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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Bibliografske podrobnosti
izdano v:Mol Ther Nucleic Acids
Main Authors: 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
Format: Artigo
Jezik:Inglês
Izdano: American Society of Gene & Cell Therapy 2018
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Article
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6023790/
https://ncbi.nlm.nih.gov/pubmed/30195799
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.04.015
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