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
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.
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.
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.
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.
Lentivirus Animation (2011): Basic description of Lentivirus Pseudotyping.
Purifying mRNA from tissue samples.