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.

 

A cake that only some of us got to enjoy!

 

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

 

Sheep lung parenchyma (cell nuclei blue) transduced with an adenoviral vector (green).

 

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

 

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

 

Proposed 3D model of the CFTR protein.