Publications
600 results found
Oceandy D, McMorran BJ, Smith SN, et al., 2002, Gene complementation of airway epithelium in the cystic fibrosis mouse is necessary and sufficient to correct the pathogen clearance and inflammatory abnormalities, HUMAN MOLECULAR GENETICS, Vol: 11, Pages: 1059-1067, ISSN: 0964-6906
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- Citations: 43
Tagawa T, Manvell M, Brown N, et al., 2002, Characterisation of LMD virus-like nanoparticles self-assembled from cationic liposomes, adenovirus core peptide μ (mu) and plasmid DNA, GENE THERAPY, Vol: 9, Pages: 564-576, ISSN: 0969-7128
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- Citations: 75
Dickinson P, Smith SN, Webb S, et al., 2002, The severe G480C cystic fibrosis mutation, when replicated in the mouse, demonstrates mistrafficking, normal survival and organ-specific bioelectrics, HUMAN MOLECULAR GENETICS, Vol: 11, Pages: 243-251, ISSN: 0964-6906
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- Citations: 25
Dickinson P, Smith SN, Webb S, et al., 2002, The severe G480C cystic fibrosis mutation, when replicated in the mouse, demonstrates mistrafficking, normal survival and organ-specific bioelectrics., Hum Mol Genet, Vol: 11, Pages: 243-251, ISSN: 0964-6906
The majority of cystic fibrosis patients produce a mutant form of CFTR (DeltaF508) which has been shown to be mislocalized in both humans and mice. G480C, another clinically 'severe' mutation, has also been demonstrated to be defective in its intracellular processing, but when allowed to traffic in Xenopus oocytes showed similar channel characteristics to that of wild-type CFTR. We have replicated the G480C mutation in the murine Cftr gene using the 'hit and run' double recombination procedure. As expected, the G480C cystic fibrosis mouse model expresses the G480C mutant transcript at a level comparable to that of wild-type CFTR: The homozygous mutant mice were fertile, had normal survival, weight, tooth colour and no evidence of caecal blockage, despite mild goblet cell hypertrophy in the intestine. Analysis of the mutant protein revealed that the majority of G480C CFTR was abnormally processed and no G480C CFTR-specific immunostaining in the apical membranes of intestinal cells was detected. The bioelectric phenotype of these mice revealed organ-specific electrophysiological effects. In contrast to DeltaF508 'hit and run' homozygotes, the classic defect of forskolin-induced chloride ion transport is not replicated in the caecum, but the response to low chloride in the nose is clearly defective in the G480C mutant animals. The mild phenotype of these G480C mutant animals combined with the defective chloride transport in the nose uniquely provides a valuable resource to test novel pharmacological agents aimed at improving trafficking and correcting the electrophysiological defect in the respiratory tract.
Griesenbach U, Cassady RL, Ferrari S, et al., 2002, The nasal epithelium as a factory for systemic protein delivery, MOLECULAR THERAPY, Vol: 5, Pages: 98-103, ISSN: 1525-0016
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- Citations: 23
Stern M, Alton EWFW, 2002, Use of liposomes in the treatment of cystic fibrosis, Gene Therapy in Lung Disease, Pages: 383-396, ISBN: 9780824708207
Cationic liposome-based gene therapy has been extensively studied for the treatment of cystic fibrosis (CF) where research has reached the stage of clinical trials. Liposomes represent just one strategy among A number used in nonviral gene therapy, ranging from intramuscular injection of naked DNA to the systemic or local administration of formulations comprising DNA and lipids, proteins, peptides, and polymers. Although the main limitation of nonviral gene transfer methods is their relatively low efficiency in vivo, both preclinical and clinical studies indicate that these methods exhibit safety profiles similar to conventional pharmaceutical and biological products. In this chapter, following A brief overview of lipid-mediated gene transfer, the progress of clinical studies for CF gene therapy are described.
