Exploring the potential role of disease-causing mutation in a gene desert: duplication of noncoding elements 5' of GRIA3 is associated with GRIA3 silencing and X-linked intellectual disability

Hum Mutat. 2012 Feb;33(2):355-8. doi: 10.1002/humu.21649. Epub 2011 Nov 28.

Abstract

GRIA3 encodes glutamate receptor ionotropic AMPA (alpha-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid) subunit 3 and has been previously involved in X-linked intellectual disability (ID). We report on a male proband with ID and epilepsy associated with a duplication mapping within a gene desert, 874-kb upstream of the GRIA3 gene. This 970-kb duplication is maternally inherited. The proband's mother has a skewed X chromosome-inactivation pattern in agreement with her normal cognitive function. Quantitative polymerase chain reaction analysis indicates absence of GRIA3 mRNA in the proband lymphocytes relative to a wild-type control. Centromeric to the duplicated region, comparative genomic analysis showed a 2268-bp evolutionarily conserved region that could be a critical transcription factor binding-site for GRIA3 expression. The repositioning of distant-acting sequences, rather a missense/nonsense mutation, is considered to be causative for GRIA3-linked ID. This study illustrates the importance of high-resolution array-Comparative Genomic Hybridization analysis in exploring the potential role of disease-causing mutation in functional noncoding sequences.

Publication types

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

MeSH terms

  • 5' Untranslated Regions*
  • Child
  • Comparative Genomic Hybridization
  • Exons
  • Gene Duplication*
  • Gene Silencing*
  • Genetic Diseases, X-Linked / genetics*
  • Humans
  • Intellectual Disability / genetics*
  • Male
  • Mutation*
  • Receptors, AMPA / genetics*
  • X Chromosome Inactivation

Substances

  • 5' Untranslated Regions
  • Receptors, AMPA
  • glutamate receptor ionotropic, AMPA 3