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KM 11060 is a small molecule that corrects the processing of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation carried by 90% of cystic fibrosis patients.1 It increases cell surface expression and restores chloride transport function of F508del-CFTRs in BHK cells at a concentration of 10 μM. KM 11060 restores F508del-CFTR function, as measured by cAMP-stimulated iodide efflux, in CFBE41o- human airway epithelial cells at a concentration of 10 nM. It also restores the secretory response to approximately 51% of wild-type levels ex vivo in ileum isolated from F508del-CFTR mice at a concentration of 20 μM.
WARNING This product is not for human or veterinary use.
1. Structural analog of sildenafil identified as a novel corrector of the F508del-