title: Use of nonviral vectors for cystic fibrosis gene therapy.
abstract: Over the last decade, three groups within the United Kingdom (Edinburgh, Oxford, and Imperial College, London) have undertaken key studies in the development of clinical gene therapy for cystic fibrosis. In 2001, catalyzed by the Cystic Fibrosis Trust, these groups came together to form the United Kingdom Cystic Fibrosis Gene Therapy Consortium. The Consortium has removed duplication and competition, developed core facilities playing to the respective strengths of the centers, and introduced the joint strategy described in this article. This is driven by a clinical trial program, with a product pipeline and the necessary development of novel preclinical and human assays. The program is milestone-related, has a structure that lies between the pharmaceutical industry and academia, and has as its endpoint negotiations with industry to undertake a phase III clinical trial of the identified product.
authors.affiliation: Imperial College London, National Heart & Lung Institute, Department of Gene Therapy, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK. e.alton@imperial.ac.uk
authors.forename: Eric W F W
authors.initials: EW
authors.lastname: Alton
mesh_terms: D002986:Clinical Trials as Topic; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics / Q000627:therapeutic use*; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans; D008297:Male; D011379:Prognosis; D018570:Risk Assessment; D012680:Sensitivity and Specificity; D016896:Treatment Outcome; D006113:United Kingdom
chemical_list: C505032:CFTR protein, human; D019005:Cystic Fibrosis Transmembrane Conductance Regulator