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...

Celý popis

Uloženo v:
Podrobná bibliografie
Vydáno v:J Clin Invest
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society for Clinical Investigation 2018
Témata:
Research Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky