SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Mueller, Christian; Berry, James D.; McKenna-Yasek, Diane M.; Gernoux, Gwladys; Owegi, Margaret A.; Pothier, Lindsay M.; Douthwright, Catherine L.; Gelevski, Dario; Luppino, Sarah D.; Blackwood, Meghan; Wightman, Nicholas S.; Oakley, Derek H.; Frosch, Matthew P.; Flotte, Terrence R.; Cudkowicz, Merit E.; Brown, Robert H.

doi:10.1056/nejmoa2005056

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

Summary Two patients with familial amyotrophic lateral sclerosis (ALS) and mutations in the gene encoding superoxide dismutase 1 (SOD1) were treated with a single intrathecal infusion of adeno-associated virus encoding a microRNA targeting SOD1. In Patient 1, SOD1 levels in spinal cord tissue as …