Syndromic true hermaphroditism due to an R-spondin1 (RSPO1) homozygous mutation

Hum Mutat. 2008 Feb;29(2):220-6. doi: 10.1002/humu.20665.

Abstract

XX true hermaphroditism, also know as ovotesticular disorder of sexual development (DSD), is a disorder of gonadal development characterized by the presence of both ovarian and testicular tissue in a 46,XX individual. The genetic basis for XX true hermaphroditism and sex reversal syndromes unrelated to SRY translocation is still mostly unclear. We report mutational analysis of the RSPO1 gene in a 46,XX woman with true hermaphroditism, palmoplantar keratoderma, congenital bilateral corneal opacities, onychodystrophy, and hearing impairment. R-spondin1 is a member of the R-spondin protein family and its pivotal role in sex determination has been recently described. We identified a homozygous splice-donor-site mutation in the RSPO1 gene in our patient. We found that the c.286+1G>A mutation led to an aberrantly spliced mRNA (r.95_286del), which is presumably translated into a partially functional protein (p.Ile32_Ile95del). Our case demonstrates for the first time, to our knowledge, that XX true hermaphroditism can be caused by a single gene mutation. The reported findings represent a further step toward a complete understanding of the complex mechanisms leading to DSDs.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Amino Acid Sequence
  • Base Sequence
  • DNA Mutational Analysis
  • Female
  • Gonads / cytology
  • Homozygote*
  • Humans
  • Molecular Sequence Data
  • Mutation / genetics*
  • Ovotesticular Disorders of Sex Development / genetics*
  • RNA Splicing
  • RNA, Messenger / genetics
  • RNA, Messenger / metabolism
  • Syndrome
  • Thrombospondins / chemistry
  • Thrombospondins / genetics*

Substances

  • RNA, Messenger
  • RSPO1 protein, human
  • Thrombospondins