The role of the endocannabinoid system in autism spectrum disorders: Evidence from mouse studies

Susanna Pietropaolo, Luigi Bellocchio, Inés Bouzón-Arnáiz, Benjamin K. Yee

Research output: Chapter in book / Conference proceedingChapter in an edited book (as author)Academic researchpeer-review

15 Citations (Scopus)


A substantive volume of research on autism spectrum disorder (ASD) has emerged in recent years adding to our understanding of the etiopathological process. Preclinical models in mice and rats have been highly instrumental in modeling and dissecting the contributions of a multitude of known genetic and environmental risk factors. However, the translation of preclinical data into suitable drug targets must overcome three critical hurdles: (i) ASD comprises a highly heterogeneous group of conditions that can markedly differ in terms of their clinical presentation and symptoms, (ii) the plethora of genetic and environmental risk factors suggests a complex, non-unitary, etiopathology, and (iii) the lack of consensus over the myriad of preclinical models, with respect to both construct validity and face validity. Against this backdrop, this Chapter traces how the endocannabinoid system (ECS) has emerged as a promising target for intervention with predictive validity. Recent supportive preclinical evidence is summarized, especially studies in mice demonstrating the emergence of ASD-like behaviors following diverse genetic or pharmacological manipulations targeting the ECS. The critical relevance of ECS to the complex pathogenesis of ASD is underscored by its multiple roles in modulating neuronal functions and shaping brain development. Finally, we argue that important lessons have been learned from the novel mouse models of ASD, which not only stimulate game-changing innovative treatments but also foster a consensual framework to integrate the diverse approaches applied in the search of novel treatments for ASD.

Original languageEnglish
Title of host publicationProgress in Molecular Biology and Translational Science
EditorsMirolyuba Ilieva, Way Kwok-Wai Lau
PublisherElsevier B.V.
Number of pages26
ISBN (Print)9780128212424
Publication statusPublished - 23 May 2020

Publication series

NameProgress in Molecular Biology and Translational Science
ISSN (Print)1877-1173
ISSN (Electronic)1878-0814


  • Autism
  • Cannabinoids
  • CB1 receptor
  • Developmental disorders
  • Mouse models
  • Social interactions

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology


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