Cell culture models of non-viral transgene expression are not indicative of in vivo lung outcome. Oliveira, C. A. et al. Molecular Therapy 20 S1 Download
Large-Scale cGMP Manufacture of a Plasmid Vector for Cystic Fibrosis Gene Therapy Clinical Trials (2012). Cai, Y. et al., Molecular Therapy, 20, Abstract 761.
Mutliple Doses of Lipid Mediated Gene Therapy Nebulised to the Mouse Lung Show Robust and Sustained CFTR Expression. Hyde, S. C. et al. Pediatic Pulmonology 46 S Download
Safety & expression of a single dose of lipid-mediated CFTR gene therapy to the upper & lower airways of patients with CF. Davies, J. C. et al. Pediatric Pulmonology 46 S33
Toxicology studies in support of the UK CF Gene Therapy Consortium's multi-dose clinical trial. Griesenbach, U. et al. Pediatric Pulmonology 46 S33
Safety and expression of a single dose of lipidmediated CFTR gene therapy to the upper and lower airways of patients with Cystic Fibrosis. Davies, G. et al. Thorax December 2011 Vol 66 Suppl 4, Abstract T4.
The importance of appropriate reference equations for spirometry: lessons learned from the Cystic Fibrosis gene therapy "Run-In" study. Davies, G. et al. Thorax December 2011 Vol 66 Suppl 4, Abstract P234.
Self-reactive T cells to CFTR in Cystic Fibrosis (CF) and non-CF humans: implications for gene therapy. Limberis, M.P.. et al., Molecular Therapy, 19, Abstract 108.
The nuclear pore dilating agent TCHD increases gene transfer into differentiated airway epithelium ex vivo, but has no effect in vivo. Griesenbach, U. et al., Molecular Therapy, 19, Abstract 165.
Strain-specific differences in pulmonary gene transfer efficiency: relevance for toxicology studies in mice. Legakis, G. et al., Molecular Therapy, 19, Abstract 272.
Knockdown of ENaCα, as a treatment for Cystic Fibrosis lung disease (2011). Harding-Smith, R. E. et al., Molecular Therapy, 19, Abstract 884.
Longitudinal assessment of biomarkers for clinical trials of novel therapeutic agents: the Run-In study. Alton, E. W. F. W. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract S18.
Lung clearance index, FEV1 and CT findings in Cystic Fibrosis: data from the UK CF Gene Therapy Consortium Run-in study. Sheridan, H S. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract S22.
Quantitative biological imaging of plasmid DNA in live human airway epithelial cells following nonviral gene transfer. Singh, C. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract P102.
Inflammatory markers: data from the UK CF Gene Thearpy Consortium Run-in study. Innes, J. A. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract P106.
Pulmonary imaging techniques to identify suitable patients and act as outcome measures in the UK CF Gene Therapy Consortium clinical programme. Davies, J. C. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract P107.
Exercise capacity and physical activity in patients with CF: data from the UK CF Gene Therapy Consortium (UKCFGTC) 'Run-In' study. Saunders, C. J. et al. Thorax December 2010 Vol 65 Suppl 4, Abstract P109.
