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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 1 (SOD1) are a leading cause of ALS, responsible for up to 20% of familial cases. Although the exact mechanism by which mutant SOD1...

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Pubblicato in:Front Mol Neurosci
Autori principali: Stoica, Lorelei, Sena-Esteves, Miguel
Natura: Artigo
Lingua:Inglês
Pubblicazione: Frontiers Media S.A. 2016
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4969298/
https://ncbi.nlm.nih.gov/pubmed/27531973
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fnmol.2016.00056
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