Publications
Below is a list of Scientific publications detailing work undertaken by the gene therapy consortium.
2019
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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
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Gelinas JF et al., Microbiology and Molecular Biology Reviews
Microbiol Mol Biol Rev. 2018 Jan 10;82(1). pii: e00051-17
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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.
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2017
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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.
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Ng-Blichfeldt JP et al., Thorax
Thorax. 2017 Jun;72(6):510-521. doi: 10.1136/thoraxjnl-2016-208846.
2016
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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.
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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.
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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.
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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
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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
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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.
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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
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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
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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
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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
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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.