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 (2024)

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 of observation. ANN NEUROL 2024;95:754-759.

Keyphrases

cell proliferation
mouse model
signaling pathway
poor prognosis
sars cov
temporal lobe epilepsy
genome wide
copy number
gene expression
dna methylation
transcription factor
combination therapy
neural network
smoking cessation
replacement therapy