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The UK Cystic Fibrosis Gene Therapy Consortium
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What is Cystic Fibrosis?
History of Cystic Fibrosis
Discovery of the CFTR Gene
What causes Cystic Fibrosis?
CFTR Protein Structure
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Why use Gene Therapy to Treat CF?
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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
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Nonviral Vector Development
Aerosol Delivery of Gene Transfer Vectors
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Lentiviral Production
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Journal Articles
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Public Presentations
Papers 3
Gene therapy in cystic fibrosis.
Armstrong DK et al., Arch Dis Child. 2014 May;99(5):465-8. doi: 10.1136/archdischild-2012-302158. Epub 2014 Jan 24.
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
Abstracts 2
Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
Repeated Administration of the Non-Viral Gene Transfer Agent pGM169/GL67A Does Not Induce Anti-CFTR or Anti-Plasmid Immunoresponses.
Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
Bacterial Lysate (2009): Neutralisation of a bacterial lysate.
More...
E.coli from a large scale industrial production of our clinical trial plasmid pGM169.