Papers 13

  1. 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.
  2. 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.
  3. 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.
  4. 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.
  5. 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.
  6. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Alton EW et al., In Press
  7. 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.
  8. 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.
  9. 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.
  10. 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.
  11. 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.
  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. 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.

Abstracts 14

  1. 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)
  2. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
  3. 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)
  4. 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)
  5. 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)
  6. 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)
  7. 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)
  8. 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)
  9. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
  10. 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)
  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. 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)
  13. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
    Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)
  14. 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)

 

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)

 

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

 

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

 

Pellets of DNA following precipitation.