Papers 70

  1. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis.
    Hyde SC et al., Gene Ther. 2000 Jul;7(13):1156-65.
  2. Ignoring the nonsense: a phase II trial in cystic fibrosis.
    Hyde SC et al., Lancet. 2008 Aug 30;372(9640):691-2. doi: 10.1016/S0140-6736(08)61169-1. Epub 2008 Aug 20.
  3. 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.
  4. Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy.
    Hyde SC et al., Nature. 1993 Mar 18;362(6417):250-5.
  5. Structural model of ATP-binding proteins associated with cystic fibrosis, multidrug resistance and bacterial transport.
    Hyde SC et al., Nature. 1990 Jul 26;346(6282):362-5.
  6. Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects.
    Pringle IA et al., Expert Opin Biol Ther. 2009 Aug;9(8):991-1003. doi: 10.1517/14712590903055029.
  7. 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.
  8. 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.
  9. Binding protein-dependent transport systems.
    Higgins CF et al., J Bioenerg Biomembr. 1990 Aug;22(4):571-92.
  10. Separation of drug transport and chloride channel functions of the human multidrug resistance P-glycoprotein.
    Gill DR et al., Cell. 1992 Oct 2;71(1):23-32.
  11. Delivery of genes into the CF airway.
    Gill DR et al., Thorax. 2014 Oct;69(10):962-4. doi: 10.1136/thoraxjnl-2014-205835. Epub 2014 Jul 11.
  12. Identification and functional characterization of cytoplasmic determinants of plasmid DNA nuclear import.
    Munkonge FM et al., J Biol Chem. 2009 Sep 25;284(39):26978-87. doi: 10.1074/jbc.M109.034850. Epub 2009 Jul 28.
  13. Progress and prospects: the design and production of plasmid vectors.
    Gill DR et al., Gene Ther. 2009 Feb;16(2):165-71. doi: 10.1038/gt.2008.183. Epub 2009 Jan 8.
  14. Chloride secretion in the trachea of null cystic fibrosis mice: the effects of transfection with pTrial10-CFTR2.
    MacVinish LJ et al., J Physiol. 1997 Mar 15;499 ( Pt 3):677-87.
  15. Strategies for long-term expression of transgenes in the respiratory epithelium.
    Gill DR et al., Curr Opin Mol Ther. 2010 Aug;12(4):386-93.
  16. 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.
  17. The development of gene therapy for diseases of the lung.
    Gill DR et al., Cell Mol Life Sci. 2004 Feb;61(3):355-68.
  18. The significance of plasmid DNA preparations contaminated with bacterial genomic DNA on inflammatory responses following delivery of lipoplexes to the murine lung.
    Bazzani RP et al., Biomaterials. 2011 Dec;32(36):9854-65. doi: 10.1016/j.biomaterials.2011.08.092. Epub 2011 Sep 23.
  19. The multidrug resistance and cystic fibrosis genes have complementary patterns of epithelial expression.
    Trezise AE et al., EMBO J. 1992 Dec;11(12):4291-303.
  20. Aerosol delivery of DNA/liposomes to the lung for cystic fibrosis gene therapy.
    Davies LA et al., Hum Gene Ther Clin Dev. 2014 Jun;25(2):97-107. doi: 10.1089/humc.2014.019. Epub 2014 May 27.
  21. Optimising non-viral gene delivery in a tumour spheroid model.
    Mellor HR et al., J Gene Med. 2006 Sep;8(9):1160-70.
  22. Identification of transfected cell types following non-viral gene transfer to the murine lung.
    Davies LA et al., J Gene Med. 2007 Mar;9(3):184-96.
  23. Detection of plasmid DNA vectors following gene transfer to the murine airways.
    Pringle IA et al., Gene Ther. 2005 Aug;12(15):1206-14.
  24. Specific inhibitors distinguish the chloride channel and drug transporter functions associated with the human multidrug resistance P-glycoprotein.
    Mintenig GM et al., Receptors Channels. 1993;1(4):305-13.
  25. Volume-regulated chloride channels associated with the human multidrug-resistance P-glycoprotein.
    Valverde MA et al., Nature. 1992 Feb 27;355(6363):830-3.
  26. Towards gene therapy for cystic fibrosis: a clinical progress report.
    Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2.
  27. 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.
  28. Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways.
    Rose AC et al., Gene Ther. 2002 Oct;9(19):1312-20.
  29. Electrohydrodynamic comminution: a novel technique for the aerosolisation of plasmid DNA.
    Davies LA et al., Pharm Res. 2005 Aug;22(8):1294-304. Epub 2005 Aug 3.
  30. A novel mixing device for the reproducible generation of nonviral gene therapy formulations.
    Davies LA et al., Biotechniques. 2010 Sep;49(3):666-8. doi: 10.2144/000113498.
  31. Drug efflux mediated by the human multidrug resistance P-glycoprotein is inhibited by cell swelling.
