Papers 24

1. 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.
2. 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.
3. 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.
4. 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.
5. 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.
6. 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.
7. 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.
8. Assessment of selected media supplements to improve F/HN lentiviral vector production yields.
   Gelinas JF et al., 7: 10198, DOI:10.1038/s41598-017-07893-3
9. The development of gene therapy for diseases of the lung.
   Gill DR et al., Cell Mol Life Sci. 2004 Feb;61(3):355-68.
10. Optimising non-viral gene delivery in a tumour spheroid model.
    Mellor HR et al., J Gene Med. 2006 Sep;8(9):1160-70.
11. 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.
12. 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.
13. 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.
14. 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.
15. 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.
16. 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.
17. 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.
18. 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.
19. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
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. 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.
22. 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.
23. 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
24. 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 54

1. Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Sheep Lung.
   Davies LA et al.,The American Society of Gene Therapy Annual Conference (2007)
2. Transgene Expression in the Mouse Lung following Administration of Gene Transfer Vectors Expressing EGFP.
   Davies LA et al.,The European Cystic Fibrosis Conference (2004)
3. 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)
4. Aerosol Delivery of Gene Therapeutics to the Lung.
   Davies LA et al.,1000 years of pharmaceutical aerosols – What remains to be done? (2009)
5. 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)
6. Aerosol Delivery of Concentrated pDNA/PEI Formulations to the Murine Lung.
   Davies LA et al.,The American Society of Gene Therapy Annual Conference (2006)
7. Concentrated PEI Formulations Retain Biological Efficacy Following Long-Term Storage at 4°C.
   Davies LA et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
8. 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)
9. Optimisation of Aerosol Delivery of Lipid/DNA Complexes for Clinical Studies.
   Davies LA et al.,The American Society of Gene Therapy Annual Conference (2008)
10. 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)
11. Concentrated PEI Gene Transfer formulations retain biological efficacy following long-term storage at 4°C.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2010)
12. 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)
13. 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)
14. Avoiding the Nuclear Barrier: The Development of mRNA as a Gene Transfer Agent.
    Painter H et al.,The North American Cystic Fibrosis Conference (2005)
15. 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)
16. The use of Lentiviral vectors to treat airway disease.
    Harding-Smith RE et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
17. Cystic Fibrosis Gene Therapy Clinical Trial Demonstrates Beneficial Effect on Lung Function.
    Pringle IA et al.,Radcliffe Department of Medicine, Annual Symposium (2016)
18. 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)
19. 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)
20. 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)
21. 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)
22. 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)
23. 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)
24. Long-Term Stability of Aqueous pDNA/PEI Complexes.
    Nunez-Alonso GA et al.,The American Society of Gene Therapy Annual Conference (2007)
25. 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)
26. 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)
27. 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)
28. 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)
29. Salt Concentration and Liposomal DNA Therapeutic Formulations.
    Nicholls P et al.,The American Society of Gene Therapy Annual Conference (2009)
30. Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2008)
31. 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)
32. Lung antibody factory to provide long-term passive immunity to influenza.
    Tan TK et al.,Radcliffe Department of Medicine, Annual Symposium (2015)
33. 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)
34. Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease.
    Pringle IA et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
35. 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)
36. 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)
37. 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)
38. Optimising Gene Transfer Products for Evaluation in the Mouse Nasal Epithelium.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2005)
39. 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)
40. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
41. 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)
42. 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)
43. 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)
44. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
45. 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)
46. 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)
47. 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)
48. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
49. 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)
50. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
51. 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)
52. Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies.
    Gill DR et al.,The North American Cystic Fibrosis Conference (2008)
53. 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)
54. 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)