Disorders in protein glycosylation and potential therapy: tip of an iceberg?

J Pediatr. 1998 Nov;133(5):593-600. doi: 10.1016/s0022-3476(98)70096-4.

Abstract

Genetic defects in glycoprotein metabolism usually result in neurologic symptoms, but newly discovered defects in glycoprotein biosynthesis (the carbohydrate-deficient glycoprotein syndromes) also present as severe gastrointestinal disorders with hypoglycemia, protein-losing enteropathy, and hepatic pathology. Glycosylation disorders may be more widespread than previously thought and can be detected by using a simple, but underutilized, serum test. Some patients may benefit from promising dietary therapies now in clinical trials.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Child
  • Congenital Disorders of Glycosylation / diagnosis
  • Congenital Disorders of Glycosylation / diet therapy
  • Congenital Disorders of Glycosylation / genetics*
  • Glycoproteins / blood
  • Glycoproteins / genetics
  • Glycosylation
  • Humans
  • Mannose / administration & dosage
  • Transferrin / genetics
  • Transferrin / metabolism

Substances

  • Glycoproteins
  • Transferrin
  • Mannose