title: Cystic fibrosis--what are the prospects for a cure?
abstract: Significant improvements in the treatment of cystic fibrosis over the last few decades have altered this lethal disease in children to a multisystem disorder with survival into adult life now common. In most developed countries the numbers of adult cystic fibrosis patients outnumber children. This is mainly due to improvements in care during early life. The principal cause of morbidity and mortality is pulmonary disease, and so the focus of new treatments has targeted the lungs. Identification of the underlying gene defect in the cystic fibrosis transmembrane conductance regulator has ushered in a new era in cystic fibrosis research, with prospects of a cure. In this article, we review the most exciting recent advances that correct defects in cellular processing, chloride channel function and gene therapy.
authors.forename: Shankar
authors.initials: S
authors.lastname: Kumar
authors.forename: Anand
authors.initials: A
authors.lastname: Tana
authors.forename: Anu
authors.initials: A
authors.lastname: Shankar
mesh_terms: D000328:Adult; D000627:Aminophenols / Q000627:therapeutic use; D000631:Aminopyridines / Q000627:therapeutic use; D052117:Benzodioxoles / Q000627:therapeutic use; D002648:Child; D003550:Cystic Fibrosis / Q000188:drug therapy / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D015363:Quinolones / Q000627:therapeutic use
chemical_list: D000627:Aminophenols; D000631:Aminopyridines; D052117:Benzodioxoles; D015363:Quinolones; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C545203:ivacaftor; C569105:lumacaftor