Therapeutic AAV9-mediated Suppression of Mutant SOD1 Slows Disease Progression and Extends Survival in Models of Inherited ALS

Mutations in superoxide dismutase 1 (SOD1) are linked to familial amyotrophic lateral sclerosis (ALS) resulting in progressive motor neuron death through one or more acquired toxicities. Involvement of wild-type SOD1 has been linked to sporadic ALS, as misfolded SOD1 has been reported in affected ti...

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Hlavní autoři: Foust, Kevin D, Salazar, Desirée L, Likhite, Shibi, Ferraiuolo, Laura, Ditsworth, Dara, Ilieva, Hristelina, Meyer, Kathrin, Schmelzer, Leah, Braun, Lyndsey, Cleveland, Don W, Kaspar, Brian K
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2013
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3863799/
https://ncbi.nlm.nih.gov/pubmed/24008656
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.211
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