Papers 121

  1. 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.
  2. 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.
  3. 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.
  4. Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice.
    Alton EW et al., Nat Genet. 1993 Oct;5(2):135-42.
  5. Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.
    Alton EW et al., Lancet. 1999 Mar 20;353(9157):947-54.
  6. Gene therapy for asthma: inspired research or unnecessary effort?
    Alton EW et al., Gene Ther. 1999 Feb;6(2):155-6.
  7. Biomarkers for cystic fibrosis: are we progressing?
    Alton EW et al., Am J Respir Crit Care Med. 2007 Apr 15;175(8):750-1.
  8. Milking gene therapy.
    Alton EW et al., Nat Med. 1998 Oct;4(10):1121-2.
  9. Towards gene therapy for cystic fibrosis: a clinical progress report.
    Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2.
  10. 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.
  11. 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
  12. 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
  13. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
  14. 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.
  15. 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.
  16. 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.
  17. Progress in gene and cell therapy for cystic fibrosis lung disease.
    Griesenbach U et al., Curr Pharm Des. 2012;18(5):642-62.
  18. Design of gene therapy trials in CF patients.
    Davies JC et al., Methods Mol Biol. 2011;741:55-68. doi: 10.1007/978-1-61779-117-8_5.
  19. 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.
  20. Gene therapy for cystic fibrosis.
    Davies JC et al., Proc Am Thorac Soc. 2010 Nov;7(6):408-14. doi: 10.1513/pats.201004-029AW.
  21. 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.
  22. Monitoring respiratory disease severity in cystic fibrosis.
    Davies JC et al., Respir Care. 2009 May;54(5):606-17.
  23. 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.
  24. Cystic fibrosis.
    Davies JC et al., BMJ. 2007 Dec 15;335(7632):1255-9.
  25. Airway gene therapy.
    Davies JC et al., Adv Genet. 2005;54:291-314.
  26. Recent progress in gene therapy for cystic fibrosis.
    Griesenbach U et al., Curr Opin Mol Ther. 2001 Aug;3(4):385-9.
  27. 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.
  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. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
    Caplen NJ et al., Nat Med. 1995 Jan;1(1):39-46.
  30. Measurement of halide efflux from cultured and primary airway epithelial cells using fluorescence indicators.
    Munkonge FM et al., J Cyst Fibros. 2004 Aug;3 Suppl 2:171-6.
  31. Recent advances in understanding and managing cystic fibrosis transmembrane conductance regulator dysfunction.
    Griesenbach U et al., F1000Prime Rep. 2015; 7: 64.
  32. Lung clearance index in primary ciliary dyskinesia and bronchiectasis.
    Irving SJ et al., Am J Respir Crit Care Med. 2014 May 1;189(9):1147-8. doi: 10.1164/rccm.201402-0206LE.
  33. Airway remodelling and its relationship to inflammation in cystic fibrosis.
    Regamey N et al., Thorax. 2011 Jul;66(7):624-9. doi: 10.1136/thx.2009.134106. Epub 2010 Oct 1.
  34. Computed tomography and cystic fibrosis: promises and problems.
    Aziz ZA et al., Thorax. 2007 Feb;62(2):181-6.
  35. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2006 Jul;13(14):1061-7.
  36. Advances in cystic fibrosis gene therapy.
    Griesenbach U et al., Curr Opin Pulm Med. 2004 Nov;10(6):542-6.
  37. Gene therapy for cystic fibrosis: an example for lung gene therapy.
    Griesenbach U et al., Gene Ther. 2004 Oct;11 Suppl 1:S43-50.
  38. Update on gene therapy for cystic fibrosis.
    Griesenbach U et al., Curr Opin Mol Ther. 2003 Oct;5(5):489-94.
  39. Prospects for gene therapy in lung disease.
    Davies JC et al., Curr Opin Pharmacol. 2001 Jun;1(3):272-7.
  40. Gene therapy for cystic fibrosis.
    Davies JC et al., J Gene Med. 2001 Sep-Oct;3(5):409-17.
  41. The pathogenic consequences of a single mutated CFTR gene.
    Griesenbach U et al., Thorax. 1999 Aug;54 Suppl 2:S19-23.
  42. Prospects for gene therapy for cystic fibrosis.
    Davies JC et al., Mol Med Today. 1998 Jul;4(7):292-9.
  43. Lung clearance index in CF: a sensitive marker of lung disease severity.
    Davies JC et al., Thorax. 2008 Feb;63(2):96-7. doi: 10.1136/thx.2007.082768.
  44. Progress and prospects: gene therapy clinical trials (part 1).
    Alexander BL et al., Gene Ther. 2007 Oct;14(20):1439-47.
  45. Cystic Fibrosis Gene Therapy in the UK and elsewhere
    Griesenbach U et al., Hum Gene Ther. 2015 May;26(5):266-75.
  46. Multiple breath washouts in children can be shortened without compromising quality.
    Ahmad F et al., Eur Respir J. 2015 Dec;46(6):1814-6. doi: 10.1183/13993003.00791-2015. Epub 2015 Oct 9.
  47. Gene therapy in cystic fibrosis.
    Armstrong DK et al., Arch Dis Child. 2014 May;99(5):465-8. doi: 10.1136/archdischild-2012-302158. Epub 2014 Jan 24.
  48. Cystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function.
    Simmonds NJ et al., Eur Respir J. 2011 May;37(5):1076-82. doi: 10.1183/09031936.00079010. Epub 2010 Sep 16.
  49. Sendai virus for gene therapy and vaccination.
    Griesenbach U et al., Curr Opin Mol Ther. 2005 Aug;7(4):346-52.
  50. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2002 Oct;9(20):1344-50.
  51. Does mass spectrometric breath analysis detect Pseudomonas aeruginosa in cystic fibrosis?
    Pabary R et al., Eur Respir J. 2016 Mar;47(3):994-7. doi: 10.1183/13993003.00944-2015. Epub 2016 Feb 4.
  52. 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.
  53. Airway remodelling in children with cystic fibrosis.
    Hilliard TN et al., Thorax. 2007 Dec;62(12):1074-80. Epub 2007 May 25.
  54. 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.
  55. 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.
  56. A molecular comparison of microbial communities in bronchiectasis and cystic fibrosis.
    Duff RM et al., Eur Respir J. 2013 Apr;41(4):991-3. doi: 10.1183/09031936.00052712.
  57. 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.
