Adeno-associated viral vector delivered RNAi for gene therapy of SOD1 Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease caused by progressive loss of upper and lower motor neurons. Mutations in superoxide dismutase (SOD1) are a leading cause of ALS, responsible for up to twenty percent of familial cases. Although the exact mechanism by which mut...

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Bibliographic Details
Main Authors: Lorelei Stoica, Miguel Sena-Esteves
Format: Article
Language:English
Published: Frontiers Media S.A. 2016-08-01
Series:Frontiers in Molecular Neuroscience
Subjects:
ALS
Online Access:http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00056/full