Maternal transmission of a rare GABRB3 signal peptide variant is associated with autism

Mol Psychiatry. 2011 Jan;16(1):86-96. doi: 10.1038/mp.2009.118. Epub 2009 Nov 24.

Abstract

Maternal 15q11-q13 duplication is the most common copy number variant in autism, accounting for ∼1-3% of cases. The 15q11-q13 region is subject to epigenetic regulation, and genomic copy number losses and gains cause genomic disorders in a parent-of-origin-specific manner. One 15q11-q13 locus encodes the GABA(A) receptor β3 subunit gene (GABRB3), which has been implicated by several studies in both autism and absence epilepsy, and the co-morbidity of epilepsy in autism is well established. We report that maternal transmission of a GABRB3 signal peptide variant (P11S), previously implicated in childhood absence epilepsy, is associated with autism. An analysis of wild-type and mutant β3 subunit-containing α1β3γ2 or α3β3γ2 GABA(A) receptors shows reduced whole-cell current and decreased β3 subunit protein on the cell surface due to impaired intracellular β3 subunit processing. We thus provide the first evidence of an association between a specific GABA(A) receptor defect and autism, direct evidence that this defect causes synaptic dysfunction that is autism relevant and the first maternal risk effect in the 15q11-q13 autism duplication region that is linked to a coding variant.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autistic Disorder / genetics*
  • Chromosomes, Human, Pair 15*
  • Female
  • Genome-Wide Association Study
  • Germ-Line Mutation
  • Humans
  • Male
  • Pedigree
  • Polymorphism, Single Nucleotide
  • Receptors, GABA-A / genetics*

Substances

  • GABRB3 protein, human
  • Receptors, GABA-A