PTEN c.511C>T nonsense mutation in a BRRS family disrupts a potential exonic splicing enhancer and causes exon skipping

Jpn J Clin Oncol. 2006 Dec;36(12):814-21. doi: 10.1093/jjco/hyl107. Epub 2006 Oct 16.

Abstract

Bannayan-Riley-Ruvalcaba syndrome (BRRS) is an autosomal dominant disorder characterized by macrocephaly, intestinal hamartomatous polyps, lipomas and pigmented macules of the glans penis. We identified a Thai family affected with BRRS. In addition to typical manifestations of BRRS, the proband has a large hepatic AVM which is rarely found in BRRS. The molecular analysis revealed affected members were heterozygous for an exon skipping-associated nonsense mutation c.511C>T in the PTEN gene. The mutation was previously assumed to be deleterious by causing a change to a termination codon, Q171X. We, herein, found that another pathogenic effect was splicing related by disrupting a potential exonic splicing enhancer (ESE) and causing an entire exon 6 skipping. The results prompted us to investigate other reported missense/nonsense mutations in the PTEN gene. We found that they do not colocalize with ESE sites, suggesting that most of their pathogenic effects are not through ESE disruption.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Exons / genetics*
  • Female
  • Hamartoma Syndrome, Multiple / diagnosis
  • Hamartoma Syndrome, Multiple / genetics*
  • Humans
  • Male
  • Middle Aged
  • Mutation, Missense / genetics*
  • PTEN Phosphohydrolase / genetics*
  • Pedigree
  • Phenotype
  • RNA Splicing / genetics*
  • Regulatory Sequences, Ribonucleic Acid / genetics*

Substances

  • Regulatory Sequences, Ribonucleic Acid
  • PTEN Phosphohydrolase
  • PTEN protein, human