Research Topics
| U GriesenbachSummaryAffiliation: Imperial College Country: UK Publications
| Collaborators
|
Detail Information
Publications
Gene therapy progress and prospects: cystic fibrosisU Griesenbach
Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK
Gene Ther 13:1061-7. 2006..Developments in viral and non-viral vectors, as well as recent alternative strategies such as gene repair, trans-splicing and stem cell therapy will be reviewed...- Secreted Gaussia luciferase as a sensitive reporter gene for in vivo and ex vivo studies of airway gene transferUta 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... - Progress in gene and cell therapy for cystic fibrosis lung diseaseUta Griesenbach
Reader Associate Professor in Molecular Medicine, Department of Gene Therapy, Imperial College London, Emmanuel Kaye Building, Manresa Road, London SW3 6LR
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... - Current status and future directions of gene and cell therapy for cystic fibrosisUta 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... - Effect of tolerance induction to immunodominant T-cell epitopes of Sendai virus on gene expression following repeat administration to lungU Griesenbach
Department of Gene Therapy, NHLI, Imperial College, Edinburgh, London, UK
Gene Ther 13:449-56. 2006..Multiple immune mechanisms operate to eradicate viruses from the lung, and these findings indicate that impeding the adaptive T-cell response to the immunodominant viral epitope is not sufficient to prevent the process... - Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivoUta 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... - In vivo imaging of gene transfer to the respiratory tractUta 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... - Validation of nasal potential difference measurements in gut-corrected CF knockout miceUta 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... - The role of doxorubicin in non-viral gene transfer in the lungUta Griesenbach
Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, Manresa Road, London SW36LR, UK
Biomaterials 30:1971-7. 2009.... - Limitations of the murine nose in the development of nonviral airway gene transferUta 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... - The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airwaysUta 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... - Quantification of periciliary fluid height in human airway biopsies is feasible, but not suitable as a biomarkerUta Griesenbach
Department of Gene Therapy, Imperial College London, UK
Am J Respir Cell Mol Biol 44:309-15. 2011..quot; 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... - Cystic fibrosis gene therapy: successes, failures and hopes for the futureUta 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... - Validation of recombinant Sendai virus in a non-natural host modelU Griesenbach
Department of Gene Therapy, Imperial College London, National Heart and Lung Institute, Manresa Road, London, UK
Gene Ther 18:182-8. 2011..In conclusion, the SeV vector should be strongly considered for lung-related applications requiring a single administration of the vector even though it might not be suitable for diseases requiring repeat administration... - Sendai virus for gene therapy and vaccinationUta 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... - Pre-clinical and clinical endpoint assays for cystic fibrosis gene therapyUta Griesenbach
Department of Gene Therapy, Faculty of Medicine, Imperial College London, UK
J Cyst Fibros 4:89-100. 2005.... - Gene transfer to the lung: lessons learned from more than 2 decades of CF gene therapyUta 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... - Advances in cystic fibrosis gene therapyUta 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... - Update on gene therapy for cystic fibrosisUta Griesenbach
Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College London, Manresa Road, London, SW3 6LR, UK
Curr Opin Mol Ther 5:489-94. 2003..Steady progress has been made over the last two years and key papers, including recent advances in viral and nonviral gene transfer agents, will be reviewed here... - Inflammation in cystic fibrosis airways: relationship to increased bacterial adherenceP Scheid
Dept. of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK
Eur Respir J 17:27-35. 2001..The authors conclude that the stimulus produced by Pseudomonas aeruginosa is the predominant inflammatory trigger in their models... - Intravenously administered oligonucleotides can be delivered to conducting airway epithelium via the bronchial circulationE Holder
Department of Gene Therapy, Faculty of Medicine, Imperial College, Manrisa Road, London, UK
Gene Ther 13:1628-38. 2006..ODNs may be relevant to CF in a variety of ways and these data suggest one way towards implementing their use... - A defective nontransmissible recombinant Sendai virus mediates efficient gene transfer to airway epithelium in vivoS Ferrari
Department of Gene Therapy, National Heart and Lung Institute, Imperial College Faculty of Medicine, London, UK
Gene Ther 11:1659-64. 2004.... - Sendai virus-mediated CFTR gene transfer to the airway epitheliumS Ferrari
Department of Gene Therapy, Faculty of Medicine, Imperial College, National Heart and Lung Institute, London, UK
Gene Ther 14:1371-9. 2007..Further modifications to the vector or the host may make it easier to translate these studies into clinical trials of cystic fibrosis... - Use of ultrasound to enhance nonviral lung gene transfer in vivoS Xenariou
Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, London, UK
Gene Ther 14:768-74. 2007..We have thus established proof of principle that US can increase nonviral gene transfer, in the air-filled murine lung... - Bactofection of lung epithelial cells in vitro and in vivo using a genetically modified Escherichia coliM D B Larsen
Department of Gene Therapy, Faculty of Medicine, National Heart and Lung Institute, Imperial College, London, UK
Gene Ther 15:434-42. 2008..In conclusion, although proof-of-principle for lung bactofection has been demonstrated, levels were low and further modification to the bacterial vector, vector administration and the plasmids will be required... - Gene therapy progress and prospects: cystic fibrosisU Griesenbach
Department of Gene Therapy, National Heart and Lung Institute, Imperial College, Faculty of Medicine, London, UK
