A novel homozygous DPH1 mutation causes intellectual disability and unique craniofacial features

J Hum Genet. 2018 Apr;63(4):487-491. doi: 10.1038/s10038-017-0404-9. Epub 2018 Feb 6.

Abstract

Biallelic mutations of the gene encoding diphthamide biosynthesis 1 (DPH1, NM_001383.3) cause developmental delay, dysmorphic features, sparse hair, and short stature (MIM *603527). Only two missense DPH1 mutations have been reported to date. Here, we describe a consanguineous family with two siblings both showing developmental delay, agenesis of the corpus callosum, dysmorphic facial features, sparse hair, brachycephaly, and short stature. By wholeexome sequencing, a homozygous frameshift mutation in DPH1 (c.1227delG, p.[Ala411Argfs*91]) was identified, which is likely responsible for the familial condition. The unique clinical features of the affected siblings are cleft palate and absent renal findings.

Publication types

  • Case Reports

MeSH terms

  • Brain / abnormalities
  • Brain / diagnostic imaging
  • Child
  • Child, Preschool
  • Consanguinity
  • Craniofacial Abnormalities / diagnosis*
  • Craniofacial Abnormalities / genetics*
  • Female
  • Genetic Association Studies
  • Homozygote*
  • Humans
  • Intellectual Disability / diagnosis*
  • Intellectual Disability / genetics*
  • Magnetic Resonance Imaging
  • Male
  • Minor Histocompatibility Antigens / genetics*
  • Mutation*
  • Pedigree
  • Phenotype*
  • Siblings
  • Tomography, X-Ray Computed
  • Tumor Suppressor Proteins / genetics*

Substances

  • DPH1 protein, human
  • Minor Histocompatibility Antigens
  • Tumor Suppressor Proteins