Papers 22

  1. Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways.
    Sumner-Jones SG et al., Gene Ther. 2006 Dec;13(24):1703-13. Epub 2006 Jul 20.
  2. Lack of repeat transduction by recombinant adeno-associated virus type 5/5 vectors in the mouse airway.
    Sumner-Jones SG et al., J Virol. 2007 Nov;81(22):12360-7. Epub 2007 Sep 12.
  3. Limitations of the murine nose in the development of nonviral airway gene transfer.
    Griesenbach U et al., Am J Respir Cell Mol Biol. 2010 Jul;43(1):46-54. doi: 10.1165/rcmb.2009-0075OC. Epub 2009 Jul 31.
  4. Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes.
    Davies LA et al., Mol Ther. 2008 Jul;16(7):1283-90. doi: 10.1038/mt.2008.96. Epub 2008 May 20.
  5. Assessment of CFTR function after gene transfer in vitro and in vivo.
    Griesenbach U et al., Methods Mol Biol. 2008;433:229-42. doi: 10.1007/978-1-59745-237-3_14.
  6. Coating of adeno-associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera
    Carlisle RC et al., J Gene Med. 2008 Apr;10(4):400-11. doi: 10.1002/jgm.1161.
  7. Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung.
    Pringle IA et al., J Gene Med. 2007 May;9(5):369-80.
  8. Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice.
    Davies LA et al., Mol Ther. 2008 May;16(5):812-8. doi: 10.1038/mt.2008.25. Epub 2008 Mar 11.
  9. Transgene sequences free of CG dinucleotides lead to high level, long-term expression in the lung independent of plasmid backbone design.
    Bazzani RP et al., 2016 Jul;93:20-6.
  10. Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung.
    McLachlan G et al., Gene Ther. 2011 Oct;18(10):996-1005. doi: 10.1038/gt.2011.55. Epub 2011 Apr 21.
  11. The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways.
    Griesenbach U et al., Biomaterials. 2010 Mar;31(9):2665-72. doi: 10.1016/j.biomaterials.2009.12.005. Epub 2009 Dec 21.
  12. CpG-free plasmid expression cassettes for cystic fibrosis gene therapy.
    Pringle IA et al., Biomaterials. 2012 Oct;33(28):6833-42. doi: 10.1016/j.biomaterials.2012.06.009. Epub 2012 Jun 22.
  13. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
    Hyde SC et al., Nat Biotechnol. 2008 May;26(5):549-51. doi: 10.1038/nbt1399. Epub 2008 Apr 27.
  14. The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes.
    Davies LA et al., Biomaterials. 2012 Aug;33(22):5618-27. doi: 10.1016/j.biomaterials.2012.04.019. Epub 2012 May 8.
  15. An immunocytochemical assay to detect human CFTR expression following gene transfer.
    Davidson H et al., Mol Cell Probes. 2009 Dec;23(6):272-80. doi: 10.1016/j.mcp.2009.07.001. Epub 2009 Jul 15.
  16. 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.
  17. Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.
    Alton EW et al., Gene Ther. 2014 Jan;21(1):89-95. doi: 10.1038/gt.2013.61. Epub 2013 Nov 7.
  18. Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis.
    Clark KL et al., Mol Ther Nucleic Acids. 2013 Jan 15;2:e65. doi: 10.1038/mtna.2012.57.
  19. The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep.
    Alton EW et al., Biomaterials. 2013 Dec;34(38):10267-77. doi: 10.1016/j.biomaterials.2013.09.023. Epub 2013 Oct 3.
  20. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Alton EW et al., Thorax. 2017 Feb;72(2):137-147. doi: 10.1136/thoraxjnl-2016-208406.
  21. 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.
  22. 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 30

