Molecular diagnosis of type 1c glycogen storage disease

Hum Genet. 1999 Mar;104(3):275-7. doi: 10.1007/s004390050948.

Abstract

Glycogen storage disease type 1 (GSD 1) results from deficiency of the microsomal multicomponent glucose-6-phosphatase system. Malfunction of the catalytic subunit characterises GSD 1a. GSD 1b and GSD 1c are characterised by defective microsomal glucose-6-phosphate or pyrophosphate/phosphate transport, respectively. Recently, a gene encoding a microsomal transporter protein has been found to be mutated in GSD 1b and 1c patients. Here, we report the genomic sequence of the transporter gene and the detection of a homozygous 2-bp deletion (1211delCT) and a homozygous donor splice site mutation (317+1G-->T) in two GSD 1c patients, confirming that GSD 1c is allelic to GSD 1b.

MeSH terms

  • Antiporters
  • Chromosomes, Human, Pair 11 / genetics
  • Cloning, Molecular
  • DNA / chemistry
  • DNA / genetics
  • DNA Mutational Analysis
  • Diagnosis, Differential
  • Exons
  • Genes / genetics
  • Glycogen Storage Disease Type I / enzymology
  • Glycogen Storage Disease Type I / genetics*
  • Glycogen Storage Disease Type I / pathology
  • Humans
  • Introns
  • Molecular Sequence Data
  • Monosaccharide Transport Proteins
  • Phosphotransferases / genetics*
  • Sequence Analysis, DNA

Substances

  • Antiporters
  • Monosaccharide Transport Proteins
  • SLC37A4 protein, human
  • glucose 6-phosphate(transporter)
  • DNA
  • Phosphotransferases

Associated data

  • GENBANK/AF116862
  • GENBANK/AF116863
  • GENBANK/AF116864