Phenotypic heterogeneity and mosaicism in Xia-Gibbs syndrome: Five Danish patients with novel variants in AHDC1

Eur J Med Genet. 2021 Sep;64(9):104280. doi: 10.1016/j.ejmg.2021.104280. Epub 2021 Jul 3.

Abstract

Xia-Gibbs syndrome (XGS) is a neurodevelopmental disorder characterized by intellectual disability, developmental delay, seizures, hypotonia, obstructive sleep apnoea and mild facial dysmorphism. Heterozygosity for loss-of-function variants in AHDC1, encoding the AT-hook DNA binding motif containing protein 1, were discovered in 2014 as the likely genetic cause of Xia-Gibbs syndrome. We present five patients with Xia-Gibbs syndrome caused by previously unreported variants in AHDC1. Two of the patients share a frameshift variant: c.2849del (p.(Pro950Argfs*192)) in AHDC1. Despite sharing this variant, the two patients show remarkable phenotypic differences underscoring the clinical heterogeneity of Xia-Gibbs syndrome. In addition, we present a case of Xia-Gibbs syndrome caused by mosaicism for an AHDC1 variant.

Keywords: AHDC1; Dysmorphism; Reverse phenotyping; Whole exome sequencing; Xia-gibbs syndrome.

Publication types

  • Case Reports

MeSH terms

  • Adolescent
  • Adult
  • Craniofacial Abnormalities / genetics*
  • Craniofacial Abnormalities / pathology
  • DNA-Binding Proteins / genetics*
  • Developmental Disabilities / genetics*
  • Developmental Disabilities / pathology
  • Female
  • Foot Deformities / genetics*
  • Foot Deformities / pathology
  • Frameshift Mutation
  • Humans
  • Male
  • Muscle Hypotonia / genetics*
  • Muscle Hypotonia / pathology
  • Phenotype*
  • Syndrome
  • Young Adult

Substances

  • AHDC1 protein, human
  • DNA-Binding Proteins