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Article

In vivo adenine base editing ameliorates Dravet syndrome phenotypes in a mouse model.

Science translational medicine - 13 May 2026

Nelson Andrew T, Hill Sophie F, Simon Matthew, Clatot Jérôme, Sakai Holt A, Xie Jun, Sousa Alexander A, An Meirui, Gao Guangping, Lutz Cathleen M, Goldberg Ethan M, Liu David R

Abstract excerpt

Dravet syndrome (DS) is a severe neurodevelopmental disorder characterized by drug-resistant epilepsy, temperature-sensitive seizures, cognitive impairment, and a high incidence of sudden unexpected death in epilepsy (SUDEP). DS is caused by loss-of-function variants in SCN1A, which encodes the α subunit of the voltage-gated sodium channel (Nav1.1). Current approved treatments manage symptoms of DS but do not...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 42127220
DOI: 10.1126/scitranslmed.adx5999

Genes

SCN1A

Variants

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 8 Sept 2026.

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