Eric W F W Alton

Summary

Affiliation: Imperial College
Country: UK

Publications

  1. pmc Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
    Eric W F W Alton
    Imperial College London, London, UK Electronic address
    Lancet Respir Med 3:684-91. 2015
  2. ncbi request reprint Ex Vivo and In Vivo Lentivirus-Mediated Transduction of Airway Epithelial Progenitor Cells
    Giulia Leoni
    National Heart and Lung Institute, Imperial College, London, United Kingdom, Emmanuel Kaye Building 1b, Manresa Rd, London, SW3 6LR UK
    Curr Gene Ther 15:581-90. 2015
  3. ncbi request reprint Use of nonviral vectors for cystic fibrosis gene therapy
    Eric W F W Alton
    Imperial College London, National Heart and Lung Institute, Department of Gene Therapy, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK
    Proc Am Thorac Soc 1:296-301. 2004
  4. doi request reprint Assessment of CFTR function after gene transfer in vitro and in vivo
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine, Imperial College London, UK
    Methods Mol Biol 433:229-42. 2008
  5. doi request reprint Limitations of the murine nose in the development of nonviral airway gene transfer
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK
    Am J Respir Cell Mol Biol 43:46-54. 2010
  6. doi request reprint Nasal abnormalities in cystic fibrosis mice independent of infection and inflammation
    Tom N Hilliard
    Department of Gene Therapy, Emmanuel Kaye Building, 1B Manresa Road, London SW3 6LR, UK
    Am J Respir Cell Mol Biol 39:19-25. 2008
  7. pmc The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London SW3 6LR, UK
    Biomaterials 31:2665-72. 2010
  8. doi request reprint Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis
    Nicolas Regamey
    Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK
    Thorax 67:164-70. 2012
  9. doi request reprint The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
    Eric W F W Alton
    Department of Gene Therapy, Imperial College London, Manresa Road, London SW3 6LR, UK
    Biomaterials 34:10267-77. 2013
  10. doi request reprint Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
    Uta Griesenbach
    Department of Gene Therapy, Imperial College London, UK
    J Gene Med 14:491-500. 2012

Detail Information

Publications65

  1. pmc Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
    Eric W F W Alton
    Imperial College London, London, UK Electronic address
    Lancet Respir Med 3:684-91. 2015
    ..We aimed to assess the efficacy of non-viral CFTR gene therapy in patients with cystic fibrosis...
  2. ncbi request reprint Ex Vivo and In Vivo Lentivirus-Mediated Transduction of Airway Epithelial Progenitor Cells
    Giulia Leoni
    National Heart and Lung Institute, Imperial College, London, United Kingdom, Emmanuel Kaye Building 1b, Manresa Rd, London, SW3 6LR UK
    Curr Gene Ther 15:581-90. 2015
    ..These results indicate that our lentiviral vector can transduce progenitor basal cells in vivo, although transduction required denudation of the surface epithelium prior to vector administration. ..
  3. ncbi request reprint Use of nonviral vectors for cystic fibrosis gene therapy
    Eric W F W Alton
    Imperial College London, National Heart and Lung Institute, Department of Gene Therapy, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK
    Proc Am Thorac Soc 1:296-301. 2004
    ..The program is milestone-related, has a structure that lies between the pharmaceutical industry and academia, and has as its endpoint negotiations with industry to undertake a phase III clinical trial of the identified product...
  4. doi request reprint Assessment of CFTR function after gene transfer in vitro and in vivo
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine, Imperial College London, UK
    Methods Mol Biol 433:229-42. 2008
    ..Here, we describe pre-clinical methods related to assessing correction of the CF chloride transport defect...
  5. doi request reprint Limitations of the murine nose in the development of nonviral airway gene transfer
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK
    Am J Respir Cell Mol Biol 43:46-54. 2010
    ..At the current levels of gene transfer efficiency achievable with nonviral vectors, the murine nose is of limited value as a stepping stone to human trials...
  6. doi request reprint Nasal abnormalities in cystic fibrosis mice independent of infection and inflammation
    Tom N Hilliard
    Department of Gene Therapy, Emmanuel Kaye Building, 1B Manresa Road, London SW3 6LR, UK
    Am J Respir Cell Mol Biol 39:19-25. 2008
    ..There are significant histologic changes in the nasal mucosa of adult CF mice, not associated with increased lumenal inflammation or bacterial content, and which are not present perinatally. These may be novel therapeutic targets...
