CFTR: New insights into structure and function and implications for modulation by small molecules
Publication date
2019-11-20
Authors
Kleizen, Bertrand
Hunt, John F.
Callebaut, Isabelle
Hwang, Tzyh Chang
Sermet-Gaudelus, Isabelle
Hafkemeyer, Sylvia
Sheppard, David N.
Editors
Advisors
Supervisors
Document Type
Article
Metadata
Show full item recordCollections
License
taverne
Abstract
Structural biology and functional studies are a powerful combination to elucidate fundamental knowledge about the cystic fibrosis transmembrane conductance regulator (CFTR). Here, we discuss the latest findings, including how clinically-approved drugs restore function to mutant CFTR, leading to better clinical outcomes for people with cystic fibrosis (CF). Despite the prospect of regulatory approval of a CFTR-targeting therapy for most CF mutations, strenuous efforts are still needed to fully comprehend CFTR structure-and-function for the development of better drugs to enable people with CF to live full and active lives.
Keywords
ABC-transporter, Clinical drugs, Cystic fibrosis, F508del, Membrane protein, Mutations, Therapy, Taverne, Pediatrics, Perinatology, and Child Health, Pulmonary and Respiratory Medicine, SDG 3 - Good Health and Well-being
Citation
Kleizen, B, Hunt, J F, Callebaut, I, Hwang, T C, Sermet-Gaudelus, I, Hafkemeyer, S & Sheppard, D N 2019, 'CFTR : New insights into structure and function and implications for modulation by small molecules', Journal of Cystic Fibrosis, vol. 19, no. suppl. 1, pp. s19-s24. https://doi.org/10.1016/j.jcf.2019.10.021