title: Gene therapy for cystic fibrosis.
abstract: Cystic fibrosis (CF) is associated with significant morbidity and mortality, despite significant advances in conventional treatment. The field of gene therapy has progressed rapidly since the cystic fibrosis transmembrane conductance regulator (CFTR) gene was cloned. In this review we discuss current knowledge on the underlying molecular defect in CF, and the progress in gene transfer studies from the early in vitro work through to clinical trials, including the development of endpoints to assess efficacy. We highlight the problems encountered, and likely future directions of the field.
authors.affiliation: Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK. j.c.davies@ic.ac.uk
authors.forename: J C
authors.initials: JC
authors.lastname: Davies
authors.forename: D M
authors.initials: DM
authors.lastname: Geddes
authors.forename: E W
authors.initials: EW
authors.lastname: Alton
mesh_terms: D000256:Adenoviridae / Q000235:genetics; D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics*; D016503:Drug Delivery Systems / Q000379:methods; D015316:Genetic Therapy; D006801:Humans; D008081:Liposomes
chemical_list: C505032:CFTR protein, human; D008081:Liposomes; D019005:Cystic Fibrosis Transmembrane Conductance Regulator