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)