  7. pmc The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London SW3 6LR, UK
    Biomaterials 31:2665-72. 2010
    ..0001, n=18). This study suggests that contact time of non-viral gene transfer agents is a key factor for gene delivery, and suggests two methods which may be translatable for use in man...
  8. doi request reprint Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis
    Nicolas Regamey
    Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK
    Thorax 67:164-70. 2012
    ..Studies in cystic fibrosis (CF) generally focus on inflammation present in the airway lumen. Little is known about inflammation occurring in the airway wall, the site ultimately destroyed in end-stage disease...
  9. doi request reprint The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep
    Eric W F W Alton
    Department of Gene Therapy, Imperial College London, Manresa Road, London SW3 6LR, UK
    Biomaterials 34:10267-77. 2013
    ..In conclusion, nine doses of pGM169/GL67A were well tolerated with no significant evidence of toxicity that would preclude adoption of a similar strategy in CF patients...
  10. doi request reprint Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium
    Uta Griesenbach
    Department of Gene Therapy, Imperial College London, UK
    J Gene Med 14:491-500. 2012
    ..Previous studies have shown that TCHD can increase lipid-mediated transfection in vitro...
  11. doi request reprint Quantification of periciliary fluid height in human airway biopsies is feasible, but not suitable as a biomarker
    Uta Griesenbach
    Department of Gene Therapy, Imperial College London, UK
    Am J Respir Cell Mol Biol 44:309-15. 2011
    ..However, power calculations indicate that this assay can only be considered as a biomarker in large, late-phase clinical trials, because sample sizes required to achieve sufficient power are comparatively large...
  12. doi request reprint Increased airway smooth muscle mass in children with asthma, cystic fibrosis, and non-cystic fibrosis bronchiectasis
    Nicolas Regamey
    Department of Gene Therapy, National Heart and Lung Institute, 1B Manresa Road, London SW3 6LR, UK
    Am J Respir Crit Care Med 177:837-43. 2008
    ..Structural alterations to airway smooth muscle (ASM) are a feature of asthma and cystic fibrosis (CF) in adults...
  13. doi request reprint The role of doxorubicin in non-viral gene transfer in the lung
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW36LR, UK
    Biomaterials 30:1971-7. 2009
    ....
  14. doi request reprint Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transfer
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK
    Biomaterials 32:2614-24. 2011
    ..This has allowed us to validate that GL67A, which is currently in clinical use, can generate significant amounts of recombinant protein in fully differentiated human air liquid interface cultures and the ovine lung in vivo...
  15. ncbi request reprint Low-frequency ultrasound increases non-viral gene transfer to the mouse lung
    Stefania Xenariou
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, London, UK
    Acta Biochim Biophys Sin (Shanghai) 42:45-51. 2010
    ..We have thus, established that low-frequency US can enhance lung gene transfer with naked pDNA and this enhancement is more effective than the previously reported 1 MHz US...
  16. doi request reprint Validation of nasal potential difference measurements in gut-corrected CF knockout mice
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College London, London, United Kingdom
    Am J Respir Cell Mol Biol 39:490-6. 2008
    ..These data should allow a more informed use of CF animals in future studies...
  17. pmc Identification and functional characterization of cytoplasmic determinants of plasmid DNA nuclear import
    Felix M Munkonge
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College London, London SW3 6LR, United Kingdom
    J Biol Chem 284:26978-87. 2009
    ..Increasing the potential for exogenously added pDNA to bind intracellular transport cofactors may enhance the potency of non-viral gene transfer...
  18. ncbi request reprint Evaluation of the porcine ameroid constrictor model of myocardial ischemia for therapeutic angiogenesis studies
    Peter W Radke
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, London, UK
    Endothelium 13:25-33. 2006
    ..Angiographic assessment of the degree of coronary collateralization, however, is not associated with myocardial blood flow or function and should not be used as a primary outcome measure of therapeutic angiogenesis studies in this model...
