Article
A novel triple combination of pharmacological chaperones improves F508del-CFTR correction.
Scientific reports - 30 Jul 2018
Carlile Graeme W, Yang Qi, Matthes Elizabeth, Liao Jie, Radinovic Stevo, Miyamoto Carol, Robert Renaud, Hanrahan John W, Thomas David Y
Abstract excerpt
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 drugs will be needed to achieve substantial clinical benefit. Here we examine if multiple sites for pharmacological...
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