Papers 121
- Gene therapy for asthma: inspired research or unnecessary effort?
Alton EW et al., Gene Ther. 1999 Feb;6(2):155-6. - Biomarkers for cystic fibrosis: are we progressing?
Alton EW et al., Am J Respir Crit Care Med. 2007 Apr 15;175(8):750-1. - Milking gene therapy.
Alton EW et al., Nat Med. 1998 Oct;4(10):1121-2. - 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. - 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. - 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. - 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. - Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.
Alton EW et al., Lancet. 1999 Mar 20;353(9157):947-54. - Towards gene therapy for cystic fibrosis: a clinical progress report.
Alton EW et al., Gene Ther. 1998 Mar;5(3):291-2. - Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice.
Alton EW et al., Nat Genet. 1993 Oct;5(2):135-42. - Genetic medicines for CF: Hype versus reality.
Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543. - 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 - 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. - 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 - Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy.
Griesenbach U et al., Adv Drug Deliv Rev. 2009 Feb 27;61(2):128-39. doi: 10.1016/j.addr.2008.09.010. Epub 2008 Dec 24. - Design of gene therapy trials in CF patients.
Davies JC et al., Methods Mol Biol. 2011;741:55-68. doi: 10.1007/978-1-61779-117-8_5. - Current status and future directions of gene and cell therapy for cystic fibrosis.
Griesenbach U et al., BioDrugs. 2011 Apr 1;25(2):77-88. doi: 10.2165/11586960-000000000-00000. - Gene therapy for cystic fibrosis.
Davies JC et al., Proc Am Thorac Soc. 2010 Nov;7(6):408-14. doi: 10.1513/pats.201004-029AW. - Cystic fibrosis: ferreting with fibroblasts for cystic fibrosis.
Griesenbach U et al., Gene Ther. 2009 Jan;16(1):1-2. doi: 10.1038/gt.2008.155. Epub 2008 Oct 2. - Monitoring respiratory disease severity in cystic fibrosis.
Davies JC et al., Respir Care. 2009 May;54(5):606-17. - Recent progress in gene therapy for cystic fibrosis.
Griesenbach U et al., Curr Opin Mol Ther. 2001 Aug;3(4):385-9. - Moving forward: cystic fibrosis gene therapy.
Griesenbach U et al., Hum Mol Genet. 2013 Oct 15;22(R1):R52-8. doi: 10.1093/hmg/ddt372. Epub 2013 Aug 4. - Cystic fibrosis.
Davies JC et al., BMJ. 2007 Dec 15;335(7632):1255-9. - Progress in gene and cell therapy for cystic fibrosis lung disease.
Griesenbach U et al., Curr Pharm Des. 2012;18(5):642-62. - Airway gene therapy.
Davies JC et al., Adv Genet. 2005;54:291-314. - Cystic fibrosis gene therapy: successes, failures and hopes for the future.
Griesenbach U et al., Expert Rev Respir Med. 2009 Aug;3(4):363-71. doi: 10.1586/ers.09.25. - Recent advances in understanding and managing cystic fibrosis transmembrane conductance regulator dysfunction.
Griesenbach U et al., F1000Prime Rep. 2015; 7: 64. - Measurement of halide efflux from cultured and primary airway epithelial cells using fluorescence indicators.
Munkonge FM et al., J Cyst Fibros. 2004 Aug;3 Suppl 2:171-6. - Expert opinion in biological therapy: update on developments in lung gene transfer.
Griesenbach U et al., Expert Opin Biol Ther. 2013 Mar;13(3):345-60. doi: 10.1517/14712598.2013.735656. Epub 2013 Jan 5. - Oral contraceptives do not appear to affect cystic fibrosis disease severity.
Kernan NG et al., Eur Respir J. 2013 Jan;41(1):67-73. doi: 10.1183/09031936.00018712. Epub 2012 May 3. - Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis.
Caplen NJ et al., Nat Med. 1995 Jan;1(1):39-46. - Advances in cystic fibrosis gene therapy.
Griesenbach U et al., Curr Opin Pulm Med. 2004 Nov;10(6):542-6. - The pathogenic consequences of a single mutated CFTR gene.
Griesenbach U et al., Thorax. 1999 Aug;54 Suppl 2:S19-23. - Gene therapy progress and prospects: cystic fibrosis.
Griesenbach U et al., Gene Ther. 2006 Jul;13(14):1061-7. - Prospects for gene therapy in lung disease.
Davies JC et al., Curr Opin Pharmacol. 2001 Jun;1(3):272-7. - Gene therapy for cystic fibrosis: an example for lung gene therapy.
