Human MAMLD1 Gene Variations Seem Not Sufficient to Explain a 46,XY DSD Phenotype

PLoS One. 2015 Nov 16;10(11):e0142831. doi: 10.1371/journal.pone.0142831. eCollection 2015.

Abstract

MAMLD1 is thought to cause disordered sex development in 46,XY patients. But its role is controversial because some MAMLD1 variants are also detected in normal individuals, several MAMLD1 mutations have wild-type activity in functional tests, and the male Mamld1-knockout mouse has normal genitalia and reproduction. Our aim was to search for MAMLD1 variations in 108 46,XY patients with disordered sex development, and to test them functionally. We detected MAMDL1 variations and compared SNP frequencies in controls and patients. We tested MAMLD1 transcriptional activity on promoters involved in sex development and assessed the effect of MAMLD1 on androgen production. MAMLD1 expression in normal steroid-producing tissues and mutant MAMLD1 protein expression were also assessed. Nine MAMLD1 mutations (7 novel) were characterized. In vitro, most MAMLD1 variants acted similarly to wild type. Only the L210X mutation showed loss of function in all tests. We detected no effect of wild-type or MAMLD1 variants on CYP17A1 enzyme activity in our cell experiments, and Western blots revealed no significant differences for MAMLD1 protein expression. MAMLD1 was expressed in human adult testes and adrenals. In conclusion, our data support the notion that MAMLD1 sequence variations may not suffice to explain the phenotype in carriers and that MAMLD1 may also have a role in adult life.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adrenal Glands / embryology
  • Adrenal Glands / metabolism
  • Adult
  • Animals
  • Cell Line
  • Cell Line, Tumor
  • Child, Preschool
  • Cohort Studies
  • DNA-Binding Proteins / genetics*
  • Disorder of Sex Development, 46,XY / genetics*
  • Female
  • Gene Expression Regulation
  • Genetic Variation*
  • Genotype
  • HEK293 Cells
  • Heterozygote
  • Humans
  • Infant
  • Infant, Newborn
  • Male
  • Mice
  • Middle Aged
  • Mutation
  • Nuclear Proteins / genetics*
  • Phenotype
  • Polymorphism, Single Nucleotide
  • Promoter Regions, Genetic
  • Species Specificity
  • Steroid 17-alpha-Hydroxylase / genetics
  • Steroids / chemistry
  • Testis / embryology
  • Testis / metabolism
  • Transcription Factors / genetics*

Substances

  • DNA-Binding Proteins
  • MAMLD1 protein, human
  • Nuclear Proteins
  • Steroids
  • Transcription Factors
  • CYP17A1 protein, human
  • Steroid 17-alpha-Hydroxylase

Grants and funding

This work was supported by grants of the Swiss National Science Foundation (320030-146127) to CEF, the Instituto de Salud Carlos III, Madrid, Spain CIBERER U-712 to MFC, the University and Research Management and Evaluation Agency, Barcelona, Spain (2009SGR31) to LA, and the private Foundation Bangerter-Rhyner, Basel, Switzerland to NC. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.