Abnormal glycosylation with hypersialylated O-glycans in patients with Sialuria

Biochim Biophys Acta. 2006 Jun;1762(6):598-607. doi: 10.1016/j.bbadis.2006.03.009.

Abstract

Sialuria is an inborn error of metabolism characterized by coarse face, hepatomegaly and recurrent respiratory tract infections. The genetic defect in this disorder results in a loss of feedback control of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine-kinase by CMP-N-acetylneuraminic acid (CMP-NeuAc) resulting in a substantial overproduction of cytoplasmic free sialic acid. This study addresses fibroblast CMP-NeuAc levels and N- and O-glycan sialylation of serum proteins from Sialuria patients. CMP-NeuAc levels were measured with HPLC in fibroblasts. Isoelectric focusing (IEF) of serum transferrin and of apolipoprotein C-III (apoC-III) was performed on serum of three Sialuria patients. Isoforms of these proteins can be used as specific markers for the biosynthesis of N- and core 1 O-glycans. Furthermore, total N- and O-linked glycans from serum proteins were analyzed by HPLC. HPLC showed a clear overproduction of CMP-NeuAc in fibroblasts of a Sialuria patient. Minor changes were found for serum N-glycans and hypersialylation was found for core 1 O-glycans on serum apoC-III and on total serum O-glycans in Sialuria patients. HPLC showed an increased ratio of disialylated over monosialylated core 1 O-glycans. The hypersialylation of core 1 O-glycans is due to the increase of NeuAcalpha2,6-containing structures (mainly NeuAcalpha2-3Galbeta1-3[NeuAcalpha2-6]GalNAc). This may relate to KM differences between GalNAc-alpha2,6-sialyltransferase and alpha2,3-sialyltransferases. This is the first study demonstrating that the genetic defect in Sialuria results in a CMP-NeuAc overproduction. Subsequently, increased amounts of alpha2,6-linked NeuAc were found on serum core 1 O-glycans from Sialuria patients. N-glycosylation of serum proteins seems largely unaffected. Sialuria is the first metabolic disorder presenting with hypersialylated O-glycans.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Apolipoprotein C-III
  • Apolipoproteins C / analysis
  • Apolipoproteins C / blood
  • Chromatography, High Pressure Liquid
  • Glycoproteins / blood
  • Glycosylation
  • Humans
  • Isoelectric Focusing
  • N-Acetylneuraminic Acid / metabolism*
  • Nucleotides / analysis
  • Nucleotides / isolation & purification
  • Polysaccharides / blood
  • Polysaccharides / metabolism*
  • Protein Isoforms
  • Sialic Acid Storage Disease / metabolism*
  • Transferrin / analysis

Substances

  • Apolipoprotein C-III
  • Apolipoproteins C
  • Glycoproteins
  • Nucleotides
  • Polysaccharides
  • Protein Isoforms
  • Transferrin
  • N-Acetylneuraminic Acid