Longitudinal assessment of biomarkers for clinical trials of novel therapeutic agents; the Run-in Study. Alton, E. W. F. W. et al. Pediatric Pulmonology 33 223
SIV vector pseudotyped with SeV-F/HN envelope proteins produces long lasting expression in the murine lung, is readministrable and transfects human airway models. Griesenbach, U. et al. Pediatric Pulmonology 33 238
Assessment of the nuclear pore dilating agent TCHD in differentiated airway epithelium. Griesenbach, U. et al. Pediatric Pulmonology 33 240
Quantitative biological imaging of plasmid DNA in live human airway epithelial cells following non-viral gene transfer. Singh, C. et al. Pediatric Pulmonology 33 263
Development, Production and Evaluation of clinical grade CFTR Expression Plasmid for CF Lung Gene Therapy. Gill, D. R. et al. Pediatric Pulmonology 33 270
Concentrated PEI Gene Transfer formulations retain biological efficacy following long-term storage at 4˚C. Hyde, S. C. et al. Pediatric Pulmonology 33 Abstract 275
Pulmonary imaging techniques to identify suitable patients and act as outcome measures in the UK CF Gene Therapy Consortium clinical programme. Davies, J. C. et al. Pediatric Pulmonology 33 436
Exercise capacity and physical activity in patients with CF: data from the UK CF Gene Therapy Consortium 'Run-In' Study. Sheridan, H. S. et al. Pediatric Pulmonology 33 450
Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4°C. Davies, L. A. et al. Molecular Therapy 18 S123
Duration of expression from CpG-Free plasmids following hydrodynamic delivery to the mouse. Pringle, I. A. et al. Molecular Therapy 18 S283
Genomic DNA reduction for therapeutic DNA manufacture. Cai, Y. et al. Molecular Therapy 18 S288
SIV vector pseudotyped with SeV-F/HN envelope proteins produces long lasting expression in the murine lung, is readministrable and transfects human airway models. Griesenbach, U. et al. Molecular Therapy 18 S337
Repeat aerosol delivery of concentrated PEI/pDNA to the Sheep Lung. McLachlan, G. et al. Molecular Therapy 18 880
A clinical study to evaluate the safety and efficacy of pGM169/GL67A administered to the nose and lungs of individuals with cystic fibrosis. Hyde, S. C. et al. Molecular Therapy 18 Late Breaking
Non-viral gene expression in the lung using the mini-CFTR promoter. Connolly, M. M. et al. Human Gene Therapy 20 403
Inflammation-Free shRNA Expression Vectors for Cystic Fibrosis Gene Therapy. Lawton, A. E. et al. Human Gene Therapy 20 404
Successful Transfection of Human Air Liquid Interface Cultures with the Non-Viral Gene Transfer Agent GI67A . Griesenbach, U. et al. Human Gene Therapy 20
Secreted Gaussla Luciferase Is a Sensitive Reporter of Gene Transfer In the Sheep Lung. Griesenbach, U. et al. Human Gene Therapy 20
Evaluation of safety and gene expression with a single dose of pGM169/GL67A administered to the nose and lund of individuals with CF: The UK CF Gene Therapy Consortium "Pilot Study". Davies, J. C. et al. Pediatric Pulmonology 44 Abstract 268
Optimisation of molecular assays for clinical trial of GL67A/pGM169 delivery to nose and lung of CF patients. Sumner-Jones, S. G. et al. Pediatric Pulmonology 44 Abstract 269
3-Step TaqMan RT-PCR: Ultimate mRNA Detection Sensitivity For CF Gene Therapy Clinical Trials. McCormick, D. et al. Pediatric Pulmonology 44 Abstract 271
Success and utility of sputum induction in non-expectorating CF subjects: data from the UK CF Gene Therapy Run-in Study. Voase, N. W. et al. Pediatric Pulmonology 44 Abstract 359
Optimising sputum processing for analysis of inflammatory markers in CF: Removal of dithiothreitol by dialysis does not improve detection of cytokines. Donovan, J. et al. Pediatric Pulmonology 44 Abstract 413
The Effect of pDNA Quality on Gene Transfer Outcome In Vivo. Bazzani, R. P. et al. Molecular Therapy 17 S20
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. Molecular Therapy 17 S123
Optimising harvest of bronchial brush biopsy samples to maximise cell and RNA yeild in gene therapy studies. Vrettou, C. et al. Molecular Therapy 17 S125
Exploiting Single-Cell-PCR Technology To Determine the Percentage of Transfected Cells in the Respiratory Epithelium. Pringle, I. A. et al. Molecular Therapy 17 S125
A Novel Mixing Device for the Reproducible Manufacture of Non-Viral Gene Therapy Formulations. Davies, L. A. et al. Molecular Therapy 17 S188
Salt Concentration and Liposomal DNA Therapeutic Formulations. Nicholls, P. et al. Molecular Therapy 17 S227
Repeated Exposure to pDNA/PEI Aerosols Results in Minimal Detectable Toxicity in the Mouse Lungs . Nunez-Alonso, G. A. et al. Molecular Therapy 17 S123
Calculating the percentage of cells transfected following non-viral delivery to the respiratory epithelium. Pringle, I. A. et al. Molecular Therapy 17 S227
Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies. Gill, D. R. et al. Pediatric Pulmonology 31 307
Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy. Hyde, S. C. et al. Pediatric Pulmonology 31 307
Optimisation of the harvest and handling of bronchial brushing samples to maximise RNA yield in gene therapy studies. McLachlan, G. et al. Pediatric Pulmonology 31
Transcriptional profiles of CF and non-CF nasal and bronchial epithelium. Ogilvie, VC. et al. Pediatric Pulmonology 31
Carboxymethylecellulose increases non-viral gene transfer in mouse airways. Griesenbach, U. et al. Pediatric Pulmonology 31, Abstract 293
Doxorubicin increases non-viral gene transfer in the lung. Griesenbach, U. et al. Pediatric Pulmonology 31, Abstract 291
Secreted Gaussia Luciferase is a sensitive reporter of gene transfer in the sheep lung.Griesenbach, U. et al. Pediatric Pulmonology 31, Abstract 292
Successful Transfection of human air liquid interface cultures with the nonviral gene transfer agent GL67A. Griesenbach, U. et al. Pediatric Pulmonology 31, Abstract 251
Validation of lung function values obtained with a hand-held PIKO6 device in adults and children with CF. Saunders, C. et al. Pediatric Pulmonology 31, Abstract 496
Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice. Davies, L. et al.
