Papers 4

  1. 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.
  2. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
  3. 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.
  4. 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.

Abstracts 14

  1. A clinical study to evaluate the safety and efficacy of pGM169/GL67A administered to the nose and lungs of individuals with cystic fibrosis.
    Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2010)
  2. 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)
  3. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  4. 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)
  5. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
  6. 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)
  7. 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)
  8. Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung.
    Sumner-Jones SG et al.,The European Society of Gene and Cell Therapy (2012)
  9. Longitudinal assessment of biomarkers for clinical trials of novel therapeutic agents: the Run-In study.
    Alton EW et al.,British Thoracic Society Winter Meeting (2010)
  10. 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)
  11. 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)
  12. Moving lentiviral-based gene therapy into a first-in-man CF trial.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)
  13. 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)
  14. Repeated Administration of the Non-Viral Gene Transfer Agent pGM169/GL67A Does Not Induce Anti-CFTR or Anti-Plasmid Immunoresponses.
    Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015)

 

Pellets of DNA following precipitation.

 

Purifying mRNA from tissue samples.

 

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