Central and peripheral delivery of AAV9-SMN target different pathomechanisms in a mouse model of spinal muscular atrophy
Aoife Reilly, Marc‐Olivier Deguise, Ariane Beauvais, Rebecca Yaworski, Simon Thebault, Daniel R. Tessier, Vincent Tabard‐Cossa, Niko Hensel, Bernard Laurent Schneider, Rashmi Kothary
The paper compares IV and ICV delivery of AAV9-SMN in SMA mice, showing different SMN restoration patterns and treatment outcomes.
bioRxiv · Nov 8, 2021 · PreprintMolecular Biology