    Sardini A et al., J Cell Sci. 1994 Dec;107 ( Pt 12):3281-90.
  32. 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.
  33. A second dose of a CFTR cDNA-liposome complex is as effective as the first dose in restoring cAMP-dependent chloride secretion to null CF mice trachea.
    Goddard CA et al., Gene Ther. 1997 Nov;4(11):1231-6.
  34. 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.
  35. Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter.
    Gill DR et al., Gene Ther. 2001 Oct;8(20):1539-46.
  36. Tamoxifen blocks chloride channels. A possible mechanism for cataract formation.
    Zhang JJ et al., J Clin Invest. 1994 Oct;94(4):1690-7.
  37. Electroporation-mediated interleukin-10 overexpression in skeletal muscle reduces acute rejection in rat cardiac allografts.
    Tavakoli R et al., J Gene Med. 2006 Feb;8(2):242-8.
  38. Self-reactive CFTR T cells in humans: implications for gene therapy.
    Calcedo R et al., Hum Gene Ther Clin Dev. 2013 Sep;24(3):108-15. doi: 10.1089/humc.2012.249. Epub 2013 Jul 19.
  39. 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.
  40. Human-Mouse Chimeras With Normal Expression and Function Reveal That Major Domain Swapping is Tolerated by P-glycoprotein (ABCB1).
    Pluchino KM et al., Biochemistry , 2016 Feb 23;55(7):1010-23.
  41. A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis.
    Alton EW et al., Thorax. 2013 Nov;68(11):1075-7. doi: 10.1136/thoraxjnl-2013-203309. Epub 2013 Mar 22.
  42. A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial.
    Alton EW et al., AJRCCM, Volume 192, Pages 1389-1392
  43. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
  44. Effects of a novel archaeal tetraether-based colipid on the in vivo gene transfer activity of two cationic amphiphiles.
    Le Gall T et al., Mol Pharm. 2014 Sep 2;11(9):2973-88. doi: 10.1021/mp4006276. Epub 2014 Jul 30.
  45. Human-specific cystic fibrosis transmembrane conductance regulator antibodies detect in vivo gene transfer to ovine airways.
    Davidson H et al., Am J Respir Cell Mol Biol. 2006 Jul;35(1):72-83. Epub 2006 Feb 23.
  46. 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.
  47. 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.
  48. Keratinocyte growth factor therapy in murine oleic acid-induced acute lung injury.
    Ulrich K et al., Am J Physiol Lung Cell Mol Physiol. 2005 Jun;288(6):L1179-92. Epub 2005 Jan 28.
  49. Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo.
    Griesenbach U et al., Respir Res. 2006 Feb 15;7:26.
  50. Rapid identification of novel functional promoters for gene therapy.
    Pringle IA et al., J Mol Med (Berl). 2012 Dec;90(12):1487-96. doi: 10.1007/s00109-012-0928-6. Epub 2012 Jul 6.
  51. 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.
  52. Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segments.
    Emerson M et al., Mol Ther. 2003 Oct;8(4):646-53.
  53. Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.
    Holder E et al., J Gene Med. 2010 Jan;12(1):55-63. doi: 10.1002/jgm.1413.
  54. Identification of a Cryptic Bacterial Promoter in Mouse (mdr1a) P-Glycoprotein cDNA.
    Pluchino KM et al., PLoS One. 2015 Aug 26;10(8):e0136396. doi: 10.1371/journal.pone.0136396.
  55. 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.
  56. 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.
  57. Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.
    Griesenbach U et al., Am J Respir Crit Care Med. 2012 Nov 1;186(9):846-56. doi: 10.1164/rccm.201206-1056OC. Epub 2012 Sep 6.
  58. 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.
  59. 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.
  60. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
  61. 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.
  62. 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.
  63. A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis.
    Gill DR et al., Gene Ther. 1997 Mar;4(3):199-209.
  64. Sendai virus-mediated CFTR gene transfer to the airway epithelium.
    Ferrari S et al., Gene Ther. 2007 Oct;14(19):1371-9. Epub 2007 Jun 28.
  65. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Alton EW et al., In Press
  66. 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.
  67. Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation.
    Horsley AR et al., Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.
  68. 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.
  69. 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
  70. 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.

