Mutation analysis of a Japanese patient with fucosidosis

J Hum Genet. 1999;44(5):323-6. doi: 10.1007/s100380050169.

Abstract

Fucosidosis is a rare autosomal recessive disorder resulting from a deficiency of alpha-L-fucosidase. Recently, various mutations have been reported in this disease, but it is difficult to elucidate the phenotype from the genetic mutations. We report a patient with chronic infantile type fucosidosis, with a compound heterozygote of a nonsense mutation (W148X, Trp at codon 148 to stop codon) and a large deletion, including all exons. This is the first report of a large deletion demonstrated in fucosidosis. It is interesting that this patient has a relatively mild clinical course despite the absence of the mRNA. This case also indicates the difficulty in determining the phenotype from the genotype in fucosidosis.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Amino Acid Substitution
  • Asian People
  • Base Sequence
  • Chromosome Mapping
  • Chromosomes, Human, Pair 1*
  • Codon, Terminator
  • Exons
  • Female
  • Fucosidosis / enzymology
  • Fucosidosis / genetics*
  • Humans
  • In Situ Hybridization, Fluorescence
  • Japan
  • Male
  • Mutation, Missense*
  • Phenotype
  • Sequence Deletion*
  • alpha-L-Fucosidase / genetics*

Substances

  • Codon, Terminator
  • alpha-L-Fucosidase