Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome.

Guy M LenkPaymaan Jafar-NejadSophie F HillLucas D HuffmanCorrine E SmolenJacy L WagnonHayley PetitWenxi YuJulie ZiobroKritika BhatiaJack ParentRoman J GigerFrank RigoMiriam H Meisler

Published in: Annals of neurology (2020)

Reduction of Scn8a transcript by 25 to 50% delayed seizure onset and lethality in mouse models of SCN8A encephalopathy and Dravet syndrome. Reduction of SCN8A transcript is a promising approach to treatment of intractable childhood epilepsies. Ann Neurol 2020;87:339-346.

Keyphrases

mouse model
early onset
case report
rna seq
childhood cancer