Article

Antisense oligonucleotide therapy for <i>KCNT1</i> encephalopathy

2020-11-14

Abstract excerpt

<h4>ABSTRACT</h4> Developmental and epileptic encephalopathies (DEE) are characterized by pharmacoresistant seizures with concomitant intellectual disability. Epilepsy of infancy with migrating focal seizures (EIMFS) is one of the most severe of these syndromes. De novo mutations in ion channels, including gain-of-function variants in KCNT1 , have been found to play a major role in the etiology of EIMFS. Here,...

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Literature Corpus work: d9bbebd9-3144-5173-bca2-e8b9a656d9dc
DOI: 10.1101/2020.11.12.379164

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