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  1. Article

    Open Access

    The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins

    We have shown that a lentiviral vector (rSIV.F/HN) pseudotyped with the F and HN proteins from Sendai virus generates high levels of intracellular proteins after lung transduction. Here, we evaluate the use of...

    Michael C. Paul-Smith, Kamila M. Pytel, Jean-François Gelinas… in Gene Therapy (2018)

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  2. No Access

    Article

    CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

    Pulmonary delivery of plasmid DNA (pDNA)/cationic liposome complexes is associated with an acute unmethylated CG dinucleotide (CpG)-mediated inflammatory response and brief duration of transgene expression. We...

    Stephen C Hyde, Ian A Pringle, Syahril Abdullah, Anna E Lawton… in Nature Biotechnology (2008)

  3. No Access

    Protocol

    Assessment of CFTR Function after Gene Transfer In Vitro and In Vivo

    Cystic fibrosis (CF) a monogenic lethal disease and, therefore, ideally suited for the development of gene therapy. The first clinical trials were carried out shortly after cloning the CF gene in 1989. Since t...

    Uta Griesenbach, Felix M. Munkonge, Stephanie Sumner-Jones… in Gene Therapy Protocols (2008)

  4. Article

    Open Access

    Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo

    The cationic lipid Genzyme lipid (GL) 67 is the current "gold-standard" for in vivo lung gene transfer. Here, we assessed, if GL67 mediated uptake of siRNAs and asODNs into airway epithelium in vivo.

    Uta Griesenbach, Chris Kitson, Escudero Sara Garcia, Raymond Farley… in Respiratory Research (2006)

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  5. Article

    Open Access

    Beta-defensin genomic copy number is not a modifier locus for cystic fibrosis

    Human beta-defensin 2 (DEFB4, also known as DEFB2 or hBD-2) is a salt-sensitive antimicrobial protein that is expressed in lung epithelia. Previous work has shown that it is encoded in a cluster of beta-defens...

    Edward J Hollox, Jane Davies, Uta Griesenbach… in Journal of Negative Results in BioMedicine (2005)

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  6. No Access

    Article

    Efficient gene transfer to airway epithelium using recombinant Sendai virus

    Clinical studies of gene therapy for cystic fibrosis (CF) suggest that the key problem is the efficiency of gene transfer to the airway epithelium. The availability of relevant vector receptors, the transient ...

    Yoshikazu Yonemitsu, Christopher Kitson, Stefano Ferrari… in Nature Biotechnology (2000)

  7. No Access

    Article

    Gene therapy for asthma: inspired research or unnecessary effort?

    Eric WFW Alton, Uta Griesenbach, Duncan M Geddes in Gene Therapy (1999)

  8. No Access

    Chapter

    Cystic Fibrosis

    Cystic fibrosis (CF) is the most common lethal autosomal recessive single gene disorder in Caucasians, with an incidence of 1 in 1500 to 1 in 6500 in various populations [1]. The phenotype is complex with invo...

    Uta Griesenbach, Duncan M. Geddes, Eric W. F. W. Alton in Molecular Biology of the Lung (1999)

  9. No Access

    Article

    Milking gene therapy

    Oral administration of an adeno-associated virus vector that encodes the enzyme ß-galactosidase effectively reverses lactose intolerance in rats (pages 1131–1135

    Eric W.F.W. Alton, Uta Griesenbach, Duncan M. Geddes in Nature Medicine (1998)