Gene dosage of DAX-1, determining in sexual differentiation: duplication of DAX-1 in two sisters with gonadal dysgenesis

Mol Biol Rep. 2019 Jun;46(3):2971-2978. doi: 10.1007/s11033-019-04758-y. Epub 2019 Mar 16.

Abstract

Two sisters phenotypically normal females, presenting with tumor abdominal mass with histopathological findings of teratoma and gonadoblastoma associated to 46,XY male-to-female sex reversal syndrome, secondary to a duplication in DAX-1, possibly inherited of maternal gonadal mosaicism. Copy number variation and functional effects of the duplication were done by MLPA multiplex ligation-dependent probe amplification and real time PCR. DAX-1, also known as dosage sensitive sex reversal gene (DSS), is considered the most likely candidate gene involved in XY gonadal dysgenesis when overexpressed. The excess of DAX-1 gene disturbs testicular development by down regulation of SF-1, WT1, and SOX9. This is the first report of 46,XY sex reversal in two siblings who have a maternally inherited duplication of DAX-1 associated with reduced levels of expression of downstream genes as SOX9-SF1.

Keywords: DAX-1; DSD; Disorders of sex development; Gonadoblastoma; Sex reversal syndrome; Simple gonadal dysgenesis.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Child
  • DAX-1 Orphan Nuclear Receptor / genetics*
  • DAX-1 Orphan Nuclear Receptor / metabolism
  • DNA Copy Number Variations
  • DNA-Binding Proteins / genetics
  • Female
  • Gene Dosage / genetics
  • Gene Duplication
  • Gonadal Dysgenesis / genetics*
  • Gonadal Dysgenesis, 46,XY / genetics
  • Gonadoblastoma / genetics
  • Humans
  • Pedigree
  • Sex Determination Analysis / methods
  • Sex Determination Processes / genetics*
  • Sex Differentiation
  • Sexual Maturation / genetics
  • Siblings
  • Teratoma
  • Testis / abnormalities

Substances

  • DAX-1 Orphan Nuclear Receptor
  • DNA-Binding Proteins
  • NR0B1 protein, human

Supplementary concepts

  • 46, XY female
  • Anorchia