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
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
Optimizing aerosol gene delivery and expression in the ovine lung.
McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes.
Davies LA et al., Mol Ther. 2008 Jul;16(7):1283-90. doi: 10.1038/mt.2008.96. Epub 2008 May 20.
Validation of recombinant Sendai virus in a non-natural host model.
Griesenbach U et al., Gene Ther. 2011 Feb;18(2):182-8. doi: 10.1038/gt.2010.131. Epub 2010 Oct 21.
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.
Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer.
Griesenbach U et al., Biomaterials. 2011 Apr;32(10):2614-24. doi: 10.1016/j.biomaterials.2010.12.001. Epub 2011 Jan 15.
The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep.
Alton EW et al., Biomaterials. 2013 Dec;34(38):10267-77. doi: 10.1016/j.biomaterials.2013.09.023. Epub 2013 Oct 3.
Optimising Harvest of Bronchial Brush Biopsy Samples To Maximise Cell and RNA Yield in Gene Therapy Studies.
Vrettou C et al.,The American Society of Gene Therapy Annual Conference (2009)
Persistent Gene Expression in the Ovine Lung from a Human Elongation Factor 1 Alpha Promoter Plasmid Following Non-Viral Gene Delivery.
McLachlan G et al.,The American Society of Gene Therapy Annual Conference (2007)
3-Step TaqMan RT-PCR: Ultimate mRNA Detection Sensitivity For CF Gene Therapy Clinical Trials.
McCormick D et al.,The North American Cystic Fibrosis Conference (2009)
Repeat Aerosol Delivery of Concentrated PEI/pDNA to the Sheep Lung.
McLachlan G et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
Quantifying non-viral gene transfer by real-time PCR: a cautionary tale.
Coles R et al.,The European Society of Gene and Cell Therapy Conference (2009)
Optimisation of molecular assays for clinical trial of GL67A/pGM169 delivery to nose and lung of CF patients.
Sumner-Jones SG et al.,The North American Cystic Fibrosis Conference (2009)
Secreted Gaussia Luciferase Is a More Sensitive Reporter Than Firefly Luciferase for Non- Viral Gene Transfer to Airway Epithelium Ex Vivo and In Vivo.
Griesenbach U et al.,The American Society of Gene Therapy Annual Conference (2009)
Lentivirus Animation (2011): Basic description of Lentivirus Pseudotyping.
Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.
Mouse lung large airway (cell nuclei blue) transduced with an adenoviral vector (green).
DNA fragments being cut from an agarose gel exposed to UV.