De novo mutation in POLG leads to haplotype insufficiency and Alpers syndrome

Mitochondrion. 2009 Sep;9(5):340-5. doi: 10.1016/j.mito.2009.05.002. Epub 2009 Jun 6.

Abstract

Mutations in POLG are a major contributor to pediatric and adult mitochondrial diseases. However, the consequences of many POLG mutations are not well understood. We investigated the molecular cause of Alpers syndome in a patient harboring the POLG mutations A467T in trans with c.2157+5_+6 gc-->ag in intron 12. Analysis of transcripts arising from the c.2157+5_+6 gc-->ag allele revealed alternative splicing with an insertion of 30 intronic nucleotides leading to a premature termination codon. These transcripts were subsequently removed through nonsense-mediated decay, leading to haplotype insufficiency due to expression of the A467T allele and decreased expression of the c.2157+5_+6 gc-->ag allele, which is likely responsible for the Alpers syndrome phenotype.

Publication types

  • Case Reports
  • Research Support, N.I.H., Extramural
  • Research Support, N.I.H., Intramural

MeSH terms

  • Base Sequence
  • Codon, Nonsense
  • DNA Polymerase gamma
  • DNA-Directed DNA Polymerase / deficiency*
  • Diffuse Cerebral Sclerosis of Schilder / genetics*
  • Haplotypes
  • Humans
  • Infant
  • Male
  • Molecular Sequence Data
  • Mutagenesis, Insertional
  • Mutation*
  • Point Mutation
  • RNA Splicing
  • RNA Stability

Substances

  • Codon, Nonsense
  • DNA Polymerase gamma
  • DNA-Directed DNA Polymerase
  • POLG protein, human