Type 1 Gaucher disease: molecular, biochemical, and clinical characterization of patients from northern Portugal

Biochem Med Metab Biol. 1993 Feb;49(1):97-107. doi: 10.1006/bmmb.1993.1011.

Abstract

We report the study of 16 catholic type 1 Gaucher disease patients originating from a well-defined region in the north of Portugal where a relatively high incidence is observed. The patients were screened for mutations: 3060G-->A, 5841A-->G, 5976C-->G, and 6433T-->C, which enabled the identification of 27 of the 32 mutated alleles. Four different genotypes were identified, namely 5841G/6433C (n = 6), 5841G/5841G (n = 5), 5841G/? (n = 4), and 6433C/? (n = 1). All but one of the patients carried at least one 5841G mutated allele, making its frequency 62.5%, which is similar to that described for Ashkenazi Jewish patients. The 5841G homozygotes presented an overall milder clinical profile, whereas no clear genotype/phenotype correlation could be established for heterozygous patients. On the basis of residual glucocerebrosidase activity, no distinction could be made between 5841G homozygotes and 5841G/6433C compound heterozygotes. Patients that had at least one 5841G allele (encoding the Ser 370 mutated enzyme) all presented a cell-type-specific residual glucocerebrosidase activity as well as an increased molecular activity when measured in the presence of the physiological activators.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Alleles
  • Base Sequence
  • Child
  • Child, Preschool
  • Female
  • Gaucher Disease / enzymology
  • Gaucher Disease / genetics*
  • Genotype
  • Glucosylceramidase / genetics
  • Glucosylceramidase / metabolism
  • Humans
  • Hydrogen-Ion Concentration
  • Male
  • Middle Aged
  • Molecular Sequence Data
  • Mutation

Substances

  • Glucosylceramidase