The UK Cystic Fibrosis Gene Therapy Consortium
Who are we
Profiles of the Consortium Strategy Group
Frequently Asked Questions
All Trials Registered Initiative
About this Website
Research Assistant in Genome Editing and Gene Therapy (Oxford)
The Cystic Fibrosis Trust
Just Gene Therapy
What is Cystic Fibrosis?
History of Cystic Fibrosis
Discovery of the CFTR Gene
What causes Cystic Fibrosis?
CFTR Protein Structure
Introduction to Gene Therapy
Why use Gene Therapy to Treat CF?
Other CF Gene Therapy Groups
Successful Applications of Gene Therapy
CF Gene Therapy Clinical Trials
GL67A pGM169 - Our first clinical trial product
The Tracking Study
The Run In Study
Single Dose Clinical Trial (2009-2011)
Multi Dose Clinical Trial (2012-2014)
Multi Dose Clinical Trial Protocol
Public Meeting, 30 May 2015
Nonviral Vector Development
Aerosol Delivery of Gene Transfer Vectors
Lentiviral Vector Development
Self-reactive CFTR T cells in humans: implications for gene therapy.
Calcedo R et al., Hum Gene Ther Clin Dev. 2013 Sep;24(3):108-15. doi: 10.1089/humc.2012.249. Epub 2013 Jul 19.
A randomised, double-blind, placebo-controlled trial of repeated nebulisation of non-viral cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy in patients with cystic fibrosis.
Alton EW et al., Efficacy and Mechanism Evaluation (2016) Volume: 3 Issue: 5
Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.
Alton EW et al., Lancet Respir Med. 2015 Sep;3(9):684-91. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3.
Self-Reactive T Cells to CFTR in Cystic Fibrosis (CF) and Non-CF Humans: Implications for Gene Therapy.
Limberis MP et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
Immune Responses to Single and Repeated Administration of pGM169/GL67A, The UK CF Gene Therapy Consortium Clinical Trials.
Griesenbach U et al.,British Thoracic Society Winter Meeting (2014)
Repeated Administration of the Non-Viral Gene Transfer Agent pGM169/GL67A Does Not Induce Anti-CFTR or Anti-Plasmid Immunoresponses.
Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
Bacterial Lysate (2009): Neutralisation of a bacterial lysate.
Mouse lung large airway (cell nuclei blue) transduced with an adenoviral vector (green).
Sheep lung parenchyma (cell nuclei blue) transduced with an adenoviral vector (green).