  19. pmc Antipseudomonal Bacteriophage Reduces Infective Burden and Inflammatory Response in Murine Lung
    Rishi Pabary
    National Heart and Lung Institute, Imperial College London, London, United Kingdom Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, United Kingdom
    Antimicrob Agents Chemother 60:744-51. 2016
    ..Bacteriophages are efficacious in reducing both the bacterial load and inflammation in a murine model of P. aeruginosa lung infection. This study provides proof of concept for future clinical trials in patients with CF. ..
  20. ncbi request reprint Quality, size, and composition of pediatric endobronchial biopsies in cystic fibrosis
    Nicolas Regamey
    Department of Paediatric Respiratory Medicine, Royal Brompton Hospital, London, UK
    Chest 131:1710-7. 2007
    ..We prospectively assessed how the quality of biopsy specimens obtained from children with CF compare with those from children with other airway diseases...
  21. pmc Airway remodelling in children with cystic fibrosis
    Tom N Hilliard
    Department of Gene Therapy, National Heart and Lung Institute, Imperial College, London, UK
    Thorax 62:1074-80. 2007
    ..A study was undertaken to determine changes in airway remodelling in children with CF compared with appropriate disease and healthy controls...
  22. ncbi request reprint In vivo imaging of gene transfer to the respiratory tract
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK
    Biomaterials 29:1533-40. 2008
    ..45, p<0.05). To our knowledge these studies show for the first time that this non-invasive method of assessing pulmonary gene transfer is viable for evaluating non-viral gene transfer agents...
  23. pmc Assessment of F/HN-pseudotyped lentivirus as a clinically relevant vector for lung gene therapy
    Uta Griesenbach
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London, UK
    Am J Respir Crit Care Med 186:846-56. 2012
    ....
  24. doi request reprint A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis
    Eric W F W Alton
    UK CF Gene Therapy Consortium, Edinburgh, London, Oxford, UK
    Thorax 68:1075-7. 2013
    ..We have recently embarked on a large, multi-dose clinical trial of a non-viral, liposome-based formulation powered for the first time to detect clinical benefit. The article describes the details of the protocol. ..
  25. ncbi request reprint Exploring the mechanisms of macrolides in cystic fibrosis
    Amanda C Equi
    Department of Gene Therapy, National Heart and Lung Institute, Imperial College, London, UK
    Respir Med 100:687-97. 2006
    ..aeruginosa did not decrease. We conclude that these are unlikely to be significant contributing mechanisms accounting for the consistent beneficial results observed in clinical trials of macrolides in CF...
  26. ncbi request reprint Sendai virus for gene therapy and vaccination
    Uta Griesenbach
    Imperial College London, NHLI, UK
    Curr Opin Mol Ther 7:346-52. 2005
    ..In addition, the potential of SeV vector for vaccination has been explored. Data on the use of SeV for gene therapy and vaccination since January 2004 are reviewed and recent improvements in SeV vectorology are discussed...
  27. ncbi request reprint Potential difference measurements in the lower airway of children with and without cystic fibrosis
    Jane C Davies
    Department of Gene Therapy, National Heart and Lung Institute, Imperial College London, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK
    Am J Respir Crit Care Med 171:1015-9. 2005
    ..Potential difference measurements could form a useful functional endpoint assay for future studies of either the CFTR gene or protein-based therapies in future trials in the pediatric age group...
  28. ncbi request reprint The effect of varying tonicity on nasal epithelial ion transport in cystic fibrosis
    Michael G Davies
    Department of Gene Therapy, Faculty of Medicine, Imperial College at the National Heart and Lung Institute, London SW3 6LR, UK
    Am J Respir Crit Care Med 171:760-3. 2005
    ..For the hypotonic solution, we found that hypotonicity inhibited CFTR-mediated chloride secretion in the non-CF group. These data suggest that CFTR plays a role in the recognition and regulation of airway fluid tonicity...
  29. pmc Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College, London, UK
    Respir Res 7:26. 2006
    ..The cationic lipid Genzyme lipid (GL) 67 is the current "gold-standard" for in vivo lung gene transfer. Here, we assessed, if GL67 mediated uptake of siRNAs and asODNs into airway epithelium in vivo...
  30. doi request reprint Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapy
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College London, Manresa Road, London SW36LR, UK
    Adv Drug Deliv Rev 61:128-39. 2009
    ..This review will focus on CF as an example for lung gene therapy, but lessons learned may be applicable to other target diseases...
