Publications
600 results found
Alton EWFW, Boyd C, Cunningham S, et al., 2010, LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS: THE RUN-IN STUDY, British-Thoracic-Society-Winter-Meeting 2010, Publisher: B M J PUBLISHING GROUP, Pages: A11-A11, ISSN: 0040-6376
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- Citations: 1
Davies JC, Alton EWFW, 2010, Gene therapy for cystic fibrosis., Proc Am Thorac Soc, Vol: 7, Pages: 408-414
The report of the first patients with cystic fibrosis (CF) to receive cystic fibrosis transmembrane conductance regulator gene (CFTR) therapy appeared in 1993, and since then there have been more than 20 clinical trials of both viral and nonviral gene transfer agents. These have largely been single dose to either nose or lower airway and have been designed around molecular or bioelectrical outcome measures. Both transgene mRNA and partial correction of chloride secretion have been reported, although sodium hyperabsorption has not been improved. The U.K. Cystic Fibrosis Gene Therapy Consortium is focused on a clinical program to establish whether these proof-of-principle measures translate into clinical benefit. 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.
Griesenbach U, Sumner-Jones SG, Holder E, et al., 2010, Limitations of the Murine Nose in the Development of Nonviral Airway Gene Transfer, AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY, Vol: 43, Pages: 46-54, ISSN: 1044-1549
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- Citations: 12
Mitomo K, Griesenbach U, Inoue M, et al., 2010, Toward Gene Therapy for Cystic Fibrosis Using a Lentivirus Pseudotyped With Sendai Virus Envelopes, MOLECULAR THERAPY, Vol: 18, Pages: 1173-1182, ISSN: 1525-0016
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- Citations: 91
Griesenbach U, Meng C, Farley R, et al., 2010, The use of carboxymethylcellulose gel to increase non-viral gene transfer in mouse airways, Biomaterials, Vol: 31, Pages: 2665-2672, ISSN: 1878-5905
Xenariou S, Liang H-D, Griesenbach U, et al., 2010, Low-frequency ultrasound increases non-viral gene transfer to the mouse lung, ACTA BIOCHIMICA ET BIOPHYSICA SINICA, Vol: 42, Pages: 45-51, ISSN: 1672-9145
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- Citations: 10
Holder E, Stevenson B, Farley R, et al., 2010, Detection of <i>CFTR</i> transgene mRNA expression in respiratory epithelium isolated from the murine nasal cavity, JOURNAL OF GENE MEDICINE, Vol: 12, Pages: 55-63, ISSN: 1099-498X
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- Citations: 2
Naderi K, Donovan J, Leaver N, et al., 2010, VITAMIN D-DEFICIENT CF CHILDREN HAVE REDUCED LEVELS OF ANTIMICROBIAL DEFENCE MOLECULES IN THE AIRWAYS, PEDIATRIC PULMONOLOGY, Pages: 279-279, ISSN: 8755-6863
Alton EW, Boyd C, Cunningham S, et al., 2010, LONGITUDINAL ASSESSMENT OF BIOMARKERS FOR CLINICAL TRIALS OF NOVEL THERAPEUTIC AGENTS; THE RUN-IN STUDY, PEDIATRIC PULMONOLOGY, Pages: 298-298, ISSN: 8755-6863
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- Citations: 1
Griesenbach U, Inoue M, Meng C, et al., 2010, SIV VECTOR PSEUDOTYPED WITH SEV-F/HN ENVELOPE PROTEINS PRODUCES LONG LASTING EXPRESSION IN THE MURINE LUNG, IS READMINISTRABLE & TRANSFECTS HUMAN AIRWAY MODELS, PEDIATRIC PULMONOLOGY, Pages: 304-304, ISSN: 8755-6863
Donovan J, Ives A, Hogg C, et al., 2010, COMPARISON OF LOCAL & SYSTEMIC INFLAMMATORY RESPONSE IN PATIENTS WITH CYSTIC FIBROSIS & PRIMARY CILIARY DYSKINESIA, PEDIATRIC PULMONOLOGY, Pages: 266-266, ISSN: 8755-6863
Singh C, Munkonge F, Smith S, et al., 2010, QUANTITATIVE BIOLOGICAL IMAGING OF PLASMID DNA IN LIVE HUMAN AIRWAY EPITHELIAL CELLS FOLLOWING NON-VIRAL GENE TRANSFER, PEDIATRIC PULMONOLOGY, Pages: 313-313, ISSN: 8755-6863
Ives A, Irving S, Hogg C, et al., 2010, LUNG CLEARANCE INDEX AND STRUCTURAL LUNG DISEASE IN CYSTIC FIBROSIS: A COMPARISON WITH PRIMARY CILIARY DYSKINESIA, PEDIATRIC PULMONOLOGY, Pages: 361-362, ISSN: 8755-6863
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- Citations: 1
Kernan NG, Cullinan P, Alton EW, et al., 2010, ORAL CONTRACEPTIVE USE DOES NOT AFFECT CF DISEASE SEVERITY, PEDIATRIC PULMONOLOGY, Pages: 250-250, ISSN: 8755-6863
Griesenbach U, Wilson KM, Farley R, et al., 2010, ASSESSMENT OF THE NUCLEAR PORE DILATING AGENT TCHD IN DIFFERENTIATED AIRWAY EPITHELIUM, PEDIATRIC PULMONOLOGY, Pages: 304-305, ISSN: 8755-6863
Tan H, Regamey N, Hilliard T, et al., 2009, EXPLORING THE PHENOTYPE OF THE LYMPHOCYTIC INFILTRATE IN THE CF AIRWAY: DO IL-17+CELLS PLAY A ROLE IN DISEASE PATHOGENESIS?, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A110-A111, ISSN: 0040-6376
Duff R, Simmonds N, Pantelidis P, et al., 2009, MOLECULAR DETECTION OF COMPLEX MICROBIAL COMMUNITIES IN SPUTA OF PATIENTS WITH CYSTIC FIBROSIS AND NON-CF BRONCHIECTASIS, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A63-A63, ISSN: 0040-6376
