Publications
Below is a list of Scientific publications detailing work undertaken by the gene therapy consortium.
2019
Emerging gene therapies for cystic fibrosis.
Miah KM et al., Expert Review of Respiratory Medicine
Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.
2018
Gelinas JF et al., Microbiology and Molecular Biology Reviews
Microbiol Mol Biol Rev. 2018 Jan 10;82(1). pii: e00051-17
Lessons learned from lung and liver in-vivo gene therapy: implications for the future.
van Haasteren J, Hyde SC, Gill DR.
Expert Opin Biol Ther. 2018 Sep;18(9):959-972.
The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins.
Paul-Smith MC et al., Gene Therapy.
Gene Ther. 2018 Aug;25(5):345-358. doi: 10.1038/s41434-018-0025-8.
2017
Assessment of selected media supplements to improve F/HN lentiviral vector production yields.
Gelinas JF et al., Scientific Reports
Sci Rep. 2017 Aug 31;7(1):10198. doi: 10.1038/s41598-017-07893-3.
Ng-Blichfeldt JP et al., Thorax
Thorax. 2017 Jun;72(6):510-521. doi: 10.1136/thoraxjnl-2016-208846.
2016
Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis.
Alton EW et al., Thorax
Thorax. 2017 Feb;72(2):137-147. doi: 10.1136/thoraxjnl-2016-208406.
Genetic medicines for CF: Hype versus reality.
Alton EW et al., Pediatric Pulmonololgy
Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
Alton EW et al., Efficacy and Mechanism Evaluation
Efficacy and Mechanism Evaluation (2016) Volume: 3 Issue: 5
Lund-Palau H et al., Expert Review Of Respiratory Medicine
Expert Rev Respir Med. 2016 Jun;10(6):685-97. doi: 10.1080/17476348.2016.1177460. Epub 2016 May 13.
Bazzani RP et al., Biomaterials
Biomaterials. 2016 Jul;93:20-26. doi: 10.1016/j.biomaterials.2016.03.029. Epub 2016 Mar 22.
Does mass spectrometric breath analysis detect Pseudomonas aeruginosa in cystic fibrosis?
Pabary R et al., The European Respiratory Journal
Eur Respir J. 2016 Mar;47(3):994-7. doi: 10.1183/13993003.00944-2015. Epub 2016 Feb 4.
Pluchino KM et al., Biochemistry
Biochemistry , 2016 Feb 23;55(7):1010-23. doi: 10.1021/acs.biochem.5b01064. Epub 2016 Feb 10.
704. Enhanced Lentiviral Production Through Rational Design of Mammalian Host Cells.
Gelinas JF et al., Molecular Therapy
Molecular Therapy, 2016 May;Volume 24,Supplement 1, page S278. doi: 10.1016/S1525-0016(16)33512-2.
2015
Alton EW et al., American Journal of Respiratory and Critical Care Medicine
AJRCCM, Volume 192, Pages 1389-1392
Ex vivo and in vivo lentivirus-mediated transduction of airway epithelial progenitor cells.
Leoni G et al., Current Gene Therapy
Curr Gene Ther. 2015;15(6):581-90.
Multiple breath washouts in children can be shortened without compromising quality.
Ahmad F et al., The European Respiratory Journal
Eur Respir J. 2015 Dec;46(6):1814-6. doi: 10.1183/13993003.00791-2015. Epub 2015 Oct 9.
Identification of a Cryptic Bacterial Promoter in Mouse (mdr1a) P-Glycoprotein cDNA.
Pluchino KM et al., PLoS One.
PLoS One. 2015 Aug 26;10(8):e0136396. doi: 10.1371/journal.pone.0136396.
Alton EW et al., The Lancet Respiratory Medicine
Lancet Respir Med. 2015 Sep;3(9):684-91. doi: 10.1016/S2213-2600(15)00245-3. Epub 2015 Jul 3.
Griesenbach U et al., F1000Prime Reports
F1000Prime Rep. 2015; 7: 64.
Special Focus Issue on the Annual Meeting of the British Society for Gene and Cell Therapy.
Nicklin SA et al., Human Gene Therapy
Human Gene Therapy. May 2015, 26(5): 247-248.
Cystic Fibrosis Gene Therapy in the UK and elsewhere.
Griesenbach U et al., Human Gene Therapy
Hum Gene Ther. 2015 May;26(5):266-75.
Reid PA et al., American Journal of Respiratory and Critical Care Medicine
Am J Respir Crit Care Med. 2015 Jan 15;191(2):233-236.
2014
Cyanide levels found in infected cystic fibrosis sputum inhibit airway ciliary function.
Nair C et al., The European Respiratory Journal
Eur Respir J. 2014 Nov;44(5):1253-61. doi: 10.1183/09031936.00097014. Epub 2014 Sep 3.
