Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology.

Huntington disease (HD) is a fatal, neurodegenerative genetic disorder with aggregation of mutant Huntingtin protein (mutHTT) in the brain as a key pathological mechanism. There are currently no disease modifying therapies for HD; however, -lowering therapies hold promise. Recombinant adeno-associated virus serotype 5 expressing a microRNA that targets mRNA (AAV5-miHTT) is in development for the treatment of HD with promising results in rodent and minipig HD models.

Cannabinoid antagonist SLV326 induces convulsive seizures and changes in the interictal EEG in rats.

Cannabinoid CB1 antagonists have been investigated for possible treatment of e.g. obesity-related disorders.