Article

PV interneuron-targeted CRISPRa rescue of <i>SCN1A</i> haploinsufficiency in Dravet syndrome

2026-07-14

Abstract excerpt

Dravet syndrome is a severe epileptic encephalopathy caused by SCN1A haploinsufficiency, which leads to reduced Na V 1.1 expression in parvalbumin (PV)-expressing interneurons and disrupted excitatory-inhibitory balance in the brain. We developed an AAV-based CRISPR activation system (AAV9-E2-dCas9-VP64) to selectively upregulate SCN1A from its endogenous locus in PV interneurons. An in vitro saturating guide R...

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Literature Corpus work: 389d8d7f-087b-5617-816c-7bd9ca06415d
DOI: 10.64898/2026.07.12.737793

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