Papers 76

1. Advances in cystic fibrosis gene therapy.
   Griesenbach U et al., Curr Opin Pulm Med. 2004 Nov;10(6):542-6.
2. Cystic fibrosis gene therapy: successes, failures and hopes for the future.
   Griesenbach U et al., Expert Rev Respir Med. 2009 Aug;3(4):363-71. doi: 10.1586/ers.09.25.
3. Quantification of periciliary fluid height in human airway biopsies is feasible, but not suitable as a biomarker.
   Griesenbach U et al., Am J Respir Cell Mol Biol. 2011 Mar;44(3):309-15. doi: 10.1165/rcmb.2009-0265OC. Epub 2010 Apr 23.
4. Recent progress in gene therapy for cystic fibrosis.
   Griesenbach U et al., Curr Opin Mol Ther. 2001 Aug;3(4):385-9.
5. The role of doxorubicin in non-viral gene transfer in the lung.
   Griesenbach U et al., Biomaterials. 2009 Apr;30(10):1971-7. doi: 10.1016/j.biomaterials.2008.12.037. Epub 2009 Jan 18.
6. Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.
   Griesenbach U et al., Adv Drug Deliv Rev. 2009 Feb 27;61(2):128-39. doi: 10.1016/j.addr.2008.09.010. Epub 2008 Dec 24.
7. 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.
8. Validation of nasal potential difference measurements in gut-corrected CF knockout mice.
   Griesenbach U et al., Am J Respir Cell Mol Biol. 2008 Oct;39(4):490-6. doi: 10.1165/rcmb.2007-0385OC. Epub 2008 May 5.
9. The nasal epithelium as a factory for systemic protein delivery.
   Griesenbach U et al., Mol Ther. 2002 Feb;5(2):98-103.
10. In vivo imaging of gene transfer to the respiratory tract.
    Griesenbach U et al., Biomaterials. 2008 Apr;29(10):1533-40. Epub 2007 Dec 21.
11. Cytoplasmic deposition of NFkappaB decoy oligonucleotides is insufficient to inhibit bleomycin-induced pulmonary inflammation.
    Griesenbach U et al., Gene Ther. 2002 Aug;9(16):1109-15.
12. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2002 Oct;9(20):1344-50.
13. Update on gene therapy for cystic fibrosis.
    Griesenbach U et al., Curr Opin Mol Ther. 2003 Oct;5(5):489-94.
14. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2006 Jul;13(14):1061-7.
15. 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.
16. Gene therapy for cystic fibrosis: an example for lung gene therapy.
    Griesenbach U et al., Gene Ther. 2004 Oct;11 Suppl 1:S43-50.
17. Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lung.
    Griesenbach U et al., Gene Ther. 2006 Mar;13(5):449-56.
18. Sendai virus for gene therapy and vaccination.
    Griesenbach U et al., Curr Opin Mol Ther. 2005 Aug;7(4):346-52.
19. Validation of recombinant Sendai virus in a non-natural host model.
    Griesenbach U et al., Gene Ther. 2011 Feb;18(2):182-8. doi: 10.1038/gt.2010.131. Epub 2010 Oct 21.
20. 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.
21. Anti-inflammatory gene therapy directed at the airway epithelium.
    Griesenbach U et al., Gene Ther. 2000 Feb;7(4):306-13.
22. Current status and future directions of gene and cell therapy for cystic fibrosis.
    Griesenbach U et al., BioDrugs. 2011 Apr 1;25(2):77-88. doi: 10.2165/11586960-000000000-00000.
23. 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.
24. 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.
25. Recent advances in understanding and managing cystic fibrosis transmembrane conductance regulator dysfunction.
    Griesenbach U et al., F1000Prime Rep. 2015; 7: 64.
26. Moving forward: cystic fibrosis gene therapy.
    Griesenbach U et al., Hum Mol Genet. 2013 Oct 15;22(R1):R52-8. doi: 10.1093/hmg/ddt372. Epub 2013 Aug 4.
27. Cystic Fibrosis Gene Therapy in the UK and elsewhere
    Griesenbach U et al., Hum Gene Ther. 2015 May;26(5):266-75.
28. Cystic fibrosis: ferreting with fibroblasts for cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2009 Jan;16(1):1-2. doi: 10.1038/gt.2008.155. Epub 2008 Oct 2.
29. Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.
    Griesenbach U et al., J Gene Med. 2012 Jul;14(7):491-500. doi: 10.1002/jgm.2643.
30. 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.
31. Progress in gene and cell therapy for cystic fibrosis lung disease.
    Griesenbach U et al., Curr Pharm Des. 2012;18(5):642-62.
32. Expert opinion in biological therapy: update on developments in lung gene transfer.
    Griesenbach U et al., Expert Opin Biol Ther. 2013 Mar;13(3):345-60. doi: 10.1517/14712598.2013.735656. Epub 2013 Jan 5.
33. The pathogenic consequences of a single mutated CFTR gene.
    Griesenbach U et al., Thorax. 1999 Aug;54 Suppl 2:S19-23.
34. Special Focus Issue on the Annual Meeting of the British Society for Gene and Cell Therapy
    Nicklin SA et al., Human Gene Therapy. May 2015, 26(5): 247-248.
35. Magnetic nanoparticles as gene delivery agents: enhanced transfection in the presence of oscillating magnet arrays.
    McBain SC et al., Nanotechnology. 2008 Oct 8;19(40):405102. doi: 10.1088/0957-4484/19/40/405102. Epub 2008 Aug 20.
