<i>SOD1</i> Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

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

doi:10.1056/nejmoa2005056

Public

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

Published in New England Journal of Medicine • Jul 8, 2020

Authors:

Christian Mueller

James D. Berry

Diane McKenna‐Yasek

Abstract

) 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. Levels of ...

Subject

Adeno-associated virus

microRNA

Biology

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