GeneMedicine Group Conference Abstracts

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RDM Inaugural Symposium, March 25 2013

Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease. Pringle I.A. et al.

The use of Lentiviral vectors to treat airway disease. Harding-Smith R. et al.

 

The American Society of Gene and Cell Therapy Annual Conference. Philidelphia. May 15 - 19, 2012

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. Cai, Y. et al. Molecular Therapy 20 S1

 

The North American Cystic Fibrosis Conference, Anaheim, November 3-5, 2011

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 S34 Download

 

The American Society of Gene and Cell Therapy Annual Conference. Seattle. May 18 - 21, 2011

Knockdown of ENaCα, as a treatment for Cystic Fibrosis lung disease (2011). Harding-Smith, R. E. et al., Molecular Therapy, 19, Abstract. Download

 

The North American Cystic Fibrosis Conference, Baltimore, October 21-23, 2010

Development, Production and Evaluation of clinical grade CFTR Expression Plasmid for CF Lung Gene Therapy. Gill, D. R. et al. Pediatric Pulmonology 33 315 Download

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 Download

 

 

The American Society of Gene and Cell Therapy Annual Conference. Washington DC. May 19 - 23, 2010

Genomic DNA Reduction for Therapeutic DNA Manufacture. Cai, Y. et al. Molecular Therapy 18 S288 Download

Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4°C. Davies, L. A. et al. Molecular Therapy 18 S123 Download

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 Download

Duration of Expression from CpG-Free Plasmids Following Hydrodynamic Delivery to the Mouse. Pringle, I. A. et al. Molecular Therapy 18 S283 Download

 

The British Society of Gene Therapy Annual Conference. London, April 21-23, 2009.

Non-viral gene expression in the lung using the mini-CFTR promoter. Connolly, M. M. et al. Human Gene Therapy 20 403 Download

Inflammation-Free shRNA Expression Vectors for Cystic Fibrosis Gene Therapy. Lawton, A. E. et al. Human Gene Therapy 20 404 Download

 

The North American Cystic Fibrosis Conference. Minneapolis. October 14-19 2009.

3-Step TaqMan RT-PCR: Ultimate mRNA Detection Sensitivity For CF Gene Therapy Clinical Trials. McCormick, D. et al. Pediatric Pulmonology 44 Abstract 271 Download

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 Download

 

The American Society of Gene Therapy Annual Conference. San Diego, May 26-30, 2009.

The Effect of pDNA Quality on Gene Transfer Outcome In Vivo. Bazzani, R. P. et al. Molecular Therapy 17 S20 Download

A Novel Mixing Device for the Reproducible Manufacture of Non-Viral Gene Therapy Formulations. Davies, L. A. et al. Molecular Therapy 17 S188 Download

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 Download

Salt Concentration and Liposomal DNA Therapeutic Formulations. Nicholls, P. et al. Molecular Therapy 17 S227 Download

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 Download

Calculating the percentage of cells transfected following non-viral delivery to the respiratory epithelium. Pringle, I. A. et al. Molecular Therapy 17 S227 Download

 

The North American Cystic Fibrosis Conference. Orlando. October 23-25, 2008.

Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies. Gill, D. R. et al. Pediatric Pulmonology 31 307 Download

Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy. Hyde, S. C. et al. Pediatric Pulmonology 31 307 Download

 

The British Society of Gene Therapy Annual Conference. Edinburgh, April 7-9, 2008.

Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice. Davies, L. et al. Download

Development of highly sensitive TaqMan RT-PCR assays for quantifying vector mRNA in human samples. Sumner-Jones, S. G. et al. Download

 

The American Society of Gene and Cell Therapy Annual Conference. Boston. May 28 - 01 June, 2008

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

 

The North American Cystic Fibrosis Conference. Anaheim. October 3-6, 2007.

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

 

The American Society of Gene and Cell Therapy Annual Conference. Seattle. May 30 - 03 June, 2007.

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

 

North American Cystic Fibrosis Conference Denver Colorado November 2-5 2006.

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

 

British Society of Gene Therapy Conference. London , March 28 - 30, 2006.

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

 

American Society of Gene Therapy 9th Annual Meeting Baltimore. May-June 2006.

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

 

The American Society of Gene Therapy Annual Conference. St Louis. June 2005

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

 

The European Society of Gene and Cell Therapy Conference. Prague, October 2005.

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

 

The North American Cystic Fibrosis Conference. Baltimore, October 2005.

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

 

The European CF Conference, Birmingham, June 2004

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

 

The American Society of Gene Therapy Annual Conference. Minneapolis. June 2004

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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