Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles.
Identifieur interne : 000418 ( Ncbi/Curation ); précédent : 000417; suivant : 000419Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles.
Auteurs : T L Fink [États-Unis] ; P J Klepcyk ; S M Oette ; C R Gedeon ; S L Hyatt ; T H Kowalczyk ; R C Moen ; M J CooperSource :
- Gene therapy [ 0969-7128 ] ; 2006.
Descripteurs français
- KwdFr :
- Cellules épithéliales (enzymologie), Expression des gènes, Humains, Lignée cellulaire, Luciferases (analyse), Luciferases (génétique), Microscopie électronique à transmission, Mucoviscidose (), Mucoviscidose (métabolisme), Nanostructures, Nanotechnologie, Plasmides (génétique), Poumon (enzymologie), Protéines à fluorescence verte (analyse), Protéines à fluorescence verte (génétique), Thérapie génétique (), Transfection ().
- MESH :
- analyse : Luciferases, Protéines à fluorescence verte.
- enzymologie : Cellules épithéliales, Poumon.
- génétique : Luciferases, Plasmides, Protéines à fluorescence verte.
- métabolisme : Mucoviscidose.
- Expression des gènes, Humains, Lignée cellulaire, Microscopie électronique à transmission, Mucoviscidose, Nanostructures, Nanotechnologie, Thérapie génétique, Transfection.
English descriptors
- KwdEn :
- Cell Line, Cystic Fibrosis (metabolism), Cystic Fibrosis (therapy), Epithelial Cells (enzymology), Gene Expression, Genetic Therapy (methods), Green Fluorescent Proteins (analysis), Green Fluorescent Proteins (genetics), Humans, Luciferases (analysis), Luciferases (genetics), Lung (enzymology), Microscopy, Electron, Transmission, Nanostructures, Nanotechnology, Plasmids (genetics), Transfection (methods).
- MESH :
- chemical , analysis : Green Fluorescent Proteins, Luciferases.
- enzymology : Epithelial Cells, Lung.
- chemical , genetics : Green Fluorescent Proteins, Luciferases, Plasmids.
- metabolism : Cystic Fibrosis.
- methods : Genetic Therapy, Transfection.
- therapy : Cystic Fibrosis.
- Cell Line, Gene Expression, Humans, Microscopy, Electron, Transmission, Nanostructures, Nanotechnology.
Abstract
Nanoparticles consisting of single molecules of DNA condensed with polyethylene glycol-substituted lysine 30-mers efficiently transfect lung epithelium following intrapulmonary administration. Nanoparticles formulated with lysine polymers having different counterions at the time of DNA mixing have distinct geometric shapes: trifluoroacetate or acetate counterions produce ellipsoids or rods, respectively. Based on intracytoplasmic microinjection studies, nanoparticle ellipsoids having a minimum diameter less than the 25 nm nuclear membrane pore efficiently transfect non-dividing cells. This 25 nm size restriction corresponds to a 5.8 kbp plasmid when compacted into spheroids, whereas the 8-11 nm diameter of rod-like particles is smaller than the nuclear pore diameter. In mice, up to 50% of lung cells are transfected after dosing with a rod-like compacted 6.9 kbp lacZ expression plasmid, and correction of the CFTR chloride channel was observed in humans following intranasal administration of a rod-like compacted 8.3 kbp plasmid. To further investigate the potential size and shape limitations of DNA nanoparticles for in vivo lung delivery, reporter gene activity of ellipsoidal and rod-like compacted luciferase plasmids ranging in size between 5.3 and 20.2 kbp was investigated. Equivalent molar reporter gene activities were observed for each formulation, indicating that microinjection size limitations do not apply to the in vivo gene transfer setting.
DOI: 10.1038/sj.gt.3302761
PubMed: 16525478
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Klepcyk</name></author><author><name sortKey="Oette, S M" sort="Oette, S M" uniqKey="Oette S" first="S M" last="Oette">S M Oette</name></author><author><name sortKey="Gedeon, C R" sort="Gedeon, C R" uniqKey="Gedeon C" first="C R" last="Gedeon">C R Gedeon</name></author><author><name sortKey="Hyatt, S L" sort="Hyatt, S L" uniqKey="Hyatt S" first="S L" last="Hyatt">S L Hyatt</name></author><author><name sortKey="Kowalczyk, T H" sort="Kowalczyk, T H" uniqKey="Kowalczyk T" first="T H" last="Kowalczyk">T H Kowalczyk</name></author><author><name sortKey="Moen, R C" sort="Moen, R C" uniqKey="Moen R" first="R C" last="Moen">R C Moen</name></author><author><name sortKey="Cooper, M J" sort="Cooper, M J" uniqKey="Cooper M" first="M J" last="Cooper">M J Cooper</name></author></analytic><series><title level="j">Gene therapy</title><idno type="ISSN">0969-7128</idno><imprint><date when="2006" 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des gènes</term><term>Humains</term><term>Lignée cellulaire</term><term>Microscopie électronique à transmission</term><term>Mucoviscidose</term><term>Nanostructures</term><term>Nanotechnologie</term><term>Thérapie génétique</term><term>Transfection</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Nanoparticles consisting of single molecules of DNA condensed with polyethylene glycol-substituted lysine 30-mers efficiently transfect lung epithelium following intrapulmonary administration. Nanoparticles formulated with lysine polymers having different counterions at the time of DNA mixing have distinct geometric shapes: trifluoroacetate or acetate counterions produce ellipsoids or rods, respectively. Based on intracytoplasmic microinjection studies, nanoparticle ellipsoids having a minimum diameter less than the 25 nm nuclear membrane pore efficiently transfect non-dividing cells. This 25 nm size restriction corresponds to a 5.8 kbp plasmid when compacted into spheroids, whereas the 8-11 nm diameter of rod-like particles is smaller than the nuclear pore diameter. In mice, up to 50% of lung cells are transfected after dosing with a rod-like compacted 6.9 kbp lacZ expression plasmid, and correction of the CFTR chloride channel was observed in humans following intranasal administration of a rod-like compacted 8.3 kbp plasmid. To further investigate the potential size and shape limitations of DNA nanoparticles for in vivo lung delivery, reporter gene activity of ellipsoidal and rod-like compacted luciferase plasmids ranging in size between 5.3 and 20.2 kbp was investigated. Equivalent molar reporter gene activities were observed for each formulation, indicating that microinjection size limitations do not apply to the in vivo gene transfer setting.</div></front></TEI></record>Pour manipuler ce document sous Unix (Dilib)
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