TargetRx Atlas

MRNA basic information

  • mRNA ID:ENST00000216117
  • Gene Name:HMOX1
  • mRNA database:Ensembl
  • mRNA expression :up-regulated
  • mRNA Method:qPCR
  • mRNA Pathway:NA
  • Evidence:validated
  • (mRNA-drug)

Drug basic information

  • Drug ID:DB06151
  • Drug Name:Acetylcysteine
  • Drug Method:up-regulated the gene expression

Disease basic infommation

  • Disease:childhood cerebral adrenoleukodystrophy
  • Tissue:tissues

Other information

  • Title:Mechanisms of Antioxidant Induction with High-Dose N-Acetylcysteine in Childhood Cerebral Adrenoleukodystrophy
  • Journal:Cns Drugs
  • Published:2015
  • PubMed ID:26670322
  • Abstract:Background:Childhood cerebral adrenoleukodystrophy (CCALD), a progressive demyelinating disease affecting school-aged boys, causes death within a few years. Oxidative stress is an important contributing factor. N-acetylcysteine (NAC; 280 mg/kg/day) added as adjunctive therapy to reduced-intensity hematopoietic cell transplantation (HCT) improves survival in advanced cases. However, the mechanisms underlying the benefits of NAC are unclear. Objective:The aim of this study was to understand the mechanism of action of NAC in the setting of HCT in CCALD. Methods:Immunoassays were carried out to determine changes in heme oxygenase-1 (HO-1) and ferritin expression in plasma samples collected from boys with CCALD at three different timepoints during the course of transplantation. In addition, the induction of HO-1 was also confirmed in normal fibroblasts following incubation with 10-100 μmol/L NAC for 4 h. Results:Following NAC therapy we observed an increase in expression of the antioxidants HO-1 (~4-fold) and its effector ferritin (~160-fold) in patient samples as compared with baseline. We also observed that NAC exposure significantly increased HO-1 expression in fibroblasts. Conclusion:Our data suggest that HO-1 is a possible target protein of NAC and a mediator of its cytoprotective effects in these patients.
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