Development of highly sensitive TaqMan RT-PCR assays for quantifying vector mRNA in human samples. Sumner-Jones, S. G. et al.
Optimisation of Aerosol Delivery of Lipid/DNA Complexes for Clinical Studies. Davies, L. A. et al. Molecular Therapy 16 S160 Download
Challenges in the Process Development of a Novel Zero CpG CFTR Plasmid for Human Clinical Use. Hebel, H. L. et al. Molecular Therapy 16 S110 Download
Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung. Pringle, I. A. et al. Molecular Therapy 16 S341 Download
Identification of Novel Naturally CpG-Free Human and Murine Promoters for Non-viral Gene Therapy. Pringle, I. A. et al. Molecular Therapy 16 S182 Download
Near-Single Copy mRNA Quantification from a TaqMan RT-PCR Assay for an Aerosol Gene Therapy Clinical Trial. Sumner-Jones, S. G. et al. Molecular Therapy 16 S309 Download
Influence of CpG-dinuceotide motifs on the duration of duration of gene expression from plasmid vectors after in vivo lung delivery. Gill, D. R. et al. Pediatric Pulmonology 42 307 Download
EnaC Knockdown in the Mouse Lung using RNAI. Hyde, S. C. et al. Pediatric Pulmonology 42 306 Download
CpG-Dependent Inflammatory Response after Delivery of Lipid/pDNA Complexes to Murine Lungs. Bazzani, R. P. et al. Molecular Therapy 15 S388 Download
Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Sheep Lung. Davies, L. A. et al. Molecular Therapy 15 S407 Download
Influence of the Human and Murine CMV Enhancer on the Duration of Expression from CpG-Free pDNA Vectors in the Mouse Lung. Green, A.-M. et al. Molecular Therapy 15 S385 Download
CpGs Influence the Duration of Gene Expression from Plasmid Vectors after In Vivo Lung Delivery. Lawton, A. E. et al. Molecular Therapy 15 S130 Download
Persistent Gene Expression in the Ovine Lung from a Human Elongation Factor 1 Alpha Promoter Plasmid Following Non-Viral Gene Delivery. McLachlan, G. et al. Molecular Therapy 15 S162 Download
Long-Term Stability of Aqueous pDNA/PEI Complexes. Nunez-Alonso, G. A. et al. Molecular Therapy 15 S361 Download
Generation of a CpG-Free Clinical Trial Plasmid for Cystic Fibrosis Lung Gene Therapy. Pringle, I. A. et al. Molecular Therapy 15 S389 Download
Complete but not Partial Reduction of Plasmid CpG Content Increases Transgene Expression and Eliminates the Inflammatory Response Associated with Delivery of Non-Viral Vectors to the Lung. Hyde, S. C. et al. Pediatric Pulmonology 41 310 Download
EGFP expression in the mouse lung following administration of gene transfer vectors expressing EGFP from the UbC promoter. Davies, L. A. et al. Download
CpG depletion results in increased duration of gene expression from plasmid DNA vectors in vivo. Lawton, A. E. et al. Download
Complete but not partial reduction of plasmid CpG content reduces the inflammatory response associated with delivery of GL67/pDNA complexes to the mouse lung. Pringle, I. A. et al. Download
Reduced reporter activity upon repeated administration of adeno-associated virus 5 in murine airways. Sumner-Jones, S. G. et al. Download
Novel CpG depleted and codon optimised CFTR cDNAs maintain the structure and fuction of CFTR protein. Varathalingam, A. et al. Download
Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Murine Lung. Davies, L. A. et al. Molecular Therapy 13 S267 Download
Development of Zero-CpG Plasmids with Reduced Inflammatory Responses Following Delivery of Lipid/pDNA Complexes to the Mouse Lung. Pringle, I. A. et al. Molecular Therapy 13 S304 Download
Repeat Administration of Polyethylenimine (PEI) Aerosols of Plasmid DNA to the Murine Lung is Associated with a Loss of Gene Transfer Efficiency. Davies, L. A. et al. Molecular Therapy 11 S137 Download
Development of Quantitative TaqMan RT-PCR for the Evaluation of Non-Viral Mediated Gene Transfer to the airways. Pringle, I. A. et al. Molecular Therapy 11 S143 Download
Using Real-Time (TaqMan) PCR to Genotype Offspring of Transgenic CF-null Mice- A Core Facility of the UK Cystic Fibrosis Gene Therapy Consortium. Smith, R. L. et al. Molecular Therapy 11 S141 Download
Development of zero-CpG plasmids for noviral lung gene therapy. Pringle, I. A. et al. Download
Repeat administration of adenoassociated virus 5 in murine airways. Sumner-Jones, S. G. et al. Download
Optimising Gene Transfer Products for Evaluation in the Mouse Nasal Epithelium. Hyde, S. C. et al. Pediatric Pulmonology 40 291 Download
CpG Depletion Results in Increased Duration of Gene Expression from Plasmid DNA Vectors In vivo. Lawton, A. E. et al. Pediatric Pulmonology 40 280 Download
Avoiding the Nuclear Barrier: The Development of mRNA as a Gene Transfer Agent. Painter, H. et al. Pediatric Pulmonology 40 281 Download
Novel CPG-Depleted and Codon-Optimised CFTR CDNAs Maintain the Structure and Function of CFTR Protein. Varathalingam, A. et al. Pediatric Pulmonology 40 275 Download
Transgene Expression in the Mouse Lung following Administration of Gene Transfer Vectors Expressing EGFP. Davies, L. A. et al. Journal of Cystic Fibrosis 3 S29 Download
Evaluating Gene Transfer Products for Gene Expression in Mouse Nasal Epithelium. Jones, B. L. et al. Journal of Cystic Fibrosis 3 S29 Download
Direct Electroporation of the Murine Lung greatly Enhances Reporter Gene Expression following Intranasal Delivery of Plasmid DNA. Pringle, I. A. et al. Journal of Cystic Fibrosis 3 S29 Download
Using real-time (Taqman) RT-PCR to measure gene expression- a core facility of the UK . Smith, R. L. et al. Journal of Cystic Fibrosis 3 S5 Download
Repeat Administration of Adeno-associated Virus in Murine Airways. Sumner-Jones, S. G. et al. Journal of Cystic Fibrosis 3 S29 Download
Use of Ciliated Cell Specific Promoter FoxJ1 in Gene Transfer Vectors for the Airway Epithelium. Lawton, A. E. et al. Molecular Therapy 9 S55 Download
Topical Delivery of mRNA to the Murine Lung and Nasal Epithelium. Painter, H. et al. Molecular Therapy 9 S187 Download
Duration of Reporter Gene Expression from Naked pDNA in the Mouse Lung following Direct Electroporation and Development of Wire Electrodes for Sheep Lung Electroporation Studies. Pringle, I. A. et al. Molecular Therapy 9 S56 Download
Delivery of NF-κβ Decoy Related Oligodeoxynucleotides Reduces Pro-Inflammatory Cytokine Responses Associated with Plasmid DNA/Lipid Mediated Gene Transfer to Murine Lungs. Varathalingam, A. et al. Molecular Therapy 9 S177 Download
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