Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770

Cystic fibrosis (CF) is a lethal recessive genetic disease caused primarily by the F508del mutation in the CF transmembrane conductance regulator (CFTR). The potentiator VX-770 was the first CFTR modulator approved by the FDA for treatment of CF patients with the gating mutation G551D. Orkambi is a...

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Vydáno v:Am J Physiol Lung Cell Mol Physiol
Hlavní autoři: Gentzsch, Martina, Ren, Hong Y., Houck, Scott A., Quinney, Nancy L., Cholon, Deborah M., Sopha, Pattarawut, Chaudhry, Imron G., Das, Jhuma, Dokholyan, Nikolay V., Randell, Scott H., Cyr, Douglas M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Physiological Society 2016
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC5142211/
https://ncbi.nlm.nih.gov/pubmed/27402691
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1152/ajplung.00186.2016
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