Griesenbach U, Geddes DM, Alton EW, 2002, Genetics and pathogenesis of cystic fibrosis, Textbook of respiratory cell and molecular biology, Pages: 403-418, ISBN: 9789058231789
Davies JC, Lloyd T, Geddes DM, et al., 2001, A murine model of <i>B-cepacia</i> syndrome, THORAX, Vol: 56, Pages: 37-38, ISSN: 0040-6376
Davies JC, Potter M, Bush A, et al., 2001, Bone marrow stem cells do not repopulate the healthy respiratory epithelium, THORAX, Vol: 56, Pages: 23-23, ISSN: 0040-6376
Griesenbach U, Cassady RL, Cain RJ, et al., 2001, The effect of NFkB decoy oligonucleotides in two murine models of pulmonary inflammation, THORAX, Vol: 56, Pages: 45-45, ISSN: 0040-6376
Fox E, Griesenbach U, Rogers DF, et al., 2001, Successful oligodeoxynucleotide transfer to the airway epithelium following intravenous delivery, THORAX, Vol: 56, Pages: 2-2, ISSN: 0040-6376
Griesenbach U, Kren BT, Stern M, et al., 2001, Conversion of wild-type CFTR to the G551D mutation in primary rat hepatocytes using RNA/DNA oligonucleotides, THORAX, Vol: 56, Pages: 44-45, ISSN: 0040-6376
Griesenbach U, Cassady RL, Ferrari S, et al., 2001, A novel approach to the production of serum proteins using recombinant Sendai virus, THORAX, Vol: 56, Pages: 45-45, ISSN: 0040-6376
Munkonge FM, Xenariou S, Hillery E, et al., 2001, Regulation of plasmid DNA entry into digitonin-permeabilised HeLa cell nuclei by phosphorylation, MOLECULAR BIOLOGY OF THE CELL, Vol: 12, Pages: 106A-107A, ISSN: 1059-1524
Ferrari S, Kitson C, Farley R, et al., 2001, Mucus altering agents as adjuncts for nonviral gene transfer to airway epithelium, GENE THERAPY, Vol: 8, Pages: 1380-1386, ISSN: 0969-7128
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- Citations: 92
Griesenbach U, Alton EWFW, 2001, Recent progress in gene therapy for cystic fibrosis, CURRENT OPINION IN MOLECULAR THERAPEUTICS, Vol: 3, Pages: 385-389, ISSN: 1464-8431
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- Citations: 5
Davies JC, Geddes DM, Alton EW, 2001, Prospects for gene therapy in lung disease., Curr Opin Pharmacol, Vol: 1, Pages: 272-277, ISSN: 1471-4892
The past decade has brought significant advances in the field of gene therapy for both inherited and acquired diseases, especially with regard to respiratory disease. Barriers to gene transfer posed by the lung have led to the development of modifications of both vector and host in an attempt to increase the efficiency of transfer. Recently, progress has been made in both laboratory and clinical studies of gene therapy for cystic fibrosis, alpha1-antitrypsin deficiency and lung cancer.
Stewart L, Manvell M, Hillery E, et al., 2001, Cationic lipids for gene therapy part 4 -: Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to <i>in vitro</i> and <i>in vivo</i> gene delivery efficiency, JOURNAL OF THE CHEMICAL SOCIETY-PERKIN TRANSACTIONS 2, Pages: 624-632, ISSN: 1472-779X
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- Citations: 34
Scheid P, Kempster L, Griesenbach U, et al., 2001, Inflammation in cystic fibrosis airways: relationship to increased bacterial adherence, EUROPEAN RESPIRATORY JOURNAL, Vol: 17, Pages: 27-35, ISSN: 0903-1936
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- Citations: 53
L Stewart, M Manvell, E Hillery, et al., 2001, Physico-chemical analysis of cationic liposome-DNA complexes (lipoplexes) with respect to in vitro and in vivo gene delivery efficacy, J Chem Soc, Vol: 2, Pages: 624-632
Davies JC, Geddes DM, Alton EWFW, 2001, Gene therapy for cystic fibrosis, JOURNAL OF GENE MEDICINE, Vol: 3, Pages: 409-417, ISSN: 1099-498X
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- Citations: 45
Stern M, Ulrich K, Geddes DM, et al., 2000, Controlled-release DNA preparations to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo, THORAX, Vol: 55, Pages: A5-A5, ISSN: 0040-6376
Davies JC, Davies MG, Smith S, et al., 2000, Confirmation of abnormal chloride ion secretion in the lower airway of children with cystic fibrosis, THORAX, Vol: 55, Pages: A67-A67, ISSN: 0040-6376
Davies JC, Booth C, Alton EWFW, et al., 2000, Increased frequency of mannose-binding lectin polymorphisms in CF patients colonised with Burkholderia cepacia, THORAX, Vol: 55, Pages: A12-A12, ISSN: 0040-6376
Griesenbach U, Judd D, Ferrari S, et al., 2000, Sendai virus-mediated overexpression of interleukin 10 following pulmonary and intramuscular adminstration, THORAX, Vol: 55, Pages: A5-A5, ISSN: 0040-6376
Davies MG, Davies JC, Geddes DM, et al., 2000, Nasal epithelial pH is lower in patients with cystic fibrosis., THORAX, Vol: 55, Pages: A66-A66, ISSN: 0040-6376
Smith SN, Geddes DM, Alton EWFW, 2000, 96 well-based fluorescence functional CFTR assay for gene medicine, THORAX, Vol: 55, Pages: A67-A67, ISSN: 0040-6376
Yonemitsu Y, Kitson C, Ferrari S, et al., 2000, Efficient gene transfer to airway epithelium using recombinant Sendai virus, NATURE BIOTECHNOLOGY, Vol: 18, Pages: 970-973, ISSN: 1087-0156
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- Citations: 182
Alton E, Geddes D, Gill D, et al., 2000, Measure and interpretation of CF gene therapy trial results, MOLECULAR THERAPY, Vol: 2, Pages: 3-3, ISSN: 1525-0016
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- Citations: 3
Kitson C, Alton E, 2000, Gene therapy for cystic fibrosis, EXPERT OPINION ON INVESTIGATIONAL DRUGS, Vol: 9, Pages: 1523-1535, ISSN: 1354-3784
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- Citations: 11
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