Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A

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 e...

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Bibliographic Details

Published in:	Cells
Main Authors:	Miquéias Lopes-Pacheco, Mafalda Bacalhau, Sofia S. Ramalho, Iris A. L. Silva, Filipa C. Ferreira, Graeme W. Carlile, David Y. Thomas, Carlos M. Farinha, John W. Hanrahan, Margarida D. Amaral
Format:	Article
Language:	English
Published:	MDPI AG 2022-01-01
Subjects:	cystic fibrosis drug discovery F508del-CFTR genetic revertants intestinal organoids low temperature
Online Access:	https://www.mdpi.com/2073-4409/11/1/136