  1. Repeat Administration of Adeno-associated Virus in Murine Airways.
    Sumner-Jones SG et al.,The European Cystic Fibrosis Conference (2004)
  2. Reduced reporter activity upon repeated administration of adeno-associated virus 5 in murine airways.
    Sumner-Jones SG et al.,British Society of Gene Therapy Conference (2006)
  3. Optimisation of molecular assays for clinical trial of GL67A/pGM169 delivery to nose and lung of CF patients.
    Sumner-Jones SG et al.,The North American Cystic Fibrosis Conference (2009)
  4. Repeat administration of adenoassociated virus 5 in murine airways.
    Sumner-Jones SG et al.,The European Society of Gene and Cell Therapy Conference (2005)
  5. Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung.
    Sumner-Jones SG et al.,The European Society of Gene and Cell Therapy (2012)
  6. Near-Single Copy mRNA Quantification from a TaqMan RT-PCR Assay for an Aerosol Gene Therapy Clinical Trial.
    Sumner-Jones SG et al.,The American Society of Gene Therapy Annual Conference (2008)
  7. Development of highly sensitive TaqMan RT-PCR assays for quantifying vector mRNA in human samples.
    Sumner-Jones SG et al.,The British Society of Gene Therapy Annual Conference (2008)
  8. Cell culture models of non-viral transgene expression are not indicative of in vivo lung outcome (2012)
    Oliveira CA et al.,The American Society of Gene and Cell Therapy Annual Conference (2012)
  9. Mutliple Doses of Lipid Mediated Gene Therapy Nebulised to the Mouse Lung Show Robust and Sustained CFTR Expression.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2011)
  10. Direct Electroporation of the Murine Lung greatly Enhances Reporter Gene Expression following Intranasal Delivery of Plasmid DNA.
    Pringle IA et al.,The European Cystic Fibrosis Conference (2004)
  11. Evaluating Gene Transfer Products for Gene Expression in Mouse Nasal Epithelium.
    Jones BL et al.,The European Cystic Fibrosis Conference (2004)
  12. Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease.
    Pringle IA et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
  13. Topical Delivery of mRNA to the Murine Lung and Nasal Epithelium.
    Painter H et al.,The American Society of Gene Therapy Annual Conference (2004)
  14. 3-Step TaqMan RT-PCR: Ultimate mRNA Detection Sensitivity For CF Gene Therapy Clinical Trials.
    McCormick D et al.,The North American Cystic Fibrosis Conference (2009)
  15. Optimising Gene Transfer Products for Evaluation in the Mouse Nasal Epithelium.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2005)
  16. Use of Ciliated Cell Specific Promoter FoxJ1 in Gene Transfer Vectors for the Airway Epithelium.
    Lawton AE et al.,The American Society of Gene Therapy Annual Conference (2004)
  17. The use of Lentiviral vectors to treat airway disease.
    Harding-Smith RE et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
  18. Cystic Fibrosis Gene Therapy Clinical Trial Demonstrates Beneficial Effect on Lung Function.
    Pringle IA et al.,Radcliffe Department of Medicine, Annual Symposium (2016)
  19. Avoiding the Nuclear Barrier: The Development of mRNA as a Gene Transfer Agent.
    Painter H et al.,The North American Cystic Fibrosis Conference (2005)
  20. Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Sheep Lung.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2007)
  21. Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice.
    Davies L et al.,The British Society of Gene Therapy Annual Conference (2008)
  22. Development, Production and Evaluation of clinical grade CFTR Expression Plasmid for CF Lung Gene Therapy
    Gill DR et al.,The North American Cystic Fibrosis Conference (2010)
  23. Optimising Harvest of Bronchial Brush Biopsy Samples To Maximise Cell and RNA Yield in Gene Therapy Studies.
    Vrettou C et al.,The American Society of Gene Therapy Annual Conference (2009)
  24. Quantifying non-viral gene transfer by real-time PCR: a cautionary tale.
    Coles R et al.,The European Society of Gene and Cell Therapy Conference (2009)
  25. Repeat Aerosol Delivery of Concentrated PEI/pDNA to the Sheep Lung.
    McLachlan G et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  26. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
  27. Clinical Development of an Optimal F/HN Pseudotyped SIV Vector for Cystic Fibrosis Lung Gene Therapy.
    Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014)
  28. Repeat Administration of Gl67A/pGM169 Is Feasible, Safe, and Produces Endogenous Levels of CFTR Expression After 12 Doses.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  29. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
  30. Preparation for a First-in-Man Lentivirus Trial in Cystic Fibrosis Patients.
    Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2016)

 

E.coli from a large scale industrial production of our clinical trial plasmid pGM169.

 

A pellet of E.coli containing a plasmid expressing a pink fluorescent protein.

 

Purifying mRNA from tissue samples.

 

Human airway liquid interface cultures transduced with a lentivirus expressing Luciferase.

 

Large scale lentivirus production in suspension culture.

 

DNA fragments being cut from an agarose gel exposed to UV.

 

Schematic diagram of the large human airways.

 

Sheep lung parenchyma (cell nuclei blue) transduced with an adenoviral vector (green).

 

A CFTR Western blot, to confirm protein production in cell culture.