Molecular genetic heterogeneity of phosphoglycerate kinase (PGK) deficiency

Muscle Nerve Suppl. 1995:3:S45-9. doi: 10.1002/mus.880181411.

Abstract

Phosphoglycerate kinase (PGK; EC 2.7.2.3) is a glycolytic enzyme encoded by a single gene on the X chromosome and ubiquitously expressed. Hereditary PGK deficiency can cause hemolytic anemia, central nervous system dysfunction, and/or myopathy characterized by exercise intolerance, cramps, and myoglobinuria. So far, 20 PGK variants with reduced PGK activity have been identified, 8 of them in patients with myopathy. Six missense mutations and one splice-junction mutation have been identified in 7 patients, 2 of whom had myopathy. However, the biochemical and molecular bases for clinical heterogeneity in PGK deficiency remain unknown.

Publication types

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

MeSH terms

  • Base Sequence
  • Glycogen Storage Disease / genetics*
  • Humans
  • Molecular Sequence Data
  • Muscles / enzymology*
  • Mutation
  • Phosphoglycerate Kinase / deficiency*
  • Phosphoglycerate Kinase / genetics*

Substances

  • Phosphoglycerate Kinase