Abstracts 108

  1. A clinical study to evaluate the safety and efficacy of pGM169/GL67A administered to the nose and lungs of individuals with cystic fibrosis.
    Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  2. Optimising Gene Transfer Products for Evaluation in the Mouse Nasal Epithelium.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2005)
  3. EnaC Knockdown in the Mouse Lung using RNAI.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2007)
  4. Concentrated PEI Gene Transfer formulations retain biological efficacy following long-term storage at 4C.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2010)
  5. 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)
  6. CpG Free Plasmids For Enhanced Duration & Reduced Toxicity Of Non-Viral Airway Gene Transfer.
    Hyde SC et al.,European Society of Gene and Cell Therapy Conference (2008)
  7. Non-Viral Vector Development For Reduced Inflammation and Sustained Pulmonary Gene Expression.
    Hyde SC et al.,The American Society of Gene Therapy Annual Conference (2008)
  8. Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2008)
  9. 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 SC et al.,North American Cystic Fibrosis Conference (2006)
  10. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
  11. Non-Viral CFTR Gene Delivery To The Lungs Of Cystic Fibrosis Patients.
    Hyde SC et al.,The European Society of Gene and Cell Therapy (2013)
  12. Translating Gene Therapy Research Ideas to the Clinical Trials.
    Hyde SC et al.,Malaysian Tissue Engineering and Regenerative Medicine Scientific Meeting (2012)
  13. Scalable, Animal-Free, Suspension-Based Production of SIV Lentiviral Vectors.
    Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
  14. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
  15. Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Murine Lung.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2006)
  16. Repeat Administration of Polyethylenimine (PEI) Aerosols of Plasmid DNA to the Murine Lung is Associated with a Loss of Gene Transfer Efficiency.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2005)
  17. Gene Therapy: Barriers to Translation.
    Davie N et al.,The British Society of Gene Therapy Annual Conference (2014)
  18. Delivery of Therapeutic Monoclonal Antibody Genes for Prophylaxis of Respiratory Syncytial Virus Infection.
    Antepowicz A et al.,The American Society of Gene and Cell Therapy Annual Conference (2017)
  19. Long-Term Stability of Aqueous pDNA/PEI Complexes.
    Nunez-Alonso GA et al.,The American Society of Gene Therapy Annual Conference (2007)
  20. Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4C.
    Davies LA et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  21. Knockdown of ENaCα, as a treatment for Cystic Fibrosis lung disease
    Harding-Smith RE et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
  22. 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)
  23. Repeat administration of adenoassociated virus 5 in murine airways.
    Sumner-Jones SG et al.,The European Society of Gene and Cell Therapy Conference (2005)
  24. Lentiviral-Mediated ENaCα Knockdown, as a Treatment for Cystic Fibrosis Lung Disease
    Harding-Smith RE et al.,The American Society of Gene and Cell Therapy Annual Conference (2013)
  25. Alpha-1-antitrypsin gene therapy for lung disease.
    Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2014)
  26. The Significance of Academia-Industry Collaboration in Translational Research: A Survey of Over 300 PIs Who Have Received Industry Funding.
    Davie NL et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
  27. Dissecting enhancer/promoter function for high-level, persistent transgene expression
    Gill DR et al.,The European Society of Gene and Cell Therapy Conference (2016)
  28. Challenges in the Process Development of a Novel Zero CpG CFTR Plasmid for Human Clinical Use.
    Hebel HL et al.,The American Society of Gene Therapy Annual Conference (2008)
  29. Salt Concentration and Liposomal DNA Therapeutic Formulations.
    Nicholls P et al.,The American Society of Gene Therapy Annual Conference (2009)
  30. Topical Delivery of mRNA to the Murine Lung and Nasal Epithelium.
    Painter H et al.,The American Society of Gene Therapy Annual Conference (2004)
  31. Avoiding the Nuclear Barrier: The Development of mRNA as a Gene Transfer Agent.
    Painter H et al.,The North American Cystic Fibrosis Conference (2005)
  32. EGFP expression in the mouse lung following administration of gene transfer vectors expressing EGFP from the UbC promoter.
    Davies LA et al.,British Society of Gene Therapy Conference (2006)
  33. Inflammation-Free shRNA Expression Vectors for Cystic Fibrosis Gene Therapy.
    Lawton AE et al.,The British Society of Gene Therapy Annual Conference (2009)
  34. Transgene Expression in the Mouse Lung following Administration of Gene Transfer Vectors Expressing EGFP.
    Davies LA et al.,The European Cystic Fibrosis Conference (2004)
  35. Evaluating Gene Transfer Products for Gene Expression in Mouse Nasal Epithelium.
    Jones BL et al.,The European Cystic Fibrosis Conference (2004)
  36. Using real-time (Taqman) RT-PCR to measure gene expression- a core facility of the UK .
