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
Bacterial DNA is implicated in the inflammatory response to delivery of DNA/DOTAP to mouse lungs.
McLachlan G et al., Gene Ther. 2000 Mar;7(5):384-92.
Plasmid DNA molecules complexed with cationic liposomes are protected from degradation by nucleases and shearing by aerosolisation.
Crook K et al., Gene Ther. 1996 Sep;3(9):834-9.
Evaluation in vitro and in vivo of cationic liposome-expression construct complexes for cystic fibrosis gene therapy.
McLachlan G et al., Gene Ther. 1995 Nov;2(9):614-22.
Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
Caplen NJ et al., Nat Med. 1995 Jan;1(1):39-46.
Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
Porteous DJ et al., Gene Ther. 1997 Mar;4(3):210-8.
Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP.
McLachlan G et al., Gene Ther. 1996 Dec;3(12):1113-23.
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
Alton EW et al., Thorax. 2017 Feb;72(2):137-147. doi: 10.1136/thoraxjnl-2016-208406.
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.
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
Moving lentiviral-based gene therapy into a first-in-man CF trial.
Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
Lentivirus Animation (2011): Basic description of Lentivirus Pseudotyping.
Large scale lentivirus production in suspension culture.
A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)