Publications
297 results found
Alton EWFW, Baker A, Baker E, et al., 2013, The safety profile of a cationic lipid-mediated cystic fibrosis gene transfer agent following repeated monthly aerosol administration to sheep, BIOMATERIALS, Vol: 34, Pages: 10267-10277, ISSN: 0142-9612
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- Citations: 32
Alton EWFW, Boyd AC, Cheng SH, et al., 2013, A randomised, double-blind, placebo-controlled phase IIB clinical trial of repeated application of gene therapy in patients with cystic fibrosis, THORAX, Vol: 68, Pages: 1075-1077, ISSN: 0040-6376
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- Citations: 46
Griesenbach U, Alton EWFW, 2013, Moving forward: cystic fibrosis gene therapy, HUMAN MOLECULAR GENETICS, Vol: 22, Pages: R52-R58, ISSN: 0964-6906
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- Citations: 53
Alton EW, Boyd AC, Cheng SH, et al., 2013, A PHASE 2B DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL OF NON-VIRAL MEDIATED GENE THERAPY FOR CF, PEDIATRIC PULMONOLOGY, Vol: 48, Pages: 293-294, ISSN: 8755-6863
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- Citations: 1
Calcedo R, Griesenbach U, Dorgan DJ, et al., 2013, Self-Reactive CFTR T Cells in Humans: Implications for Gene Therapy, HUMAN GENE THERAPY CLINICAL DEVELOPMENT, Vol: 24, Pages: 108-115, ISSN: 2324-8637
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- Citations: 7
Horsley AR, Davies JC, Gray RD, et al., 2013, Changes in physiological, functional and structural markers of cystic fibrosis lung disease with treatment of a pulmonary exacerbation, Thorax, Vol: 68, Pages: 532-539, ISSN: 1468-3296
Background Clinical trials in cystic fibrosis (CF) have been hindered by the paucity of well characterised and clinically relevant outcome measures.Aim To evaluate a range of conventional and novel biomarkers of CF lung disease in a multicentre setting as a contributing study in selecting outcome assays for a clinical trial of CFTR gene therapy.Methods A multicentre observational study of adult and paediatric patients with CF (>10 years) treated for a physician-defined exacerbation of CF pulmonary symptoms. Measurements were performed at commencement and immediately after a course of intravenous antibiotics. Disease activity was assessed using 46 assays across five key domains: symptoms, lung physiology, structural changes on CT, pulmonary and systemic inflammatory markers.Results Statistically significant improvements were seen in forced expiratory volume in 1 s (p<0.001, n=32), lung clearance index (p<0.01, n=32), symptoms (p<0.0001, n=37), CT scores for airway wall thickness (p<0.01, n=31), air trapping (p<0.01, n=30) and large mucus plugs (p=0.0001, n=31), serum C-reactive protein (p<0.0001, n=34), serum interleukin-6 (p<0.0001, n=33) and serum calprotectin (p<0.0001, n=31).Discussion We identify the key biomarkers of inflammation, imaging and physiology that alter alongside symptomatic improvement following treatment of an acute CF exacerbation. These data, in parallel with our study of biomarkers in patients with stable CF, provide important guidance in choosing optimal biomarkers for novel therapies. Further, they highlight that such acute therapy predominantly improves large airway parameters and systemic inflammation, but has less effect on airway inflammation.
Alton EW, Davies JC, Griesenbach U, 2013, Foreword: Current & emerging pharmaceutical treatments for cystic fibrosis lung disease, ISBN: 9781780841489
This book contains contributions on cystic fibrosis (CF) lung disease from leading international investigators. In eight chapters, they describe exciting progress in each of their respective fields, from pathogenesis through symptomatic treatments to addressing the basic defect. Each describes an iterative process in the best traditions of science; hypotheses being tested, rejected or improved. All of the authors have made major contributions to the cause of CF research and treatment over many years, and in this book the Editors have encouraged them to speculate and to be controversial where necessary, providing genuine insight through their experience and expertise.