    Smith RL et al.,The European Cystic Fibrosis Conference (2004)
  37. Repeat Administration of Adeno-associated Virus in Murine Airways.
    Sumner-Jones SG et al.,The European Cystic Fibrosis Conference (2004)
  38. 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)
  39. 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.,British Thoracic Society Winter Meeting (2011)
  40. Persistent gene expression in the murine lung is dependent on transgene CpG content.
    Oliveira C et al.,3rd Minicircle & DNA Vector Conference. (2014)
  41. Cystic Fibrosis Gene Therapy Clinical Trial Demonstrates Beneficial Effect on Lung Function.
    Pringle IA et al.,Radcliffe Department of Medicine, Annual Symposium (2016)
  42. Development of lung and muscle protein factories to deliver therapeutic monoclonal antibodies
    Antepowicz A et al.,The British Society of Gene Therapy Annual Conference (2016)
  43. Development of Recombinant Adeno-Associated Viral Vector (rAAV) for Passive Immunisation Against Ebola.
    Tan TK et al.,The American Society of Gene and Cell Therapy Annual Conference (2017)
  44. CpGs Influence the Duration of Gene Expression from Plasmid Vectors after In Vivo Lung Delivery.
    Lawton AE et al.,The American Society of Gene Therapy Annual Conference (2007)
  45. Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Sheep Lung.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2007)
  46. Duration of Expression from CpG-Free Plasmids Following Hydrodynamic Delivery to the Mouse.
    Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  47. Genomic DNA Reduction for Therapeutic DNA Manufacture.
    Cai Y et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  48. Development of Quantitative TaqMan RT-PCR for the Evaluation of Non-Viral Mediated Gene Transfer to the airways.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2005)
  49. 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.,The American Society of Gene Therapy Annual Conference (2004)
  50. 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)
  51. Longitudinal assessment of biomarkers for clinical trials of novel therapeutic agents: the Run-In study.
    Alton EW et al.,British Thoracic Society Winter Meeting (2010)
  52. Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease.
    Pringle IA et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
  53. Cholesterol as an enhancer of lentiviral vector production in serum-free transient transfection.
    Gelinas JF et al.,The European Society of Gene and Cell Therapy (2013)
  54. Minicircles Are Similar To Plasmids In Providing High Level, Long-Term Expression In The Lung .
    Gill DR et al.,The American Society of Gene and Cell Therapy Annual Conference (2016)
  55. CpG-Dependent Inflammatory Response after Delivery of Lipid/pDNA Complexes to Murine Lungs.
    Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2007)
  56. 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 RL et al.,The American Society of Gene Therapy Annual Conference (2005)
  57. 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 IA et al.,The American Society of Gene Therapy Annual Conference (2004)
  58. CpG Depletion Results in Increased Duration of Gene Expression from Plasmid DNA Vectors In vivo.
    Lawton AE et al.,The North American Cystic Fibrosis Conference (2005)
  59. CpG depletion results in increased duration of gene expression from plasmid DNA vectors in vivo.
    Lawton AE et al.,British Society of Gene Therapy Conference (2006)
  60. Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies.
    Gill DR et al.,The North American Cystic Fibrosis Conference (2008)
  61. 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)
  62. Lung antibody factory to provide long-term passive immunity to influenza.
    Tan TK et al.,Radcliffe Department of Medicine, Annual Symposium (2015)
  63. Optimisation of Aerosol Delivery of Lipid/DNA Complexes for Clinical Studies.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2008)
  64. Repeated Exposure to pDNA/PEI Aerosols Results in Minimal Detectable Toxicity in the Mouse Lungs .
    Nunez-Alonso GA et al.,The American Society of Gene Therapy Annual Conference (2009)
  65. A Novel Mixing Device for the Reproducible Manufacture of Non-Viral Gene Therapy Formulations.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2009)
  66. The Effect of pDNA Quality on Gene Transfer Outcome In Vivo.
    Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2009)
  67. Influence of CpG-dinuceotide motifs on the duration of duration of gene expression from plasmid vectors after in vivo lung delivery.
    Gill DR et al.,The North American Cystic Fibrosis Conference (2007)
  68. 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)
  69. Large-Scale cGMP Manufacture of a Plasmid Vector for Cystic Fibrosis Gene Therapy Clinical Trials.
    Cai Y et al.,The American Society of Gene and Cell Therapy Annual Conference (2012)
  70. The use of Lentiviral vectors to treat airway disease.
    Harding-Smith RE et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
  71. Lung antibody factory to provide long-term passive immunity to influenza.
