Papers 5

  1. The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes.
    Davies LA et al., Biomaterials. 2012 Aug;33(22):5618-27. doi: 10.1016/j.biomaterials.2012.04.019. Epub 2012 May 8.
  2. 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.
  3. 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.
  4. 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
  5. 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.

Abstracts 6

  1. 3-Step TaqMan RT-PCR: Ultimate mRNA Detection Sensitivity For CF Gene Therapy Clinical Trials.
    McCormick D et al.,The North American Cystic Fibrosis Conference (2009)
  2. Optimisation of molecular assays for clinical trial of GL67A/pGM169 delivery to nose and lung of CF patients.
    Sumner-Jones SG et al.,The North American Cystic Fibrosis Conference (2009)
  3. Near-Single Copy mRNA Quantification from a TaqMan RT-PCR Assay for an Aerosol Gene Therapy Clinical Trial.
    Sumner-Jones SG et al.,The American Society of Gene Therapy Annual Conference (2008)
  4. Development of highly sensitive TaqMan RT-PCR assays for quantifying vector mRNA in human samples.
    Sumner-Jones SG et al.,The British Society of Gene Therapy Annual Conference (2008)
  5. 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)
  6. Repeat Aerosol Delivery of Concentrated PEI/pDNA to the Sheep Lung.
    McLachlan G et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)

 

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

 

Schematic diagram of the large human airways.

 

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