Article

Optimizing Lentiviral Vector-Based Delivery of <i>SCN1A</i> Transgenes to Mammalian Cells

2026-05-01

Abstract excerpt

The SCN1A gene encodes Na V 1.1, a voltage-gated sodium channel protein that is necessary for neuronal excitability and whose loss-of-function mutations cause Dravet syndrome, a treatment-resistant childhood onset epilepsy. Gene replacement strategies for this syndrome are challenged by the large size of SCN1A and difficulty achieving stable cellular expression. Lentiviral vectors (LVVs) offer sufficient packag...

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Literature Corpus work: 6ea4bd36-fc93-546d-8a30-4f414fd12f4d
DOI: 10.64898/2026.05.01.722074

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