Griesenbach U et al., Gene Ther. 2004 Oct;11 Suppl 1:S43-50. - Gene therapy for cystic fibrosis.
Davies JC et al., J Gene Med. 2001 Sep-Oct;3(5):409-17. - Computed tomography and cystic fibrosis: promises and problems.
Aziz ZA et al., Thorax. 2007 Feb;62(2):181-6. - Update on gene therapy for cystic fibrosis.
Griesenbach U et al., Curr Opin Mol Ther. 2003 Oct;5(5):489-94. - Cystic Fibrosis Gene Therapy in the UK and elsewhere
Griesenbach U et al., Hum Gene Ther. 2015 May;26(5):266-75. - Airway remodelling and its relationship to inflammation in cystic fibrosis.
Regamey N et al., Thorax. 2011 Jul;66(7):624-9. doi: 10.1136/thx.2009.134106. Epub 2010 Oct 1. - Progress and prospects: gene therapy clinical trials (part 1).
Alexander BL et al., Gene Ther. 2007 Oct;14(20):1439-47. - 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. - Multiple breath washouts in children can be shortened without compromising quality.
Ahmad F et al., Eur Respir J. 2015 Dec;46(6):1814-6. doi: 10.1183/13993003.00791-2015. Epub 2015 Oct 9. - Prospects for gene therapy for cystic fibrosis.
Davies JC et al., Mol Med Today. 1998 Jul;4(7):292-9. - Lung clearance index in primary ciliary dyskinesia and bronchiectasis.
Irving SJ et al., Am J Respir Crit Care Med. 2014 May 1;189(9):1147-8. doi: 10.1164/rccm.201402-0206LE. - Gene therapy progress and prospects: cystic fibrosis.
Griesenbach U et al., Gene Ther. 2002 Oct;9(20):1344-50. - Sendai virus for gene therapy and vaccination.
Griesenbach U et al., Curr Opin Mol Ther. 2005 Aug;7(4):346-52. - Gene therapy in cystic fibrosis.
Armstrong DK et al., Arch Dis Child. 2014 May;99(5):465-8. doi: 10.1136/archdischild-2012-302158. Epub 2014 Jan 24. - Does mass spectrometric breath analysis detect Pseudomonas aeruginosa in cystic fibrosis?
Pabary R et al., Eur Respir J. 2016 Mar;47(3):994-7. doi: 10.1183/13993003.00944-2015. Epub 2016 Feb 4. - Cystic fibrosis and survival to 40 years: a study of cystic fibrosis transmembrane conductance regulator function.
Simmonds NJ et al., Eur Respir J. 2011 May;37(5):1076-82. doi: 10.1183/09031936.00079010. Epub 2010 Sep 16. - Validation of nasal potential difference measurements in gut-corrected CF knockout mice.
Griesenbach U et al., Am J Respir Cell Mol Biol. 2008 Oct;39(4):490-6. doi: 10.1165/rcmb.2007-0385OC. Epub 2008 May 5. - Beta-defensin genomic copy number is not a modifier locus for cystic fibrosis.
Hollox EJ et al., J Negat Results Biomed. 2005 Dec 7;4:9. - Emerging significance of plasmid DNA nuclear import in gene therapy.
Munkonge FM et al., Adv Drug Deliv Rev. 2003 Jun 16;55(6):749-60. - Airway remodelling in children with cystic fibrosis.
Hilliard TN et al., Thorax. 2007 Dec;62(12):1074-80. Epub 2007 May 25. - A molecular comparison of microbial communities in bronchiectasis and cystic fibrosis.
Duff RM et al., Eur Respir J. 2013 Apr;41(4):991-3. doi: 10.1183/09031936.00052712. - Normal nasal mucociliary clearance in CF children: evidence against a CFTR-related defect.
McShane D et al., Eur Respir J. 2004 Jul;24(1):95-100. - Bone marrow stem cells do not repopulate the healthy upper respiratory tract.
Davies JC et al., Pediatr Pulmonol. 2002 Oct;34(4):251-6. - Magnetic nanoparticles as gene delivery agents: enhanced transfection in the presence of oscillating magnet arrays.
McBain SC et al., Nanotechnology. 2008 Oct 8;19(40):405102. doi: 10.1088/0957-4484/19/40/405102. Epub 2008 Aug 20. - Cytoplasmic deposition of NFkappaB decoy oligonucleotides is insufficient to inhibit bleomycin-induced pulmonary inflammation.
