A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis (2013). Alton, E. W. et al., Thorax, 68, 1075-1077.
Toxicology study assessing efficacy and safety of repeated administration of lipid/DNA complexes to mouse lung (2013). Alton, E. W. et al., Gene Ther, Epub ahead of print.
Self-Reactive CFTR T Cells in Humans: Implications for Gene Therapy (2013). Calcedo, R. et al., Hum Gene Ther Clin Dev, 24, 108-115.
Pharmacological Characterization of a Novel ENaCalpha siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis (2013). Clark, K. L. et al., Mol Ther Nucleic Acids, 2, e65.
Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy (2012). Griesenbach, U. et al., Am J Respir Crit Care Med, E-Pub Ahead of Print.
Clinical Expert Panel on Monitoring Potential Lung Toxicity of Inhaled Oligonucleotides: Consensus Points and Recommendations (2012). Alton, E. W. et al., Nucleic Acid Ther, E-Pub Ahead of Print.
Rapid identification of novel functional promoters for gene therapy (2012). Pringle, I.A. et al., Journal of Molecular Medicine, June 5, [Epub ahead of print].
Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium (2012). Griesenbach, U. et al., J Gene Med, 14, 491-500.
CpG-free plasmid expression cassettes for cystic fibrosis gene therapy (2012). Pringle, I.A. et al., Biomaterials, 33, 6833-68428.
The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes (2012). Davies, L. A. et al., Biomaterials, 33, 5618-27.
Progress in gene and cell therapy for cystic fibrosis lung disease (2012). Griesenbach, U. et al., Curr Pharm Des. 18(5):642-62
The significance of plasmid DNA preparations contaminated with bacterial genomic DNA on inflammatory responses following delivery of lipoplexes to the murine lung (2011). Bazzani, R. P. et al., Biomaterials, 32, 9854-9865.
Differential global gene expression in cystic fibrosis nasal and bronchial epithelium. Ogilvie, V et al. Genomics 2011 Nov;98(5):327-36.
Design of gene therapy trials in CF patients. Davies, JC. et al. Methods Mol Biol. 2011;741:55-68.
Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung (2011). McLachlan, G. et al. Gene Ther. 2011 Oct;18(10):996-1005.
Current status and future directions of gene and cell therapy for cystic fibrosis (2011). Griesenbach, U. & Alton, E. W., BioDrugs, 2011 Apr 1; 25, 77-88.
Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer (2011). Griesenbach, U. et al., Biomaterials, 32, 2614-2624.
Validation of recombinant Sendai virus in a non-natural host model (2011). Griesenbach, U. et al. Gene Ther. 2011 Feb;18(2):182-8.
Airway remodelling and its relationship to inflammation in cystic fibrosis (2011). Regamey, N. et al. Thorax 2011 Jul;66(7):624-9.
Quantification of Periciliary Fluid (PCL) Height in Human Airway Biopsies is Feasible, but not Suitable as a Biomarker (2011). Griesenbach, U. et al., Am J Respir Cell Mol Biol. 2011 Mar;44(3):309-15
Low-frequency ultrasound increases non-viral gene transfer to the mouse lung (2010). Xenariou, S. et al., Acta Biochim Biophys Sin (Shanghai), 42, 45-51. Download
A novel mixing device for the reproducible generation of nonviral gene therapy formulations (2010). Davies, L. A. et al., Biotechniques, 49, 666-668. Download
Cystic fibrosis and survival to 40 years: a study of CFTR function (2010). Simmonds, N. J. et al., Eur Respir J.
Gene therapy for cystic fibrosis (2010). Davies, J. C. & Alton, E. W., Proc Am Thorac Soc, 7, 408-414.
Strategies for long-term expression of transgenes in the respiratory epithelium (2010). Gill, D. R. et al., Curr Opin Mol Ther, 12, 386-393.
SELDI-TOF biomarker signatures for cystic fibrosis, asthma and chronic obstructive pulmonary disease (2010). Gomes-Alves, P. et al., Clin Biochem, 43, 168-177.
Sputum trace metals are biomarkers of inflammatory and suppurative lung disease (2010). Gray, R. D. et al., Chest, 137, 635-641.
The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways (2010). Griesenbach, U. et al., Biomaterials, 31, 2665-2672.
Limitations of the murine nose in the development of nonviral airway gene transfer (2010). Griesenbach, U. et al., Am J Respir Cell Mol Biol, 43, 46-54.
Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity (2010). Holder, E. et al., J Gene Med, 12, 55-63.
Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes (2010). Mitomo, K. et al., Mol Ther, 18, 1173-1182.
Cystic fibrosis: ferreting with fibroblasts for cystic fibrosis (2009). Griesenbach, U. & Alton, E. W., Gene Ther, 16, 1-2. Download
Identification of protein cofactors necessary for sequence-specific plasmid DNA nuclear import (2009). Miller, A. M. et al., Mol Ther, 17, 1897-1903. Download
Identification and functional characterization of cytoplasmic determinants of plasmid DNA nuclear import (2009). Munkonge, F. M. et al., J Biol Chem, 284, 26978-26987. Download
An immunocytochemical assay to detect human CFTR expression following gene transfer (2009). Davidson, H. et al., Mol Cell Probes, 23, 272-280.
Monitoring respiratory disease severity in cystic fibrosis (2009). Davies, J. C. & Alton, E. W., Respir Care, 54, 606-617.
Mannose-binding lectin is present in the infected airway: a possible pulmonary defence mechanism (2009). Fidler, K. J. et al., Thorax, 64, 150-155.
Progress and prospects: the design and production of plasmid vectors (2009). Gill, D. R. et al., Gene Ther, 16, 165-171.
Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy (2009). Griesenbach, U. & Alton, E. W., Adv Drug Deliv Rev, 61, 128-139.
Cystic fibrosis gene therapy: successes, failures and hopes for the future (2009). Griesenbach, U. & Alton, E. W., Expert Rev Respir Med, 3, 363-371.
The role of doxorubicin in non-viral gene transfer in the lung (2009). Griesenbach, U. et al., Biomaterials, 30, 1971-1977.
Non-viral vectors in cystic fibrosis gene therapy: recent developments and future prospects (2009). Pringle, I. A. et al., Expert Opin Biol Ther, 9, 991-1003.
Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes (2008). Davies, L. A. et al., Mol Ther, 16, 1283-1290. Download
Adenovirus-mediated in utero expression of CFTR does not improve survival of CFTR knockout mice (2008). Davies, L. A. et al., Mol Ther, 16, 812-818. Download
Sputum proteomics in inflammatory and suppurative respiratory diseases (2008). Gray, R. D. et al., Am J Respir Crit Care Med, 178, 444-452. Download
Validation of nasal potential difference measurements in gut-corrected CF knockout mice (2008). Griesenbach, U. et al., Am J Respir Cell Mol Biol, 39, 490-496. Download
Nasal abnormalities in cystic fibrosis mice independent of infection and inflammation (2008). Hilliard, T. N. et al., Am J Respir Cell Mol Biol, 39, 19-25. Download
Increased airway smooth muscle mass in children with asthma, cystic fibrosis, and non-cystic fibrosis bronchiectasis (2008). Regamey, N. et al., Am J Respir Crit Care Med, 177, 837-843. Download
Lung clearance index in CF: a sensitive marker of lung disease severity (2008). Davies, J. C. et al., Thorax, 63, 96-97.
In vivo imaging of gene transfer to the respiratory tract (2008). Griesenbach, U. et al., Biomaterials, 29, 1533-1540.
Assessment of CFTR function after gene transfer in vitro and in vivo (2008). Griesenbach, U. et al., Methods Mol Biol, 433, 229-242.
Lung clearance index is a sensitive, repeatable and practical measure of airways disease in adults with cystic fibrosis (2008). Horsley, A. R. et al., Thorax, 63, 135-140.
Ignoring the nonsense: a phase II trial in cystic fibrosis (2008). Hyde, S. C. & Gill, D. R., Lancet, 372, 691-692.
CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression (2008). Hyde, S. C. et al., Nat Biotechnol, 26, 549-551.
Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coli (2008). Larsen, M. D. et al., Gene Ther, 15, 434-442.
Biomarkers for cystic fibrosis lung disease: application of SELDI-TOF mass spectrometry to BAL fluid (2008). MacGregor, G. et al., J Cyst Fibros, 7, 352-358.
CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector (2008). Rakonczay, Z., Jr. et al., J Cell Physiol, 214, 442-455.
Tripod-like cationic lipids as novel gene carriers (2008). Unciti-Broceta, A. et al., J Med Chem, 51, 4076-4084.
