PTCH1 isoform 1b is the major transcript in the development of basal cell nevus syndrome

J Hum Genet. 2018 Sep;63(9):965-969. doi: 10.1038/s10038-018-0485-0. Epub 2018 Jun 21.

Abstract

Basal cell nevus syndrome (BCNS) is an autosomal dominant disorder most commonly caused by a germline mutation in the PTCH1 gene. PTCH1 is known to have different isoforms with different functional properties and expression patterns among tissues. We detected a novel, pathogenic de novo mutation in PTCH1 isoform 1b (c.114delG) in a BCNS patient. Furthermore, we elucidated the specific expression pattern of PTCH1 isoforms in normal skin, BCC and peripheral blood by studying expression of different PTCH1 isoforms. Human skin showed expression of isoforms 1b and 1d, while peripheral blood additionally showed 1a and 1e expression. BCCs showed expression of all isoforms. Here we report a patient with a novel, isoform 1b specific mutation in PTCH1 and thereby distinguish PTCH1 isoform 1b as the major transcript in the development of BCNS.

Publication types

  • Case Reports

MeSH terms

  • Basal Cell Nevus Syndrome* / genetics
  • Basal Cell Nevus Syndrome* / metabolism
  • Basal Cell Nevus Syndrome* / pathology
  • Child
  • Gene Expression Regulation, Neoplastic*
  • Germ-Line Mutation*
  • Humans
  • Male
  • Patched-1 Receptor* / biosynthesis
  • Patched-1 Receptor* / genetics
  • Protein Isoforms / biosynthesis
  • Protein Isoforms / genetics
  • Skin Neoplasms* / genetics
  • Skin Neoplasms* / metabolism
  • Skin Neoplasms* / pathology

Substances

  • PTCH1 protein, human
  • Patched-1 Receptor
  • Protein Isoforms