Translating genetic and preclinical findings into autism therapies

Maria Chahrour, Robin J. Kleiman, M. Chiara Manzini

Research output: Contribution to journalArticlepeer-review

5 Scopus citations


Autism spectrum disorder (ASD) is a complex neurodevelopmental disorder characterized by social deficits and repetitive/restrictive interests. ASD is associated with multiple comorbidities, including intellectual disability, anxiety, and epilepsy. Evidence that ASD is highly heritable has spurred major efforts to unravel its genetics, revealing possible contributions from hundreds of genes through rare and common variation and through copy-number changes. In this perspective, we provide an overview of the current state of ASD genetics and of how genetic research has spurred the development of in vivo and in vitro models using animals and patient cells to evaluate the impact of genetic mutations on cellular function leading to disease. Efforts to translate these findings into successful therapies have yet to bear fruit. We discuss how the valuable insight into the disorder provided by these new models can be used to better understand ASD and develop future clinical trials.

Original languageEnglish (US)
Pages (from-to)335-343
Number of pages9
JournalDialogues in Clinical Neuroscience
Issue number4
StatePublished - Dec 1 2017


  • Autism spectrum disorder
  • Genetics
  • Genomics
  • Neurodevelopmental disorder
  • Therapy

ASJC Scopus subject areas

  • Psychiatry and Mental health
  • Biological Psychiatry


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