Journal Articles Conference Presentations Book Chapters Public Presentations

Papers 22

  1. Lung clearance index in CF: a sensitive marker of lung disease severity.
    Davies JC et al., Thorax. 2008 Feb;63(2):96-7. doi: 10.1136/thx.2007.082768.
  2. Measurement of Serum Calprotectin in Stable Patients Predicts Exacerbation and Lung Function Decline in Cystic Fibrosis.
    Reid PA et al., Am J Respir Crit Care Med. 2015 Jan 15;191(2):233-236.
  3. Sputum and serum calprotectin are useful biomarkers during CF exacerbation.
    Gray RD et al., J Cyst Fibros. 2010 May;9(3):193-8. doi: 10.1016/j.jcf.2010.01.005. Epub 2010 Mar 17.
  4. Sputum trace metals are biomarkers of inflammatory and suppurative lung disease.
    Gray RD et al., Chest. 2010 Mar;137(3):635-41. doi: 10.1378/chest.09-1047. Epub 2009 Oct 3.
  5. Towards gene therapy for cystic fibrosis: a clinical progress report.
    Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2.
  6. Sputum proteomics in inflammatory and suppurative respiratory diseases.
    Gray RD et al., Am J Respir Crit Care Med. 2008 Sep 1;178(5):444-52. doi: 10.1164/rccm.200703-409OC. Epub 2008 Jun 19.
  7. Biomarkers for cystic fibrosis lung disease: application of SELDI-TOF mass spectrometry to BAL fluid.
    MacGregor G et al., J Cyst Fibros. 2008 Sep;7(5):352-8. doi: 10.1016/j.jcf.2007.12.005. Epub 2008 Feb 1.
  8. A Phase I/IIa Safety and Efficacy Study of Nebulized Liposome-mediated Gene Therapy for Cystic Fibrosis Supports a Multidose Trial.
    Alton EW et al., AJRCCM, Volume 192, Pages 1389-1392
  9. Genetic medicines for CF: Hype versus reality.
    Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
  10. Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis.
    Horsley AR et al., Thorax. 2008 Feb;63(2):135-40. Epub 2007 Aug 3.
  11. Self-reactive CFTR T cells in humans: implications for gene therapy.
    Calcedo R et al., Hum Gene Ther Clin Dev. 2013 Sep;24(3):108-15. doi: 10.1089/humc.2012.249. Epub 2013 Jul 19.
  12. 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.
  13. 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.
  14. 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.
  15. 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.
  16. Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP.
    McLachlan G et al., Gene Ther. 1996 Dec;3(12):1113-23.
  17. Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
    Porteous DJ et al., Gene Ther. 1997 Mar;4(3):210-8.
  18. 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.
  19. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Alton EW et al., In Press
  20. Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation.
    Horsley AR et al., Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.
  21. 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
  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.

Abstracts 14

  1. 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)
  2. 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)
  3. Reliability of Measurements Using Innocor Beath by Breath Analyser During a Maximal Exercise Test in Cystic Fibrosis Patients.
    Bayfield KJ et al.,British Thoracic Society Winter Meeting (2014)
  4. Lung clearance index, FEV1 and CT findings in Cystic Fibrosis: data from the UK CF Gene Therapy Consortium Run-in study.
    Sheridan HS et al.,British Thoracic Society Winter Meeting (2010)
  5. 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)
  6. Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
    Alton EW et al.,British Thoracic Society Winter Meeting (2012)
  7. Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
    Armstrong DK et al.,British Thoracic Society Winter Meeting (2014)
  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. The Importance of Appropriate Reference Sources for Spirometry: Lessons Learned from the UK Cystic Fibrosis Gene Therapy
    Davies G et al.,The North American Cystic Fibrosis Conference (2012)
  10. 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)
  11. 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)
  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. 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)

Aerosol Gene Delivery. (2010): An Aerotech II nebuliser generating a PEI/pDNA aerosol. More...

 

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

 

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

 

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

 

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

 

Proposed 3D model of the CFTR protein.

 

Pellets of DNA following precipitation.