inhibitors
OT  - pluripotent stem cells
COIS- The authors declared no potential conflicts of interest.
EDAT- 2020/12/07 06:00
MHDA- 2022/03/23 06:00
PMCR- 2020/10/14
CRDT- 2020/12/06 21:39
PHST- 2020/05/05 00:00 [received]
PHST- 2020/08/28 00:00 [revised]
PHST- 2020/09/13 00:00 [accepted]
PHST- 2020/12/07 06:00 [pubmed]
PHST- 2022/03/23 06:00 [medline]
PHST- 2020/12/06 21:39 [entrez]
PHST- 2020/10/14 00:00 [pmc-release]
AID - SCT312849 [pii]
AID - 10.1002/sctm.20-0198 [doi]
PST - ppublish
SO  - Stem Cells Transl Med. 2021 Mar;10(3):455-464. doi: 10.1002/sctm.20-0198. Epub 
      2020 Oct 14.