Publications
297 results found
Arias IM, Van Mil SWC, Potschka H, et al., 2011, Hot topics, recent advances and novel approaches for clinical intervention, The ABC Transporters of Human Physiology and Disease: Genetics and Biochemistry of ATP Binding Cassette Transporters, Pages: 397-444, ISBN: 9789814280068
Hyde SC, Sumner-Jones SG, Pringle IA, et al., 2011, MULTIPLE DOSES OF LIPID-MEDIATED GENE THERAPY NEBULISED TO THE MOUSE LUNG SHOW ROBUST AND SUSTAINED CFTR EXPRESSION, PEDIATRIC PULMONOLOGY, Pages: 281-281, ISSN: 8755-6863
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- Citations: 1
Griesenbach U, Alton EWFW, 2011, Current Status and Future Directions of Gene and Cell Therapy for Cystic Fibrosis, BIODRUGS, Vol: 25, Pages: 77-88, ISSN: 1173-8804
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- Citations: 28
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 (Basingstoke)
Collie D, Tennant P, Gordon C, et al., 2011, The Uk Cystic Fibrosis Gene Therapy Consortium: Normal Values And Reproducibility Of Forced Expiratory Flow Volume Curves In Sheep, Publisher: AMER THORACIC SOC, ISSN: 1073-449X
Davies JC, Davies G, Gill D, 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 CF, PEDIATRIC PULMONOLOGY, Pages: 281-281, ISSN: 8755-6863
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- Citations: 2
Griesenbach U, McLachlan G, Collie D, et al., 2011, TOXICOLOGY STUDIES IN SUPPORT OF THE UK CF GENE THERAPY CONSORTIUM'S MULTI-DOSE CLINICAL TRIAL, PEDIATRIC PULMONOLOGY, Pages: 298-298, ISSN: 8755-6863
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- Citations: 1
Griesenbach U, Alton EWFW, Inoue M, et al., 2010, Viral vectors for cystic fibrosis gene therapy: What does the future hold?, Virus Adaptation and Treatment, Vol: 2, Pages: 159-171
Gene transfer to the airway epithelium has been more difficult than originally anticipated, largely because of significant extra- and intracellular barriers in the lung. In general, viral vectors are more adapted to overcoming these barriers than nonviral gene transfer agents and are, therefore, more efficient in transferring genes into recipient cells. Viral vectors derived from adenovirus, adeno-associated virus, and Sendai virus, which all have a natural tropism for the airway epithelium, have been evaluated for cystic fibrosis (CF) gene therapy. Although these vectors transduce airway epithelial cells efficiently, gene expression is transient and repeated dministration is inefficient. They are, therefore, unlikely to be suitable for CF gene therapy. More recently, lentiviruses (LV) have been assessed for lung gene transfer. In contrast to retroviruses, they transduce nondividing cells and randomly integrate into the genome. However, LVs do not have a natural tropism for the lung, and a significant amount of effort has been put into seudotyping these vectors with proteins suitable for airway gene transfer. Several studies have shown that LV-mediated transduction leads to persistent gene expression (for the lifetime of the animal) in the airways and, importantly repeated administration is feasible. Thus, appropriately pseudotyped LV vectors are promising candidates for CF gene therapy. Here, we will review preclinical and clinical research related to viral CF gene therapy. © 2010 Griesenbach et al, publisher and licensee Dove Medical Press Ltd.
Innes JA, Donovan J, Soussi S, et al., 2010, INFLAMMATORY MARKERS: DATA FROM THE UK CF GENE THERAPY CONSORTIUM RUN-IN STUDY, Publisher: B M J PUBLISHING GROUP, Pages: A122-A123, ISSN: 0040-6376
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
Kernan NG, Cullinan P, Alton EWFW, et al., 2010, ORAL CONTRACEPTIVE USE DOES NOT AFFECT CF DISEASE SEVERITY, Publisher: B M J PUBLISHING GROUP, Pages: A121-A121, ISSN: 0040-6376
Singh C, Munkonge FM, Smith SN, et al., 2010, QUANTITATIVE BIOLOGICAL IMAGING OF PLASMID DNA IN LIVE HUMAN AIRWAY EPITHELIAL CELLS FOLLOWING NON-VIRAL GENE TRANSFER, Publisher: B M J PUBLISHING GROUP, Pages: A121-A121, ISSN: 0040-6376
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
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
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
Gill DR, Pringle IA, Lawton AE, et al., 2010, DEVELOPMENT, PRODUCTION AND EVALUATION OF CLINICAL GRADE CFTR EXPRESSION PLASMID FOR CF LUNG GENE THERAPY, PEDIATRIC PULMONOLOGY, Pages: 315-315, ISSN: 8755-6863
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
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
Griesenbach U, Soussi S, Newman N, et al., 2010, INFLAMMATORY MARKERS: DATA FROM THE UK CF GENE THERAPY CONSORTIUM RUN-IN STUDY, PEDIATRIC PULMONOLOGY, Pages: 360-361, ISSN: 8755-6863
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
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
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
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
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.
Griesenbach U, Roberts MJ, Vicente CC, et al., 2009, Secreted Gaussia Luciferase Is a More Sensitive Reporter Than Firefly Luciferase for Non-Viral Gene Transfer to Airway Epithelium <i>Ex Vivo</i> and <i>In Vivo</i>, 12th Annual Meeting of the American Society of Gene Therapy, Publisher: NATURE PUBLISHING GROUP, Pages: S123-S124, ISSN: 1525-0016
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