Biomarkers for cystic fibrosis: are we progressing? (2007). Alton, E. W. et al., Am J Respir Crit Care Med, 175, 750-751. Download
Optimizing aerosol gene delivery and expression in the ovine lung (2007). McLachlan, G. et al., Mol Ther, 15, 348-354. Download
Quality, size, and composition of pediatric endobronchial biopsies in cystic fibrosis (2007). Regamey, N. et al., Chest, 131, 1710-1717. Download
Chimeric constructs endow the human CFTR Cl- channel with the gating behavior of murine CFTR (2007). Scott-Ward, T. S. et al., Proc Natl Acad Sci U S A, 104, 16365-16370. Download
Lack of repeat transduction by recombinant adeno-associated virus type 5/5 vectors in the mouse airway (2007). Sumner-Jones, S. G. et al., J Virol, 81, 12360-12367. Download
Human-specific cystic fibrosis transmembrane conductance regulator antibodies detect in vivo gene transfer to ovine airways (2006). Davidson, H. et al., Am J Respir Cell Mol Biol, 35, 72-83. Download
Progress and prospects: gene therapy clinical trials (part 1) (2007). Alexander, B. L. et al., Gene Ther, 14, 1439-1447.
Computed tomography and cystic fibrosis: promises and problems (2007). Aziz, Z. A. et al., Thorax, 62, 181-186.
Expression and maturation of Sendai virus vector-derived CFTR protein: functional and biochemical evidence using a GFP-CFTR fusion protein (2007). Ban, H. et al., Gene Ther, 14, 1688-1694.
Cystic fibrosis (2007). Davies, J. C. et al., BMJ, 335, 1255-1259.
Identification of transfected cell types following non-viral gene transfer to the murine lung (2007). Davies, L. A. et al., J Gene Med, 9, 184-196.
Sendai virus-mediated CFTR gene transfer to the airway epithelium (2007). Ferrari, S. et al., Gene Ther, 14, 1371-1379.
Airway remodelling in children with cystic fibrosis (2007). Hilliard, T. N. et al., Thorax, 62, 1074-1080.
Electroporation enhances reporter gene expression following delivery of naked plasmid DNA to the lung (2007). Pringle, I. A. et al., J Gene Med, 9, 369-380.
Use of ultrasound to enhance nonviral lung gene transfer in vivo (2007). Xenariou, S. et al., Gene Ther, 14, 768-774.
Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lung (2006). Griesenbach, U. et al., Gene Ther, 13, 449-456. Download
Gene therapy progress and prospects: cystic fibrosis (2006). Griesenbach, U. et al., Gene Ther, 13, 1061-1067. Download
Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo (2006). Griesenbach, U. et al., Respir Res, 7, 26. Download
Intravenously administered oligonucleotides can be delivered to conducting airway epithelium via the bronchial circulation (2006). Holder, E. et al., Gene Ther, 13, 1628-1638. Download
Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways (2006). Sumner-Jones, S. G. et al., Gene Ther, 13, 1703-1713. Download
Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivo (2006). Xenariou, S. et al., Gene Ther, 13, 1545-1552. Download
Exploring the mechanisms of macrolides in cystic fibrosis (2006). Equi, A. C. et al., Respir Med, 100, 687-697.
Nondisruptive, sequence-specific coupling of fluorochromes to plasmid DNA (2006). Hillery, E. et al., Anal Biochem, 352, 169-175.
Optimising non-viral gene delivery in a tumour spheroid model (2006). Mellor, H. R. et al., J Gene Med, 8, 1160-1170.
Electroporation-mediated interleukin-10 overexpression in skeletal muscle reduces acute rejection in rat cardiac allografts (2006). Tavakoli, R. et al., J Gene Med, 8, 242-248.
Potential difference measurements in the lower airway of children with and without cystic fibrosis (2005). Davies, J. C. et al., Am J Respir Crit Care Med, 171, 1015-1019. Download
The efficacy of a 'master switch gene' HIF-1alpha in a porcine model of chronic myocardial ischaemia (2005). Heinl-Green, A. et al., Eur Heart J, 26, 1327-1332. Download
Beta-defensin genomic copy number is not a modifier locus for cystic fibrosis (2005). Hollox, E. J. et al., J Negat Results Biomed, 4, 9. Download
Detection of plasmid DNA vectors following gene transfer to the murine airways (2005). Pringle, I. A. et al., Gene Ther, 12, 1206-1214. Download
Keratinocyte growth factor therapy in murine oleic acid-induced acute lung injury (2005). Ulrich, K. et al., Am J Physiol Lung Cell Mol Physiol, 288, L1179-1192. Download
Airway gene therapy (2005). Davies, J. C. & Alton, E. W., Adv Genet, 54, 291-314.
Electrohydrodynamic comminution: a novel technique for the aerosolisation of plasmid DNA (2005). Davies, L. A. et al., Pharm Res, 22, 1294-1304.
