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Papers 58

  1. 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.
  2. Monitoring respiratory disease severity in cystic fibrosis.
    Davies JC et al., Respir Care. 2009 May;54(5):606-17.
  3. 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.
  4. Cystic fibrosis.
    Davies JC et al., BMJ. 2007 Dec 15;335(7632):1255-9.
  5. 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.
  6. Airway gene therapy.
    Davies JC et al., Adv Genet. 2005;54:291-314.
  7. 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.
  8. Prospects for gene therapy in lung disease.
    Davies JC et al., Curr Opin Pharmacol. 2001 Jun;1(3):272-7.
  9. Bone marrow stem cells do not repopulate the healthy upper respiratory tract.
    Davies JC et al., Pediatr Pulmonol. 2002 Oct;34(4):251-6.
  10. Gene therapy for cystic fibrosis.
    Davies JC et al., J Gene Med. 2001 Sep-Oct;3(5):409-17.
  11. 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.
  12. Prospects for gene therapy for cystic fibrosis.
    Davies JC et al., Mol Med Today. 1998 Jul;4(7):292-9.
  13. 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.
  14. 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.
  15. 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.
  16. Computed tomography and cystic fibrosis: promises and problems.
    Aziz ZA et al., Thorax. 2007 Feb;62(2):181-6.
  17. 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.
  18. Exploring the mechanisms of macrolides in cystic fibrosis.
    Equi AC et al., Respir Med. 2006 Apr;100(4):687-97. Epub 2005 Sep 26.
  19. Biomarkers for cystic fibrosis: are we progressing?
    Alton EW et al., Am J Respir Crit Care Med. 2007 Apr 15;175(8):750-1.
  20. Airway surface pH in subjects with cystic fibrosis.
    McShane D et al., Eur Respir J. 2003 Jan;21(1):37-42.
  21. 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.
  22. Cystic fibrosis: to ion transport and beyond.
    Bush A et al., Eur Respir J. 2010 Nov;36(5):991-2. doi: 10.1183/09031936.00056310.
  23. 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.
  24. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
  25. 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.
  26. 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.
  27. Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
    Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35.
  28. 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.
  29. 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.
  30. 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.
  31. 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.
  32. 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
  33. 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.
  34. Airway remodelling in children with cystic fibrosis.
    Hilliard TN et al., Thorax. 2007 Dec;62(12):1074-80. Epub 2007 May 25.
  35. 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.
  36. 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.
  37. 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.
  38. 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.
  39. 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.
  40. 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.
  41. 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.
  42. 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.
  43. 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.
  44. Endobronchial biopsy in childhood.
    Regamey N et al., Chest. 2008 Jan;133(1):312; author reply 313. doi: 10.1378/chest.07-1735.
  45. 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.
  46. 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.
  47. 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.
  48. 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.
  49. 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.
  50. 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.
  51. 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.
  52. 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.
  53. 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
  54. 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.
  55. 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.
  56. 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.
  57. 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.
  58. 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 28

  1. Current issues around LCI (Lung Clearance Index).
    Davies JC et al.,UK Cystic Fibrosis Conference (2015)
  2. 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)
  3. 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)
  4. Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
  5. 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)
  6. 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)
  7. 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)
  8. 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)
  9. 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)
  10. 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)
  11. 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)
  12. 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)
  13. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  14. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
  15. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
  16. 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)
  17. 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)
  18. Optimising Harvest of Bronchial Brush Biopsy Samples To Maximise Cell and RNA Yield in Gene Therapy Studies.
    Vrettou C et al.,The American Society of Gene Therapy Annual Conference (2009)
  19. 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)
  20. 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)
  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. 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)
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