Adeno-associated virus-mediated restoration of NaV1.1 in targeted brain regions ameliorates epilepsy in symptomatic Dravet syndrome mice.
OBJECTIVE Dravet syndrome (DS) is a developmental and epileptic encephalopathy primarily caused by haploinsufficiency of the SCN1A gene, which encodes the alpha subunit of NaV1.1 voltage-gated sodium channel. The disease manifests with febrile and spontaneous seizures, developmental delay, cognitive impairment, and inc...