Griesenbach U et al., Gene Ther. 2002 Aug;9(16):1109-15. - 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. - Airway surface pH in subjects with cystic fibrosis.
McShane D et al., Eur Respir J. 2003 Jan;21(1):37-42. - Anti-inflammatory gene therapy directed at the airway epithelium.
Griesenbach U et al., Gene Ther. 2000 Feb;7(4):306-13. - Reduction in the adherence of Pseudomonas aeruginosa to native cystic fibrosis epithelium with anti-asialoGM1 antibody and neuraminidase inhibition.
Davies JC et al., Eur Respir J. 1999 Mar;13(3):565-70. - Exploring the mechanisms of macrolides in cystic fibrosis.
Equi AC et al., Respir Med. 2006 Apr;100(4):687-97. Epub 2005 Sep 26. - Expression and maturation of Sendai virus vector-derived CFTR protein: functional and biochemical evidence using a GFP-CFTR fusion protein.
Ban H et al., Gene Ther. 2007 Dec;14(24):1688-94. Epub 2007 Sep 27. - Cyanide levels found in infected cystic fibrosis sputum inhibit airway ciliary function.
Nair C et al., Eur Respir J. 2014 Nov;44(5):1253-61. doi: 10.1183/09031936.00097014. Epub 2014 Sep 3. - Mannose-binding lectin is present in the infected airway: a possible pulmonary defence mechanism.
Fidler KJ et al., Thorax. 2009 Feb;64(2):150-5. doi: 10.1136/thx.2008.100073. Epub 2008 Nov 6. - Effects of intramyocardial pVEGF165 delivery on regional myocardial blood flow: evidence for a spatial 'delivery-efficacy' mismatch.
Radke PW et al., Gene Ther. 2004 Aug;11(16):1249-55. - CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector.
Rakonczay Z Jr et al., J Cell Physiol. 2008 Feb;214(2):442-55. - Intravenously administered oligonucleotides can be delivered to conducting airway epithelium via the bronchial circulation.
Holder E et al., Gene Ther. 2006 Dec;13(23):1628-38. Epub 2006 Jun 22. - Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis.
Regamey N et al., Thorax. 2012 Feb;67(2):164-70. doi: 10.1136/thoraxjnl-2011-200585. Epub 2011 Oct 18. - Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium.
Griesenbach U et al., J Gene Med. 2012 Jul;14(7):491-500. doi: 10.1002/jgm.2643. - Pseudomonas aeruginosa infection in cystic fibrosis: pathophysiological mechanisms and therapeutic approaches.
Lund-Palau H et al., Expert Rev Respir Med. 2016 Jun;10(6):685-97. doi: 10.1080/17476348.2016.1177460. Epub 2016 May 13. - 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. - Increased airway smooth muscle mass in children with asthma, cystic fibrosis, and non-cystic fibrosis bronchiectasis.
Regamey N et al., Am J Respir Crit Care Med. 2008 Apr 15;177(8):837-43. doi: 10.1164/rccm.200707-977OC. Epub 2008 Jan 24. - Endobronchial biopsy in childhood.
Regamey N et al., Chest. 2008 Jan;133(1):312; author reply 313. doi: 10.1378/chest.07-1735. - 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. - Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice.
Davies LA et al., Mol Ther. 2008 May;16(5):812-8. doi: 10.1038/mt.2008.25. Epub 2008 Mar 11. - Quality, size, and composition of pediatric endobronchial biopsies in cystic fibrosis.
Regamey N et al., Chest. 2007 Jun;131(6):1710-7. Epub 2007 Feb 22. - Critical appraisal of the mouse model of myocardial infarction.
Degabriele NM et al., Exp Physiol. 2004 Jul;89(4):497-505. Epub 2004 May 6. - Ex vivo and in vivo lentivirus-mediated transduction of airway epithelial progenitor cells.
Leoni G et al., Curr Gene Ther. 2015;15(6):581-90. - Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo.
Xenariou S et al., Gene Ther. 2006 Nov;13(21):1545-52. Epub 2006 Jun 1. - The role of doxorubicin in non-viral gene transfer in the lung.
Griesenbach U et al., Biomaterials. 2009 Apr;30(10):1971-7. doi: 10.1016/j.biomaterials.2008.12.037. Epub 2009 Jan 18. - Lung clearance index and high-resolution computed tomography scores in primary ciliary dyskinesia.
Irving SJ et al., Am J Respir Crit Care Med. 2013 Sep 1;188(5):545-9. doi: 10.1164/rccm.201304-0800OC. - Assessment of CFTR function after gene transfer in vitro and in vivo.
