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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
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Article
Acesso em linha: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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