AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet Syndrome
Juan F. Antinao Diaz, Ellie M. Chilcott, Amanda Almacellas‐Barbanoj, Anna Keegan, Sonam Gurung, Valda Pauzuolyte, Zak Waddington, Maria Kyriacou, Amy McTague, Judith Helen Cross, Stéphanie Schorge, Gabriele Lignani, Simon N. Waddington, 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.
Molecular Therapy — Nucleic Acids · May 1, 2026Peer-reviewedMolecular Biology