36. Vascular oligonucleotide transfer facilitated by a polymer-coated stent.
    Radke PW et al., Hum Gene Ther. 2005 Jun;16(6):734-40.
37. Gene therapy for asthma: inspired research or unnecessary effort?
    Alton EW et al., Gene Ther. 1999 Feb;6(2):155-6.
38. Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo.
    Xenariou S et al., Gene Ther. 2006 Nov;13(21):1545-52. Epub 2006 Jun 1.
39. Critical appraisal of the mouse model of myocardial infarction.
    Degabriele NM et al., Exp Physiol. 2004 Jul;89(4):497-505. Epub 2004 May 6.
40. Milking gene therapy.
    Alton EW et al., Nat Med. 1998 Oct;4(10):1121-2.
41. Use of ultrasound to enhance nonviral lung gene transfer in vivo.
    Xenariou S et al., Gene Ther. 2007 May;14(9):768-74. Epub 2007 Feb 15.
42. 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.
43. 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.
44. Intravenously administered oligonucleotides can be delivered to conducting airway epithelium via the bronchial circulation.
    Holder E et al., Gene Ther. 2006 Dec;13(23):1628-38. Epub 2006 Jun 22.
45. Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.
    Mitomo K et al., Mol Ther. 2010 Jun;18(6):1173-82. doi: 10.1038/mt.2010.13. Epub 2010 Mar 23.
46. Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli.
    Larsen MD et al., Gene Ther. 2008 Mar;15(6):434-42. doi: 10.1038/sj.gt.3303090. Epub 2008 Jan 24.
47. A defective nontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo.
    Ferrari S et al., Gene Ther. 2004 Nov;11(22):1659-64.
48. Beta-defensin genomic copy number is not a modifier locus for cystic fibrosis.
    Hollox EJ et al., J Negat Results Biomed. 2005 Dec 7;4:9.
49. Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.
    Xenariou S et al., Acta Biochim Biophys Sin (Shanghai). 2010 Jan;42(1):45-51.
50. Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
    Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35.
51. Expression and maturation of Sendai virus vector-derived CFTR protein: functional and biochemical evidence using a GFP-CFTR fusion protein.
    Ban H et al., Gene Ther. 2007 Dec;14(24):1688-94. Epub 2007 Sep 27.
52. The effect of mucolytic agents on gene transfer across a CF sputum barrier in vitro.
    Stern M et al., Gene Ther. 1998 Jan;5(1):91-8.
53. Oral contraceptives do not appear to affect cystic fibrosis disease severity.
    Kernan NG et al., Eur Respir J. 2013 Jan;41(1):67-73. doi: 10.1183/09031936.00018712. Epub 2012 May 3.
54. Emerging significance of plasmid DNA nuclear import in gene therapy.
    Munkonge FM et al., Adv Drug Deliv Rev. 2003 Jun 16;55(6):749-60.
55. Effects of intramyocardial pVEGF165 delivery on regional myocardial blood flow: evidence for a spatial 'delivery-efficacy' mismatch.
    Radke PW et al., Gene Ther. 2004 Aug;11(16):1249-55.
56. Efficient gene transfer to airway epithelium using recombinant Sendai virus.
    Yonemitsu Y et al., Nat Biotechnol. 2000 Sep;18(9):970-3.
57. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
58. 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
59. Nasal abnormalities in cystic fibrosis mice independent of infection and inflammation.
    Hilliard TN et al., Am J Respir Cell Mol Biol. 2008 Jul;39(1):19-25. doi: 10.1165/rcmb.2007-0284OC. Epub 2008 Jan 31.
60. 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.
61. 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.
62. 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.
63. CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector.
    Rakonczay Z Jr et al., J Cell Physiol. 2008 Feb;214(2):442-55.
64. Ex vivo and in vivo lentivirus-mediated transduction of airway epithelial progenitor cells.
    Leoni G et al., Curr Gene Ther. 2015;15(6):581-90.
65. Progress and prospects: gene therapy clinical trials (part 1).
    Alexander BL et al., Gene Ther. 2007 Oct;14(20):1439-47.
66. 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.
67. 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.
68. 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.
69. 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.
70. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
71. 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.
72. 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.
73. 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.
74. 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.
75. 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
76. 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 34

1. 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)
2. SIV Vector Pseudotyped with SeV-F/HN Envelope Proteins Produces Long Lasting Expression in the Murine Lung, Is Readministrable and Transfects Human Airway Models.
   Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
3. The nuclear pore dilating Agent TCHD increases gene transfer into differentiated airway epithelium ex vivo, but has no efect in vivo.
   Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
4. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
   Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)
5. 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)
6. A Phase IIb double-blind, placebo-controlled trial of non-viral mediated gene therapy for cystic fibrosis.
   Griesenbach U et al.,The British Society of Gene Therapy Annual Conference (2015)
7. 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)
8. 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)
9. Moving lentiviral-based gene therapy into a first-in-man CF trial.
   Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
10. 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)
11. 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)
12. Self-Reactive T Cells to CFTR in Cystic Fibrosis (CF) and Non-CF Humans: Implications for Gene Therapy.
    Limberis MP et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
13. Alpha-1-antitrypsin gene therapy for lung disease.
    Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2014)
14. 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)
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. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
17. 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)
18. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
19. 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)
20. 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)
21. 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)
22. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW 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)
24. Strain-Specific Differences in Pulmonary Gene Transfer Efficiency: Relevance for Toxicology Studies in Mice.
    Legakis G et al.,The American Society of Gene and Cell Therapy Annual Conference (2011)
25. 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)
26. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
27. 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)
28. 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)
29. 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)
30. 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)
31. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
32. 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)
33. 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)
34. 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)