Mucolipidosis III: two patients displaying genetic pleiotropism

S Afr Med J. 1977 Jan 29;51(5):140-4.

Abstract

Two Cape Coloured siblings with typical features of Hurler's syndrome, but without mucopolysacchariduria or mucopolysaccharide accumulation in tissues, are presented. The clinical features, in conjunction with raised beta-D-galactosidase and alpha-L-fucosidase levels in fibroblast cultures from one of the patients, suggest the diagnosis of a mucolipidosis. Theories relating to the intracellular deficiency and extracellular excess of lysosomal enzymes in these conditions are reviewed. Phenotypical and cell culture differences between 2 siblings who display the same overall clinical syndrome, illustrate the genetic pleiotropism inherent in this group of diseases.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Child
  • Female
  • Fibroblasts / enzymology
  • Glycoside Hydrolases / analysis
  • Humans
  • Lysosomes / enzymology
  • Mucolipidoses / enzymology
  • Mucolipidoses / genetics*
  • Phenotype

Substances

  • Glycoside Hydrolases