The molecular genetic basis of muscle phosphoglycerate mutase (PGAM) deficiency

Am J Hum Genet. 1993 Mar;52(3):472-7.

Abstract

The glycolytic enzyme phosphoglycerate mutase (PGAM) is a dimer, and mature human skeletal muscle contains almost exclusively the MM form of the enzyme, PGAM-M. In 1981, we identified a patient with PGAM-M deficiency, and three additional patients have since been described. All presented with exercise intolerance, cramps, and myoglobinuria. We report two new patients with PGAM-M deficiency and describe the molecular lesions in five patients--four African-Americans and one Caucasian. Three patients were homozygous for an identical G-to-A transition converting an encoded Trp to an in-frame stop codon (codon 78). A fourth patient was heterozygous for this mutation and also carried an A-to-C mutation converting Glu to Ala (codon 89). The fifth patient, the only Caucasian, was homozygous for a different point mutation, a C-to-T mutation, converting Arg to Trp (codon 90).

Publication types

  • Case Reports
  • Comparative Study
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Adolescent
  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • Bisphosphoglycerate Mutase / deficiency*
  • Bisphosphoglycerate Mutase / genetics*
  • Carbohydrate Metabolism, Inborn Errors / enzymology
  • Carbohydrate Metabolism, Inborn Errors / genetics*
  • Child
  • Codon / genetics
  • Exons
  • Female
  • Humans
  • Isoenzymes / deficiency*
  • Isoenzymes / genetics*
  • Male
  • Molecular Sequence Data
  • Muscles / enzymology*
  • Mutation*
  • Oligodeoxyribonucleotides
  • Pedigree
  • Polymerase Chain Reaction / methods
  • Reference Values

Substances

  • Codon
  • Isoenzymes
  • Oligodeoxyribonucleotides
  • Bisphosphoglycerate Mutase