Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

Mutations in superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS. Given the gain of toxic function in this dominantly inherited disease, lowering SOD1 mRNA and protein is predicted to provide therapeutic benefit. An early generation antisense oligonucleotide (ASO) targeting SOD1 wa...

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Gepubliceerd in:J Clin Invest
Hoofdauteurs: McCampbell, Alex, Cole, Tracy, Wegener, Amy J., Tomassy, Giulio S., Setnicka, Amy, Farley, Brandon J., Schoch, Kathleen M., Hoye, Mariah L., Shabsovich, Mark, Sun, Linhong, Luo, Yi, Zhang, Mingdi, Comfort, Nicole, Wang, Bin, Amacker, Jessica, Thankamony, Sai, Salzman, David W., Cudkowicz, Merit, Graham, Danielle L., Bennett, C. Frank, Kordasiewicz, Holly B., Swayze, Eric E., Miller, Timothy M.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society for Clinical Investigation 2018
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Research Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6063493/
https://ncbi.nlm.nih.gov/pubmed/30010620
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI99081
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