Anti-inflammatory Activity of MTL-CEBPA, a Small Activating RNA Drug, in LPS-Stimulated Monocytes and Humanized Mice.


Journal

Molecular therapy : the journal of the American Society of Gene Therapy
ISSN: 1525-0024
Titre abrégé: Mol Ther
Pays: United States
ID NLM: 100890581

Informations de publication

Date de publication:
08 05 2019
Historique:
received: 29 11 2018
revised: 19 02 2019
accepted: 20 02 2019
pubmed: 11 3 2019
medline: 24 3 2020
entrez: 11 3 2019
Statut: ppublish

Résumé

Excessive or inappropriate inflammatory responses can cause serious and even fatal diseases. The CCAAT/enhancer-binding protein alpha (CEBPA) gene encodes C/EBPα, a transcription factor that plays a fundamental role in controlling maturation of the myeloid lineage and is also expressed during the late phase of inflammatory responses when signs of inflammation are decreasing. MTL-CEBPA, a small activating RNA targeting for upregulation of C/EBPα, is currently being evaluated in a phase 1b trial for treatment of hepatocellular carcinoma. After dosing, subjects had reduced levels of pro-inflammatory cytokines, and we therefore hypothesized that MTL-CEBPA has anti-inflammatory potential. The current study was conducted to determine the effects of C/EBPα saRNA - CEBPA-51 - on inflammation in vitro and in vivo after endotoxin challenge. CEBPA-51 led to increased expression of the C/EBPα gene and inhibition of pro-inflammatory cytokines in THP-1 monocytes previously stimulated by E. coli-derived lipopolysaccharide (LPS). Treatment with MTL-CEBPA in an LPS-challenged humanized mouse model upregulated C/EBPα mRNA, increased neutrophils, and attenuated production of several key pro-inflammatory cytokines, including TNF-α, IL-6, IL-1β, and IFN-γ. In addition, a Luminex analysis of mouse serum revealed that MTL-CEBPA reduced pro-inflammatory cytokines and increased the anti-inflammatory cytokine IL-10. Collectively, the data support further investigation of MTL-CEBPA in acute and chronic inflammatory diseases where this mechanism has pathogenic importance.

Identifiants

pubmed: 30852139
pii: S1525-0016(19)30059-0
doi: 10.1016/j.ymthe.2019.02.018
pmc: PMC6520465
pii:
doi:

Substances chimiques

Anti-Inflammatory Agents 0
CCAAT-Enhancer-Binding Proteins 0
CEBPA protein, human 0
IL10 protein, human 0
IL1B protein, human 0
Interleukin-1beta 0
Lipopolysaccharides 0
RNA, Messenger 0
Tumor Necrosis Factor-alpha 0
Interleukin-10 130068-27-8
RNA 63231-63-0

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

999-1016

Subventions

Organisme : NCI NIH HHS
ID : P30 CA033572
Pays : United States
Organisme : NIAID NIH HHS
ID : R01 AI029329
Pays : United States
Organisme : NIAID NIH HHS
ID : R01 AI042552
Pays : United States
Organisme : NHLBI NIH HHS
ID : R01 HL074704
Pays : United States

Informations de copyright

Copyright © 2019 The Author(s). Published by Elsevier Inc. All rights reserved.

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Auteurs

Jiehua Zhou (J)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA.

Haitang Li (H)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA.

Xin Xia (X)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA.

Alberto Herrera (A)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA; Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA.

Nicolette Pollock (N)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA; Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA.

Vikash Reebye (V)

Department of Surgery and Cancer, Imperial College London, London SW7 5NH, UK.

Mikael H Sodergren (MH)

Department of Surgery and Cancer, Imperial College London, London SW7 5NH, UK.

Stephanie Dorman (S)

Department of Surgery and Cancer, Imperial College London, London SW7 5NH, UK.

Bruce H Littman (BH)

Translational Medicine Associates, LLC, Savannah, GA 31302, USA.

Declan Doogan (D)

MiNA Therapeutics, Ltd., London W12 0BZ, UK.

Kai-Wen Huang (KW)

Department of Surgery and Hepatitis Research Center, National Taiwan University Hospital, College of Medicine, Taipei 10617, Taiwan.

Robert Habib (R)

MiNA Therapeutics, Ltd., London W12 0BZ, UK.

David Blakey (D)

MiNA Therapeutics, Ltd., London W12 0BZ, UK.

Nagy A Habib (NA)

Department of Surgery and Cancer, Imperial College London, London SW7 5NH, UK; MiNA Therapeutics, Ltd., London W12 0BZ, UK. Electronic address: nagy.habib@imperial.ac.uk.

John J Rossi (JJ)

Department of Molecular and Cellular Biology, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA; Irell and Manella Graduate School of Biological Sciences, Beckman Research Institute, City of Hope, Duarte, CA 91010, USA. Electronic address: jrossi@coh.org.

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Classifications MeSH