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Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease; survival in ALS is typically 3–5 years. No treatment extends patient survival by more than three months. Approximately 20% of familial ALS and 1–3% of sporadic ALS patients carry a mutation in the gene encoding superoxide dism...

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Publicat a:Hum Gene Ther
Autors principals: Borel, Florie, Gernoux, Gwladys, Cardozo, Brynn, Metterville, Jake P., Toro Cabreja, Gabriela C., Song, Lina, Su, Qin, Gao, Guang Ping, Elmallah, Mai K., Brown, Robert H., Mueller, Christian
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc. 2016
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4741242/
https://ncbi.nlm.nih.gov/pubmed/26710998
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.122
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