Homozygous deletion of exon 18 leads to degradation of the lysosomal alpha-glucosidase precursor and to the infantile form of glycogen storage disease type II

Clin Genet. 1996 Jun;49(6):325-8. doi: 10.1111/j.1399-0004.1996.tb03801.x.

Abstract

We describe two unrelated Dutch patients with typical symptoms of infantile glycogen storage disease type II (GSD II) and virtual absence of acid alpha-glucosidase activity in leukocytes and cultured skin fibroblasts. The patients were identified as homozygotes for a deletion of exon 18 of the acid alpha-glucosidase gene (GAA). The in-frame deletion manifests at the protein level in a characteristic way: the enzyme precursor is smaller than normal and degraded in the endoplasmic reticulum or Golgi complex. These case present an evident example of a genotype-phenotype correlation in glycogen storage disease type II.

Publication types

  • Case Reports

MeSH terms

  • Chromosome Deletion*
  • Enzyme Precursors / metabolism*
  • Exons*
  • Glycogen Storage Disease Type II / genetics*
  • Homozygote*
  • Humans
  • Infant, Newborn
  • Lysosomes / enzymology*
  • Male
  • Pedigree
  • alpha-Glucosidases / deficiency*
  • alpha-Glucosidases / metabolism

Substances

  • Enzyme Precursors
  • alpha-Glucosidases