  58. 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.
  59. Normal nasal mucociliary clearance in CF children: evidence against a CFTR-related defect.
    McShane D et al., Eur Respir J. 2004 Jul;24(1):95-100.
  60. Airway surface pH in subjects with cystic fibrosis.
    McShane D et al., Eur Respir J. 2003 Jan;21(1):37-42.
  61. Bone marrow stem cells do not repopulate the healthy upper respiratory tract.
    Davies JC et al., Pediatr Pulmonol. 2002 Oct;34(4):251-6.
  62. 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.
  63. Cyanide levels found in infected cystic fibrosis sputum inhibit airway ciliary function.
    Nair C et al., Eur Respir J. 2014 Nov;44(5):1253-61. doi: 10.1183/09031936.00097014. Epub 2014 Sep 3.
  64. Mannose-binding lectin is present in the infected airway: a possible pulmonary defence mechanism.
    Fidler KJ et al., Thorax. 2009 Feb;64(2):150-5. doi: 10.1136/thx.2008.100073. Epub 2008 Nov 6.
  65. 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.
  66. Exploring the mechanisms of macrolides in cystic fibrosis.
    Equi AC et al., Respir Med. 2006 Apr;100(4):687-97. Epub 2005 Sep 26.
  67. 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.
  68. Anti-inflammatory gene therapy directed at the airway epithelium.
    Griesenbach U et al., Gene Ther. 2000 Feb;7(4):306-13.
  69. Reduction in the adherence of Pseudomonas aeruginosa to native cystic fibrosis epithelium with anti-asialoGM1 antibody and neuraminidase inhibition.
    Davies JC et al., Eur Respir J. 1999 Mar;13(3):565-70.
  70. 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.
  71. Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis.
    Regamey N et al., Thorax. 2012 Feb;67(2):164-70. doi: 10.1136/thoraxjnl-2011-200585. Epub 2011 Oct 18.
  72. Increased airway smooth muscle mass in children with asthma, cystic fibrosis, and non-cystic fibrosis bronchiectasis.
    Regamey N et al., Am J Respir Crit Care Med. 2008 Apr 15;177(8):837-43. doi: 10.1164/rccm.200707-977OC. Epub 2008 Jan 24.
  73. 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.
  74. 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.
  75. Endobronchial biopsy in childhood.
    Regamey N et al., Chest. 2008 Jan;133(1):312; author reply 313. doi: 10.1378/chest.07-1735.
  76. Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP.
    McLachlan G et al., Gene Ther. 1996 Dec;3(12):1113-23.
  77. Pseudomonas aeruginosa infection in cystic fibrosis: pathophysiological mechanisms and therapeutic approaches.
    Lund-Palau H et al., Expert Rev Respir Med. 2016 Jun;10(6):685-97. doi: 10.1080/17476348.2016.1177460. Epub 2016 May 13.
  78. 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.
  79. Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis.
    Horsley AR et al., Thorax. 2008 Feb;63(2):135-40. Epub 2007 Aug 3.
  80. Quality, size, and composition of pediatric endobronchial biopsies in cystic fibrosis.
    Regamey N et al., Chest. 2007 Jun;131(6):1710-7. Epub 2007 Feb 22.
  81. 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.
  82. 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.
  83. 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.
  84. 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.
  85. Lung clearance index and high-resolution computed tomography scores in primary ciliary dyskinesia.
    Irving SJ et al., Am J Respir Crit Care Med. 2013 Sep 1;188(5):545-9. doi: 10.1164/rccm.201304-0800OC.
  86. 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.
  87. 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.
  88. 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.
  89. 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.
  90. Vascular oligonucleotide transfer facilitated by a polymer-coated stent.
    Radke PW et al., Hum Gene Ther. 2005 Jun;16(6):734-40.
  91. Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
    Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35.
  92. 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.
  93. 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.
  94. 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.
  95. 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.
  96. CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium.
    Davies JC et al., Am J Respir Cell Mol Biol. 1997 Jun;16(6):657-63.
  97. 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.
  98. 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.
  99. The nasal epithelium as a factory for systemic protein delivery.
    Griesenbach U et al., Mol Ther. 2002 Feb;5(2):98-103.
  100. 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.
  101. 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.
  102. 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.
  103. Potential difference measurements in the lower airway of children with and without cystic fibrosis.
    Davies JC et al., Am J Respir Crit Care Med. 2005 May 1;171(9):1015-9. Epub 2005 Jan 7.
  104. 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.
  105. 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.
  106. 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.
  107. 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.
  108. 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.
  109. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
  110. Efficient gene transfer to airway epithelium using recombinant Sendai virus.
    Yonemitsu Y et al., Nat Biotechnol. 2000 Sep;18(9):970-3.
  111. 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.
  112. 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.
  113. 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.
  114. 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.
  115. 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.
  116. 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.
  117. 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.
  118. 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.
  119. 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.
  120. 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.
  121. 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 43