  31. ncbi request reprint The efficacy of a 'master switch gene' HIF-1alpha in a porcine model of chronic myocardial ischaemia
    Amanda Heinl-Green
    Department of Gene Therapy, Faculty of Medicine, The National Heart and Lung Institute, Imperial College London, Manresa Road, London SW3 6LR, UK
    Eur Heart J 26:1327-32. 2005
    ..Therapeutic angiogenesis is a potential new treatment for patients unsuitable for conventional revascularization strategies. We investigated angiogenesis via a 'master switch gene' hypoxia inducible factor (HIF-1alpha)...
  32. pmc Lung clearance index and high-resolution computed tomography scores in primary ciliary dyskinesia
    Samantha J Irving
    Department of Paediatrics, Royal Brompton and Harefield NHS Foundation Trust, London, United Kingdom
    Am J Respir Crit Care Med 188:545-9. 2013
    ..We hypothesized LCI would be equally sensitive to lung disease in primary ciliary dyskinesia (PCD)...
  33. doi request reprint Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation
    Alex R Horsley
    UK Cystic fibrosis Gene Therapy Consortium, London, UK
    Thorax 68:532-9. 2013
    ..Clinical trials in cystic fibrosis (CF) have been hindered by the paucity of well characterised and clinically relevant outcome measures...
  34. ncbi request reprint Advances in cystic fibrosis gene therapy
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College, London, UK
    Curr Opin Pulm Med 10:542-6. 2004
    ..The authors review the most recent advances in preclinical airway gene transfer and summarize the results from the latest clinical trials...
  35. ncbi request reprint Nondisruptive, sequence-specific coupling of fluorochromes to plasmid DNA
    Elizabeth Hillery
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College London, London SW3 6LR, UK
    Anal Biochem 352:169-75. 2006
    ..Thus, this method allows for the sequence-specific coupling of commercially available fluorochromes to DNA expression vectors while retaining biological function...
  36. ncbi request reprint Progress in gene and cell therapy for cystic fibrosis lung disease
    Uta Griesenbach
    Department of Gene Therapy, Imperial College London, Emmanuel Kaye Building, Manresa Road, London
    Curr Pharm Des 18:642-62. 2012
    ..Recent studies in viral and non-viral vector developments, as well as cell therapy will be discussed and an update on clinical gene therapy studies will be provided here...
  37. ncbi request reprint Vascular oligonucleotide transfer facilitated by a polymer-coated stent
    Peter W Radke
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College London, SW3 6LR, UK
    Hum Gene Ther 16:734-40. 2005
    ..Rapid intravascular release of ODN before implantation and potential vascular barriers for gene transfer are most likely responsible for the currently unsatisfactory in vivo release kinetics...
  38. doi request reprint Gene therapy for cystic fibrosis
    Jane C Davies
    Department of Gene Therapy, Imperial College London, Manresa Road, London SW3 6LR, UK
    Proc Am Thorac Soc 7:408-14. 2010
    ..Here, we review the published literature, discuss the limitations to gene therapy in the CF airway, and consider issues influencing the design of clinical trial programs...
  39. doi request reprint Design of gene therapy trials in CF patients
    Jane C Davies
    Department of Gene Therapy, Imperial College London, SW3 6LR, London, UK
    Methods Mol Biol 741:55-68. 2011
    ..We highlight the logistic and regulatory complexities of such a clinical programme and finally, we look to the future and consider possible alternative strategies...
  40. ncbi request reprint Airway gene therapy
    Jane C Davies
    Department of Gene Therapy, Imperial College London, London SW3 6LR, United Kingdom
    Adv Genet 54:291-314. 2005
    ..This chapter reviews these issues, strategies aimed at overcoming them, and progress into clinical trials with non-viral vectors in a variety of pulmonary diseases...
  41. pmc Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
    Eric W F W Alton
    Department of Gene Therapy, National Heart and Lung Institute, Imperial College London, London, UK
    Thorax 72:137-147. 2017
    ..The data support the progression of the F/HN-pseudotyped lentiviral vector into a first-in-man CF trial in 2017...
