Reduced heparan sulfate accumulation in enterocytes contributes to protein-losing enteropathy in a congenital disorder of glycosylation

Am J Pathol. 2000 Dec;157(6):1917-25. doi: 10.1016/S0002-9440(10)64830-4.

Abstract

Intestinal biopsy in a boy with gastroenteritis-induced protein-losing enteropathy (PLE) showed loss of heparan sulfate (HS) and syndecan-1 core protein from the basolateral surface of the enterocytes, which improved after PLE subsided. Isoelectric focusing analysis of serum transferrin indicated a congenital disorder of glycosylation (CDG) and subsequent analysis showed three point mutations in the ALG6 gene encoding an alpha1,3-glucosyltransferase needed for the addition of the first glucose to the dolichol-linked oligosaccharide. The maternal mutation, C998T, causing an A333V substitution, has been shown to cause CDG-Ic, whereas the two paternal mutations, T391C (Y131H) and C924A (S308R) have not previously been reported. The mutations were tested for their ability to rescue faulty N:-linked glycosylation of carboxypeptidase Y in an ALG6-deficient Saccharomyces cerevisiae strain. Normal human ALG6 rescues glycosylation and A333V partially rescues, whereas the combined paternal mutations (Y131H and S308R) are ineffective. Underglycosylation resulting from each of these mutations is much more severe in rapidly dividing yeast. Similarly, incomplete protein glycosylation in the patient is most severe in rapidly dividing enterocytes during gastroenteritis-induced stress. Incomplete N:-linked glycosylation of an HS core protein and/or other biosynthetic enzymes may explain the selective localized loss of HS and PLE.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biopsy
  • Carboxypeptidases / metabolism
  • Enterocytes / metabolism*
  • Fibroblasts / metabolism
  • Glucosyltransferases / deficiency
  • Glycosylation
  • Heparitin Sulfate / metabolism*
  • Histocytochemistry
  • Humans
  • Immunohistochemistry
  • Infant
  • Intestinal Mucosa / metabolism*
  • Intestinal Mucosa / pathology
  • Male
  • Membrane Proteins*
  • Protein-Losing Enteropathies / congenital*
  • Protein-Losing Enteropathies / metabolism*
  • Protein-Losing Enteropathies / pathology
  • Saccharomyces cerevisiae / metabolism

Substances

  • Membrane Proteins
  • Heparitin Sulfate
  • ALG6 protein, human
  • Glucosyltransferases
  • dolichyl pyrophosphate Man(9)GlcNAc(2) alpha1,3-glucosyltransferase
  • Carboxypeptidases