Davies JC, Davies G, Voase N, et al., 2009, EVALUATION OF SAFETY AND GENE EXPRESSION WITH A SINGLE DOSE OF PGM169/GL67A ADMINISTERED TO THE NOSE AND LUNG OF INDIVIDUALS WITH CYSTIC FIBROSIS: THE UK CF GENE THERAPY CONSORTIUM "PILOT STUDY'', Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A70-A70, ISSN: 0040-6376
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- Citations: 4
Voase NWG, Davies G, Reid PA, et al., 2009, SUCCESS AND UTILITY OF SPUTUM INDUCTION IN NON-EXPECTORATING CF SUBJECTS: DATA FROM THE UK CF GENE THERAPY RUN-IN STUDY, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A110-A110, ISSN: 0040-6376
Griesenbach U, Mitomo K, Inoue M, et al., 2009, TOWARDS GENE THERAPY FOR CYSTIC FIBROSIS USING A LENTIVIRUS PSEUDOTYPED WITH SENDAI VIRUS ENVELOPES, Winter Meeting of the British-Thoracic-Society, Publisher: B M J PUBLISHING GROUP, Pages: A69-A70, ISSN: 0040-6376
Davidson H, Wilson A, Gray RD, et al., 2009, An immunocytochemical assay to detect human CFTR expression following gene transfer, MOLECULAR AND CELLULAR PROBES, Vol: 23, Pages: 272-280, ISSN: 0890-8508
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- Citations: 8
Miller AM, Munkonge FM, Alton EWFW, et al., 2009, Identification of Protein Cofactors Necessary for Sequence-specific Plasmid DNA Nuclear Import, MOLECULAR THERAPY, Vol: 17, Pages: 1897-1903, ISSN: 1525-0016
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- Citations: 26
Munkonge FM, Amin V, Hyde SC, et al., 2009, Identification and Functional Characterization of Cytoplasmic Determinants of Plasmid DNA Nuclear Import, JOURNAL OF BIOLOGICAL CHEMISTRY, Vol: 284, Pages: 26978-26987
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- Citations: 36
Griesenbach U, Alton EWFW, 2009, Cystic fibrosis gene therapy: successes, failures and hopes for the future., Expert Rev Respir Med, Vol: 3, Pages: 363-371
Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early years of gene therapy, CF was at the forefront of this field. Since cloning of the CF gene in 1989, 25 Phase I/II clinical trials involving approximately 420 CF patients have been carried out using a variety of viral and nonviral gene transfer agents. Most early trials focused on the nasal epithelium as a surrogate for the lung to allow for easy access and sampling, and, importantly, to ensure safety. Once an acceptable safety profile had been established, gene transfer agents were administered directly into the lung. Although many of these trials established proof-of-principle for gene transfer in the airways, a gene therapy-based treatment has not yet been developed. 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.
Radke PW, Heinl-Green A, Frass OM, et al., 2009, Evaluation of the porcine ameroid constrictor model of myocardial ischemia for therapeutic angiogenesis studies, ENDOTHELIUM-JOURNAL OF ENDOTHELIAL CELL RESEARCH, Vol: 13, Pages: 25-33, ISSN: 1062-3329
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- Citations: 17
GRAHAM A, STEEL DM, WILSON R, et al., 2009, EFFECTS OF PURIFIED PSEUDOMONAS RHAMNOLIPIDS ON BIOELECTRIC PROPERTIES OF SHEEP TRACHEAL EPITHELIUM, EXPERIMENTAL LUNG RESEARCH, Vol: 19, Pages: 77-89, ISSN: 0190-2148
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- Citations: 28
ROGERS DF, ALTON E, DEWAR A, et al., 2009, IMPAIRED STIMULUS-EVOKED MUCUS SECRETION IN CYSTIC-FIBROSIS BRONCHI, EXPERIMENTAL LUNG RESEARCH, Vol: 19, Pages: 37-53, ISSN: 0190-2148
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- Citations: 11
Davies JC, Alton EWFW, 2009, Monitoring Respiratory Disease Severity in Cystic Fibrosis, 43rd Respiratory Care Journal Conference on Respiratory Care and Cystic Fibrosis, Publisher: DAEDALUS ENTERPRISES INC, Pages: 606-615, ISSN: 0020-1324
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- Citations: 39
Davies JC, Alton EW, 2009, Monitoring respiratory disease severity in cystic fibrosis., Respir Care, Vol: 54, Pages: 606-617, ISSN: 0020-1324
Measurements of disease severity provide a guide for the physician to tailor therapies, for the patient and family to gauge progress, and are required for clinical trials. For many respiratory diseases, including cystic fibrosis, sensitive, noninvasive measurements are few, and some of those that are available are applicable only to certain subgroups of patients or lack sufficient sensitivity. We discuss currently available measurements in 4 groups: physiology, infection, inflammation, and radiology. For each group we highlight strengths and weaknesses, ask how we could improve upon these, and provide details of alternative methods.
Nunez-Alonso GA, Davies LA, Bazzani RP, et al., 2009, Repeated Exposure to pDNA/PEI Aerosols Results in Minimal Detectable Toxicity in the Mouse Lungs, 12th Annual Meeting of the American Society of Gene Therapy, Publisher: NATURE PUBLISHING GROUP, Pages: S123-S123, ISSN: 1525-0016
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- Citations: 1
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