A de novo truncating mutation in ASXL1 associated with segmental overgrowth

J Genet. 2019 Dec:98:108. doi: 10.1007/s12041-019-1155-5.

Abstract

Mutations in genes involved in chromatin remodelling have been implicated in broad phenotypes of congenital abnormalities and neurodevelopment. However, limited genotype-phenotype correlations are available for some of the rarest genetic disorders that affect chromatin regulation. We hereby describe a 12-year-old girl presented at birth with severe hypotonia, developmental delay, a mid-line capillary malformation and distinctive craniofacial features. During the natural history of her disease, the girl developed severe spasticity and drug-resistant seizures, leading to a diagnosis of Bohring-Opitz syndrome (BOS). We performed whole-exome sequencing (WES) and identified a de novo mutation in ASXL1 (c.2033dupG) which results in the introduction of a premature stop codon (p.R678fs*6). ASXL1 encodes a polycomb repressive complex protein implicated in chromatin regulation and de novo mutations are a known cause of BOS. Phenotypes with segmental craniofacial overgrowth associated to midline capillary malformations enlarge the clinical spectrum of BOS at onset and further expand the differential diagnosis in ASXL1 mutation carriers.

Publication types

  • Case Reports

MeSH terms

  • Base Sequence
  • Brain / diagnostic imaging
  • Child
  • Codon, Nonsense / genetics
  • Craniosynostoses / diagnostic imaging
  • Craniosynostoses / genetics*
  • Craniosynostoses / physiopathology
  • Exome Sequencing
  • Female
  • Genetic Association Studies
  • Genetic Diseases, Inborn / genetics
  • Genetic Predisposition to Disease / genetics*
  • Humans
  • Intellectual Disability / diagnostic imaging
  • Intellectual Disability / genetics*
  • Intellectual Disability / physiopathology
  • Muscle Hypotonia
  • Mutation*
  • Pedigree
  • Phenotype
  • Repressor Proteins / genetics*
  • Repressor Proteins / metabolism*

Substances

  • ASXL1 protein, human
  • Codon, Nonsense
  • Repressor Proteins

Supplementary concepts

  • Bohring syndrome