Oligosaccharyltransferase-subunit mutations in nonsyndromic mental retardation

Am J Hum Genet. 2008 May;82(5):1150-7. doi: 10.1016/j.ajhg.2008.03.021. Epub 2008 May 1.

Abstract

Mental retardation (MR) is the most frequent handicap among children and young adults. Although a large proportion of X-linked MR genes have been identified, only four genes responsible for autosomal-recessive nonsyndromic MR (AR-NSMR) have been described so far. Here, we report on two genes involved in autosomal-recessive and X-linked NSMR. First, autozygosity mapping in two sibs born to first-cousin French parents led to the identification of a region on 8p22-p23.1. This interval encompasses the gene N33/TUSC3 encoding one subunit of the oligosaccharyltransferase (OTase) complex, which catalyzes the transfer of an oligosaccharide chain on nascent proteins, the key step of N-glycosylation. Sequencing N33/TUSC3 identified a 1 bp insertion, c.787_788insC, resulting in a premature stop codon, p.N263fsX300, and leading to mRNA decay. Surprisingly, glycosylation analyses of patient fibroblasts showed normal N-glycan synthesis and transfer, suggesting that normal N-glycosylation observed in patient fibroblasts may be due to functional compensation. Subsequently, screening of the X-linked N33/TUSC3 paralog, the IAP gene, identified a missense mutation (c.932T-->G, p.V311G) in a family with X-linked NSMR. Recent studies of fucosylation and polysialic-acid modification of neuronal cell-adhesion glycoproteins have shown the critical role of glycosylation in synaptic plasticity. However, our data provide the first demonstration that a defect in N-glycosylation can result in NSMR. Together, our results demonstrate that fine regulation of OTase activity is essential for normal cognitive-function development, providing therefore further insights to understand the pathophysiological bases of MR.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Amino Acid Sequence
  • Female
  • Genes, Recessive
  • Genetic Predisposition to Disease*
  • Glycosylation
  • Hexosyltransferases / genetics*
  • Humans
  • Intellectual Disability / genetics*
  • Male
  • Membrane Proteins / genetics*
  • Mental Retardation, X-Linked / genetics
  • Molecular Sequence Data
  • Mutation
  • Pedigree
  • Protein Subunits / genetics
  • Siblings
  • Tumor Suppressor Proteins / genetics*

Substances

  • Membrane Proteins
  • Protein Subunits
  • TUSC3 protein, human
  • Tumor Suppressor Proteins
  • Hexosyltransferases
  • dolichyl-diphosphooligosaccharide - protein glycotransferase