Recombination events between the p47-phox gene and its highly homologous pseudogenes are the main cause of autosomal recessive chronic granulomatous disease

Blood. 2000 Mar 15;95(6):2150-6.

Abstract

Chronic granulomatous disease (CGD) is an inherited disease caused by defects in the superoxide-generating nicotinamide adenine dinucleotide phosphate (NADPH) oxidase of phagocytes. Genetic lesions in any of 4 components of this antimicrobial enzyme have been detected. Family-specific mutations are found in 3 of 4 forms of CGD due to deficiencies of the gp91-phox, p22-phox, and p67-phox genes. In p47-phox-deficient CGD (autosomal recessive form A47 degrees ) patients, a GT deletion (triangle upGT) at the beginning of exon 2 of the p47-phox gene has been reported in 19 of 20 alleles. This GT deletion is also characteristic for the recently identified p47-phox pseudogenes. To explore a possible link between these findings, a sequence analysis of 28 unrelated, racially diverse A47 degrees CGD patients and 37 healthy individuals was performed. The GT deletion in exon 2 was present on all alleles in 25 patients. Only 3 patients but all healthy individuals contained the GTGT and triangle upGT sequences. A total of 22 patients carried additional pseudogene-specific intronic sequences on all alleles, either only in intron 1 or in intron 1 and intron 2, which lead to different types of chimeric DNA strands. It is concluded that recombination events between the p47-phox gene and its highly homologous pseudogenes result in the incorporation of triangle upGT into the p47-phox gene, thereby leading to the high frequency of GT deletion in A47 degrees CGD patients. (Blood. 2000;95:2150-2156)

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Exons
  • Gene Deletion
  • Genes, Recessive
  • Granulomatous Disease, Chronic / genetics*
  • Heterozygote
  • Humans
  • Introns
  • Molecular Sequence Data
  • NADPH Oxidases
  • Phosphoproteins / genetics*
  • Polymorphism, Single-Stranded Conformational
  • Pseudogenes / genetics*
  • Recombination, Genetic*
  • Restriction Mapping
  • Reverse Transcriptase Polymerase Chain Reaction
  • Sequence Homology, Nucleic Acid

Substances

  • Phosphoproteins
  • NADPH Oxidases
  • neutrophil cytosolic factor 1