Rescue of Mutant CFTR Trafficking Defect by the Investigational Compound MCG1516A

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
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
Series:Cells
Subjects:
cystic fibrosis
drug discovery
F508del-CFTR
genetic revertants
intestinal organoids
low temperature
Online Access:https://www.mdpi.com/2073-4409/11/1/136

Internet

https://www.mdpi.com/2073-4409/11/1/136

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