4-phenylbutyrate promoted wildtype GABA receptor trafficking, reduced Endoplasmic reticulum stress and mitigated seizures in Gabrg2 mice associated with Dravet syndrome.

GABA receptor subunit gene mutations are major causes of various epilepsy syndromes, including severe kinds such as Dravet syndrome. Although the GABA receptor is a major target for antiseizure medications, treating GABA receptor mutations with the receptor channel modulators is ineffective. Here we determined the effect of a novel treatment with 4-phenylbutyrate (PBA) in the Gabrg2 knockin mice associated with Dravet syndrome.