title: A combination therapy for cystic fibrosis.
abstract: The most prevalent form of cystic fibrosis arises from an amino acid deletion in the cystic fibrosis transmembrane conductance regulator, CFTR. A recently approved treatment for individuals homozygous for this mutation combines a chemical corrector, which helps CFTR fold, and a potentiator that increases CFTR channel activity.
authors.affiliation: Department of Biological Sciences, University of Pittsburgh, Pittsburgh, PA 15260, USA. Electronic address: jbrodsky@pitt.edu.
authors.forename: Jeffrey L
authors.initials: JL
authors.lastname: Brodsky
authors.affiliation: Department of Cell Biology, University of Pittsburgh School of Medicine, Pittsburgh, PA 15261, USA.
authors.forename: Raymond A
authors.initials: RA
authors.lastname: Frizzell
mesh_terms: D000627:Aminophenols / Q000627:therapeutic use*; D000631:Aminopyridines / Q000627:therapeutic use*; D052117:Benzodioxoles / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics / Q000266:history; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000378:metabolism; D004338:Drug Combinations; D017353:Gene Deletion; D049673:History, 20th Century; D049674:History, 21st Century; D006801:Humans; D015363:Quinolones / Q000627:therapeutic use*
chemical_list: D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; D004338:Drug Combinations; D015363:Quinolones; C000599212:lumacaftor, ivacaftor drug combination; D019005:Cystic Fibrosis Transmembrane Conductance Regulator