Antisense oligonucleotide eluforsen improves CFTR function in F508del cystic fibrosis

BACKGROUND: Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. In this study we assessed the effect of antisense oligonucleotide eluforsen on CFTR biological activity measured by Nasal Potential Difference (NPD) in patients with the most common mut...

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Detalhes bibliográficos
Publicado no:J Cyst Fibros
Main Authors: Sermet-Gaudelus, Isabelle, Clancy, John P., Nichols, David P., Nick, Jerry A., De Boeck, Kris, Solomon, George M., Mall, Marcus A., Bolognese, James, Bouisset, Florilene, den Hollander, Wilhelmina, Paquette-Lamontagne, Nicolas, Tomkinson, Nigel, Henig, Noreen, Elborn, J. Stuart, Rowe, Steven M.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7227803/
https://ncbi.nlm.nih.gov/pubmed/30467074
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jcf.2018.10.015
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