A novel patient with White-Sutton syndrome refines the mutational and clinical repertoire of the POGZ-related phenotype and suggests further observations

Am J Med Genet A. 2020 Jul;182(7):1791-1795. doi: 10.1002/ajmg.a.61605. Epub 2020 May 2.

Abstract

A rare developmental delay (DD)/intellectual disability (ID) syndrome with craniofacial dysmorphisms and autistic features, termed White-Sutton syndrome (WHSUS, MIM#614787), has been recently described, identifying truncating mutations in the chromatin regulator POGZ (KIAA0461, MIM#614787). We describe a further WHSUS patient harboring a novel nonsense de novo POGZ variant, which afflicts a protein domain with transposase activity less frequently impacted by mutational events (DDE domain). This patient displays additional physical and behavioral features, these latter mimicking Smith-Magenis syndrome (SMS, MIM#182290). Considering sleep-wake cycle anomalies and abnormal behavior manifested by this boy, we reinforced the clinical resemblance between WHSUS and SMS, being both chromatinopathies. In addition, using the DeepGestalt technology, we identified a different facial overlap between WHSUS patients with mutations in the DDE domain (Group 1) and individuals harboring variants in other protein domains/regions (Group 2). This report further delineates the clinical and molecular repertoire of the POGZ-related phenotype, adding a novel patient with uncommon clinical and behavioral features and provides the first computer-aided facial study of WHSUS patients.

Keywords: POGZ; Face2Gene; White-Sutton syndrome; chromatinopathies.

Publication types

  • Case Reports

MeSH terms

  • Child, Preschool
  • Codon, Nonsense / genetics
  • Craniofacial Abnormalities / diagnosis
  • Craniofacial Abnormalities / diagnostic imaging
  • Craniofacial Abnormalities / genetics
  • Craniofacial Abnormalities / physiopathology
  • Developmental Disabilities / diagnosis
  • Developmental Disabilities / diagnostic imaging
  • Developmental Disabilities / genetics*
  • Developmental Disabilities / physiopathology
  • Exome / genetics
  • Female
  • Humans
  • Intellectual Disability / diagnosis
  • Intellectual Disability / diagnostic imaging
  • Intellectual Disability / genetics*
  • Intellectual Disability / physiopathology
  • Male
  • Mutation / genetics
  • Phenotype
  • Smith-Magenis Syndrome / diagnosis
  • Smith-Magenis Syndrome / diagnostic imaging
  • Smith-Magenis Syndrome / genetics*
  • Smith-Magenis Syndrome / physiopathology
  • Transposases / genetics*

Substances

  • Codon, Nonsense
  • POGZ protein, human
  • Transposases