Papers 15

  1. Sendai virus for gene therapy and vaccination.
    Griesenbach U et al., Curr Opin Mol Ther. 2005 Aug;7(4):346-52.
  2. CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector.
    Rakonczay Z Jr et al., J Cell Physiol. 2008 Feb;214(2):442-55.
  3. 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.
  4. 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.
  5. 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.
  6. The nasal epithelium as a factory for systemic protein delivery.
    Griesenbach U et al., Mol Ther. 2002 Feb;5(2):98-103.
  7. 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.
  8. Validation of recombinant Sendai virus in a non-natural host model.
    Griesenbach U et al., Gene Ther. 2011 Feb;18(2):182-8. doi: 10.1038/gt.2010.131. Epub 2010 Oct 21.
  9. 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.
  10. 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.
  11. Efficient gene transfer to airway epithelium using recombinant Sendai virus.
    Yonemitsu Y et al., Nat Biotechnol. 2000 Sep;18(9):970-3.
  12. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Alton EW et al., In Press
  13. 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.
  14. 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.
  15. 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.

Abstracts 13

  1. Scalable, Animal-Free, Suspension-Based Production of SIV Lentiviral Vectors.
    Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015)
  2. Production of FVIII in the lungs.
    Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015)
  3. SIV Vector Pseudotyped with SeV-F/HN Envelope Proteins Produces Long Lasting Expression in the Murine Lung, Is Readministrable and Transfects Human Airway Models.
    Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  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. 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)
  6. 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)
  7. 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)
  8. Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
    Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
  9. 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)
  10. 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)
  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)

 

Schematic diagram of the large human airways.

 

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

 

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

 

Proposed 3D model of the CFTR protein.

 

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