Molecular BiologybioRxivPreprint
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…·Rashmi Kothary
The paper compares IV and ICV delivery of AAV9-SMN in SMA mice, showing different SMN restoration patterns and treatment outcomes.
1 citation