Article

AAV delivery of RNA editing machinery rescues SUDEP and seizure phenotype in a mouse model of Dravet Syndrome

2025-07-18

Abstract excerpt

<h4>ABSTRACT</h4> Dravet syndrome (DS) is a severe childhood genetic epilepsy, caused by de novo heterozygous mutations in SCN1A , resulting in a loss-of-function of the voltage-gated sodium ion channel, Nav1.1. Nav1.1 is expressed in the brain, and at a lower level, in the heart. DS manifests in the first year of life. Patients exhibit tonic-clonic seizures, febrile seizures, cognitive decline, developmental d...

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Literature Corpus work: fc7b357c-5162-5457-a6b9-18addfc00852
DOI: 10.1101/2025.07.18.664895

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