Papers 58
- 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.
- Monitoring respiratory disease severity in cystic fibrosis.
Davies JC et al., Respir Care. 2009 May;54(5):606-17.
- 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.
Davies JC et al., BMJ. 2007 Dec 15;335(7632):1255-9.
- 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.
- Airway gene therapy.
Davies JC et al., Adv Genet. 2005;54:291-314.
- 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.
- Prospects for gene therapy in lung disease.
Davies JC et al., Curr Opin Pharmacol. 2001 Jun;1(3):272-7.
- Bone marrow stem cells do not repopulate the healthy upper respiratory tract.
Davies JC et al., Pediatr Pulmonol. 2002 Oct;34(4):251-6.
- Gene therapy for cystic fibrosis.
Davies JC et al., J Gene Med. 2001 Sep-Oct;3(5):409-17.
- 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.
- Prospects for gene therapy for cystic fibrosis.
Davies JC et al., Mol Med Today. 1998 Jul;4(7):292-9.
- 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.
- Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation.
Horsley AR et al., Thorax. 2013 Jun;68(6):532-9. doi: 10.1136/thoraxjnl-2012-202538. Epub 2013 Feb 9.
- 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.
- Computed tomography and cystic fibrosis: promises and problems.
Aziz ZA et al., Thorax. 2007 Feb;62(2):181-6.
- 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.
- Exploring the mechanisms of macrolides in cystic fibrosis.
Equi AC et al., Respir Med. 2006 Apr;100(4):687-97. Epub 2005 Sep 26.
- Biomarkers for cystic fibrosis: are we progressing?
Alton EW et al., Am J Respir Crit Care Med. 2007 Apr 15;175(8):750-1.
- Airway surface pH in subjects with cystic fibrosis.
McShane D et al., Eur Respir J. 2003 Jan;21(1):37-42.
- 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.
- Cystic fibrosis: to ion transport and beyond.
Bush A et al., Eur Respir J. 2010 Nov;36(5):991-2. doi: 10.1183/09031936.00056310.
- 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.
- Genetic medicines for CF: Hype versus reality.
Alton EW et al., Pediatr Pulmonol. 2016 Oct;51(S44):S5-S17. doi: 10.1002/ppul.23543.
- 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.
- 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.
- Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence.
Scheid P et al., Eur Respir J. 2001 Jan;17(1):27-35.
- 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.
- 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.
- 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.
- 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.
- 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
- 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.
- Airway remodelling in children with cystic fibrosis.
Hilliard TN et al., Thorax. 2007 Dec;62(12):1074-80. Epub 2007 May 25.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Endobronchial biopsy in childhood.
Regamey N et al., Chest. 2008 Jan;133(1):312; author reply 313. doi: 10.1378/chest.07-1735.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression.
Hyde SC et al., Nat Biotechnol. 2008 May;26(5):549-51. doi: 10.1038/nbt1399. Epub 2008 Apr 27.
- 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.
- An immunocytochemical assay to detect human CFTR expression following gene transfer.
Davidson H et al., Mol Cell Probes. 2009 Dec;23(6):272-80. doi: 10.1016/j.mcp.2009.07.001. Epub 2009 Jul 15.
- 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 Ther. 2011 Oct;18(10):996-1005. doi: 10.1038/gt.2011.55. Epub 2011 Apr 21.
- 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.
- Limitations of the murine nose in the development of nonviral airway gene transfer.
Griesenbach U et al., Am J Respir Cell Mol Biol. 2010 Jul;43(1):46-54. doi: 10.1165/rcmb.2009-0075OC. Epub 2009 Jul 31.
Abstracts 28
- Current issues around LCI (Lung Clearance Index).
Davies JC et al.,UK Cystic Fibrosis Conference (2015)
- 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)
- 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 rSIV-F/HN: a new Lentivirus vector for CF gene therapy.
Hyde SC et al.,The North American Cystic Fibrosis Conference (2015)
- 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)
- 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)
- 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)
- 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)
- 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)
- 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)
- Immune Responses to Single and Repeated Administration of pGM169/GL67A, The UK CF Gene Therapy Consortium Clinical Trials.
Griesenbach U et al.,British Thoracic Society Winter Meeting (2014)
- 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)
- Update on The UK CF Gene Therapy Consortium Multidose, Non-Viral, Gene Therapy Trial.
Alton EW et al.,British Thoracic Society Winter Meeting (2012)
- Development of an optimal F/HN pseudotyped SIV vector for CF gene therapy.
Hyde SC et al.,British Thoracic Society Winter Meeting (2014)
- Moving lentiviral-based gene therapy into a first-in-man CF trial.
Griesenbach U 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.,The North American Cystic Fibrosis Conference (2012)
- 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)
- Optimising Harvest of Bronchial Brush Biopsy Samples To Maximise Cell and RNA Yield in Gene Therapy Studies.
Vrettou C et al.,The American Society of Gene Therapy Annual Conference (2009)
- 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)
- 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)
- 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)
- 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)
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