Mapping of sudden infant death with dysgenesis of the testes syndrome (SIDDT) by a SNP genome scan and identification of TSPYL loss of function

Proc Natl Acad Sci U S A. 2004 Aug 10;101(32):11689-94. doi: 10.1073/pnas.0401194101. Epub 2004 Jul 23.

Abstract

We have identified a lethal phenotype characterized by sudden infant death (from cardiac and respiratory arrest) with dysgenesis of the testes in males [Online Mendelian Inheritance in Man (OMIM) accession no. 608800]. Twenty-one affected individuals with this autosomal recessive syndrome were ascertained in nine separate sibships among the Old Order Amish. High-density single-nucleotide polymorphism (SNP) genotyping arrays containing 11,555 single-nucleotide polymorphisms evenly distributed across the human genome were used to map the disease locus. A genome-wide autozygosity scan localized the disease gene to a 3.6-Mb interval on chromosome 6q22.1-q22.31. This interval contained 27 genes, including two testis-specific Y-like genes (TSPYL and TSPYL4) of unknown function. Sequence analysis of the TSPYL gene in affected individuals identified a homozygous frameshift mutation (457_458insG) at codon 153, resulting in truncation of translation at codon 169. Truncation leads to loss of a peptide domain with strong homology to the nucleosome assembly protein family. GFP-fusion expression constructs were constructed and illustrated loss of nuclear localization of truncated TSPYL, suggesting loss of a nuclear localization patch in addition to loss of the nucleosome assembly domain. These results shed light on the pathogenesis of a disorder of sexual differentiation and brainstem-mediated sudden death, as well as give insight into a mechanism of transcriptional regulation.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Active Transport, Cell Nucleus / genetics
  • Adult
  • Chromosome Mapping*
  • DNA-Binding Proteins / genetics*
  • DNA-Binding Proteins / physiology
  • Family Health
  • Female
  • Frameshift Mutation
  • Genome, Human*
  • Gonadal Dysgenesis / ethnology
  • Gonadal Dysgenesis / genetics*
  • Humans
  • Infant
  • Male
  • Nuclear Proteins / genetics*
  • Nuclear Proteins / physiology
  • Nucleosomes / genetics
  • Pedigree
  • Sex-Determining Region Y Protein
  • Sudden Infant Death / ethnology
  • Sudden Infant Death / genetics*
  • Transcription Factors / genetics*
  • Transcription Factors / physiology

Substances

  • DNA-Binding Proteins
  • Nuclear Proteins
  • Nucleosomes
  • Sex-Determining Region Y Protein
  • Transcription Factors

Associated data

  • OMIM/608800