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
The significance of plasmid DNA preparations contaminated with bacterial genomic DNA on inflammatory responses following delivery of lipoplexes to the murine lung.
Bazzani RP et al., Biomaterials. 2011 Dec;32(36):9854-65. doi: 10.1016/j.biomaterials.2011.08.092. Epub 2011 Sep 23.
Transgene sequences free of CG dinucleotides lead to high level, long-term expression in the lung independent of plasmid backbone design.
Bazzani RP et al., 2016 Jul;93:20-6.
Strategies for long-term expression of transgenes in the respiratory epithelium.
Gill DR et al., Curr Opin Mol Ther. 2010 Aug;12(4):386-93.
The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes.
Davies LA et al., Biomaterials. 2012 Aug;33(22):5618-27. doi: 10.1016/j.biomaterials.2012.04.019. Epub 2012 May 8.
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
Hyde SC et al., Nat Biotechnol. 2008 May;26(5):549-51. doi: 10.1038/nbt1399. Epub 2008 Apr 27.
CpG-Dependent Inflammatory Response after Delivery of Lipid/pDNA Complexes to Murine Lungs.
Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2007)
The Effect of pDNA Quality on Gene Transfer Outcome In Vivo.
Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2009)
Persistent gene expression in the murine lung is dependent on transgene CpG content.
Oliveira C et al.,3rd Minicircle & DNA Vector Conference. (2014)
Minicircles Are Similar To Plasmids In Providing High Level, Long-Term Expression In The Lung .
Gill DR et al.,The American Society of Gene and Cell Therapy Annual Conference (2016)
Repeated Exposure to pDNA/PEI Aerosols Results in Minimal Detectable Toxicity in the Mouse Lungs .
Nunez-Alonso GA et al.,The American Society of Gene Therapy Annual Conference (2009)
Genomic DNA Reduction for Therapeutic DNA Manufacture.
Cai Y et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
Lentivirus Animation (2011): Basic description of Lentivirus Pseudotyping.
A CFTR Western blot, to confirm protein production in cell culture.
Proposed 3D model of the CFTR protein.