Rapid cystic fibrosis lung-function decline and in-vitro CFTR modulation

New treatments targeting the underlying defect in cystic fibrosis (CF) have dramatically improved outcomes for select cystic fibrosis transmembrane conductance regulator (CFTR) gene mutations. Highly effective modulators are available for ivacaftor-responsive variants (10-15% of CF patients) [1] and/or a copy of the most common CFTR variant, F508del (up to 85%) [2,3]. In-vitro CFTR function quantification and modulation using human nasal epithelial (HNE) cells indicate clear and direct relationships between in-vitro and short-term in-vivo responses (e.g.: CFTR-dependent chloride transport, mucociliary clearance, lung function, sweat chloride concentration) [4 –6].

Source: Journal of Cystic Fibrosis - May 3, 2021 Category: Respiratory Medicine Authors: Emrah Gecili, Weiji Su, Cole Brokamp, Eleni-Rosalina Andrinopoulou, Francis J. LaRosa III, Teresa Pestian, John P. Clancy, George M. Solomon, John J. Brewington, Rhonda D. Szczesniak Tags: Letter to the Editor Source Type: research