Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy
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R. Kothary | Vincent Tabard-Cossa | B. Schneider | A. Reilly | S. Thebault | D. Tessier | M. Deguise | A. Beauvais | Niko Hensel | R. Yaworski