    Tan TK et al.,The American Society of Gene and Cell Therapy Annual Conference (2016)
  72. Identification of Novel Naturally CpG-Free Human and Murine Promoters for Non-viral Gene Therapy.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2008)
  73. Development of Zero-CpG Plasmids with Reduced Inflammatory Responses Following Delivery of Lipid/pDNA Complexes to the Mouse Lung.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2006)
  74. 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)
  75. 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 IA et al.,British Society of Gene Therapy Conference (2006)
  76. Pre-existing immunity to human parainfluenza virus (hPIV) does not affect rSIV.F/HN-mediated transduction efficiency.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2016)
  77. A phase IIb Double-Blind Placebo-Controlled Trial of Non-Viral Gene Transfer for Cystic Fibrosis.
    Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014)
  78. 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.,The American Society of Gene Therapy Annual Conference (2007)
  79. 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.,The American Society of Gene Therapy Annual Conference (2007)
  80. Novel CPG-Depleted and Codon-Optimised CFTR CDNAs Maintain the Structure and Function of CFTR Protein.
    Varathalingam A et al.,The North American Cystic Fibrosis Conference (2005)
  81. 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)
  82. Novel CpG depleted and codon optimised CFTR cDNAs maintain the structure and fuction of CFTR protein.
    Varathalingam A et al.,British Society of Gene Therapy Conference (2006)
  83. Non-viral gene expression in the lung using the mini-CFTR promoter.
    Connolly MM et al.,The British Society of Gene Therapy Annual Conference (2009)
  84. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
  85. 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)
  86. Update on the UK CF Gene Therapy Consortium Multidose, Non-viral, Gene Therapy Trial
    Alton EW et al.,The North American Cystic Fibrosis Conference (2012)
  87. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
  88. 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)
  89. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  90. Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus.
    Pytel KM et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
  91. Therapeutic levels of alpha-1-antitrypsin following gene therapy with F/HN pseudotyped simian immunodeficiency virus.
    Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2015)
  92. 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)
  93. 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)
  94. 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)
  95. 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)
  96. 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)
  97. Immune Responses to Single and Repeated Administration of pGM169/GL67A, The UK CF Gene Therapy Consortium Clinical Trials.
    Griesenbach U et al.,British Thoracic Society Winter Meeting (2014)
  98. 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)
  99. Generation of a CpG-Free Clinical Trial Plasmid for Cystic Fibrosis Lung Gene Therapy.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2007)
  100. F/HN Pseudotyped Lentivirus Generates Therapeutically Relevant and Long-Lasting Alpha-1-Antitrypsin Expression in Mouse Lung.
    Paul-Smith MC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
  101. 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)
  102. Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2008)
  103. Calculating the percentage of cells transfected following non-viral delivery to the respiratory epithelium.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2009)
  104. Assessment of F/HN-Pseudotyped Lentivirus in a Clinically Relevant Vector for Lung Gene Therapy
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2012)
  105. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
    Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)
  106. A randomized, double-blind, placebo-controlled trial of repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis.
    Alton EW et al.,The North American Cystic Fibrosis Conference (2015)
  107. 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)
  108. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)

 

Schematic diagram of the large human airways.

 

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

 

Large scale lentivirus production in suspension culture.

 

Proposed 3D model of the CFTR protein.

 

Mouse lung large airway (cell nuclei blue) transduced with an adenoviral vector (green).

 

Purifying mRNA from tissue samples.

 

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

 

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

 

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

 

A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)

 

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

 

A cake that only some of us got to enjoy!

 

Light microscope image of a human airway liquid interface cultures. Dark patches are mucous.

 

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

 

Pellets of DNA following precipitation.