Microcephaly, short stature, and limb abnormality disorder due to novel autosomal biallelic DONSON mutations in two German siblings

Eur J Hum Genet. 2018 Sep;26(9):1282-1287. doi: 10.1038/s41431-018-0128-0. Epub 2018 May 14.

Abstract

Recently, variants in DONSON have been reported to cause different disorders of the microcephalic primordial dwarfism spectrum. Using whole-exome sequencing, we identified two novel, compound heterozygous DONSON variants in a pair of siblings, one of whom was previously diagnosed with Fanconi anemia. This occurred because the present cases exhibited clinical findings in addition to those of the microcephalic primordial dwarfism disorder, including severe limb malformations. These findings suggest that the DONSON and Fanconi anemia proteins could have supplementary roles in developmental processes as they have in the maintenance of genomic integrity, resulting in related disease phenotypes.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aborted Fetus / pathology
  • Cell Cycle Proteins / genetics*
  • Dwarfism / genetics*
  • Dwarfism / pathology
  • Female
  • Heterozygote
  • Humans
  • Infant
  • Limb Deformities, Congenital / genetics*
  • Limb Deformities, Congenital / pathology
  • Microcephaly / genetics*
  • Microcephaly / pathology
  • Mutation
  • Nuclear Proteins / genetics*
  • Phenotype*
  • Syndrome

Substances

  • Cell Cycle Proteins
  • DONSON protein, human
  • Nuclear Proteins