A girl with West syndrome and autistic features harboring a de novo TBL1XR1 mutation

J Hum Genet. 2014 Oct;59(10):581-3. doi: 10.1038/jhg.2014.71. Epub 2014 Aug 7.

Abstract

Recently, de novo mutations in TBL1XR1 were found in two patients with autism spectrum disorders. Here, we report on a Japanese girl presenting with West syndrome, Rett syndrome-like and autistic features. Her initial development was normal until she developed a series of spasms at 5 months of age. Electroencephalogram at 7 months showed a pattern of hypsarrhythmia, which led to a diagnosis of West syndrome. Stereotypic hand movements appeared at 8 months of age, and autistic features such as deficits in communication, hyperactivity and excitability were observed later, at 4 years and 9 months. Whole exome sequencing of the patient and her parents revealed a de novo TBL1XR1 mutation [c.209 G>A (p.Gly70Asp)] occurring at an evolutionarily conserved amino acid in an F-box-like domain. Our report expands the clinical spectrum of TBL1XR1 mutations to West syndrome with Rett-like features, together with autistic features.

Publication types

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

MeSH terms

  • Autistic Disorder / diagnosis
  • Autistic Disorder / genetics*
  • DNA Mutational Analysis
  • Exons
  • Female
  • Humans
  • Infant
  • Mutation, Missense*
  • Nuclear Proteins / genetics*
  • Receptors, Cytoplasmic and Nuclear / genetics*
  • Repressor Proteins / genetics*
  • Rett Syndrome / diagnosis
  • Rett Syndrome / genetics*
  • Spasms, Infantile / diagnosis
  • Spasms, Infantile / genetics*

Substances

  • Nuclear Proteins
  • Receptors, Cytoplasmic and Nuclear
  • Repressor Proteins
  • TBL1XR1 protein, human