rss
Arch Dis Child 99:465-468 doi:10.1136/archdischild-2012-302158
  • Review

Gene therapy in cystic fibrosis

  1. Eric W FW Alton1,3,5
  1. 1UK CF Gene Therapy Consortium
  2. 2Department of Respiratory and Sleep Medicine, Royal Hospital for Sick Children, Edinburgh, UK
  3. 3Department of Gene Therapy, Imperial College London, London, UK
  4. 4Department of Paediatric Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK
  5. 5Department of Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK
  1. Correspondence to Dr David Armstrong, Department of Respiratory and Sleep Medicine, Royal Hospital for Sick Children, 9 Sciennes Road, Edinburgh, EH9 1LF, UK; david.armstrong{at}ed.ac.uk
  • Received 25 September 2013
  • Revised 16 December 2013
  • Accepted 30 December 2013
  • Published Online First 24 January 2014

Abstract

The principal cause of morbidity and mortality in cystic fibrosis (CF) is pulmonary disease, so the focus of new treatments in this condition is primarily targeted at the lungs. Since the cloning of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene in 1989, there has been significant interest in the possibility of gene therapy as a treatment for CF. Early studies using viral vectors carrying a healthy CFTR plasmid highlighted the difficulties with overcoming the body's host defences. This article reviews the work on gene therapy in CF to date and describes the ongoing work of the UK CF Gene Therapy Consortium in investigating the potential of gene therapy as a treatment for patients with CF.