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
Rapid identification of novel functional promoters for gene therapy.
Pringle IA et al., J Mol Med (Berl). 2012 Dec;90(12):1487-96. doi: 10.1007/s00109-012-0928-6. Epub 2012 Jul 6.
CpG-free plasmid expression cassettes for cystic fibrosis gene therapy.
Pringle IA et al., Biomaterials. 2012 Oct;33(28):6833-42. doi: 10.1016/j.biomaterials.2012.06.009. Epub 2012 Jun 22.
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.
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
Alton EW et al., In Press
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)
Duration of Expression from CpG-Free Plasmids Following Hydrodynamic Delivery to the Mouse.
Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
Clinical Development of an Optimal F/HN Pseudotyped SIV Vector for Cystic Fibrosis Lung Gene Therapy.
Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014)
Calculating the percentage of cells transfected following non-viral delivery to the respiratory epithelium.
Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2009)
Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease.
Pringle IA et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
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)
Development, Production and Evaluation of clinical grade CFTR Expression Plasmid for CF Lung Gene Therapy
Gill DR et al.,The North American Cystic Fibrosis Conference (2010)
Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
The use of Lentiviral vectors to treat airway disease.
Harding-Smith RE et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
Bacterial Lysate (2009): Neutralisation of a bacterial lysate.
A cake that only some of us got to enjoy!
Proposed 3D model of the CFTR protein.