Sendai virus for gene therapy and vaccination (2005). Griesenbach, U. et al., Curr Opin Mol Ther, 7, 346-352.
Vascular oligonucleotide transfer facilitated by a polymer-coated stent (2005). Radke, P. W. et al., Hum Gene Ther, 16, 734-740.
Critical appraisal of the mouse model of myocardial infarction (2004). Degabriele, N. M. et al., Exp Physiol, 89, 497-505. Download
A defective nontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivo (2004). Ferrari, S. et al., Gene Ther, 11, 1659-1664. Download
Gene therapy for cystic fibrosis: an example for lung gene therapy (2004). Griesenbach, U. et al., Gene Ther, 11 Suppl 1, S43-50. Download
Normal nasal mucociliary clearance in CF children: evidence against a CFTR-related defect (2004). McShane, D. et al., Eur Respir J, 24, 95-100. Download
Effects of intramyocardial pVEGF165 delivery on regional myocardial blood flow: evidence for a spatial 'delivery-efficacy' mismatch (2004). Radke, P. W. et al., Gene Ther, 11, 1249-1255. Download
The development of gene therapy for diseases of the lung (2004). Gill, D. R. et al., Cell Mol Life Sci, 61, 355-368.
Advances in cystic fibrosis gene therapy (2004). Griesenbach, U. et al., Curr Opin Pulm Med, 10, 542-546.
HIV-1 Tat protein transduction domain peptide facilitates gene transfer in combination with cationic liposomes (2004). Hyndman, L. et al., J Control Release, 99, 435-444.
Measurement of halide efflux from cultured and primary airway epithelial cells using fluorescence indicators (2004). Munkonge, F. et al., J Cyst Fibros, 3 Suppl 2, 171-176.
Potent stimulation of gene expression by histone deacetylase inhibitors on transiently transfected DNA (2004). Nan, X. et al., Biochem Biophys Res Commun, 324, 348-354.
The effects of plasmid copy number and sequence context upon transfection efficiency (2004). Walker, W. E. et al., J Control Release, 94, 245-252.
Airway surface pH in subjects with cystic fibrosis (2003). McShane, D. et al., Eur Respir J, 21, 37-42. Download
Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segments (2003). Emerson, M. et al., Mol Ther, 8, 646-653.
Update on gene therapy for cystic fibrosis (2003). Griesenbach, U. et al., Curr Opin Mol Ther, 5, 489-494.
Emerging significance of plasmid DNA nuclear import in gene therapy (2003). Munkonge, F. M. et al., Adv Drug Deliv Rev, 55, 749-760.
Cytoplasmic deposition of NFkappaB decoy oligonucleotides is insufficient to inhibit bleomycin-induced pulmonary inflammation (2002). Griesenbach, U. et al., Gene Ther, 9, 1109-1115. Download
The nasal epithelium as a factory for systemic protein delivery (2002). Griesenbach, U. et al., Mol Ther, 5, 98-103. Download
Gene therapy progress and prospects: cystic fibrosis (2002). Griesenbach, U. et al., Gene Ther, 9, 1344-1350. Download
Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways (2002). Rose, A. C. et al., Gene Ther, 9, 1312-1320. Download
Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter (2001). Gill, D. R. et al., Gene Ther, 8, 1539-1546. Download
Bone marrow stem cells do not repopulate the healthy upper respiratory tract (2002). Davies, J. C. et al., Pediatr Pulmonol, 34, 251-256.
Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence (2001). Scheid, P. et al., Eur Respir J, 17, 27-35. Download
Prospects for gene therapy in lung disease (2001). Davies, J. C. et al., Curr Opin Pharmacol, 1, 272-277.
Gene therapy for cystic fibrosis (2001). Davies, J. C. et al., J Gene Med, 3, 409-417.
Recent progress in gene therapy for cystic fibrosis (2001). Griesenbach, U. & Alton, E. W., Curr Opin Mol Ther, 3, 385-389.
Genomic sequence analysis of Fugu rubripes CFTR and flanking genes in a 60 kb region conserving synteny with 800 kb of human chromosome 7 (2000). Davidson, H. et al., Genome Res, 10, 1194-1203. Download
Anti-inflammatory gene therapy directed at the airway epithelium (2000). Griesenbach, U. et al., Gene Ther, 7, 306-313. Download
Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis (2000). Hyde, S. C. et al., Gene Ther, 7, 1156-1165. Download
Bacterial DNA is implicated in the inflammatory response to delivery of DNA/DOTAP to mouse lungs (2000). McLachlan, G. et al., Gene Ther, 7, 384-392. Download
Efficient gene transfer to airway epithelium using recombinant Sendai virus (2000). Yonemitsu, Y. et al., Nat Biotechnol, 18, 970-973.
Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial (1999). Alton, E. W. et al., Lancet, 353, 947-954.
pSURF-2, a modified BAC vector for selective YAC cloning and functional analysis (1999). Boyd, A. C. et al., Biotechniques, 27, 164-170, 172, 175.
Insertion of natural intron 6a-6b into a human cDNA-derived gene therapy vector for cystic fibrosis improves plasmid stability and permits facile RNA/DNA discrimination (1999). Boyd, A. C. et al., J Gene Med, 1, 312-321.
Ovine bronchoalveolar lavage cellularity: reproducibility and the effect of multiple repeated lavage (1999). Collie, D. D. et al., Res Vet Sci, 67, 137-140.
Elimination of contaminant Escherichia coli chromosomal DNA from preparations of P1 artificial chromosome recombinants facilitates directed subcloning (1999). Davidson, H. et al., Electrophoresis, 20, 1469-1475.
Reduction in the adherence of Pseudomonas aeruginosa to native cystic fibrosis epithelium with anti-asialoGM1 antibody and neuraminidase inhibition (1999). Davies, J. et al., Eur Respir J, 13, 565-570.
Milking gene therapy (1998). Alton, E. W. et al., Nat Med, 4, 1121-1122.
Prospects for gene therapy for cystic fibrosis (1998). Davies, J. C. et al., Mol Med Today, 4, 292-299.
PCR-generated cross-over linkers for site-directed mutagenesis (1997). Boyd, A. C. & Porteous, D. J., Biotechniques, 23, 827-830.
CFTR gene transfer reduces the binding of Pseudomonas aeruginosa to cystic fibrosis respiratory epithelium (1997). Davies, J. C. et al., Am J Respir Cell Mol Biol, 16, 657-663.
Asthma prophylaxis agents alter the function of an airway epithelial chloride channel (1996). Alton, E. W. et al., Am J Respir Cell Mol Biol, 14, 380-387.
Plasmid DNA molecules complexed with cationic liposomes are protected from degradation by nucleases and shearing by aerosolisation (1996). Crook, K. et al., Gene Ther, 3, 834-839.
Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP (1996). McLachlan, G. et al., Gene Ther, 3, 1113-1123.
Lung disease in the cystic fibrosis mouse exposed to bacterial pathogens (1995). Davidson, D. J. et al., Nat Genet, 9, 351-357.
Evaluation in vitro and in vivo of cationic liposome-expression construct complexes for cystic fibrosis gene therapy (1995). McLachlan, G. et al., Gene Ther, 2, 614-622.
Bioelectric characteristics of exon 10 insertional cystic fibrosis mouse: comparison with humans (1995). Smith, S. N. et al., Am J Physiol, 268, C297-307.
Quantitative fluorescence measurements of chloride secretion in native airway epithelium from CF and non-CF subjects (1995). Stern, M. et al., Gene Ther, 2, 766-774.
Lack of inhibition by dideoxy-forskolin and verapamil of DIDS-sensitive volume-activated Cl- secretion in human squamous lung carcinoma epithelial cells (1994). Munkonge, F. M. et al., Biochim Biophys Acta, 1224, 342-348.
Drug efflux mediated by the human multidrug resistance P-glycoprotein is inhibited by cell swelling (1994). Sardini, A. et al., J Cell Sci, 107 ( Pt 12), 3281-3290.
Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice (1993). Alton, E. W. et al., Nat Genet, 5, 135-142.
Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy (1993). Hyde, S. C. et al., Nature, 362, 250-255.
Specific inhibitors distinguish the chloride channel and drug transporter functions associated with the human multidrug resistance P-glycoprotein (1993). Mintenig, G. M. et al., Receptors Channels, 1, 305-313.
Separation of drug transport and chloride channel functions of the human multidrug resistance P-glycoprotein (1992). Gill, D. R. et al., Cell, 71, 23-32.
Volume-regulated chloride channels associated with the human multidrug-resistance P-glycoprotein (1992). Valverde, M. A. et al., Nature, 355, 830-833.
Binding protein-dependent transport systems (1990). Higgins, C. F. et al., J Bioenerg Biomembr, 22, 571-592.
Structural model of ATP-binding proteins associated with cystic fibrosis, multidrug resistance and bacterial transport (1990). Hyde, S. C. et al., Nature, 346, 362-365.
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