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The UK Cystic Fibrosis Gene Therapy Consortium
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Flutterby FUNdraisers
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Cystic Fibrosis
CF
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What is Cystic Fibrosis?
History of Cystic Fibrosis
Discovery of the CFTR Gene
What causes Cystic Fibrosis?
CFTR Protein Structure
Gene Therapy
Introduction to Gene Therapy
Why use Gene Therapy to Treat CF?
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Successful Applications of Gene Therapy
CF Gene Therapy Clinical Trials
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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
Research
Nonviral Vector Development
Aerosol Delivery of Gene Transfer Vectors
Assay Development
Lentiviral Vector Development
Lentiviral Production
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Journal Articles
Conference Presentations
Book Chapters
Public Presentations
Papers 1
Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer.
Griesenbach U et al., Biomaterials. 2011 Apr;32(10):2614-24. doi: 10.1016/j.biomaterials.2010.12.001. Epub 2011 Jan 15.
Abstracts 1
Secreted Gaussia Luciferase Is a More Sensitive Reporter Than Firefly Luciferase for Non- Viral Gene Transfer to Airway Epithelium Ex Vivo and In Vivo.
Griesenbach U et al.,The American Society of Gene Therapy Annual Conference (2009)
Aerosol Gene Delivery. (2010): An Aerotech II nebuliser generating a PEI/pDNA aerosol.
More...
A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)