Long-term downregulation of the sodium channel gene Scn8a is therapeutic in mouse models of SCN8A epilepsy.

Sophie F HillWenxi YuJulie ZiobroSanjna ChalasaniFaith RegerMiriam H Meisler

Published in: Annals of neurology (2023)

Downregulation of Scn8a expression that is initiated after the onset of seizures is effective for long-term treatment in a model of SCN8A-DEE. Repeated ASO administration or a single dose of viral shRNA prevented seizures and extended survival through 12 months' observation. This article is protected by copyright. All rights reserved.

Keyphrases

cell proliferation
signaling pathway
poor prognosis
temporal lobe epilepsy
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