Article

A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies

2025-10-22

Abstract excerpt

<h4>Summary Paragraph</h4> Variants in ion channel genes are common causes of pediatric epilepsy, often leading to intractable seizures, developmental delay and other comorbidities, which increases risk of death. Pathogenic variants in the SCN8A gene, which encodes a voltage-gated sodium channel critical for action potential generation in the brain, account for ∼1% of genetic epilepsies. The voltage sensor in S...

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Literature Corpus work: 201601f4-1495-53b4-afdb-f55c2cc11298
DOI: 10.1101/2025.10.22.683934

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