Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome
Juan F. Antinao Diaz, Ellie M. Chilcott, Amanda Almacellas‐Barbanoj…·Rajvinder Karda
This paper demonstrates the use of AAV9 vectors to deliver AntagoNATs targeting a natural antisense transcript in a DS mouse model, improving survival and reducing seizures.
DOI 10.1016/j.omtn.2026.102942