We are analyzing https://link.springer.com/article/10.1186/1465-9921-7-26.

Title:
Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo | Respiratory Research
Description:
Background 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. Methods Anti-lacZ and ENaC (epithelial sodium channel) siRNA and asODN were complexed to GL67 and administered to the mouse airway epithelium in vivo Transfection efficiency and efficacy were assessed using real-time RT-PCR as well as through protein expression and functional studies. In parallel in vitro experiments were carried out to select the most efficient oligonucleotides. Results In vitro, GL67 efficiently complexed asODNs and siRNAs, and both were stable in exhaled breath condensate. Importantly, during in vitro selection of functional siRNA and asODN we noted that asODNs accumulated rapidly in the nuclei of transfected cells, whereas siRNAs remained in the cytoplasm, a pattern consistent with their presumed site of action. Following in vivo lung transfection siRNAs were only visible in alveolar macrophages, whereas asODN also transfected alveolar epithelial cells, but no significant uptake into conducting airway epithelial cells was seen. SiRNAs and asODNs targeted to β-galactosidase reduced βgal mRNA levels in the airway epithelium of K18-lacZ mice by 30% and 60%, respectively. However, this was insufficient to reduce protein expression. In an attempt to increase transfection efficiency of the airway epithelium, we increased contact time of siRNA and asODN using the in vivo mouse nose model. Although highly variable and inefficient, transfection of airway epithelium with asODN, but not siRNA, was now seen. As asODNs more effectively transfected nasal airway epithelial cells, we assessed the effect of asODN against ENaC, a potential therapeutic target in cystic fibrosis; no decrease in ENaC mRNA levels or function was detected. Conclusion This study suggests that although siRNAs and asODNs can be developed to inhibit gene expression in culture systems and certain organs in vivo, barriers to nucleic acid transfer in airway epithelial cells seen with large DNA molecules may also affect the efficiency of in vivo uptake of small nucleic acid molecules.
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asodn, sirna, transfection, cells, lung, airway, gene, enac, pubmed, article, protein, mrna, hours, vivo, lacz, google, scholar, epithelial, cas, expression, sirnas, complexed, nucleic, epithelium, nasal, rna, figure, acid, βgal, asodns, shown, min, mouse, distribution, control, data, transfer, cystic, fibrosis, stability, fitclabelled, group, efficiency, transfected, size, complexes, mice, murine, full, vitro,

Topics {✒️}

double-blind placebo-controlled trial low-pass signal-averaging filter open access article nih-3t3-lacz cells yu-hua chow small double-stranded rnas article download pdf β-galactosidase protein expression β-gal reporter kit real-time quantitative pcr small interfering rna real-time rt-pcr fitc versus biotin-streptavidin global anti-viral responses cell culture-based systems double-stranded dna oligonucleotides double-lumen polyethylene catheter asodn-mediated gene silencing lipid-complexed fitc-labelled sirna rnai-mediated gene silencing semi-confluent m1 cells epithelium-specific expression cassette fitc-labelled nucleic acids quantitative rt-pcr carried fitc-labelled sirna/gl67 complexes exhaled breath condensate k18-lacz transgenic mice express β-galactosidase screening anti-enac sirna cystic fibrosis mice directly targeting mrna antisense oligonucleotide-rna duplexes uk home office clinical respiratory medicine develop computer-assisted design independent sample t-test lung biology research gene expression mediated mark edbrooke aerosolized lipid-dna administration quantitative rt-pcr nucleic acid size pulmonary inflammatory diseases β-gal protein high auto-fluorescence epithelial cells mediated β-gal mrna small nucleic acid privacy choices/manage cookies airway gene transfer

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WebPage:
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         headline:Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
         description: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. Anti-lacZ and ENaC (epithelial sodium channel) siRNA and asODN were complexed to GL67 and administered to the mouse airway epithelium in vivo Transfection efficiency and efficacy were assessed using real-time RT-PCR as well as through protein expression and functional studies. In parallel in vitro experiments were carried out to select the most efficient oligonucleotides. In vitro, GL67 efficiently complexed asODNs and siRNAs, and both were stable in exhaled breath condensate. Importantly, during in vitro selection of functional siRNA and asODN we noted that asODNs accumulated rapidly in the nuclei of transfected cells, whereas siRNAs remained in the cytoplasm, a pattern consistent with their presumed site of action. Following in vivo lung transfection siRNAs were only visible in alveolar macrophages, whereas asODN also transfected alveolar epithelial cells, but no significant uptake into conducting airway epithelial cells was seen. SiRNAs and asODNs targeted to β-galactosidase reduced βgal mRNA levels in the airway epithelium of K18-lacZ mice by 30% and 60%, respectively. However, this was insufficient to reduce protein expression. In an attempt to increase transfection efficiency of the airway epithelium, we increased contact time of siRNA and asODN using the in vivo mouse nose model. Although highly variable and inefficient, transfection of airway epithelium with asODN, but not siRNA, was now seen. As asODNs more effectively transfected nasal airway epithelial cells, we assessed the effect of asODN against ENaC, a potential therapeutic target in cystic fibrosis; no decrease in ENaC mRNA levels or function was detected. This study suggests that although siRNAs and asODNs can be developed to inhibit gene expression in culture systems and certain organs in vivo, barriers to nucleic acid transfer in airway epithelial cells seen with large DNA molecules may also affect the efficiency of in vivo uptake of small nucleic acid molecules.
