Article
Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A.
Cells - 1 Jan 2022
Lopes-Pacheco Miquéias, Bacalhau Mafalda, Ramalho Sofia S, Silva Iris A L, Ferreira Filipa C, Carlile Graeme W, Thomas David Y, Farinha Carlos M, Hanrahan John W, Amaral Margarida D
Abstract excerpt
Although some therapeutic progress has been achieved in developing small molecules that correct F508del-CFTR defects, the mechanism of action (MoA) of these compounds remain poorly elucidated. Here, we investigated the effects and MoA of MCG1516A, a newly developed F508del-CFTR corrector. MCG1516A effects on wild-type (WT) and F508del-CFTR were assessed by immunofluorescence microscopy, and biochemical and...
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