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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Detalles Bibliográficos
Autores principales: 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: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7227803/
https://www.ncbi.nlm.nih.gov/pubmed/30467074
http://dx.doi.org/10.1016/j.jcf.2018.10.015

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