         datePublished:2006-02-15T00:00:00Z
         dateModified:2006-02-15T00:00:00Z
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         pageEnd:15
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         keywords:
            Cystic Fibrosis Transmembrane Conductance Regulator
            Cystic Fibrosis Patient
            Airway Epithelial Cell
            Airway Epithelium
            Exhale Breath Condensate
            Pneumology/Respiratory System
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                        type:PostalAddress
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                        name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
                        type:PostalAddress
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               type:Person
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                        name:GlaxoSmithKline, Middlesex, United Kingdom
                        type:PostalAddress
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               type:Person
               name:Escudero Sara Garcia
               affiliation:
                     name:Faculty of Medicine at the National Heart and Lung Institute, Imperial College
                     address:
                        name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
                        type:PostalAddress
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                     name:UK Cystic Fibrosis Gene Therapy Consortium
                     address:
                        name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
                        type:PostalAddress
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               type:Person
               name:Raymond Farley
               affiliation:
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                     address:
                        name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
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                        name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
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                        name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
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                        name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
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                        name:Gene Medicine Research Group, Nuffield Department of Clinical Laboratory Sciences, John Radcliffe Hospital, University of Oxford, UK
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ScholarlyArticle:
      headline:Inefficient cationic lipid-mediated siRNA and antisense oligonucleotide transfer to airway epithelial cells in vivo
      description: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. Anti-lacZ and ENaC (epithelial sodium channel) siRNA and asODN were complexed to GL67 and administered to the mouse airway epithelium in vivo Transfection efficiency and efficacy were assessed using real-time RT-PCR as well as through protein expression and functional studies. In parallel in vitro experiments were carried out to select the most efficient oligonucleotides. In vitro, GL67 efficiently complexed asODNs and siRNAs, and both were stable in exhaled breath condensate. Importantly, during in vitro selection of functional siRNA and asODN we noted that asODNs accumulated rapidly in the nuclei of transfected cells, whereas siRNAs remained in the cytoplasm, a pattern consistent with their presumed site of action. Following in vivo lung transfection siRNAs were only visible in alveolar macrophages, whereas asODN also transfected alveolar epithelial cells, but no significant uptake into conducting airway epithelial cells was seen. SiRNAs and asODNs targeted to β-galactosidase reduced βgal mRNA levels in the airway epithelium of K18-lacZ mice by 30% and 60%, respectively. However, this was insufficient to reduce protein expression. In an attempt to increase transfection efficiency of the airway epithelium, we increased contact time of siRNA and asODN using the in vivo mouse nose model. Although highly variable and inefficient, transfection of airway epithelium with asODN, but not siRNA, was now seen. As asODNs more effectively transfected nasal airway epithelial cells, we assessed the effect of asODN against ENaC, a potential therapeutic target in cystic fibrosis; no decrease in ENaC mRNA levels or function was detected. This study suggests that although siRNAs and asODNs can be developed to inhibit gene expression in culture systems and certain organs in vivo, barriers to nucleic acid transfer in airway epithelial cells seen with large DNA molecules may also affect the efficiency of in vivo uptake of small nucleic acid molecules.
