Papers 29

  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. 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.
  3. Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways.
    Rose AC et al., Gene Ther. 2002 Oct;9(19):1312-20.
  4. Identification of transfected cell types following non-viral gene transfer to the murine lung.
    Davies LA et al., J Gene Med. 2007 Mar;9(3):184-96.
  5. Detection of plasmid DNA vectors following gene transfer to the murine airways.
    Pringle IA et al., Gene Ther. 2005 Aug;12(15):1206-14.
  6. 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.
  7. 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.
  8. A novel mixing device for the reproducible generation of nonviral gene therapy formulations.
    Davies LA et al., Biotechniques. 2010 Sep;49(3):666-8. doi: 10.2144/000113498.
  9. 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.
  10. 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.
  11. Progress and prospects: gene therapy clinical trials (part 1).
    Alexander BL et al., Gene Ther. 2007 Oct;14(20):1439-47.
  12. 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.
  13. Rapid identification of novel functional promoters for gene therapy.
    Pringle IA et al., J Mol Med (Berl). 2012 Dec;90(12):1487-96. doi: 10.1007/s00109-012-0928-6. Epub 2012 Jul 6.
  14. 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.
  15. Optimizing aerosol gene delivery and expression in the ovine lung.
    McLachlan G et al., Mol Ther. 2007 Feb;15(2):348-54.
  16. 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.
  17. 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.
  18. 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.
  19. 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.
  20. 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
  21. Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung.
    Pringle IA et al., J Gene Med. 2007 May;9(5):369-80.
  22. 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.
  23. 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.
  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. 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.
  26. 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.
  27. 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.
  28. 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.
  29. 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 31

  1. Topical Delivery of mRNA to the Murine Lung and Nasal Epithelium.
    Painter H et al.,The American Society of Gene Therapy Annual Conference (2004)
  2. Delivery of NF-κβ Decoy Related Oligodeoxynucleotides Reduces Pro-Inflammatory Cytokine Responses Associated with Plasmid DNA/Lipid Mediated Gene Transfer to Murine Lungs.
    Varathalingam A et al.,The American Society of Gene Therapy Annual Conference (2004)
  3. 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)
  4. Transgene Expression in the Mouse Lung following Administration of Gene Transfer Vectors Expressing EGFP.
    Davies LA et al.,The European Cystic Fibrosis Conference (2004)
  5. Development of zero-CpG plasmids for noviral lung gene therapy.
    Pringle IA et al.,The European Society of Gene and Cell Therapy Conference (2005)
  6. Aerosol Characteristics of DNA/lipid Formulations for Gene Therapy Clinical Studies.
    Gill DR et al.,The North American Cystic Fibrosis Conference (2008)
  7. CpG Depletion Results in Increased Duration of Gene Expression from Plasmid DNA Vectors In vivo.
    Lawton AE et al.,The North American Cystic Fibrosis Conference (2005)
  8. CpG depletion results in increased duration of gene expression from plasmid DNA vectors in vivo.
    Lawton AE et al.,British Society of Gene Therapy Conference (2006)
  9. 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)
  10. 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)
  11. Complete but not Partial Reduction of Plasmid CpG Content Increases Transgene Expression and Eliminates the Inflammatory Response Associated with Delivery of Non-Viral Vectors to the Lung.
    Hyde SC et al.,North American Cystic Fibrosis Conference (2006)
  12. 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)
  13. CpG-Dependent Inflammatory Response after Delivery of Lipid/pDNA Complexes to Murine Lungs.
    Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2007)
  14. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  15. CpGs Influence the Duration of Gene Expression from Plasmid Vectors after In Vivo Lung Delivery.
    Lawton AE et al.,The American Society of Gene Therapy Annual Conference (2007)
  16. 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)
  17. 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)
  18. The Effect of pDNA Quality on Gene Transfer Outcome In Vivo.
    Bazzani RP et al.,The American Society of Gene Therapy Annual Conference (2009)
  19. A Novel Mixing Device for the Reproducible Manufacture of Non-Viral Gene Therapy Formulations.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2009)
  20. 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)
  21. Inflammation-free Human and Murine Promoters for Non-viral CFTR Lung Gene Therapy.
    Hyde SC et al.,The North American Cystic Fibrosis Conference (2008)
  22. 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)
  23. Complete but not partial reduction of plasmid CpG content reduces the inflammatory response associated with delivery of GL67/pDNA complexes to the mouse lung.
    Pringle IA et al.,British Society of Gene Therapy Conference (2006)
  24. Influence of CpG-dinuceotide motifs on the duration of duration of gene expression from plasmid vectors after in vivo lung delivery.
    Gill DR et al.,The North American Cystic Fibrosis Conference (2007)
  25. Development of Zero-CpG Plasmids with Reduced Inflammatory Responses Following Delivery of Lipid/pDNA Complexes to the Mouse Lung.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2006)
  26. Optimisation of Aerosol Delivery of Lipid/DNA Complexes for Clinical Studies.
    Davies LA et al.,The American Society of Gene Therapy Annual Conference (2008)
  27. Identification of Novel Naturally CpG-Free Human and Murine Promoters for Non-viral Gene Therapy.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2008)
  28. Influence of the Human and Murine CMV Enhancer on the Duration of Expression from CpG-Free pDNA Vectors in the Mouse Lung.
    Green A-M et al.,The American Society of Gene Therapy Annual Conference (2007)
  29. 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)
  30. Generation of a CpG-Free Clinical Trial Plasmid for Cystic Fibrosis Lung Gene Therapy.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2007)
  31. Towards Gene Therapy for Cystic Fibrosis: Bio-Distribution of GL67A/pGM169 DNA and mRNA Following Aerosol Delivery to the Mouse Lung.
    Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2008)

 

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

 

A frozen vial of GL67A (left) and a frozen vial of pGM169 plasmid DNA (right)

 

Pellets of DNA following precipitation.

 

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

 

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

 

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

 

Purifying mRNA from tissue samples.

 

A cake that only some of us got to enjoy!

 

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

 

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