Central and peripheral delivered AAV9-SMN are both efficient but 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 L. Schneider, Rashmi Kothary
Topics & Concepts
Spinal muscular atrophySMA*SMN1BiologyNeuroprotectionPeripheralPeripheral nervous systemCentral nervous systemMotor neuronGenetic enhancementNeurosciencePathologyMedicineSpinal cordInternal medicineDiseaseGeneMathematicsCombinatoricsBiochemistryNeurogenetic and Muscular Disorders ResearchCongenital Anomalies and Fetal SurgeryRNA modifications and cancer