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    Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy

    Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of the SMN1 gene and low SMN protein levels. Although lower motor neurons are a primary target, there is evidence that peripheral organ defe...

    Aoife Reilly, Marc-Olivier Deguise, Ariane Beauvais, Rebecca Yaworski… in Gene Therapy (2022)