Le Gall T et al., Molecular Pharmaceutics
Mol Pharm. 2014 Sep 2;11(9):2973-88. doi: 10.1021/mp4006276. Epub 2014 Jul 30.
Delivery of genes into the CF airway.
Gill DR et al., Thorax
Thorax. 2014 Oct;69(10):962-4. doi: 10.1136/thoraxjnl-2014-205835. Epub 2014 Jul 11.
Aerosol delivery of DNA/liposomes to the lung for cystic fibrosis gene therapy.
Davies LA et al., Human Gene Therapy. Clinical Development
Hum Gene Ther Clin Dev. 2014 Jun;25(2):97-107. doi: 10.1089/humc.2014.019. Epub 2014 May 27.
Lung clearance index in primary ciliary dyskinesia and bronchiectasis.
Irving SJ et al., American Journal Of Respiratory And Critical Care Medicine
Am J Respir Crit Care Med. 2014 May 1;189(9):1147-8. doi: 10.1164/rccm.201402-0206LE.
Gene therapy in cystic fibrosis.
Armstrong DK et al., Archives Of Disease In Childhood
Arch Dis Child. 2014 May;99(5):465-8. doi: 10.1136/archdischild-2012-302158. Epub 2014 Jan 24.
P203 Development Of An Optimal F/hn Pseudotyped Siv Vector For Cf Gene Therapy.
Hyde SC et al., Thorax
Thorax. 2014;69:A165-A166. doi: 10.1136/thoraxjnl-2014-206260.332
2013
Alton EW et al., Gene Therapy
Gene Ther. 2014 Jan;21(1):89-95. doi: 10.1038/gt.2013.61. Epub 2013 Nov 7.
Alton EW et al., Biomaterials
Biomaterials. 2013 Dec;34(38):10267-77. doi: 10.1016/j.biomaterials.2013.09.023. Epub 2013 Oct 3.
Moving forward: cystic fibrosis gene therapy.
Griesenbach U et al., Human Molecular Genetics
Hum Mol Genet. 2013 Oct 15;22(R1):R52-8. doi: 10.1093/hmg/ddt372. Epub 2013 Aug 4.
Lung clearance index and high-resolution computed tomography scores in primary ciliary dyskinesia.
Irving SJ et al., American Journal Of Respiratory And Critical Care Medicine
Am J Respir Crit Care Med. 2013 Sep 1;188(5):545-9. doi: 10.1164/rccm.201304-0800OC.
Self-reactive CFTR T cells in humans: implications for gene therapy.
Calcedo R et al., Human Gene Therapy. Clinical Development
Hum Gene Ther Clin Dev. 2013 Sep;24(3):108-15. doi: 10.1089/humc.2012.249. Epub 2013 Jul 19.
A molecular comparison of microbial communities in bronchiectasis and cystic fibrosis.
Duff RM et al., The European Respiratory Journal
Eur Respir J. 2013 Apr;41(4):991-3. doi: 10.1183/09031936.00052712.
Alton EW et al., Thorax
Thorax. 2013 Nov;68(11):1075-7. doi: 10.1136/thoraxjnl-2013-203309. Epub 2013 Mar 22.
Horsley AR et al., Thorax
Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.
Clark KL et al., Molecular Therapy. Nucleic Acids
Mol Ther Nucleic Acids. 2013 Jan 15;2:e65. doi: 10.1038/mtna.2012.57.
Expert opinion in biological therapy: update on developments in lung gene transfer.
Griesenbach U et al., Expert Opinion On Biological Therapy
Expert Opin Biol Ther. 2013 Mar;13(3):345-60. doi: 10.1517/14712598.2013.735656. Epub 2013 Jan 5.
2012
Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.
Griesenbach U et al., American Journal Of Respiratory And Critical Care Medicine
Am J Respir Crit Care Med. 2012 Nov 1;186(9):846-56. doi: 10.1164/rccm.201206-1056OC. Epub 2012 Sep 6.
Rapid identification of novel functional promoters for gene therapy.
Pringle IA et al., Journal Of Molecular Medicine (Berlin, Germany)
J Mol Med (Berl). 2012 Dec;90(12):1487-96. doi: 10.1007/s00109-012-0928-6. Epub 2012 Jul 6.
CpG-free plasmid expression cassettes for cystic fibrosis gene therapy.
Pringle IA et al., Biomaterials
Biomaterials. 2012 Oct;33(28):6833-42. doi: 10.1016/j.biomaterials.2012.06.009. Epub 2012 Jun 22.
Griesenbach U et al., The Journal Of Gene Medicine
J Gene Med. 2012 Jul;14(7):491-500. doi: 10.1002/jgm.2643.
Davies LA et al., Biomaterials
Biomaterials. 2012 Aug;33(22):5618-27. doi: 10.1016/j.biomaterials.2012.04.019. Epub 2012 May 8.
