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
Postdoctoral Research Assistant in Gene Therapy
Research Assistant in Gene Therapy
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
Elimination of contaminant Escherichia coli chromosomal DNA from preparations of P1 artificial chromosome recombinants facilitates directed subcloning.
Davidson H et al., Electrophoresis. 1999 Jun;20(7):1469-75.
Genomic sequence analysis of Fugu rubripes CFTR and flanking genes in a 60 kb region conserving synteny with 800 kb of human chromosome 7.
Davidson H et al., Genome Res. 2000 Aug;10(8):1194-203.
Chimeric constructs endow the human CFTR Cl- channel with the gating behavior of murine CFTR.
Scott-Ward TS et al., Proc Natl Acad Sci U S A. 2007 Oct 9;104(41):16365-70. Epub 2007 Oct 3.
Human-specific cystic fibrosis transmembrane conductance regulator antibodies detect in vivo gene transfer to ovine airways.
Davidson H et al., Am J Respir Cell Mol Biol. 2006 Jul;35(1):72-83. Epub 2006 Feb 23.
Insertion of natural intron 6a-6b into a human cDNA-derived gene therapy vector for cystic fibrosis improves plasmid stability and permits facile RNA/DNA discrimination.
Boyd AC et al., J Gene Med. 1999 Sep-Oct;1(5):312-21.
An immunocytochemical assay to detect human CFTR expression following gene transfer.
Davidson H et al., Mol Cell Probes. 2009 Dec;23(6):272-80. doi: 10.1016/j.mcp.2009.07.001. Epub 2009 Jul 15.
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
Alton EW et al., In Press
Limitations of the murine nose in the development of nonviral airway gene transfer.
Griesenbach U et al., Am J Respir Cell Mol Biol. 2010 Jul;43(1):46-54. doi: 10.1165/rcmb.2009-0075OC. Epub 2009 Jul 31.
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung.
McLachlan G et al., Gene Ther. 2011 Oct;18(10):996-1005. doi: 10.1038/gt.2011.55. Epub 2011 Apr 21.
Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation.
Horsley AR et al., Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.
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.
Non-viral gene expression in the lung using the mini-CFTR promoter.
Connolly MM et al.,The British Society of Gene Therapy Annual Conference (2009)
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.
A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)
Pellets of DNA following precipitation.
DNA fragments being cut from an agarose gel exposed to UV.