Mutant POLG2 disrupts DNA polymerase gamma subunits and causes progressive external ophthalmoplegia

Am J Hum Genet. 2006 Jun;78(6):1026-34. doi: 10.1086/504303. Epub 2006 May 4.

Abstract

DNA polymerase gamma (pol gamma ) is required to maintain the genetic integrity of the 16,569-bp human mitochondrial genome (mtDNA). Mutation of the nuclear gene for the catalytic subunit of pol gamma (POLG) has been linked to a wide range of mitochondrial diseases involving mutation, deletion, and depletion of mtDNA. We describe a heterozygous dominant mutation (c.1352G-->A/p.G451E) in POLG2, the gene encoding the p55 accessory subunit of pol gamma , that causes progressive external ophthalmoplegia with multiple mtDNA deletions and cytochrome c oxidase (COX)-deficient muscle fibers. Biochemical characterization of purified, recombinant G451E-substituted p55 protein in vitro revealed incomplete stimulation of the catalytic subunit due to compromised subunit interaction. Although G451E p55 retains a wild-type ability to bind DNA, it fails to enhance the DNA-binding strength of the p140-p55 complex. In vivo, the disease most likely arises through haplotype insufficiency or heterodimerization of the mutated and wild-type proteins, which promote mtDNA deletions by stalling the DNA replication fork. The progressive accumulation of mtDNA deletions causes COX deficiency in muscle fibers and results in the clinical phenotype.

Publication types

  • Case Reports
  • Research Support, N.I.H., Intramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age of Onset
  • Amino Acid Sequence
  • Catalytic Domain / genetics
  • DNA / metabolism
  • DNA Polymerase gamma
  • DNA, Mitochondrial / analysis
  • DNA, Mitochondrial / genetics
  • DNA-Directed DNA Polymerase / genetics*
  • DNA-Directed DNA Polymerase / metabolism*
  • Dimerization
  • Electron Transport Complex IV / analysis
  • Electron Transport Complex IV / metabolism
  • Enzyme Inhibitors / pharmacology
  • Ethylmaleimide / pharmacology
  • Female
  • Heterozygote
  • Humans
  • Middle Aged
  • Molecular Sequence Data
  • Muscle Fibers, Skeletal / enzymology
  • Muscle, Skeletal / chemistry
  • Muscle, Skeletal / enzymology
  • Mutation
  • Nucleic Acid Synthesis Inhibitors
  • Ophthalmoplegia, Chronic Progressive External / diagnosis
  • Ophthalmoplegia, Chronic Progressive External / enzymology
  • Ophthalmoplegia, Chronic Progressive External / genetics*
  • Protein Subunits / genetics
  • Protein Subunits / metabolism

Substances

  • DNA, Mitochondrial
  • Enzyme Inhibitors
  • Nucleic Acid Synthesis Inhibitors
  • Protein Subunits
  • DNA
  • Electron Transport Complex IV
  • DNA Polymerase gamma
  • DNA-Directed DNA Polymerase
  • POLG protein, human
  • Ethylmaleimide