Oral contraceptives do not appear to affect cystic fibrosis disease severity.
Kernan NG et al., The European Respiratory Journal
Eur Respir J. 2013 Jan;41(1):67-73. doi: 10.1183/09031936.00018712. Epub 2012 May 3.
Progress in gene and cell therapy for cystic fibrosis lung disease.
Griesenbach U et al., Current Pharmaceutical Design
Curr Pharm Des. 2012;18(5):642-62.
2011
Regamey N et al., Thorax
Thorax. 2012 Feb;67(2):164-70. doi: 10.1136/thoraxjnl-2011-200585. Epub 2011 Oct 18.
Bazzani RP et al., Biomaterials
Biomaterials. 2011 Dec;32(36):9854-65. doi: 10.1016/j.biomaterials.2011.08.092. Epub 2011 Sep 23.
Differential global gene expression in cystic fibrosis nasal and bronchial epithelium.
Ogilvie V et al., Genomics
Genomics. 2011 Nov;98(5):327-36. doi: 10.1016/j.ygeno.2011.06.008. Epub 2011 Jul 2.
Design of gene therapy trials in CF patients.
Davies JC et al., Methods In Molecular Biology (Clifton, N.J.)
Methods Mol Biol. 2011;741:55-68. doi: 10.1007/978-1-61779-117-8_5.
McLachlan G et al., Gene Therapy
Gene Ther. 2011 Oct;18(10):996-1005. doi: 10.1038/gt.2011.55. Epub 2011 Apr 21.
Current status and future directions of gene and cell therapy for cystic fibrosis.
Griesenbach U et al., BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals And Gene Therapy
BioDrugs. 2011 Apr 1;25(2):77-88. doi: 10.2165/11586960-000000000-00000.
Griesenbach U et al., Biomaterials
Biomaterials. 2011 Apr;32(10):2614-24. doi: 10.1016/j.biomaterials.2010.12.001. Epub 2011 Jan 15.
2010
Cystic fibrosis: to ion transport and beyond.
Bush A et al., The European Respiratory Journal
Eur Respir J. 2010 Nov;36(5):991-2. doi: 10.1183/09031936.00056310.
Gene therapy for cystic fibrosis.
Davies JC et al., Proceedings Of The American Thoracic Society
Proc Am Thorac Soc. 2010 Nov;7(6):408-14. doi: 10.1513/pats.201004-029AW.
Validation of recombinant Sendai virus in a non-natural host model.
Griesenbach U et al., Gene Therapy
Gene Ther. 2011 Feb;18(2):182-8. doi: 10.1038/gt.2010.131. Epub 2010 Oct 21.
Airway remodelling and its relationship to inflammation in cystic fibrosis.
Regamey N et al., Thorax
Thorax. 2011 Jul;66(7):624-9. doi: 10.1136/thx.2009.134106. Epub 2010 Oct 1.
A novel mixing device for the reproducible generation of nonviral gene therapy formulations.
Davies LA et al., BioTechniques
Biotechniques. 2010 Sep;49(3):666-8. doi: 10.2144/000113498.
Simmonds NJ et al., The European Respiratory Journal
Eur Respir J. 2011 May;37(5):1076-82. doi: 10.1183/09031936.00079010. Epub 2010 Sep 16.
Strategies for long-term expression of transgenes in the respiratory epithelium.
Gill DR et al., Current Opinion In Molecular Therapeutics
Curr Opin Mol Ther. 2010 Aug;12(4):386-93.
Cystic fibrosis gene therapy: successes, failures and hopes for the future.
Griesenbach U et al., Expert Review Of Respiratory Medicine
Expert Rev Respir Med. 2009 Aug;3(4):363-71. doi: 10.1586/ers.09.25.
Griesenbach U et al., American Journal Of Respiratory Cell And Molecular Biology
Am J Respir Cell Mol Biol. 2011 Mar;44(3):309-15. doi: 10.1165/rcmb.2009-0265OC. Epub 2010 Apr 23.
Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.
Mitomo K et al., Molecular Therapy : The Journal Of The American Society Of Gene Therapy
Mol Ther. 2010 Jun;18(6):1173-82. doi: 10.1038/mt.2010.13. Epub 2010 Mar 23.
Sputum and serum calprotectin are useful biomarkers during CF exacerbation.
Gray RD et al., Journal Of Cystic Fibrosis : Official Journal Of The European Cystic Fibrosis Society
J Cyst Fibros. 2010 May;9(3):193-8. doi: 10.1016/j.jcf.2010.01.005. Epub 2010 Mar 17.
Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.
Xenariou S et al., Acta Biochimica Et Biophysica Sinica
Acta Biochim Biophys Sin (Shanghai). 2010 Jan;42(1):45-51.