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
Cystic Fibrosis Gene Therapy in the UK and elsewhere
Griesenbach U et al., Hum Gene Ther. 2015 May;26(5):266-75.
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
Alton EW et al., In Press
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.
Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus.
Pytel KM et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
Production of FVIII in the lungs.
Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
Pre-existing immunity to human parainfluenza virus (hPIV) does not affect rSIV.F/HN-mediated transduction efficiency.
Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2016)
F/HN Pseudotyped Lentivirus Generates Therapeutically Relevant and Long-Lasting Alpha-1-Antitrypsin Expression in Mouse Lung.
Paul-Smith MC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
Therapeutic levels of alpha-1-antitrypsin following gene therapy with F/HN pseudotyped simian immunodeficiency virus.
Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2015)
Preparation for a First-in-Man Lentivirus Trial in Cystic Fibrosis Patients.
Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2016)
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.
Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.
A CFTR Western blot, to confirm protein production in cell culture.