Papers 45

  1. Towards gene therapy for cystic fibrosis: a clinical progress report.
    Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2.
  2. Gene therapy for cystic fibrosis: an example for lung gene therapy.
    Griesenbach U et al., Gene Ther. 2004 Oct;11 Suppl 1:S43-50.
  3. Update on gene therapy for cystic fibrosis.
    Griesenbach U et al., Curr Opin Mol Ther. 2003 Oct;5(5):489-94.
  4. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2006 Jul;13(14):1061-7.
  5. Gene therapy for cystic fibrosis.
    Davies JC et al., J Gene Med. 2001 Sep-Oct;3(5):409-17.
  6. Prospects for gene therapy in lung disease.
    Davies JC et al., Curr Opin Pharmacol. 2001 Jun;1(3):272-7.
  7. The pathogenic consequences of a single mutated CFTR gene.
    Griesenbach U et al., Thorax. 1999 Aug;54 Suppl 2:S19-23.
  8. Prospects for gene therapy for cystic fibrosis.
    Davies JC et al., Mol Med Today. 1998 Jul;4(7):292-9.
  9. Advances in cystic fibrosis gene therapy.
    Griesenbach U et al., Curr Opin Pulm Med. 2004 Nov;10(6):542-6.
  10. Gene therapy progress and prospects: cystic fibrosis.
    Griesenbach U et al., Gene Ther. 2002 Oct;9(20):1344-50.
  11. Gene therapy for asthma: inspired research or unnecessary effort?
    Alton EW et al., Gene Ther. 1999 Feb;6(2):155-6.
  12. Biomarkers for cystic fibrosis: are we progressing?
    Alton EW et al., Am J Respir Crit Care Med. 2007 Apr 15;175(8):750-1.
  13. Milking gene therapy.
    Alton EW et al., Nat Med. 1998 Oct;4(10):1121-2.
  14. Airway surface pH in subjects with cystic fibrosis.
    McShane D et al., Eur Respir J. 2003 Jan;21(1):37-42.
  15. 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.
  16. 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.
  17. Bone marrow stem cells do not repopulate the healthy upper respiratory tract.
    Davies JC et al., Pediatr Pulmonol. 2002 Oct;34(4):251-6.
  18. 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.
  19. Computed tomography and cystic fibrosis: promises and problems.
    Aziz ZA et al., Thorax. 2007 Feb;62(2):181-6.
  20. Anti-inflammatory gene therapy directed at the airway epithelium.
    Griesenbach U et al., Gene Ther. 2000 Feb;7(4):306-13.
  21. 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.
  22. 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.
  23. Exploring the mechanisms of macrolides in cystic fibrosis.
    Equi AC et al., Respir Med. 2006 Apr;100(4):687-97. Epub 2005 Sep 26.
  24. 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.
  25. 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.
  26. 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.
  27. 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.
  28. 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.
  29. Vascular oligonucleotide transfer facilitated by a polymer-coated stent.
    Radke PW et al., Hum Gene Ther. 2005 Jun;16(6):734-40.
  30. Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
    Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35.
  31. 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.
  32. 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.
  33. The nasal epithelium as a factory for systemic protein delivery.
    Griesenbach U et al., Mol Ther. 2002 Feb;5(2):98-103.
  34. 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.
  35. 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.
  36. 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.
  37. 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.
  38. 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.
  39. 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.
  40. 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.
  41. Efficient gene transfer to airway epithelium using recombinant Sendai virus.
    Yonemitsu Y et al., Nat Biotechnol. 2000 Sep;18(9):970-3.
  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. 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.
  44. 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
  45. 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.

 

Schematic diagram of the large human airways.

 

Purifying mRNA from tissue samples.

 

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

 

Pellets of DNA following precipitation.

 

E.coli from a large scale industrial production of our clinical trial plasmid pGM169.

 

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