Mechanisms of hypercholesterolaemia in glycogen storage disease type I: defective metabolism of low density lipoprotein in cultured skin fibroblasts

Eur J Clin Invest. 1990 Jun;20(3):253-60. doi: 10.1111/j.1365-2362.1990.tb01852.x.

Abstract

Hyperlipidaemia is a feature of glycogen storage disease type I (GSD-I) (Levy et al.). High levels of LDL cholesterol (200 +/- 25 mg dl-1) and apo B (387 +/- 44 mg dl-1) were found in association with hypercholesterolaemia in GSD-I. Related causative factors might be attributed to overproduction and/or delayed removal of LDL. In this study, a possible alteration in the clearance of LDL was examined. Using cultured fibroblasts for LDL receptor activity, the following observations were made: 1. GSD-I fibroblasts revealed only a slight decrease in LDL binding (65 +/- 7) when compared with controls (74 +/- 4 ng mg-1 protein), however, LDL internalization (382 +/- 24 vs. 570 +/- 52 ng mg-1 protein) and proteolytic degradation (2082 +/- 280 vs. 2916 +/- 12.5 ng mg-1 protein) were significantly affected (P less than 0.01). 2. Binding, internalization and proteolytic degradation of LDL from GSD-I were compared with that of controls, and were found to be significantly lower (P less than 0.01). 3. Substitution of control lipoprotein-deficient serum (LPDS) by GSD-I LPDS further diminished the above processes (P less than 0.05). Our results demonstrate that increased plasma cholesterol in GSD-I is due to a decreased catabolism of LDL. The data suggest that the problem may well be multifactorial, due to diminished receptor expression, abnormal LDL composition and impaired LDL receptor interaction due to a circulating inhibitory factor.

MeSH terms

  • Adolescent
  • Adult
  • Cells, Cultured
  • Child
  • Child, Preschool
  • Cholesterol, LDL / blood
  • Female
  • Fibroblasts / metabolism
  • Glycogen Storage Disease Type I / complications*
  • Glycogen Storage Disease Type I / metabolism
  • Humans
  • Hypercholesterolemia / etiology*
  • Hypercholesterolemia / metabolism
  • Lipoproteins, LDL / blood
  • Lipoproteins, LDL / metabolism*
  • Male
  • Receptors, LDL / metabolism
  • Skin / metabolism

Substances

  • Cholesterol, LDL
  • Lipoproteins, LDL
  • Receptors, LDL