  42. pmc Cystic fibrosis
    Jane C Davies
    Department of Gene Therapy, Imperial College, London
    BMJ 335:1255-9. 2007
  43. doi request reprint Oral contraceptives do not appear to affect cystic fibrosis disease severity
    Natalie G Kernan
    Dept of Gene Therapy, Imperial College London, London, UK
    Eur Respir J 41:67-73. 2013
    ..Our data suggests that the use of OCs does not affect CF disease severity...
  44. ncbi request reprint Barriers to and new approaches for gene therapy and gene delivery in cystic fibrosis
    Stefano Ferrari
    Department of Gene Therapy, National Heart and Lung Institute, Imperial College Faculty of Medicine, UK Cystic fibrosis Gene Therapy Consortium, Edinburgh London Oxford, UK, London SW3 6LR, UK
    Adv Drug Deliv Rev 54:1373-93. 2002
    ..These advances have the potential to improve the efficiency of gene delivery to the airway epithelium, thus making gene therapy a more realistic option for cystic fibrosis...
  45. doi request reprint Current status and future directions of gene and cell therapy for cystic fibrosis
    Uta Griesenbach
    Department of Gene Therapy, Imperial College London, London, UK
    BioDrugs 25:77-88. 2011
    ..In this review, we discuss recent developments with viral and non-viral vectors and cell therapy, and provide an update on clinical gene therapy studies...
  46. doi request reprint Expert opinion in biological therapy: update on developments in lung gene transfer
    Uta Griesenbach
    National Heart and Lung Institute, Imperial College London, Department of Gene Therapy, and The UK Cystic Fibrosis Gene Therapy Consortium, London, UK
    Expert Opin Biol Ther 13:345-60. 2013
    ..Although gene transfer to the lung has proven more challenging than initially anticipated, significant progress has been made over the last 10 years...
  47. doi request reprint Cyanide levels found in infected cystic fibrosis sputum inhibit airway ciliary function
    Chandrika Nair
    Dept of Life Sciences, Imperial College, London, UK
    Eur Respir J 44:1253-61. 2014
    ..If cyanide production similarly impairs mucocilliary clearance in vivo, it could explain the link with increased disease severity observed in cystic fibrosis patients with detectable cyanide in their airway. ..
  48. ncbi request reprint Critical appraisal of the mouse model of myocardial infarction
    Naomi M Degabriele
    Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
    Exp Physiol 89:497-505. 2004
    ..Power calculations indicated that, despite a certain amount of intragroup variation, the Middle Suture model may be useful for therapeutic studies to assess the effects of treatment on cardiac function and overall lesion size...
  49. ncbi request reprint Emerging significance of plasmid DNA nuclear import in gene therapy
    Felix M Munkonge
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, London, UK
    Adv Drug Deliv Rev 55:749-60. 2003
    ....
  50. ncbi request reprint Bone marrow stem cells do not repopulate the healthy upper respiratory tract
    Jane C Davies
    Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK
    Pediatr Pulmonol 34:251-6. 2002
    ..In the absence of such conditions, whole bone marrow transplantation is unlikely to provide a route for correction of the CF airway...
  51. ncbi request reprint The nasal epithelium as a factory for systemic protein delivery
    Uta Griesenbach
    Department of Gene Therapy, Imperial College School of Medicine at the National Heart and Lung Institute, London, SM5 1RU, UK
    Mol Ther 5:98-103. 2002
    ..The combination of a high-efficiency gene transfer agent (SeV) and sites that can be assessed noninvasively (nose or lung) may circumvent several current challenges to gene therapy...
  52. doi request reprint Pseudomonas aeruginosa infection in cystic fibrosis: pathophysiological mechanisms and therapeutic approaches
    Helena Lund-Palau
    a Department of Gene Therapy, National Heart and Lung Institute, Imperial College, London, UK
    Expert Rev Respir Med 10:685-97. 2016
    ..aeruginosa infection in CF. Established treatments are discussed, and an overview is offered of novel detection methods and therapeutic strategies in development. ..
  53. pmc Cystic Fibrosis Gene Therapy in the UK and Elsewhere
    Uta Griesenbach
    Department of Gene Therapy and the U K Cystic Fibrosis Gene Therapy Consortium, Imperial College, London SW3 6LR, United Kingdom
    Hum Gene Ther 26:266-75. 2015
    ..The demonstration that the molecular defect in CFTR can be corrected with small-molecule drugs, and the success of gene therapy in other monogenic diseases, is boosting interest in CF gene therapy. Developments are discussed here. ..
