A novel triple combination of pharmacological chaperones improves F508del-CFTR correction

Pharmacological chaperones (e.g. VX-809, lumacaftor) that bind directly to F508del-CFTR and correct its mislocalization are promising therapeutics for Cystic Fibrosis (CF). However to date, individual correctors provide only ~4% improvement in lung function measured as FEV1, suggesting that multiple...

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Bibliografiske detaljer
Udgivet i:Sci Rep
Main Authors: Carlile, Graeme W., Yang, Qi, Matthes, Elizabeth, Liao, Jie, Radinovic, Stevo, Miyamoto, Carol, Robert, Renaud, Hanrahan, John W., Thomas, David Y.
Format: Artigo
Sprog:Inglês
Udgivet: Nature Publishing Group UK 2018
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6065411/
https://ncbi.nlm.nih.gov/pubmed/30061653
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-018-29276-y
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