Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene that impair the function of CFTR, an epithelial chloride channel required for proper function of the lung, pancreas, and other organs. Most patients with CF carry the F508del CFTR mutation, which ca...

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Hauptverfasser: Van Goor, Fredrick, Hadida, Sabine, Grootenhuis, Peter D. J., Burton, Bill, Stack, Jeffrey H., Straley, Kimberly S., Decker, Caroline J., Miller, Mark, McCartney, Jason, Olson, Eric R., Wine, Jeffrey J., Frizzell, Ray A., Ashlock, Melissa, Negulescu, Paul A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: National Academy of Sciences 2011
Schlagworte:
Biological Sciences
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3219147/
https://ncbi.nlm.nih.gov/pubmed/21976485
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1105787108
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