Characterization and modulation of the endocannabinoid system for treating Fragile X Syndrome
Aperçu du projet
Fragile X syndrome (FXS) is the first inherited cause of intellectual disability and one of the most prevalent causes of autism spectrum disorders. Individuals with FXS do not produce a protein named FMRP. The absence of FMRP results in abnormal brain development and impaired synaptic transmission, which is the mechanism neurons use to communicate with one another. “Cortical hyperexcitability” is now widely recognized by the scientific community as the core deficit in the brain of Fragile X patients. Recent findings suggest that an alteration of the endocannabinoid (eCB) system may contribute to this deficit. Cannabidiol (CBD), a non- hallucinogenic component of the cannabis plant, can modulate the eCB system and has shown promising results in recent Fragile X clinical trials. The aim of this project is to provide a more detailed characterization of the eCB system in FXS using a human neuronal model. Using this approach, we seek to investigate the ability of CBD, and other drugs targeting this system, to correct the core deficit of the pathology. This project may allow us to identify new therapeutical strategies to change the natural course of the syndrome.
Chef d'équipe
Olivier Dionne , Université de Sherbrooke
Partenaire et Donateurs
M. Wayne and J. Coleman Family Fund