Gene Ther 9:1344-50. 2002..Here, we will review the clinical and pre-clinical progress for the last 2 years (2000-2001) and briefly speculate on future prospects for the next 2 in CF gene therapy... - Using magnetic forces to enhance non-viral gene transfer to airway epithelium in vivoS Xenariou
Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College, 1B Manresa Road, London SW3 6LR, UK
Gene Ther 13:1545-52. 2006..Thus, whereas exposure to a magnetic field improved in vitro transfection efficiency, translation to the in vivo setting remains difficult... - Gene therapy for cystic fibrosis: an example for lung gene therapyU Griesenbach
Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
Gene Ther 11:S43-50. 2004..This review will focus on CF as an example for lung gene therapy and discuss the progress made in this field over the last couple of years... - Immunological hurdles to lung gene therapyS Ferrari
Department of Gene Therapy, National Heart and Lung Institute, Imperial College Faculty of Medicine, London, UK
Clin Exp Immunol 132:1-8. 2003..A better understanding of the immunological barriers which exist in the lung might allow for a more sustained expression of the transgene and importantly help overcome the problem of readministration of viral vectors... - Recent progress in gene therapy for cystic fibrosisU Griesenbach
Department of Gene Therapy, Imperial College School of Medicine, National Heart and Lung Institute, London, UK
Curr Opin Mol Ther 3:385-9. 2001..Here, we review the progress in CF gene therapy over the last 12 months, including recent advances in viral and non-viral gene transfer agents and novel strategies, such as RNA repair and stem cell gene therapy... - Effects of intramyocardial pVEGF165 delivery on regional myocardial blood flow: evidence for a spatial 'delivery-efficacy' mismatchP W Radke
Department of Gene Therapy, National Heart and Lung Institute, Faculty of Medicine, Imperial College London, UK
Gene Ther 11:1249-55. 2004..These data, therefore, demonstrate a spatial 'delivery-efficacy' mismatch with implications for myocardial gene delivery sites and detection of treatment effects in vivo... - Cytoplasmic deposition of NFkappaB decoy oligonucleotides is insufficient to inhibit bleomycin-induced pulmonary inflammationU Griesenbach
Department of Gene Therapy, Faculty of Medicine, Imperial College, London, UK
Gene Ther 9:1109-15. 2002..We suggest that access of ODN to the nucleus of airway epithelial cells is a key problem, limiting the efficacy of such decoy strategies, as well as attempts at gene repair... - Detection of CFTR transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavityEmma Holder
Medical Genetics Section, Molecular Medicine Centre, Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK
J Gene Med 12:55-63. 2010..Only the respiratory epithelium is a satisfactory model for human airway epithelium and therefore CFTR gene transfer should be specifically assessed in respiratory epithelial cells (RECs)... - Cystic fibrosis modifier genesJane Davies
Department of Gene Therapy, Faculty of Medicine, Imperial College, Emmanuel Kaye Building, Manresa Road, London, UK
J R Soc Med 98:47-54. 2005..The aims of such studies are to increase our understanding of disease pathogenesis, to aid prognosis and ultimately to lead to the development of novel treatments... - Identification and functional characterization of cytoplasmic determinants of plasmid DNA nuclear importFelix 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... - Emerging significance of plasmid DNA nuclear import in gene therapyFelix 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.... - Assessment of CFTR function after gene transfer in vitro and in vivoUta 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... - Optimizing aerosol gene delivery and expression in the ovine lungGerry 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... - Nasal abnormalities in cystic fibrosis mice independent of infection and inflammationTom 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... - The nasal epithelium as a factory for systemic protein deliveryUta 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... - Modifier genes in cystic fibrosisJ C Davies
Department of Gene Therapy, National Lung and Heart Institute, Faculty of Medicine, Imperial College, London, UK
Pediatr Pulmonol 39:383-91. 2005..This review will summarize the field to date and discuss some of the methodological issues important in the design and interpretation of such studies... - Low-frequency ultrasound increases non-viral gene transfer to the mouse lungStefania 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... - Progress and prospects: gene therapy clinical trials (part 2)Eric Alton
Department of Gene Therapy, Emmanuel Kaye Building, NHLI, Imperial College, Manresa Road, London, UK
Gene Ther 14:1555-63. 2007..This part includes clinical trials for skin diseases, neurological disorders, HIV/AIDS, ornithine transcarbamylase deficiency, alpha(1)-antitrypsin deficiency, haemophilia and cancer... - Critical appraisal of the mouse model of myocardial infarctionNaomi 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... - Vascular oligonucleotide transfer facilitated by a polymer-coated stentPeter 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... - Transfection efficiency and toxicity following delivery of naked plasmid DNA and cationic lipid-DNA complexes to ovine lung segmentsMichael Emerson
Medical Genetics Section, School of Molecular and Clinical Medicine, and Department of Veterinary Pathology, College of Medicine and Veterinary Medicine, University of Edinburg, Edinburgh EH8 9AG, United Kingdom
Mol Ther 8:646-53. 2003..The severity of the inflammatory response appeared to correlate with the administered dose of DNA and was generally more severe for pDNA:GL67... - CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expressionStephen 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... - CFTR gene transfer to human cystic fibrosis pancreatic duct cells using a Sendai virus vectorZoltan 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... - Beta-defensin genomic copy number is not a modifier locus for cystic fibrosisEdward J Hollox
Institute of Genetics, University of Nottingham, Nottingham, UK
J Negat Results Biomed 4:9. 2005..No significant association was found...