Microarray-based gene expression profiling in patients with cryopyrin-associated periodic syndromes defines a disease-related signature and IL-1-responsive transcripts.

Objective: To analyse gene expression patterns and to define a specific gene expression signature in patients with the severe end of the spectrum of cryopyrin-associated periodic syndromes (CAPS). The molecular consequences of interleukin 1 inhibition were examined by comparing gene expression patte...

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Published in:Annals of the Rheumatic Diseases Vol. 72; no. 6; pp. 1064 - 1071
Main Authors: Balow Jr, James E, Ryan, John G, Chae, Jae Jin, Booty, Matthew G, Bulua, Ariel, Stone, Deborah, Sun, Hong-Wei, Greene, James, Barham, Beverly, Goldbach-Mansky, Raphaela, Kastner, Daniel L, Aksentijevich, Ivona, Balow, James E Jr
Format: research Journal Article
Published: Elsevier B.V. Jun2013
Online Access:View this record in EBSCOhost
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        00034967
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      jtl: Annals of the Rheumatic Diseases
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      dt: Jun2013
      vid: 72
      iid: 6
      pid: 467
      pub: Elsevier B.V.
      place: New York, New York
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        104287404
        NLM23223423
        2012117046
        10.1136/annrheumdis-2012-202082
        NLM23223423
        PMC4174357
        104287404
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        atl: Microarray-based gene expression profiling in patients with cryopyrin-associated periodic syndromes defines a disease-related signature and IL-1-responsive transcripts.
      aug:
        au:
          Balow Jr, James E
          Ryan, John G
          Chae, Jae Jin
          Booty, Matthew G
          Bulua, Ariel
          Stone, Deborah
          Sun, Hong-Wei
          Greene, James
          Barham, Beverly
          Goldbach-Mansky, Raphaela
          Kastner, Daniel L
          Aksentijevich, Ivona
          Balow, James E Jr
        affil: Inflammatory Disease Section, National Human Genome Research Institute (NHGRI), Bethesda, Maryland 20892-1820, USA
      sug:
        subj:
          Antirheumatic Agents Therapeutic Use
          Autoimmune Diseases
          Proteins Therapeutic Use
          RNA
          Adult
          Autoimmune Diseases Drug Therapy
          Case Control Studies
          Child
          Gene Expression Profiling
          Human
          Microarray Analysis
          Models, Biological
          Severity of Illness Indices
          Adult: 19-44 years
          Child: 6-12 years
      ab: Objective: To analyse gene expression patterns and to define a specific gene expression signature in patients with the severe end of the spectrum of cryopyrin-associated periodic syndromes (CAPS). The molecular consequences of interleukin 1 inhibition were examined by comparing gene expression patterns in 16 CAPS patients before and after treatment with anakinra.Methods: We collected peripheral blood mononuclear cells from 22 CAPS patients with active disease and from 14 healthy children. Transcripts that passed stringent filtering criteria (p values≤false discovery rate 1%) were considered as differentially expressed genes (DEG). A set of DEG was validated by quantitative reverse transcription PCR and functional studies with primary cells from CAPS patients and healthy controls. We used 17 CAPS and 66 non-CAPS patient samples to create a set of gene expression models that differentiates CAPS patients from controls and from patients with other autoinflammatory conditions.Results: Many DEG include transcripts related to the regulation of innate and adaptive immune responses, oxidative stress, cell death, cell adhesion and motility. A set of gene expression-based models comprising the CAPS-specific gene expression signature correctly classified all 17 samples from an independent dataset. This classifier also correctly identified 15 of 16 post-anakinra CAPS samples despite the fact that these CAPS patients were in clinical remission.Conclusions: We identified a gene expression signature that clearly distinguished CAPS patients from controls. A number of DEG were in common with other systemic inflammatory diseases such as systemic onset juvenile idiopathic arthritis. The CAPS-specific gene expression classifiers also suggest incomplete suppression of inflammation at low doses of anakinra.
      pubtype: Academic Journal
      doctype:
        research
        Journal Article
      ougenre: Article
    language: English
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