Publications
600 results found
Pabary R, Singh C, Morales S, et al., 2012, ANTI-PSEUDOMONAL BACTERIOPHAGE COCKTAIL REDUCES INFLAMMATORY RESPONSES IN THE MURINE LUNG, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A50-A51, ISSN: 0040-6376
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- Citations: 1
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
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, 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
Thursfield RM, Bush A, Alton EWFW, et al., 2012, LEVELS OF ANTIMICROBIAL PEPTIDES IN THE AIRWAY OF CHILDREN WITH CYSTIC FIBROSIS ARE NOT RELATED TO SERUM VITAMIN D CONCENTRATION, Winter Meeting of the British-Thoracic-Society 2012, Publisher: BMJ PUBLISHING GROUP, Pages: A41-A41, ISSN: 0040-6376
Simmonds N, Pabary R, Ya-Tung I, et al., 2012, THE ROLE OF NASAL POTENTIAL DIFFERENCE TESTING IN DIFFICULT CASES OF POSSIBLE CYSTIC FIBROSIS, 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, 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, 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
Thursfield RM, Bush A, Alton EW, et al., 2012, AIRWAY INFLAMMATION IS PRESENT BY 4 MONTHS IN CF INFANTS DIAGNOSED ON NEWBORN SCREENING, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 352-352, ISSN: 8755-6863
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- Citations: 2
Pabary R, Singh C, Morales S, et al., 2012, ANTI-PSEUDOMONAL BACTERIOPHAGE COCKTAIL REDUCES INFLAMMATORY RESPONSE IN THE MURINE LUNG, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 343-343, ISSN: 8755-6863
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
Coates M, Ito K, Alton EW, et al., 2012, P38MAP KINASE INHIBITOR REDUCES CYTOKINE RELEASE BY <i>PSEUDOMONAS AERUGINOSA</i>-CHALLENGED BRONCHIAL EPITHELIAL CELLS, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 307-307, ISSN: 8755-6863
Davies G, Simpson AJ, Bayfield KJ, et al., 2012, THE IMPORTANCE OF APPROPRIATE REFERENCE SOURCES FOR SPIROMETRY: LESSONS LEARNED FROM THE UK CYSTIC FIBROSIS GENE THERAPY "RUN-IN" STUDY, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 363-363, ISSN: 8755-6863
Tan H, Moody S, Murdoch J, et al., 2012, CHANGES IN NASAL MUCOSAL INFLAMMATORY CELL PHENOTYPE IN CYSTIC FIBROSIS (CF) MICE, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 266-267, ISSN: 8755-6863
Thursfield RM, Alton EW, Bush A, et al., 2012, LEVELS OF ANTIMICROBIAL PEPTIDES IN THE AIRWAY OF CHILDREN WITH CYSTIC FIBROSIS ARE NOT RELATED TO SERUM VITAMIN D CONCENTRATION, PEDIATRIC PULMONOLOGY, Vol: 47, Pages: 270-271, ISSN: 8755-6863
Nair C, Shoemark A, Donovan S, et al., 2012, THE TOXIC <i>P</i>. <i>AERUGINOSA</i> EXOPRODUCTS CYANIDE AND PYOCYANIN INHIBIT CILIARY FUNCTION OF HUMAN RESPIRATORY EPITHELIA VIA DIFFERENT MECHANISMS, Publisher: WILEY-BLACKWELL, Pages: 329-329, ISSN: 8755-6863
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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
Alton EW, Boushey HA, Garn H, et al., 2012, Clinical Expert Panel on Monitoring Potential Lung Toxicity of Inhaled Oligonucleotides: Consensus Points and Recommendations, NUCLEIC ACID THERAPEUTICS, Vol: 22, Pages: 246-254, ISSN: 2159-3337
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- Citations: 22
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
Burney TJ, Davies JC, 2012, Gene therapy for the treatment of cystic fibrosis., The Application of Clinical Genetics, Vol: 5, Pages: 29-36, ISSN: 1178-704X
Gene therapy is being developed as a novel treatment for cystic fibrosis (CF), a condition that has hitherto been widely-researched yet for which no treatment exists that halts the progression of lung disease. Gene therapy involves the transfer of correct copies of cystic fibrosis transmembrane conductance regulator (CFTR) DNA to the epithelial cells in the airways. The cloning of the CFTR gene in 1989 led to proof-of-principle studies of CFTR gene transfer in vitro and in animal models. The earliest clinical trials in CF patients were conducted in 1993 and used viral and non-viral gene transfer agents in both the nasal and bronchial airway epithelium. To date, studies have focused largely on molecular or bioelectric (chloride secretion) outcome measures, many demonstrating evidence of CFTR expression, but few have attempted to achieve clinical efficacy. As CF is a lifelong disease, turnover of the airway epithelium necessitates repeat administration. To date, this has been difficult to achieve with viral gene transfer agents due to host recognition leading to loss of expression. The UK Cystic Fibrosis Gene Therapy Consortium (Imperial College London, University of Edinburgh and University of Oxford) is currently working on a large and ambitious program to establish the clinical benefits of CF gene therapy. Wave 1, which has reached the clinic, uses a non-viral vector. A single-dose safety trial is nearing completion and a multi-dose clinical trial is shortly due to start; this will be powered for clinically-relevant changes. Wave 2, more futuristically, will look at the potential of lentiviruses, which have long-lasting expression. This review will summarize the current status of translational research in CF gene therapy.
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
Regamey N, Jeffery PK, Alton EWFW, et al., 2012, Should bronchoscopy be advocated to study airway remodelling and inflammation in adults with cystic fibrosis? Authors' response, THORAX, Vol: 67, Pages: 177-177, ISSN: 0040-6376
Burgel P-R, Martin C, Fajac I, et al., 2012, Should bronchoscopy be advocated to study airway remodelling and inflammation in adults with cystic fibrosis?, THORAX, Vol: 67, Pages: 177-177, ISSN: 0040-6376
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- Citations: 2
Regamey N, Tsartsali L, Hilliard TN, et al., 2012, Distinct patterns of inflammation in the airway lumen and bronchial mucosa of children with cystic fibrosis, THORAX, Vol: 67, Pages: 170-176, ISSN: 0040-6376
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- Citations: 58
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, Alton EWFW, Davies JC, 2012, Modern Molecular Therapies for Respiratory Disease, Pages: 309-316
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