Griesenbach U et al., Methods Mol Biol. 2008;433:229-42. doi: 10.1007/978-1-59745-237-3_14. - Quantification of periciliary fluid height in human airway biopsies is feasible, but not suitable as a biomarker.
Griesenbach U et al., Am J Respir Cell Mol Biol. 2011 Mar;44(3):309-15. doi: 10.1165/rcmb.2009-0265OC. Epub 2010 Apr 23. - Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35. - Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli.
Larsen MD et al., Gene Ther. 2008 Mar;15(6):434-42. doi: 10.1038/sj.gt.3303090. Epub 2008 Jan 24. - Nasal abnormalities in cystic fibrosis mice independent of infection and inflammation.
Hilliard TN et al., Am J Respir Cell Mol Biol. 2008 Jul;39(1):19-25. doi: 10.1165/rcmb.2007-0284OC. Epub 2008 Jan 31. - Vascular oligonucleotide transfer facilitated by a polymer-coated stent.
Radke PW et al., Hum Gene Ther. 2005 Jun;16(6):734-40. - Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lung.
Griesenbach U et al., Gene Ther. 2006 Mar;13(5):449-56. - Differential global gene expression in cystic fibrosis nasal and bronchial epithelium.
Ogilvie V et al., Genomics. 2011 Nov;98(5):327-36. doi: 10.1016/j.ygeno.2011.06.008. Epub 2011 Jul 2. - CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium.
Davies JC et al., Am J Respir Cell Mol Biol. 1997 Jun;16(6):657-63. - The effect of mucolytic agents on gene transfer across a CF sputum barrier in vitro.
Stern M et al., Gene Ther. 1998 Jan;5(1):91-8. - Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes.
Davies LA et al., Mol Ther. 2008 Jul;16(7):1283-90. doi: 10.1038/mt.2008.96. Epub 2008 May 20. - A defective nontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo.
Ferrari S et al., Gene Ther. 2004 Nov;11(22):1659-64. - The nasal epithelium as a factory for systemic protein delivery.
Griesenbach U et al., Mol Ther. 2002 Feb;5(2):98-103. - 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. - Potential difference measurements in the lower airway of children with and without cystic fibrosis.
Davies JC et al., Am J Respir Crit Care Med. 2005 May 1;171(9):1015-9. Epub 2005 Jan 7. - Low-frequency ultrasound increases non-viral gene transfer to the mouse lung.
Xenariou S et al., Acta Biochim Biophys Sin (Shanghai). 2010 Jan;42(1):45-51. - Keratinocyte growth factor therapy in murine oleic acid-induced acute lung injury.
Ulrich K et al., Am J Physiol Lung Cell Mol Physiol. 2005 Jun;288(6):L1179-92. Epub 2005 Jan 28. - Validation of recombinant Sendai virus in a non-natural host model.
Griesenbach U et al., Gene Ther. 2011 Feb;18(2):182-8. doi: 10.1038/gt.2010.131. Epub 2010 Oct 21. - Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segments.
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Xenariou S et al., Gene Ther. 2007 May;14(9):768-74. Epub 2007 Feb 15. - Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity.
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Griesenbach U et al., Biomaterials. 2008 Apr;29(10):1533-40. Epub 2007 Dec 21. - Optimizing aerosol gene delivery and expression in the ovine lung.
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Yonemitsu Y et al., Nat Biotechnol. 2000 Sep;18(9):970-3. - Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy.
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Abstracts 43
- 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) - Repeat Administration of Gl67A/pGM169 Is Feasible, Safe, and Produces Endogenous Levels of CFTR Expression After 12 Doses.
Alton EW et al.,British Thoracic Society Winter Meeting (2012) - A randomized, double-blind, placebo-controlled trial of repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis.
Alton EW et al.,The North American Cystic Fibrosis Conference (2015) - Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
Alton EW et al.,British Thoracic Society Winter Meeting (2012) - Moving gene therapy into CF patients.
Alton EW et al.,UK Cystic Fibrosis Conference (2015) - Gene Therapy: Moving to the Clinic.
Alton EW et al.,European Cystic Fibrosis Conference (2015) - 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) - Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
Hyde SC et al.,British Thoracic Society Winter Meeting (2014) - 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) - 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) - Moving lentiviral-based gene therapy into a first-in-man CF trial.
Griesenbach U et al.,The North American Cystic Fibrosis Conference (2015) - 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) - Production of rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
Hyde SC et al.,The North American Cystic Fibrosis Conference (2015) - 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) - Clinical Development of an Optimal F/HN Pseudotyped SIV Vector for Cystic Fibrosis Lung Gene Therapy.
