Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: insights into organ toxicities
Fumiaki Aihara, Mathew Mardo, Vera M. Ruda, Tony del Rio, Karine Bigot, Jochen Singer, Megumi Onishi‐Seebacher, Philippe Couttet, Keith Mansfield, Eloïse Hudry
This study examines transcriptional changes in non-human primate tissues after AAV9 vector delivery, finding significant immune-related gene upregulation in liver and DRG.
Molecular Therapy — Methods & Clinical Development · Oct 17, 2025Peer-reviewedMolecular Biology