      datePublished:2006-02-15T00:00:00Z
      dateModified:2006-02-15T00:00:00Z
      pageStart:1
      pageEnd:15
      license:https://creativecommons.org/licenses/by/2.0
      sameAs:https://doi.org/10.1186/1465-9921-7-26
      keywords:
         Cystic Fibrosis Transmembrane Conductance Regulator
         Cystic Fibrosis Patient
         Airway Epithelial Cell
         Airway Epithelium
         Exhale Breath Condensate
         Pneumology/Respiratory System
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      author:
            name:Uta Griesenbach
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                     type:PostalAddress
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            name:Escudero Sara Garcia
            affiliation:
                  name:Faculty of Medicine at the National Heart and Lung Institute, Imperial College
                  address:
                     name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
                     type:PostalAddress
                  type:Organization
                  name:UK Cystic Fibrosis Gene Therapy Consortium
                  address:
                     name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
                     type:PostalAddress
                  type:Organization
            type:Person
            name:Raymond Farley
            affiliation:
                  name:Faculty of Medicine at the National Heart and Lung Institute, Imperial College
                  address:
                     name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
                     type:PostalAddress
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                  address:
                     name:UK Cystic Fibrosis Gene Therapy Consortium, London, UK
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                  name:Faculty of Medicine at the National Heart and Lung Institute, Imperial College
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                     name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
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            name:Hazel Painter
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                  name:John Radcliffe Hospital, University of Oxford
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            name:Deborah R Gill
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                     name:Gene Medicine Research Group, Nuffield Department of Clinical Laboratory Sciences, John Radcliffe Hospital, University of Oxford, UK
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                  name:John Radcliffe Hospital, University of Oxford
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                     name:Gene Medicine Research Group, Nuffield Department of Clinical Laboratory Sciences, John Radcliffe Hospital, University of Oxford, UK
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            name:Yu-Hua Chow
            affiliation:
                  name:University of Toronto
                  address:
                     name:Programme in Lung Biology Research, Hospital for Sick Children and Department of Laboratory Medicine and Pathobiology, University of Toronto, Mississauga
                     type:PostalAddress
                  type:Organization
            type:Person
            name:Jim Hu
            affiliation:
                  name:University of Toronto
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                     name:Programme in Lung Biology Research, Hospital for Sick Children and Department of Laboratory Medicine and Pathobiology, University of Toronto, Mississauga
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            name:Mark Edbrooke
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                  name:GlaxoSmithKline
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                     name:GlaxoSmithKline, Middlesex, United Kingdom
                     type:PostalAddress
                  type:Organization
            type:Person
            name:Varrie Ogilvie
            affiliation:
                  name:University of Edinburgh
                  address:
                     name:Medical Genetics Section, University of Edinburgh, Edinburgh, UK
                     type:PostalAddress
                  type:Organization
            type:Person
            name:Gordon MacGregor
            affiliation:
                  name:University of Edinburgh
                  address:
                     name:Medical Genetics Section, University of Edinburgh, Edinburgh, UK
                     type:PostalAddress
                  type:Organization
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            name:Ronald K Scheule
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                  name:Genzyme Corporation
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                     name:Genzyme Corporation, USA
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                     name:Genzyme Corporation, USA
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                  name:Faculty of Medicine at the National Heart and Lung Institute, Imperial College
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                     name:Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College, London, UK
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