Pringle IA et al.,The American Society of Gene and Cell Therapy Annual Conference (2014) - Standardisation of Lung Clearance Index in a Multicentre Clinical Trial.
Armstrong DK et al.,British Thoracic Society Winter Meeting (2014) - Self-Reactive T Cells to CFTR in Cystic Fibrosis (CF) and Non-CF Humans: Implications for Gene Therapy.
Limberis MP et al.,The American Society of Gene and Cell Therapy Annual Conference (2011) - Alpha-1-antitrypsin gene therapy for lung disease.
Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2014) - A clinical study to evaluate the safety and efficacy of pGM169/GL67A administered to the nose and lungs of individuals with cystic fibrosis.
Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2010) - Repeated Exposure to pDNA/PEI Aerosols Results in Minimal Detectable Toxicity in the Mouse Lungs .
Nunez-Alonso GA et al.,The American Society of Gene Therapy Annual Conference (2009) - Mutliple Doses of Lipid Mediated Gene Therapy Nebulised to the Mouse Lung Show Robust and Sustained CFTR Expression.
Hyde SC et al.,The North American Cystic Fibrosis Conference (2011) - Non-viral gene expression in the lung using the mini-CFTR promoter.
Connolly MM et al.,The British Society of Gene Therapy Annual Conference (2009) - The nuclear pore dilating Agent TCHD increases gene transfer into differentiated airway epithelium ex vivo, but has no efect in vivo.
Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2011) - Novel CPG-Depleted and Codon-Optimised CFTR CDNAs Maintain the Structure and Function of CFTR Protein.
Varathalingam A et al.,The North American Cystic Fibrosis Conference (2005) - Novel CpG depleted and codon optimised CFTR cDNAs maintain the structure and fuction of CFTR protein.
Varathalingam A et al.,British Society of Gene Therapy Conference (2006) - 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) - Strain-Specific Differences in Pulmonary Gene Transfer Efficiency: Relevance for Toxicology Studies in Mice.
Legakis G et al.,The American Society of Gene and Cell Therapy Annual Conference (2011) - Scalable, Animal-Free, Suspension-Based Production of SIV Lentiviral Vectors.
Hyde SC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015) - SIV Vector Pseudotyped with SeV-F/HN Envelope Proteins Produces Long Lasting Expression in the Murine Lung, Is Readministrable and Transfects Human Airway Models.
Griesenbach U et al.,The American Society of Gene and Cell Therapy Annual Conference (2010) - Optimisation of molecular assays for clinical trial of GL67A/pGM169 delivery to nose and lung of CF patients.
Sumner-Jones SG et al.,The North American Cystic Fibrosis Conference (2009) - Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice.
Davies L et al.,The British Society of Gene Therapy Annual Conference (2008) - Pre-existing immunity to human parainfluenza virus (hPIV) does not affect rSIV.F/HN-mediated transduction efficiency.
Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2016) - 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) - 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) - Calculating the percentage of cells transfected following non-viral delivery to the respiratory epithelium.
Pringle IA et al.,The American Society of Gene Therapy Annual Conference (2009) - Production of FVIII in the lungs.
Pytel KM et al.,The British Society of Gene Therapy Annual Conference (2015) - Therapeutic levels of alpha-1-antitrypsin following gene therapy with F/HN pseudotyped simian immunodeficiency virus.
Paul-Smith M et al.,The British Society of Gene Therapy Annual Conference (2015) - 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) - Production of Therapeutically Relevant Levels of FVIII After Transduction of Lungs With F/HN-Pseudotyped Lentivirus.
Pytel KM et al.,The American Society of Gene and Cell Therapy Annual Conference (2015) - F/HN Pseudotyped Lentivirus Generates Therapeutically Relevant and Long-Lasting Alpha-1-Antitrypsin Expression in Mouse Lung.
Paul-Smith MC et al.,The American Society of Gene and Cell Therapy Annual Conference (2015) - Secreted Gaussia Luciferase Is a More Sensitive Reporter Than Firefly Luciferase for Non- Viral Gene Transfer to Airway Epithelium Ex Vivo and In Vivo.
Griesenbach U et al.,The American Society of Gene Therapy Annual Conference (2009) - 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) - Assessment of FHN-Pseudotyped Lentivirus as a Clinically Relevant Vector For Lung Gene Therapy.
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Lentiviral Production in Wave Bioreactor (2015): Suspension cell culture is used to produce large quantities of Lentiviral vector. The rocking platform generates a 'wave' in the disposable bag which keeps cells in suspension. More...
