POLD1 variants leading to reduced polymerase activity can cause hearing loss without syndromic features

Hum Mutat. 2020 May;41(5):913-920. doi: 10.1002/humu.23984. Epub 2020 Jan 30.

Abstract

DNA polymerase δ, whose catalytic subunit is encoded by POLD1, is responsible for synthesizing the lagging strand of DNA. Single heterozygous POLD1 mutations in domains with polymerase and exonuclease activities have been reported to cause syndromic deafness as a part of multisystem metabolic disorder or predisposition to cancer. However, the phenotypes of diverse combinations of POLD1 genotypes have not been elucidated in humans. We found that five members of a multiplex family segregating autosomal recessive nonsyndromic sensorineural hearing loss (NS-SNHL) have revealed novel compound heterozygous POLD1 variants (p.Gly1100Arg and a presumptive null function variant, p.Ser197Hisfs*54). The recombinant p.Gly1100Arg polymerase δ showed a reduced polymerase activity by 30-40%, but exhibited normal exonuclease activity. The polymerase activity in cell extracts from the affected subject carrying the two POLD1 mutant alleles was about 33% of normal controls. We suggest that significantly decreased polymerase δ activity, but not a complete absence, with normal exonuclease activity could lead to NS-SNHL.

Keywords: NS-SNHL; POLD1; Pol δ; exonuclease; polymerase.

Publication types

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

MeSH terms

  • Adult
  • Alleles
  • Amino Acid Sequence
  • Amino Acid Substitution
  • Biomarkers
  • DNA Polymerase III / genetics*
  • DNA Polymerase III / metabolism
  • Enzyme Activation
  • Exome Sequencing
  • Female
  • Genetic Association Studies* / methods
  • Genetic Predisposition to Disease*
  • Genetic Variation*
  • Genotype
  • Hearing Loss / diagnosis*
  • Hearing Loss / genetics*
  • Humans
  • Male
  • Mutation
  • Pedigree
  • Phenotype
  • Polymorphism, Single Nucleotide
  • Siblings
  • Syndrome

Substances

  • Biomarkers
  • POLD1 protein, human
  • DNA Polymerase III