Papers 6

1. 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.
2. 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
3. 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.
4. Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
   Alton EW et al., Thorax. 2017 Feb;72(2):137-147. doi: 10.1136/thoraxjnl-2016-208406.
5. 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
6. 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 3

1. A phase IIb Double-Blind Placebo-Controlled Trial of Non-Viral Gene Transfer for Cystic Fibrosis.
   Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014)
2. 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)
3. Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
   Griesenbach U et al.,British Thoracic Society Winter Meeting (2012)