Molecular analysis in two siblings African patients with severe form of Hunter Syndrome: identification of a novel (p.Y54X) nonsense mutation

J Trop Pediatr. 2007 Dec;53(6):434-7. doi: 10.1093/tropej/fmm056. Epub 2007 Jul 5.

Abstract

Hunter syndrome (or Mucopolysaccharidosis type II, MPS II) is an X-linked recessive disorder due to the deficiency of the iduronate-2-sulfatase (IDS) enzyme, resulting in the accumulation of heparan and dermatan sulfates in the lysosomes. The heterogeneity of clinical phenotypes, ranging from mild-to-severe forms, is a result of different mutations in the IDS gene. We report here, a novel nonsense mutation (p.Y54X) in two siblings MPS II African patients affected with a severe form of the disease. We postulated that the p.Y54X mutation which causes a loss of the IDS region highly conserved among sulfatase enzymes, could be predicted as a severe disease-causing mutation for Hunter syndrome.

Publication types

  • Case Reports

MeSH terms

  • Black People / genetics*
  • Child
  • Codon, Nonsense*
  • Glycoproteins / genetics*
  • Humans
  • Male
  • Mucopolysaccharidosis II / diagnostic imaging
  • Mucopolysaccharidosis II / genetics*
  • Radiography
  • Siblings

Substances

  • Codon, Nonsense
  • Glycoproteins
  • IDS protein, human