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.
Original language | English |
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Pages (from-to) | s19-s24 |
Journal | Journal of Cystic Fibrosis |
Volume | 19 |
Issue number | suppl. 1 |
DOIs | |
Publication status | Published - 20 Nov 2019 |
Funding
DNS is the recipient of a Vertex Innovation Award, IS-G is the recipient of two Vertex Innovation Awards and a member of the scientific boards of Eloxx, PTC Therapeutics and Vertex Therapeutics. T-CH has an ongoing service agreement with Abbvie and a sponsored research grant from Nanova. We thank the ECFS and the CF patient organizations from Belgium, France, Germany, Italy, the Netherlands and the UK for supporting the PCM. We are very grateful to laboratory colleagues for valuable discussions. Work in the authors’ laboratories was supported by The Dutch Cystic Fibrosis Foundation, NCFS (BK), The Netherlands Organization for Scientific Research (NWO, grant no. 731.017.420) (BK), Cystic Fibrosis Foundation Therapeutics (CFTR 3D Structure Consortium, J Frank, J Kappes and JFH), The French Association Vaincre La Mucoviscidose (IC), GENCI-[CINES] (grant no. 2018-A0040707206 and 2019-A0060707206) (IC), The National Institutes of Health (grant no. NIHR01DK55835 ) (T-CH), The French Association Vaincre La Mucoviscidose, Cystic Fibrosis Foundation, Agence Nationale pour la Recherche, Programme Hospitalier de Recherche Clinique (IS-G). Cystic Fibrosis Foundation Therapeutics grant no. ( SHEPPA14XX0 ) (DNS), Cystic Fibrosis Trust grant no. ( SRC005/SRC011 ) (DNS) and the Medical Research Council grant no. ( MR/S00274X/1 ) (DNS).
Keywords
- ABC-transporter
- Clinical drugs
- Cystic fibrosis
- F508del
- Membrane protein
- Mutations
- Therapy