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Article

Durable suppression of seizures in a preclinical model of KCNT1 genetic epilepsy with divalent small interfering RNA

27 Jan 2025

Abstract excerpt

OBJECTIVE: Gain-of-function variants in the KCNT1 gene, which encodes a sodium-activated potassium ion channel, drive severe early onset developmental epileptic encephalopathies including epilepsy of infancy with migrating focal seizures and sleep-related hypermotor epilepsy. No therapy provides more than sporadic or incremental improvement. Here, we report suppression of seizures in a genetic mouse model of...

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PMID: 39871703
DOI: 10.1111/epi.18278

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