title: New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator.
abstract: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are clinically available personalized medicines approved for some individuals with cystic fibrosis (CF) to target the underlying defect of disease. This review summarizes strategies used to develop CFTR modulators as therapies that improve function and availability of CFTR protein. Lessons learned from dissemination of ivacaftor across the CF population responsive to this therapy and future approaches to predict and monitor treatment response of CFTR modulators are discussed. The goal remains to expand patient-centered and personalized therapy to all patients with CF, ultimately improving life expectancy and quality of life for this disease.
authors.affiliation: Division of Pulmonary and Sleep Medicine, Department of Pediatrics, Seattle Children's Hospital, University of Washington, 4800 Sand Point Way Northeast, M/S OC.7.720, Seattle, WA 98105, USA.
authors.forename: Thida
authors.initials: T
authors.lastname: Ong
authors.affiliation: Department of Pediatrics, Seattle Children's Research Institute, Center for Clinical and Translational Research, University of Washington, 2001 8th Avenue, Suite 400, M/S CW8-5B, Seattle, WA 98121, USA. Electronic address: Bonnie.Ramsey@seattlechildrens.org.
authors.forename: Bonnie W
authors.initials: BW
authors.lastname: Ramsey
mesh_terms: D000627:Aminophenols / Q000009:adverse effects / Q000627:therapeutic use*; D000631:Aminopyridines / Q000009:adverse effects / Q000627:therapeutic use*; D052117:Benzodioxoles / Q000009:adverse effects / Q000627:therapeutic use*; D065101:Chloride Channel Agonists / Q000009:adverse effects / Q000627:therapeutic use*; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects* / Q000235:genetics; D006801:Humans; D058990:Molecular Targeted Therapy / Q000009:adverse effects / Q000379:methods*; D009154:Mutation; D015363:Quinolones / Q000009:adverse effects / Q000627:therapeutic use*
chemical_list: D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; D065101:Chloride Channel Agonists; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor; C569105:lumacaftor
keywords: CFTR modulator; Corrector; Personalized medicine; Potentiator; Therapeutics