Article
Cyclic Peptidyl Inhibitors against CAL/CFTR Interaction for Treatment of Cystic Fibrosis.
Journal of medicinal chemistry - 24 Dec 2020
Dougherty Patrick G, Wellmerling Jack H, Koley Amritendu, Lukowski Jessica K, Hummon Amanda B, Cormet-Boyaka Estelle, Pei Dehua
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, encoding for a chloride ion channel. Membrane expression of CFTR is negatively regulated by CFTR-associated ligand (CAL). We previously showed that inhibition of the CFTR/CAL interaction with a cell-permeable peptide improves the function of rescued F508del-CFTR. In this study, optimization of the...
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