Morquio B syndrome: a primary defect in beta-galactosidase

Am J Med Genet. 1983 Oct;16(2):261-75. doi: 10.1002/ajmg.1320160215.

Abstract

Fibroblasts from patients with Morquio B syndrome contain normal numbers of beta-galactosidase molecules with normal turnover but strongly reduced activity per enzyme molecule. Various substrate affinities are abnormal: the Km for methylum belliferyl (MU)-beta-galactoside is 4-10-fold elevated and affinity for keratan sulphate and oligosaccharides, isolated from Morquio B urine, was not detectable. In contrast, these substrate affinities are normal for beta-galactosidase in adult type GM1-gangliosidosis fibroblasts. Cell hybridization studies demonstrate that Morquio B syndrome and infantile and adult type GM1-gangliosidosis belong to the same complementation group. From these results we conclude that Morquio B syndrome is caused by a mutation in the structural gene for beta-galactosidase, which is allelic to the mutations in infantile and adult type GM1-gangliosidosis. Urinary excretion of keratan sulphate and oligosaccharides is abnormal in Morquio B syndrome but normal in adult type GM1-gangliosidosis. The catalytic properties of beta-galactosidase in Morquio B syndrome and GM1-gangliosidosis provide a possible explanation for the distinct clinical manifestations in these disorders.

Publication types

  • Comparative Study

MeSH terms

  • Fibroblasts / enzymology
  • Galactosidases / genetics*
  • Glycosaminoglycans / urine
  • Humans
  • Hybrid Cells / enzymology
  • Lactose Intolerance
  • Mucopolysaccharidosis IV / enzymology*
  • Mucopolysaccharidosis IV / genetics
  • Mucopolysaccharidosis IV / urine
  • Mutation
  • Neuraminidase / metabolism
  • Oligosaccharides / urine
  • beta-Galactosidase / genetics*

Substances

  • Glycosaminoglycans
  • Oligosaccharides
  • Galactosidases
  • Neuraminidase
  • beta-Galactosidase