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
Binding protein-dependent transport systems.
Higgins CF et al., J Bioenerg Biomembr. 1990 Aug;22(4):571-92.
Separation of drug transport and chloride channel functions of the human multidrug resistance P-glycoprotein.
Gill DR et al., Cell. 1992 Oct 2;71(1):23-32.
Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy.
Hyde SC et al., Nature. 1993 Mar 18;362(6417):250-5.
Towards gene therapy for cystic fibrosis: a clinical progress report.
Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2.
Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter.
Gill DR et al., Gene Ther. 2001 Oct;8(20):1539-46.
Chloride secretion in the trachea of null cystic fibrosis mice: the effects of transfection with pTrial10-CFTR2.
MacVinish LJ et al., J Physiol. 1997 Mar 15;499 ( Pt 3):677-87.
Volume-regulated chloride channels associated with the human multidrug-resistance P-glycoprotein.
Valverde MA et al., Nature. 1992 Feb 27;355(6363):830-3.
Drug efflux mediated by the human multidrug resistance P-glycoprotein is inhibited by cell swelling.
Sardini A et al., J Cell Sci. 1994 Dec;107 ( Pt 12):3281-90.
Specific inhibitors distinguish the chloride channel and drug transporter functions associated with the human multidrug resistance P-glycoprotein.
Mintenig GM et al., Receptors Channels. 1993;1(4):305-13.
The multidrug resistance and cystic fibrosis genes have complementary patterns of epithelial expression.
Trezise AE et al., EMBO J. 1992 Dec;11(12):4291-303.
Structural model of ATP-binding proteins associated with cystic fibrosis, multidrug resistance and bacterial transport.
Hyde SC et al., Nature. 1990 Jul 26;346(6282):362-5.
A second dose of a CFTR cDNA-liposome complex is as effective as the first dose in restoring cAMP-dependent chloride secretion to null CF mice trachea.
Goddard CA et al., Gene Ther. 1997 Nov;4(11):1231-6.
Tamoxifen blocks chloride channels. A possible mechanism for cataract formation.
Zhang JJ et al., J Clin Invest. 1994 Oct;94(4):1690-7.
Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis.
Hyde SC et al., Gene Ther. 2000 Jul;7(13):1156-65.
A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.
Gill DR et al., Gene Ther. 1997 Mar;4(3):199-209.
Bacterial Lysate (2009): Neutralisation of a bacterial lysate.
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).
E.coli from a large scale industrial production of our clinical trial plasmid pGM169.