As our approach relies on vector-mediated SCN1A ORF delivery, rather than transcriptional activation of a functional (and a mutated) SCN1A allele, it is potentially suitable for patients with truncation or missense mutations in SCN1A, with demonstrated efficacy in 2 DS models (Figures 5–9 and Supplemental Figure 14). Here, SCN1A is linked to Dravet syndrome.