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Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS

Of familial amyotrophic lateral sclerosis (fALS) cases, 20% are caused by mutations in the gene encoding human cytosolic Cu/Zn superoxide dismutase (hSOD1). Efficient translation of the therapeutic potential of RNAi for the treatment of SOD1-ALS patients requires the development of vectors that are...

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Detalles Bibliográficos
Autores principales:	Iannitti, Tommaso, Scarrott, Joseph M., Likhite, Shibi, Coldicott, Ian R.P., Lewis, Katherine E., Heath, Paul R., Higginbottom, Adrian, Myszczynska, Monika A., Milo, Marta, Hautbergue, Guillaume M., Meyer, Kathrin, Kaspar, Brian K., Ferraiuolo, Laura, Shaw, Pamela J., Azzouz, Mimoun
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2018
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6023790/ https://www.ncbi.nlm.nih.gov/pubmed/30195799 http://dx.doi.org/10.1016/j.omtn.2018.04.015

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