Congenital sucrase-isomaltase deficiency arising from cleavage and secretion of a mutant form of the enzyme

J Clin Invest. 2000 Jul;106(2):281-7. doi: 10.1172/JCI9677.

Abstract

Congenital sucrase-isomaltase deficiency (CSID) is an autosomal recessive human intestinal disorder that is clinically characterized by fermentative diarrhea, abdominal pain, and cramps upon ingestion of sugar. The symptoms are the consequence of absent or drastically reduced enzymatic activities of sucrase and isomaltase, the components of the intestinal integral membrane glycoprotein sucrase-isomaltase (SI). Several known phenotypes of CSID result from an altered posttranslational processing of SI. We describe here a novel CSID phenotype, in which pro-SI undergoes an unusual intracellular cleavage that eliminates its transmembrane domain. Biosynthesis of pro-SI in intestinal explants and in cells transfected with the SI cDNA of this phenotype demonstrated a cleavage occurring within the endoplasmic reticulum due to a point mutation that converts a leucine to proline at residue 340 of isomaltase. Cleaved pro-SI is transported to and processed in the Golgi apparatus and is ultimately secreted into the exterior milieu as an active enzyme. To our knowledge this is the first report of a disorder whose pathogenesis results not from protein malfolding or mistargeting, but from the conversion of an integral membrane glycoprotein into a secreted species that is lost from the cell surface.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Biological Transport
  • Biopsy
  • Carbohydrate Metabolism, Inborn Errors / genetics*
  • Cell Compartmentation
  • Child, Preschool
  • Golgi Apparatus / metabolism
  • Humans
  • Intestine, Small / ultrastructure
  • Membrane Proteins / genetics
  • Models, Molecular
  • Phenotype
  • Point Mutation*
  • Protein Precursors / metabolism
  • Protein Processing, Post-Translational
  • Sucrase-Isomaltase Complex / deficiency*
  • Sucrase-Isomaltase Complex / genetics
  • Sucrase-Isomaltase Complex / metabolism

Substances

  • Membrane Proteins
  • Protein Precursors
  • Sucrase-Isomaltase Complex