Ultra-rare mutation in long-range enhancer predisposes to thyroid carcinoma with high penetrance

PLoS One. 2013 May 14;8(5):e61920. doi: 10.1371/journal.pone.0061920. Print 2013.

Abstract

Thyroid cancer shows high heritability but causative genes remain largely unknown. According to a common hypothesis the genetic predisposition to thyroid cancer is highly heterogeneous; being in part due to many different rare alleles. Here we used linkage analysis and targeted deep sequencing to detect a novel single-nucleotide mutation in chromosome 4q32 (4q32A>C) in a large pedigree displaying non-medullary thyroid carcinoma (NMTC). This mutation is generally ultra-rare; it was not found in 38 NMTC families, in 2676 sporadic NMTC cases or 2470 controls. The mutation is located in a long-range enhancer element whose ability to bind the transcription factors POU2F and YY1 is significantly impaired, with decreased activity in the presence of the C- allele compared with the wild type A-allele. An enhancer RNA (eRNA) is transcribed in thyroid tissue from this region and is greatly downregulated in NMTC tumors. We suggest that this is an example of an ultra-rare mutation predisposing to thyroid cancer with high penetrance.

Publication types

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

MeSH terms

  • Case-Control Studies
  • Chromosomes, Human, Pair 4 / genetics
  • DNA Mutational Analysis
  • Databases, Genetic
  • Enhancer Elements, Genetic / genetics*
  • Genetic Linkage
  • Genetic Loci / genetics
  • Genetic Predisposition to Disease / genetics*
  • Genomics
  • Humans
  • Mutation*
  • Penetrance*
  • Thyroid Neoplasms / genetics*
  • Transcription, Genetic / genetics