Improvement of dolichol-linked oligosaccharide biosynthesis by the squalene synthase inhibitor zaragozic acid

J Biol Chem. 2011 Feb 25;286(8):6085-91. doi: 10.1074/jbc.M110.165795. Epub 2010 Dec 23.

Abstract

The majority of congenital disorders of glycosylation (CDG) are caused by defects of dolichol (Dol)-linked oligosaccharide assembly, which lead to under-occupancy of N-glycosylation sites. Most mutations encountered in CDG are hypomorphic, thus leaving residual activity to the affected biosynthetic enzymes. We hypothesized that increased cellular levels of Dol-linked substrates might compensate for the low biosynthetic activity and thereby improve the output of protein N-glycosylation in CDG. To this end, we investigated the potential of the squalene synthase inhibitor zaragozic acid A to redirect the flow of the polyisoprene pathway toward Dol by lowering cholesterol biosynthesis. The addition of zaragozic acid A to CDG fibroblasts with a Dol-P-Man synthase defect led to the formation of longer Dol-P species and to increased Dol-P-Man levels. This treatment was shown to decrease the pathologic accumulation of incomplete Dol pyrophosphate-GlcNAc(2)Man(5) in Dol-P-Man synthase-deficient fibroblasts. Zaragozic acid A treatment also decreased the amount of truncated protein N-linked oligosaccharides in these CDG fibroblasts. The increased cellular levels of Dol-P-Man and possibly the decreased cholesterol levels in zaragozic acid A-treated cells also led to increased availability of the glycosylphosphatidylinositol anchor as shown by the elevated cell-surface expression of the CD59 protein. This study shows that manipulation of the cellular Dol pool, as achieved by zaragozic acid A addition, may represent a valuable approach to improve N-linked glycosylation in CDG cells.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Bridged Bicyclo Compounds, Heterocyclic / pharmacology*
  • CD59 Antigens / biosynthesis
  • CD59 Antigens / genetics
  • Cells, Cultured
  • Cholesterol / biosynthesis
  • Cholesterol / genetics
  • Congenital Disorders of Glycosylation / genetics
  • Congenital Disorders of Glycosylation / metabolism*
  • Dolichols / genetics
  • Dolichols / metabolism*
  • Enzyme Inhibitors / pharmacology*
  • Farnesyl-Diphosphate Farnesyltransferase / antagonists & inhibitors*
  • Farnesyl-Diphosphate Farnesyltransferase / genetics
  • Farnesyl-Diphosphate Farnesyltransferase / metabolism
  • Gene Expression Regulation / drug effects
  • Glycosylation / drug effects
  • Humans
  • Mannosyltransferases / genetics
  • Mannosyltransferases / metabolism
  • Oligosaccharides / biosynthesis*
  • Oligosaccharides / genetics
  • Tricarboxylic Acids / pharmacology*

Substances

  • Bridged Bicyclo Compounds, Heterocyclic
  • CD59 Antigens
  • Dolichols
  • Enzyme Inhibitors
  • Oligosaccharides
  • Tricarboxylic Acids
  • CD59 protein, human
  • squalestatin 1
  • Cholesterol
  • Mannosyltransferases
  • dolichyl-phosphate beta-D-mannosyltransferase
  • Farnesyl-Diphosphate Farnesyltransferase