Molecular dynamics and theratyping in airway and gut organoids reveal R352Q-CFTR conductance defect
Sharon L. Wong, Nikhil T. Awatade, Miro A. Astore, Katelin M. Allan, Michael J. Carnell, Iveta Šlapetová, Po‐Chia Chen, Jeffry Setiadi, Elvis Pandzic, Laura K. Fawcett, John R. Widger, Renee M. Whan, Renate Griffith, Chee Y. Ooi, Serdar Kuyucak, , Shafagh A. Waters
The paper characterizes the functional and structural defects of the R352Q CFTR mutation using patient-derived organoids and molecular dynamics simulations, showing residual function restored by potentiators but not correctors.
American Journal of Respiratory Cell and Molecular Biology · Apr 26, 2022Peer-reviewedMolecular Biology