Phytocannabinoid treatment in a mouse model of West syndrome with spontaneous seizures

The phytocannabinoid (pCB), cannabidiol, is approved in the US for treatment of Lennox-Gasteaut syndrome and Dravet, syndrome, both of which are neurodevelopmental disorders with intractable epilepsy. West syndrome is an epileptic encephalopathy characterized by recurrent seizures generally resistant to traditional anti-epileptic drugs, severe neurological deficits, and early death. One of its monogenic causes is the instability of trinucleotide (GCN) repeats affecting the exon 2 of Aristaless-related homeobox (ARX)-like gene, codifying an interneuron-specific transcription factor with a key role in mammalian corticogenesis and neuronal maturation. Here we report on pCB treatment in the polyAlanine repeat mouse model, Arx(GCG)7/Y, which expresses seven GCG-triplets inserted at residue 330 of the mouse Arx gene.