title: Assessment of CFTR function after gene transfer in vitro and in vivo.
abstract: Cystic fibrosis (CF) a monogenic lethal disease and, therefore, ideally suited for the development of gene therapy. The first clinical trials were carried out shortly after cloning the CF gene in 1989. Since then, 25 trials have been carried out. Proof of principle for low-level airway gene transfer was established in most, but not all, trials. It is currently unclear whether current gene transfer efficiency will lead to improvements in clinically relevant endpoints such as inflammation or infection. In addition to addressing this important question, we and others are further improving airway gene transfer, by modifying existing and developing new gene transfer agents. Here, we describe pre-clinical methods related to assessing correction of the CF chloride transport defect.
authors.affiliation: Department of Gene Therapy, Faculty of Medicine, Imperial College London,UK.
authors.forename: Uta
authors.initials: U
authors.lastname: Griesenbach
authors.forename: Felix M
authors.initials: FM
authors.lastname: Munkonge
authors.forename: Stephanie
authors.initials: S
authors.lastname: Sumner-Jones
authors.forename: Emma
authors.initials: E
authors.lastname: Holder
authors.forename: Stephen N
authors.initials: SN
authors.lastname: Smith
authors.forename: A Christopher
authors.initials: AC
authors.lastname: Boyd
authors.forename: Deborah R
authors.initials: DR
authors.lastname: Gill
authors.forename: Stephen C
authors.initials: SC
authors.lastname: Hyde
authors.forename: David
authors.initials: D
authors.lastname: Porteous
authors.forename: Eric W F W
authors.initials: EW
authors.lastname: Alton
mesh_terms: D015056:1-Methyl-3-isobutylxanthine / Q000494:pharmacology; D000818:Animals; D002460:Cell Line; D002469:Cell Separation; D005576:Colforsin / Q000494:pharmacology; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000378:metabolism*; D004847:Epithelial Cells / Q000166:cytology / Q000187:drug effects; D006801:Humans; D017136:Ion Transport / Q000187:drug effects; D008156:Luciferases / Q000378:metabolism; D051379:Mice; D012137:Respiratory System / Q000166:cytology; D014162:Transfection / Q000379:methods*
chemical_list: D019005:Cystic Fibrosis Transmembrane Conductance Regulator; D005576:Colforsin; D008156:Luciferases; D015056:1-Methyl-3-isobutylxanthine