Identification of iduronate sulfatase gene alterations in 70 unrelated Hunter patients

Clin Genet. 1998 May;53(5):362-8. doi: 10.1111/j.1399-0004.1998.tb02746.x.

Abstract

We studied 70 unrelated Hunter patients and found a gene alteration in every patient. The molecular heterogeneity was very important. Large gene rearrangements were identified in 14 patients. Forty-three different mutations were identified in the 56 other patients and 31 were not previously described. Deletions and insertions, splice site mutations were associated with a severe phenotype as nonsense mutations except Q531X. Only a few mutations were present in several patients making difficult genotype-phenotype correlations. Mutation identification allows accurate carrier detection improving prenatal diagnosis. The mother was not found to be a carrier in five cases among the 44 sporadic cases. Haplotype analysis demonstrated a higher frequency of mutations in male meiosis.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Blotting, Southern
  • DNA / analysis
  • DNA / genetics
  • DNA Mutational Analysis
  • Family
  • Family Health
  • Female
  • Gene Deletion
  • Genes / genetics*
  • Genotype
  • Humans
  • Iduronate Sulfatase / genetics*
  • Male
  • Mucopolysaccharidosis II / enzymology
  • Mucopolysaccharidosis II / genetics*
  • Mutation / genetics
  • Phenotype

Substances

  • DNA
  • Iduronate Sulfatase