Griesenbach U, Alton EWFW, 2013, Expert opinion in biological therapy: update on developments in lung gene transfer, EXPERT OPINION ON BIOLOGICAL THERAPY, Vol: 13, Pages: 345-360, ISSN: 1471-2598
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- Citations: 19
Kernan NG, Alton EWFW, Cullinan P, et al., 2013, Oral contraceptives do not appear to affect cystic fibrosis disease severity, EUROPEAN RESPIRATORY JOURNAL, Vol: 41, Pages: 67-73, ISSN: 0903-1936
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- Citations: 20
Baty N, MacNeill SJ, Cullinan P, et al., 2012, IS THERE A GENDER DIFFERENCE IN THE UK CF POPULATION?, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A59-A59, ISSN: 0040-6376
Alton EWFW, Boyd AC, Cheng SH, et al., 2012, REPEAT ADMINISTRATION OF GL67A/PGM169 IS FEASIBLE, SAFE, AND PRODUCES ENDOGENOUS LEVELS OF CFTR EXPRESSION AFTER 12 DOSES, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A105-A105, ISSN: 0040-6376
Griesenbach U, Inoue M, Meng C, et al., 2012, ASSESSMENT OF F/HN-PSEUDOTYPED LENTIVIRUS AS A CLINICALLY RELEVANT VECTOR FOR LUNG GENE THERAPY, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A105-A105, ISSN: 0040-6376
Alton EWFW, Ashby D, Boyd C, et al., 2012, UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A58-A58, ISSN: 0040-6376
Griesenbach U, Inoue M, Meng C, et al., 2012, Assessment of F/HN-Pseudotyped Lentivirus as a Clinically Relevant Vector for Lung Gene Therapy, American Journal of Respiratory and Critical Care Medicine, Vol: 186, Pages: 846-856, ISSN: 1535-4970
Rationale: Ongoing efforts to improve pulmonary gene transfer thereby enabling gene therapy for the treatment of lung diseases, such as cystic fibrosis (CF), has led to the assessment of a lentiviral vector (simian immunodeficiency virus [SIV]) pseudotyped with the Sendai virus envelope proteins F and HN.Objectives: To place this vector onto a translational pathway to the clinic by addressing some key milestones that have to be achieved.Methods: F/HN-SIV transduction efficiency, duration of expression, and toxicity were assessed in mice. In addition, F/HN-SIV was assessed in differentiated human air–liquid interface cultures, primary human nasal epithelial cells, and human and sheep lung slices.Measurements and Main Results: A single dose produces lung expression for the lifetime of the mouse (∼2 yr). Only brief contact time is needed to achieve transduction. Repeated daily administration leads to a dose-related increase in gene expression. Repeated monthly administration to mouse lower airways is feasible without loss of gene expression. There is no evidence of chronic toxicity during a 2-year study period. F/HN-SIV leads to persistent gene expression in human differentiated airway cultures and human lung slices and transduces freshly obtained primary human airway epithelial cells.Conclusions: The data support F/HN-pseudotyped SIV as a promising vector for pulmonary gene therapy for several diseases including CF. We are now undertaking the necessary refinements to progress this vector into clinical trials.
