Neutrophil-specific granule deficiency results from a novel mutation with loss of function of the transcription factor CCAAT/enhancer binding protein epsilon

J Exp Med. 1999 Jun 7;189(11):1847-52. doi: 10.1084/jem.189.11.1847.

Abstract

Neutrophil-specific granule deficiency (SGD) is a rare disorder characterized by recurrent pyogenic infections, defective neutrophil chemotaxis and bactericidal activity, and lack of neutrophil secondary granule proteins. CCAAT/enhancer binding protein (C/EBP)epsilon, a member of the leucine zipper family of transcription factors, is expressed primarily in myeloid cells, and its knockout mouse model possesses distinctive defects, including a lack of neutrophil secondary granule proteins. Sequence analysis of the genomic DNA of a patient with SGD revealed a five-basepair deletion in the second exon of the C/EBPepsilon locus. The predicted frame shift results in a truncation of the 32-kD major C/EBPepsilon isoform, with loss of the dimerization domain, DNA binding region, and transcriptional activity. The multiple functional defects observed in these early neutrophil progenitor cells, a consequence of C/EBPepsilon deficiency, define SGD as a defect in myelopoiesis and establish the requirement for C/EBPepsilon for the promyelocyte-myelocyte transition in myeloid differentiation.

MeSH terms

  • Animals
  • Base Sequence
  • CCAAT-Enhancer-Binding Proteins
  • Cytoplasmic Granules / pathology
  • Cytoplasmic Granules / physiology
  • DNA Primers / genetics
  • DNA-Binding Proteins / genetics*
  • DNA-Binding Proteins / physiology*
  • HeLa Cells
  • Humans
  • Immunologic Deficiency Syndromes / genetics*
  • Immunologic Deficiency Syndromes / pathology
  • Immunologic Deficiency Syndromes / physiopathology
  • Mice
  • Mice, Knockout
  • Mutation*
  • Neutrophils / pathology
  • Neutrophils / physiology*
  • Nuclear Proteins / deficiency
  • Nuclear Proteins / genetics*
  • Nuclear Proteins / physiology*
  • Polymerase Chain Reaction

Substances

  • CCAAT-Enhancer-Binding Proteins
  • DNA Primers
  • DNA-Binding Proteins
  • Nuclear Proteins