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- ••An excellent review on all aspects of CF.
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- ••Review highlighting the conflicting hypotheses on lungdisease.
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- ••Concise summary of CFGT trials and their findings.
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- •Thoughtful review of CFGT and its future prospects.
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- ••A Phase I trial showing gene transfer to the nasal epitheliumusing DOTAP/pCMV-CFTR complex.
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- ••A comprehensive trial of CFGT using Genzyme's lipid GL-67in a formulation consisting of GL-67/DOPE/DMPE-PEG5000. Evidence for gene transfer was obtained using a number of assays. Importantly, the treatment resulted in mild, transient respiratory symptoms that are almost certainly attributable to the methylation-dependent innate immune defence system.
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- ••A repeat administration trial of CFGT to the nose usingDC-Chol/DOPE. No evidence of inflammation, toxicity or an immune response towards DNA/liposome complex or expressed CFTR was seen: compare with the lung trial of Alton et al. (2000) in which there was transient inflammation associated with gene delivery.
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- ••A nasal trial of CFGT using a GL-67:DOPE formulation. Genetransfer was detected in the treated nostrils and partial electrophysiological correction observed. Intriguingly, DNA alone was also found to be taken up and expressed at a measurable rate.
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- ••A pioneering lung trial of adenoviral CFGT.
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- ••A milestone study that revealed the decay of transgeneexpression with repeat dosing of recombinant CFTR adenovirus.
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- •A survey of methods of making viral vectors to GT scale.
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- •A good introduction to the complex factors that mediate and regulate CFTR maturation and turnover.
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- ••Documents the salt-sensitivity of I3-defensins.
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- ••Key work that correlates CF phenotype with level of CF7'Rexpression in the mouse. Work such as this strengthens the idea that correction to 5-10% normal CFTR levels should be therapeutic.
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- ••Pioneering work demonstrating the potential of Sendai virusas a vehicle for CFGT.
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- •Editorial signalling recent clinical successes of GT.