Disorder-associated mutations lead to functional inactivation of neuroligins

Hum Mol Genet. 2004 Jul 15;13(14):1471-7. doi: 10.1093/hmg/ddh158. Epub 2004 May 18.

Abstract

Autism is a neuro-developmental syndrome that affects 0.1-0.5% of the population. It has been proposed that alterations in neuronal circuitry and/or neuronal signaling are responsible for the behavioral and cognitive aberrations in autism patients. However, the cellular basis of such alterations is unknown. Recently, point mutations in a family of neuronal cell adhesion molecules called neuroligins have been linked to autism-spectrum disorders and mental retardation. We investigated the consequences of these disease-associated mutations on neuroligin function. We demonstrate that the point mutation at arginine 451 and a nonsense mutation at aspartate 396 of neuroligin-3 and -4 (NL3 and NL4), respectively, result in intracellular retention of the mutant proteins. Over-expression of wild-type NL3 and NL4 proteins in hippocampal neurons stimulates the formation of presynaptic terminals, whereas the disease-associated mutations result in a loss of this synaptic function. Our findings suggest that the previously identified mutations in neuroligin genes are likely to be relevant for the neuro-developmental defects in autism-spectrum disorders and mental retardation since they impair the function of a synaptic cell adhesion molecule.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Arginine / genetics
  • Aspartic Acid / genetics
  • Autistic Disorder / genetics*
  • COS Cells
  • Carrier Proteins / genetics*
  • Carrier Proteins / metabolism
  • Cell Adhesion Molecules, Neuronal
  • Chlorocebus aethiops
  • Genetic Predisposition to Disease
  • Hippocampus / metabolism
  • Humans
  • Intellectual Disability / genetics*
  • Membrane Proteins / genetics*
  • Membrane Proteins / metabolism
  • Mutation, Missense
  • Nerve Tissue Proteins / genetics*
  • Nerve Tissue Proteins / metabolism
  • Neurons / metabolism*
  • Point Mutation
  • Protein Transport

Substances

  • Carrier Proteins
  • Cell Adhesion Molecules, Neuronal
  • Membrane Proteins
  • NLGN4X protein, human
  • Nerve Tissue Proteins
  • neuroligin 3
  • Aspartic Acid
  • Arginine