Papers 38

1. pSURF-2, a modified BAC vector for selective YAC cloning and functional analysis.
   Boyd AC et al., Biotechniques. 1999 Jul;27(1):164-70, 172, 175.
2. Revisiting the mouse lung model for CF.
   Boyd AC et al., Gene Ther. 2004 May;11(9):737-8.
3. Insertion of natural intron 6a-6b into a human cDNA-derived gene therapy vector for cystic fibrosis improves plasmid stability and permits facile RNA/DNA discrimination.
   Boyd AC et al., J Gene Med. 1999 Sep-Oct;1(5):312-21.
4. PCR-generated cross-over linkers for site-directed mutagenesis.
   Boyd AC et al., Biotechniques. 1997 Nov;23(5):827-30.
5. 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
6. 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.
7. 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.
8. Genetic medicines for CF: Hype versus reality.
   Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
9. 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.
10. The effects of plasmid copy number and sequence context upon transfection efficiency.
    Walker WE et al., J Control Release. 2004 Jan 8;94(1):245-52.
11. Measurement of Serum Calprotectin in Stable Patients Predicts Exacerbation and Lung Function Decline in Cystic Fibrosis.
    Reid PA et al., Am J Respir Crit Care Med. 2015 Jan 15;191(2):233-236.
12. Sputum and serum calprotectin are useful biomarkers during CF exacerbation.
    Gray RD et al., J Cyst Fibros. 2010 May;9(3):193-8. doi: 10.1016/j.jcf.2010.01.005. Epub 2010 Mar 17.
13. 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.
14. Genomic sequence analysis of Fugu rubripes CFTR and flanking genes in a 60 kb region conserving synteny with 800 kb of human chromosome 7.
    Davidson H et al., Genome Res. 2000 Aug;10(8):1194-203.
15. Elimination of contaminant Escherichia coli chromosomal DNA from preparations of P1 artificial chromosome recombinants facilitates directed subcloning.
    Davidson H et al., Electrophoresis. 1999 Jun;20(7):1469-75.
16. 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.
17. Biomarkers for cystic fibrosis lung disease: application of SELDI-TOF mass spectrometry to BAL fluid.
    MacGregor G et al., J Cyst Fibros. 2008 Sep;7(5):352-8. doi: 10.1016/j.jcf.2007.12.005. Epub 2008 Feb 1.
18. HIV-1 Tat protein transduction domain peptide facilitates gene transfer in combination with cationic liposomes.
    Hyndman L et al., J Control Release. 2004 Oct 19;99(3):435-44.
19. 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.
20. Potent stimulation of gene expression by histone deacetylase inhibitors on transiently transfected DNA.
    Nan X et al., Biochem Biophys Res Commun. 2004 Nov 5;324(1):348-54.
21. Sputum proteomics in inflammatory and suppurative respiratory diseases.
    Gray RD et al., Am J Respir Crit Care Med. 2008 Sep 1;178(5):444-52. doi: 10.1164/rccm.200703-409OC. Epub 2008 Jun 19.
22. 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.
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.
25. Tripod-like cationic lipids as novel gene carriers.
    Unciti-Broceta A et al., J Med Chem. 2008 Jul 24;51(14):4076-84. doi: 10.1021/jm701493f. Epub 2008 Jun 26.
26. 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.
27. Sputum trace metals are biomarkers of inflammatory and suppurative lung disease.
    Gray RD et al., Chest. 2010 Mar;137(3):635-41. doi: 10.1378/chest.09-1047. Epub 2009 Oct 3.
28. Chimeric constructs endow the human CFTR Cl- channel with the gating behavior of murine CFTR.
    Scott-Ward TS et al., Proc Natl Acad Sci U S A. 2007 Oct 9;104(41):16365-70. Epub 2007 Oct 3.
29. 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.
30. SELDI-TOF biomarker signatures for cystic fibrosis, asthma and chronic obstructive pulmonary disease.
    Gomes-Alves P et al., Clin Biochem. 2010 Jan;43(1-2):168-77. doi: 10.1016/j.clinbiochem.2009.10.006. Epub 2009 Oct 20.
31. Differential global gene expression in cystic fibrosis nasal and bronchial epithelium.
    Ogilvie V et al., Genomics. 2011 Nov;98(5):327-36. doi: 10.1016/j.ygeno.2011.06.008. Epub 2011 Jul 2.
32. 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.
33. 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.
34. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
35. 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.
36. 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.
37. 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.
38. 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.

Abstracts 23

1. 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)
2. 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)
3. 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)
4. 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)
5. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
   Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
6. 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)
7. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
   Alton EW et al.,British Thoracic Society Winter Meeting (2012)
8. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
   Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
9. 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)
10. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
11. 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)
12. 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)
13. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
14. 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)
15. 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)
16. 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)
17. 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)
18. 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)
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. 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)
21. 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)
22. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
    Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)
23. 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)