Papers 24

1. 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.
2. 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.
3. 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.
4. 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.
5. Detection of plasmid DNA vectors following gene transfer to the murine airways.
   Pringle IA et al., Gene Ther. 2005 Aug;12(15):1206-14.
6. 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.
7. 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.
8. 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.
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. 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.
11. 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.
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. 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.
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. 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.
16. 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.
17. 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.
18. 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.
19. 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.
20. 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.
21. 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.
22. 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.
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 53

1. 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)
2. 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)
3. 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)
4. 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)
5. 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)
6. 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)
7. 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)
8. 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)
9. 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)
10. 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)
11. 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)
12. 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)
13. Cystic Fibrosis Gene Therapy Clinical Trial Demonstrates Beneficial Effect on Lung Function.
    Pringle IA et al.,Radcliffe Department of Medicine, Annual Symposium (2016)
14. Cystic Fibrosis Gene Therapy Clinical Trial Demonstrates Beneficial Effect on Lung Function.
    Pringle IA et al.,NDCLS Science Day (2015)
15. Development of zero-CpG plasmids for noviral lung gene therapy.
    Pringle IA et al.,The European Society of Gene and Cell Therapy Conference (2005)
16. Developing a persistent, inflammation-free gene therapy for Cystic Fibrosis lung disease.
    Pringle IA et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
17. Dissecting enhancer/promoter function for high-level, persistent transgene expression
    Gill DR et al.,The European Society of Gene and Cell Therapy Conference (2016)
18. 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)
19. 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)
20. 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)
21. 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)
22. 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)
23. The Effect of pDNA Quality on Gene Transfer Outcome In Vivo.
    Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2009)
24. 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)
25. 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)
26. Persistent gene expression in the murine lung is dependent on transgene CpG content.
    Oliveira C et al.,3rd Minicircle & DNA Vector Conference. (2014)
27. 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)
28. 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)
29. 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)
30. 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)
31. 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)
32. The use of Lentiviral vectors to treat airway disease.
    Harding-Smith RE et al.,Radcliffe Department of Medicine, Inaugural Symposium (2013)
33. 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)
34. 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)
35. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
36. 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)
37. 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)
38. 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)
39. 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)
40. 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)
41. 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)
42. 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)
43. 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)
44. Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2008)
45. 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)
46. 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)
47. 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)
48. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
49. 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)
50. 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)
51. 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)
52. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
53. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)