  1. 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)
  2. 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)
  3. 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)
  4. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  5. Moving gene therapy into CF patients.
    Alton EW et al.,UK Cystic Fibrosis Conference (2015)
  6. Gene Therapy: Moving to the Clinic.
    Alton EW et al.,European Cystic Fibrosis Conference (2015)
  7. 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)
  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. 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)
  11. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
  12. 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)
  13. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
  14. 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)
  15. 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)
  16. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
  17. 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)
  18. Alpha-1-antitrypsin gene therapy for lung disease.
    Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2014)
  19. 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)
  20. 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)
  21. 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)
  22. 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)
  23. 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)
  24. 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)
  25. 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)
  26. Reliability of Measurements Using Innocor Beath by Breath Analyser During a Maximal Exercise Test in Cystic Fibrosis Patients.
    Bayfield KJ et al.,British Thoracic Society Winter Meeting (2014)
  27. 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)
  28. 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)
  29. 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)
  30. 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)
  31. Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice.
    Davies L et al.,The British Society of Gene Therapy Annual Conference (2008)
  32. 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)
  33. The Importance of Appropriate Reference Sources for Spirometry: Lessons Learned from the UK Cystic Fibrosis Gene Therapy
    Davies G et al.,The North American Cystic Fibrosis Conference (2012)
  34. Lung clearance index, FEV1 and CT findings in Cystic Fibrosis: data from the UK CF Gene Therapy Consortium Run-in study.
    Sheridan HS et al.,British Thoracic Society Winter Meeting (2010)
  35. 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)
  36. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
  37. 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)
  38. 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)
  39. 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)
  40. 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)
  41. 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)
  42. 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)
  43. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
    Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)

 

Pellets of DNA following precipitation.

 

Large scale lentivirus production in suspension culture.

 

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

 

Schematic diagram of the large human airways.

 

A cake that only some of us got to enjoy!

 

Proposed 3D model of the CFTR protein.

 

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

 

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

 

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

 

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

 

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

 

Purifying mRNA from tissue samples.

 

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)