Correctors and Potentiators Rescue Function of the Truncated W1282X-Cystic Fibrosis Transmembrane Regulator (CFTR) Translation Product

W1282X is the fifth most common cystic fibrosis transmembrane regulator (CFTR) mutation that causes cystic fibrosis. Here, we investigated the utility of a small molecule corrector/potentiator strategy, as used for ΔF508-CFTR, to produce functional rescue of the truncated translation product of the...

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Veröffentlicht in:J Biol Chem
Hauptverfasser: Haggie, Peter M., Phuan, Puay-Wah, Tan, Joseph-Anthony, Xu, Haijin, Avramescu, Radu G., Perdomo, Doranda, Zlock, Lorna, Nielson, Dennis W., Finkbeiner, Walter E., Lukacs, Gergely L., Verkman, Alan S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Biochemistry and Molecular Biology 2017
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Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5247652/
https://ncbi.nlm.nih.gov/pubmed/27895116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.M116.764720
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