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...
Main Authors: | , |
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Format: | Article |
Language: | English |
Published: |
Frontiers Media S.A.
2016-08-01
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Series: | Frontiers in Molecular Neuroscience |
Subjects: | |
Online Access: | http://journal.frontiersin.org/Journal/10.3389/fnmol.2016.00056/full |