title: A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis.
abstract: The UK Cystic Fibrosis Gene Therapy Consortium has been working towards clinical gene therapy for patients with cystic fibrosis for several years. We have recently embarked on a large, multi-dose clinical trial of a non-viral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol.
authors.affiliation: UK CF Gene Therapy Consortium, , Edinburgh, , London, Oxford, UK.
authors.forename: Eric W F W
authors.initials: EW
authors.lastname: Alton
authors.forename: A Christopher
authors.initials: AC
authors.lastname: Boyd
authors.forename: Seng H
authors.initials: SH
authors.lastname: Cheng
authors.forename: Steve
authors.initials: S
authors.lastname: Cunningham
authors.forename: Jane C
authors.initials: JC
authors.lastname: Davies
authors.forename: Deborah R
authors.initials: DR
authors.lastname: Gill
authors.forename: Uta
authors.initials: U
authors.lastname: Griesenbach
authors.forename: Tracy
authors.initials: T
authors.lastname: Higgins
authors.forename: Stephen C
authors.initials: SC
authors.lastname: Hyde
authors.forename: J Alastair
authors.initials: JA
authors.lastname: Innes
authors.forename: Gordon D
authors.initials: GD
authors.lastname: Murray
authors.forename: David J
authors.initials: DJ
authors.lastname: Porteous
mesh_terms: D003550:Cystic Fibrosis / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000627:therapeutic use*; D004311:Double-Blind Method; D005500:Follow-Up Studies; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D011446:Prospective Studies; D016896:Treatment Outcome
chemical_list: D019005:Cystic Fibrosis Transmembrane Conductance Regulator
keywords: Cystic Fibrosis