Article
SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS.
The New England journal of medicine - 9 Jul 2020
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
Abstract excerpt
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 analyzed on autopsy were lower than corresponding levels in untreated patients with SOD1-mediated ALS and in healthy controls....
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.