Exome Sequencing of Two Siblings with Sporadic Autism Spectrum Disorder and Severe Speech Sound Disorder Suggests Pleiotropic and Complex Effects

Behav Genet. 2019 Jul;49(4):399-414. doi: 10.1007/s10519-019-09957-8. Epub 2019 Apr 4.

Abstract

Recent studies of autism spectrum disorder (ASD) and childhood apraxia of speech (CAS) have resulted in conflicting conclusions regarding the comorbidity of these disorders on phenotypic grounds. In a nuclear family with two dually affected and one unaffected offspring, whole-exome sequences were evaluated for single nucleotide and indel variants and CNVs. The affected siblings but not the unaffected sibling share a rare deleterious compound heterozygous mutation in WWOX, implicated both in ASD and motor control. In addition, one of the affected children carries a rare deleterious de novo mutation in the ASD candidate gene RIMS1. The two affected children but not their unaffected sibling inherited deleterious variants with relevance for ASD and/or CAS. WWOX, RIMS1, and several of the genes harboring the inherited variants are expressed in the brain during prenatal and early postnatal development. Results suggest compound heterozygosity as a cause of ASD and CAS, pleiotropic gene effects, and potentially additional, complex genetic effects.

Keywords: Autism spectrum disorder; Childhood apraxia of speech; Comorbidity; Complex trait; Heterogeneity; Language impairment; Multiple hits.

Publication types

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

MeSH terms

  • Adolescent
  • Adult
  • Autism Spectrum Disorder / etiology
  • Autism Spectrum Disorder / genetics*
  • Child
  • DNA Copy Number Variations / genetics
  • Exome / genetics
  • Exome Sequencing / methods
  • Family
  • Female
  • GTP-Binding Proteins / genetics
  • GTP-Binding Proteins / metabolism
  • Genetic Pleiotropy / genetics
  • Genetic Predisposition to Disease / genetics
  • Humans
  • Male
  • Multifactorial Inheritance / genetics
  • Nerve Tissue Proteins / genetics
  • Nerve Tissue Proteins / metabolism
  • Siblings
  • Speech Sound Disorder / etiology
  • Speech Sound Disorder / genetics*
  • Tumor Suppressor Proteins / genetics*
  • Tumor Suppressor Proteins / metabolism
  • WW Domain-Containing Oxidoreductase / genetics*
  • WW Domain-Containing Oxidoreductase / metabolism

Substances

  • Nerve Tissue Proteins
  • RIMS1 protein, human
  • Tumor Suppressor Proteins
  • WW Domain-Containing Oxidoreductase
  • WWOX protein, human
  • GTP-Binding Proteins