Coffin-Siris Syndrome with obesity, macrocephaly, hepatomegaly and hyperinsulinism caused by a mutation in the ARID1B gene

Eur J Hum Genet. 2014 Nov;22(11):1327-9. doi: 10.1038/ejhg.2014.25. Epub 2014 Feb 26.

Abstract

Coffin-Siris Syndrome (CSS, MIM 135900) is a rare genetic disorder, and mutations in ARID1B were recently shown to cause CSS. In this study, we report a novel ARID1B mutation identified by whole-exome sequencing in a patient with clinical features of CSS. We identified a novel heterozygous frameshift mutation c.1584delG in exon 2 of ARID1B (NM_020732.3) predicting a premature stop codon p.(Leu528Phefs*65). Sanger sequencing confirmed the c.1584delG mutation as a de novo in the proband and that it was not present either in her parents, half-sister or half-brother. Clinically, the patient presented with extreme obesity, macrocephaly, hepatomegaly, hyperinsulinism and polycystic ovarian syndrome (PCOS), which have previously not been described in CSS patients. We suggest that obesity, macrocephaly, hepatomegaly and/or PCOS may be added to the list of clinical features of ARID1B mutations, but further clinical reports are required to make a definite conclusion.

Publication types

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

MeSH terms

  • Abnormalities, Multiple / genetics*
  • Adolescent
  • DNA-Binding Proteins / genetics*
  • DNA-Binding Proteins / metabolism
  • Face / abnormalities*
  • Female
  • Frameshift Mutation
  • Hand Deformities, Congenital / genetics*
  • Hepatomegaly / genetics
  • Heterozygote
  • Humans
  • Hyperinsulinism / genetics
  • Intellectual Disability / genetics*
  • Megalencephaly / genetics
  • Micrognathism / genetics*
  • Neck / abnormalities*
  • Obesity / genetics
  • Polycystic Ovary Syndrome / genetics
  • Transcription Factors / genetics*
  • Transcription Factors / metabolism

Substances

  • ARID1B protein, human
  • DNA-Binding Proteins
  • Transcription Factors

Supplementary concepts

  • Coffin-Siris syndrome