  54. ncbi request reprint Gene complementation of airway epithelium in the cystic fibrosis mouse is necessary and sufficient to correct the pathogen clearance and inflammatory abnormalities
    Delvac Oceandy
    Institute for Molecular Biosciences, Imperial College of Medicine at the National Heart and Lung Institute, Manresa Road, London SW3 6LR, UK
    Hum Mol Genet 11:1059-67. 2002
    ..Additionally, these data indicate the general role for epithelial cell-mediated events in the response of the lung to bacterial pathogens and the importance of CFTR in mediating these processes...
  55. doi request reprint Cystic fibrosis gene therapy: successes, failures and hopes for the future
    Uta Griesenbach
    Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College London, Manresa Road, London SW3 6LR, UK
    Expert Rev Respir Med 3:363-71. 2009
    ..Here, we will summarize the key findings of these clinical studies and describe current preclinical and clinical research aimed at further developing gene therapy for CF...
  56. ncbi request reprint Measurement of halide efflux from cultured and primary airway epithelial cells using fluorescence indicators
    Felix Munkonge
    Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College London, London SW3 GLR, UK
    J Cyst Fibros 3:171-6. 2004
    ..These fluorescence methods permit the quantitative analysis of CFTR function...
  57. doi request reprint Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes
    Lee A Davies
    Nuffield Department of Clinical Laboratory Sciences, University of Oxford, John Radcliffe Hospital, Oxford, UK
    Mol Ther 16:1283-90. 2008
    ..It seems likely that similar benefits might be achievable with alternative delivery routes and with other nonviral formulations...
  58. doi request reprint CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression
    Stephen C Hyde
    Gene Medicine Research Group, Nuffield Department of Clinical Laboratory Sciences, University of Oxford, Oxford OX3 9DU, UK
    Nat Biotechnol 26:549-51. 2008
    ..Using a CpG-free pDNA expression vector, we achieved sustained (>or=56 d) in vivo transgene expression in the absence of lung inflammation...
  59. doi request reprint Endobronchial biopsy in childhood
    Nicolas Regamey
    Chest 133:312; author reply 313. 2008
  60. ncbi request reprint CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vector
    Zoltan Rakonczay
    Institute for Cell and Molecular Biosciences, University of Newcastle, Newcastle upon Tyne, United Kingdom
    J Cell Physiol 214:442-55. 2008
    ..Our data show that SeV vector is a potential CFTR gene transfer agent for human pancreatic duct cells and that expression of CFTR in CF cells is associated with a restoration of Cl- and HCO3- transport at the apical membrane...
  61. ncbi request reprint Optimizing aerosol gene delivery and expression in the ovine lung
    Gerry McLachlan
    Medical Genetics Section, School of Molecular and Clinical Medicine, University of Edinburgh, Western General Hospital, Edinburgh, UK
    Mol Ther 15:348-54. 2007
    ..Dose-related toxicity of GTA was reduced by aerosol administration compared to direct instillation. This large animal model will allow us to move toward clinical studies with greater confidence...
  62. doi request reprint Lung clearance index in CF: a sensitive marker of lung disease severity
    Jane C Davies
    Thorax 63:96-7. 2008
  63. ncbi request reprint Biomarkers for cystic fibrosis: are we progressing?
    Eric W F W Alton
    Am J Respir Crit Care Med 175:750-1. 2007
  64. pmc Beta-defensin genomic copy number is not a modifier locus for cystic fibrosis
    Edward J Hollox
    Institute of Genetics, University of Nottingham, Nottingham, UK
    J Negat Results Biomed 4:9. 2005
    ..No significant association was found...
  65. ncbi request reprint The severe G480C cystic fibrosis mutation, when replicated in the mouse, demonstrates mistrafficking, normal survival and organ-specific bioelectrics
    Paul Dickinson
    MRC Human Genetics Unit, Western General Hospital, Crewe Road, Edinburgh EH4 2XU, UK
    Hum Mol Genet 11:243-51. 2002
    ....