Alton EW, Boyd AC, Cheng SH, et al., 2012, Update on the UK CF Gene Therapy Consortium Multidoes, non-viral, gene therapy trial, Collaborative Congress of the European-Society-of-Gene-and-Cell-Therapy/French-Society-of-Cell-and-Gene-Therapy, Publisher: MARY ANN LIEBERT INC, Pages: A30-A30, ISSN: 1043-0342
Alton EWFW, Boyd CA, Cheng SH, et al., 2012, Cumulative CFTR expression following repeated aerosol delivery of non-viral pGM169/GL67A formulation to mouse lung, Collaborative Congress of the European-Society-of-Gene-and-Cell-Therapy/French-Society-of-Cell-and-Gene-Therapy, Publisher: MARY ANN LIEBERT INC, Pages: A83-A84, ISSN: 1043-0342
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- Citations: 1
Griesenbach U, Inoue M, Meng C, et al., 2012, ASSESSMENT OF F/HN-PSEUDOTYPED LENTIVIRUS IN A CLINICALLY RELEVANT VECTOR FOR LUNG GENE THERAPY, Publisher: WILEY-BLACKWELL, Pages: 295-295, ISSN: 8755-6863
Griesenbach U, Baty NJ, Cullinan P, et al., 2012, GENDER DIFFERENCES IN THE UK CF POPULATION, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 385-385, ISSN: 8755-6863
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- Citations: 1
Alton EW, Ashby D, Boyd C, et al., 2012, UPDATE ON THE UK CF GENE THERAPY CONSORTIUM MULTIDOSE, NON-VIRAL, GENE THERAPY TRIAL, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 309-310, ISSN: 8755-6863
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- Citations: 2
Griesenbach U, Wilson KM, Farley R, et al., 2012, Assessment of the nuclear pore dilating agent trans-cyclohexane-1,2-diol in differentiated airway epithelium, JOURNAL OF GENE MEDICINE, Vol: 14, Pages: 491-500, ISSN: 1099-498X
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- Citations: 6
Singh C, Munkonge FM, Smith SN, et al., 2012, Quantitative biological imaging of plasmid DNA in live human airway epithelial cells following non-viral gene transfer, Annual Conference of the British-Society-for-Gene-Therapy (BSGT), Publisher: MARY ANN LIEBERT INC, Pages: A4-A4, ISSN: 1043-0342
Griesenbach U, McLachlan G, Collie DD, et al., 2012, Toxicology studies in support of the UK CF Gene Therapy Consortium's Multi-dose Clinical Trial, Annual Conference of the British-Society-for-Gene-Therapy (BSGT), Publisher: MARY ANN LIEBERT INC, Pages: A12-A12, ISSN: 1043-0342
Griesenbach U, Alton EWFW, 2012, Progress in Gene and Cell Therapy for Cystic Fibrosis Lung Disease, CURRENT PHARMACEUTICAL DESIGN, Vol: 18, Pages: 642-662, ISSN: 1381-6128
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- Citations: 46
Davies G, Davies JC, Gill DR, et al., 2011, SAFETY AND EXPRESSION OF A SINGLE DOSE OF LIPID-MEDIATED CFTR GENE THERAPY TO THE UPPER AND LOWER AIRWAYS OF PATIENTS WITH CYSTIC FIBROSIS, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A2-A2, ISSN: 0040-6376
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- Citations: 6
Ng-Blichfeldt JP, Griffiths M, Griesenbach U, et al., 2011, THE ROLE OF THE RETINOIC ACID PATHWAY IN HUMAN LUNG REGENERATION, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A115-A115, ISSN: 0040-6376
McLachlan G, Davidson H, Holder E, et al., 2011, Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung, GENE THERAPY, Vol: 18, Pages: 996-1005, ISSN: 0969-7128
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- Citations: 76
Griesenbach U, 2011, Moving forward with cystic fibrosis gene therapy, Publisher: MARY ANN LIEBERT INC, Pages: A6-A6, ISSN: 1043-0342
Griesenbach U, Vicente CC, Roberts MJ, et al., 2011, Secreted Gaussia luciferase as a sensitive reporter gene for <i>in vivo</i> and <i>ex</i> <i>vivo</i> studies of airway gene transfer, BIOMATERIALS, Vol: 32, Pages: 2614-2624, ISSN: 0142-9612
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- Citations: 27
Griesenbach U, Soussi S, Larsen MB, et al., 2011, Quantification of Periciliary Fluid Height in Human Airway Biopsies Is Feasible, but Not Suitable as a Biomarker, AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY, Vol: 44, Pages: 309-315, ISSN: 1044-1549
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- Citations: 7
Griesenbach U, McLachlan G, Owaki T, et al., 2011, Validation of recombinant Sendai virus in a non-natural host model, GENE THERAPY, Vol: 18, Pages: 182-188, ISSN: 0969-7128
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- Citations: 14
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