Classical galactosaemia: novel insights in IgG N-glycosylation and N-glycan biosynthesis

Eur J Hum Genet. 2016 Jul;24(7):976-84. doi: 10.1038/ejhg.2015.254. Epub 2016 Jan 6.

Abstract

Classical galactosaemia (OMIM #230400), a rare disorder of carbohydrate metabolism, is caused by a deficient activity of galactose-1-phosphate uridyltransferase (EC 2.7.7.12). The pathophysiology of the long-term complications, mainly cognitive, neurological and female fertility problems remains poorly understood. The lack of validated biomarkers to determine prognosis, monitor disease progression and responses to new therapies, pose a huge challenge. We report the detailed analysis of an automated robotic hydrophilic interaction ultra-performance liquid chromatography N-glycan analytical method of high glycan peak resolution applied to serum IgG. This has revealed specific N-glycan processing defects observed in 40 adult galactosaemia patients (adults and adolescents), in comparison with 81 matched healthy controls. We have identified a significant increase in core fucosylated neutral glycans (P<0.0001) and a significant decrease in core fucosylated (P<0.001), non-fucosylated (P<0.0001) bisected glycans and, of specific note, decreased N-linked mannose-5 glycans (P<0.0001), in galactosaemia patients. We also report the abnormal expression of a number of related relevant N-glycan biosynthesis genes in peripheral blood mononuclear cells from 32 adult galactosaemia patients. We have noted significant dysregulation of two key N-glycan biosynthesis genes: ALG9 upregulated (P<0.001) and MGAT1 downregulated (P<0.01) in galactosaemia patients, which may contribute to its ongoing pathophysiology. Our data suggest that the use of IgG N-glycosylation analysis with matched N-glycan biosynthesis gene profiles may provide useful biomarkers for monitoring response to therapy and interventions. They also indicate potential gene modifying steps in this N-glycan biosynthesis pathway, of relevance to galactosaemia and related N-glycan biosynthesis disorders.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Biomarkers / blood
  • Case-Control Studies
  • Female
  • Galactosemias / blood
  • Galactosemias / genetics*
  • Galactosemias / pathology
  • Glycosylation
  • Humans
  • Immunoglobulin G / blood
  • Immunoglobulin G / metabolism*
  • Male
  • Mannosyltransferases / genetics
  • Mannosyltransferases / metabolism
  • Membrane Proteins / genetics
  • Membrane Proteins / metabolism
  • Monocytes / metabolism
  • N-Acetylglucosaminyltransferases / genetics
  • N-Acetylglucosaminyltransferases / metabolism
  • Polysaccharides / biosynthesis*
  • Protein Processing, Post-Translational*

Substances

  • Biomarkers
  • Immunoglobulin G
  • Membrane Proteins
  • Polysaccharides
  • ALG9 protein, human
  • MGAT1 protein, human
  • Mannosyltransferases
  • N-Acetylglucosaminyltransferases