New pharmacological target for Fragile X Syndrome and related disorders (Ref. TEC0089)
New therapeutic target for Fragile X Syndrome and related disorders based on an antagonist of the cannabinoid receptor CB1 for use in the prevention/treatment of diseases with neuronal dendritic abnormalities. Fragile X syndrome (FXS) is the most common form of inherited intellectual disability and autism. Acute administration of low doses of a selective CB1R antagonist/agonist in mice corrects the features associated to genetic silencing of the Fmr1 gene. Main facts of our technology:
- Possibility to use a similar targeting approach to orphan diseases with a common neurobiological substrate.
- First time that the CB1 cannabinoid receptors are demonstrated to play a critical role in this disease.