title: Genetic therapies for cystic fibrosis lung disease.
abstract: The aim of gene therapy for cystic fibrosis (CF) lung disease is to efficiently and safely express the CF transmembrane conductance regulator (CFTR) in the appropriate pulmonary cell types. Although CF patients experience multi-organ disease, the chronic bacterial lung infections and associated inflammation are the primary cause of shortened life expectancy. Gene transfer-based therapeutic approaches are feasible, in part, because the airway epithelium is directly accessible by aerosol delivery or instillation. Improvements in standard delivery vectors and the development of novel vectors, as well as emerging technologies and new animal models, are propelling exciting new research forward. Here, we review recent developments that are advancing this field of investigation.
authors.affiliation: Program in Gene Therapy, Department of Pediatrics, Carver College of Medicine, The University of Iowa, Iowa City, IA 52242, USA.
authors.forename: Patrick L
authors.initials: PL
authors.lastname: Sinn
authors.forename: Reshma M
authors.initials: RM
authors.lastname: Anthony
authors.forename: Paul B
authors.initials: PB
authors.lastname: McCray
mesh_terms: D003550:Cystic Fibrosis / Q000235:genetics / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D004848:Epithelium / Q000378:metabolism; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage / Q000235:genetics*; D006801:Humans; D007249:Inflammation / Q000378:metabolism; D008168:Lung / Q000378:metabolism
chemical_list: D019005:Cystic Fibrosis Transmembrane Conductance Regulator