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  • A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis

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Chest clinic
Audit, research and guideline update
A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
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  1. Eric W F W Alton1,2,3,
  2. A Christopher Boyd1,4,
  3. Seng H Cheng5,
  4. Steve Cunningham1,6,
  5. Jane C Davies1,2,7,
  6. Deborah R Gill1,8,
  7. Uta Griesenbach1,2,
  8. Tracy Higgins1,2,
  9. Stephen C Hyde1,8,
  10. J Alastair Innes1,4,
  11. Gordon D Murray9,
  12. David J Porteous1,4
  1. 1UK CF Gene Therapy Consortium, Edinburgh, London, Oxford, UK
  2. 2Department of Gene Therapy, Imperial College London, London, UK
  3. 3Department of Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK
  4. 4Medical Genetics Section, MRC Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK
  5. 5Department of Rare Diseases Science, Genzyme, a Sanofi Company, Framingham, Massachusetts, USA
  6. 6Department of Respiratory and Sleep Medicine, Royal Hospital for Sick Children, Edinburgh, UK
  7. 7Department of Paediatric Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK
  8. 8Gene Medicine Group, Nuffield Division of Clinical Laboratory Sciences, University of Oxford, Oxford, UK
  9. 9Centre for Population Health Sciences, University of Edinburgh, Edinburgh, UK
  1. Correspondence to Dr Jane C Davies, Department of Gene Therapy, National Heart and Lung Institute, Imperial College, Emmanuel Kaye Building, 1b Manresa Road, London SW3 6LR, UK; j.c.davies{at}imperial.ac.uk

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.

  • Cystic Fibrosis

https://doi.org/10.1136/thoraxjnl-2013-203309

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