New therapies in cystic fibrosis.
Cystic fibrosis is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that lead to abnormalities in transepithelial ion transport in the airways of affected patients. Lung disease is the major contributor to morbidity and mortality in patients with cystic fibrosis but recommended therapeutic interventions so far have focussed on symptom control rather than treatment of the underlying causes of the disease. New therapies that are currently in pre-clinical and clinical testing include CFTR pharmacotherapy, drugs targeting other ion channels, and hydrators of the cystic fibrosis airways. The current status of these and other new developments in the treatment of cystic fibrosis are reviewed.
Division of Respiratory Medicine, Hospital for Sick Children, 555 University Avenue, Toronto, Ontario M5G 1X8, Canada. felix.ratjen@sickkids.ca
Felix
F
Ratjen
Hartmut
H
Grasemann
D000818:Animals; D003550:Cystic Fibrosis / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics* / Q000378:metabolism; D015507:Drugs, Investigational / Q000627:therapeutic use*; D015316:Genetic Therapy; D006801:Humans; D017136:Ion Transport / Q000187:drug effects; D009154:Mutation / Q000235:genetics*
D015507:Drugs, Investigational; D019005:Cystic Fibrosis Transmembrane Conductance Regulator