A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model

One of the most promising therapeutic approaches for familial amyotrophic lateral sclerosis linked to superoxide dismutase 1 (SOD1) is the suppression of toxic mutant SOD1 in the affected tissues. Here, we report an innovative molecular strategy for inducing substantial, widespread, and sustained re...

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Publicat a:Mol Ther
Autors principals: Biferi, Maria Grazia, Cohen-Tannoudji, Mathilde, Cappelletto, Ambra, Giroux, Benoit, Roda, Marianne, Astord, Stéphanie, Marais, Thibaut, Bos, Corinne, Voit, Thomas, Ferry, Arnaud, Barkats, Martine
Format: Artigo
Idioma:Inglês
Publicat: American Society of Gene & Cell Therapy 2017
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5589057/
https://ncbi.nlm.nih.gov/pubmed/28663100
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.05.017
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