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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Multiple Inhibitory Factors Act in the Late Phase of HIV-1 Replication: a Systematic Review of the Literature.

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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Deficient retinoid-driven angiogenesis may contribute to failure of adult human lung regeneration in emphysema.

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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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
Efficacy and Mechanism Evaluation (2016) Volume: 3 Issue: 5    
 

Pseudomonas aeruginosa infection in cystic fibrosis: pathophysiological mechanisms and therapeutic approaches.

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.   
 

Transgene sequences free of CG dinucleotides lead to high level, long-term expression in the lung independent of plasmid backbone design.

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.   
 

Human-Mouse Chimeras With Normal Expression and Function Reveal That Major Domain Swapping is Tolerated by P-glycoprotein (ABCB1).

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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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., 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.    
 

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., 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.    
 

Recent advances in understanding and managing cystic fibrosis transmembrane conductance regulator dysfunction.

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.   
 

Measurement of Serum Calprotectin in Stable Patients Predicts Exacerbation and Lung Function Decline in Cystic Fibrosis.

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.   
 

Effects of a novel archaeal tetraether-based colipid on the in vivo gene transfer activity of two cationic amphiphiles.

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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Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung.

Alton EW et al., Gene Therapy

Gene Ther. 2014 Jan;21(1):89-95. doi: 10.1038/gt.2013.61. Epub 2013 Nov 7.    
 

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

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.    
 

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

Thorax. 2013 Nov;68(11):1075-7. doi: 10.1136/thoraxjnl-2013-203309. Epub 2013 Mar 22.   
 

Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation.

Horsley AR et al., Thorax

Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.   
 

Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis.

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.    
 

Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.

Griesenbach U et al., The Journal Of Gene Medicine
J Gene Med. 2012 Jul;14(7):491-500. doi: 10.1002/jgm.2643.   
 

The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes.

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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Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis.

Regamey N et al., Thorax
Thorax. 2012 Feb;67(2):164-70. doi: 10.1136/thoraxjnl-2011-200585. Epub 2011 Oct 18.   
 

The significance of plasmid DNA preparations contaminated with bacterial genomic DNA on inflammatory responses following delivery of lipoplexes to the murine lung.

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.   
 

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 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.   
 

Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer.

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.    
 

Cystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function.

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.   
 

Quantification of periciliary fluid height in human airway biopsies is feasible, but not suitable as a biomarker.

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.