Mucopolysaccharidosis I: Alpha-L-Iduronidase mutations in three Tunisian families

J Inherit Metab Dis. 2005;28(6):1019-26. doi: 10.1007/s10545-005-0197-4.

Abstract

Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease resulting from the defective activity of the enzyme alpha-L-iduronidase (IDUA). The disease has severe and milder phenotypic subtypes. The IDUA mutations in five MPS I patients from three unrelated families from central and southern Tunisia were determined by amplifying and sequencing each of the IDUA exons and intron-exon junctions. Two novel IDUA mutations, c.1805delTinsGAACA in exon 13 and I270S in exon 7, and two previously reported mutations, P533R and R628X, were detected. The two patients in family 1 who had the Hurler phenotype were homoallelic for the novel deletion-insertion mutation. The patient in family 2 who also had the Hurler phenotype was heteroallelic for the novel missense mutation I270S and the previously reported nonsense mutation R628X. The two patients in family 3 who had the Hurler-Scheie phenotype were homoallelic for P533R. In addition, six known IDUA polymorphisms were identified. These are the first Tunisian MPS I patients to be genotyped. The identification of these mutations and their genotype-phenotype correlations should facilitate prenatal diagnosis and counselling for MPS I in Tunisia, where a very high rate of consanguinity exists.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Alleles
  • Consanguinity
  • DNA Mutational Analysis
  • DNA, Complementary / metabolism
  • Exons
  • Family Health
  • Female
  • Gene Deletion
  • Genotype
  • Heterozygote
  • Humans
  • Iduronidase / genetics*
  • Introns
  • Leukocytes / metabolism
  • Male
  • Mucopolysaccharidosis I / diagnosis*
  • Mucopolysaccharidosis I / genetics*
  • Mutation*
  • Mutation, Missense
  • Pedigree
  • Phenotype
  • Tunisia

Substances

  • DNA, Complementary
  • Iduronidase