Plasmid size up to 20 kbp does not limit effective in vivo lung gene transfer using compacted DNA nanoparticles

Gene Therapy

Volumn 13, Issue 13, 2006, Pages 1048-1051

  Fink, T L ^a,b   Klepcyk, P J ^a,b   Oette, S M ^a   Gedeon, C R ^a   Hyatt, S L ^a   Kowalczyk, T H ^a   Moen, R C ^a   Cooper, M J ^a  

^a Copernicus Therapeutics Inc ^*  (United States)

^b CASE WESTERN RESERVE UNIVERSITY   (United States)

Author keywords

Cystic fibrosis; Lung; Nanoparticle; Non viral; Polyplex

Indexed keywords

ACETIC ACID; BETA GALACTOSIDASE; CHLORIDE CHANNEL; GLYCOL; LUCIFERASE; LYSINE; NANOPARTICLE; PLASMID DNA; POLYMER; TRANSMEMBRANE CONDUCTANCE REGULATOR; TRIFLUOROACETIC ACID;

ARTICLE; GENE ACTIVITY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; IN VIVO STUDY; LUNG ALVEOLUS CELL; LUNG ALVEOLUS EPITHELIUM; MICROINJECTION; NONHUMAN; NUCLEAR PORE; PARTICLE SIZE; PLASMID; PRIORITY JOURNAL; REPORTER GENE;

CELL LINE; CYSTIC FIBROSIS; EPITHELIAL CELLS; GENE EXPRESSION; GENE THERAPY; GREEN FLUORESCENT PROTEINS; HUMANS; LUCIFERASES; LUNG; MICROSCOPY, ELECTRON, TRANSMISSION; NANOSTRUCTURES; NANOTECHNOLOGY; PLASMIDS; TRANSFECTION;

EID: 33745224788     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302761     Document Type: Article

References (8)

1. Liu G, Li D, Pasumarthy MK, Kowalczyk TH, Gedeon C, Hyatt S et al. Nanoparticles of compacted DNA transfect post-mitotic cells. J Biol Chem 2003; 278: 32578-32586.
2. Kowalczyk TH, Pasumarthy MK, Gedeon C, Hyatt S, Payne JM, Muhammad O et al. Type of polylysine counterion influences morphology and biological function of condensed DNA. Mol Ther 2001; 3: S359 (Abstract 1015).
3. Ziady AG, Gedeon CR, Miller T, Quan W, Payne JM, Hyatt SL et al. Transfection of airway epithelium by stable PEGylated poly-L-lysine DNA nanoparticles in vivo. Mol Ther 2003; 8: 936-947.
4. Konstan MW, Davis PB, Wagener JS, Hilliard KA, Stern RC, Milgram LJH et al. Compacted DNA nanoparticles administered to the nasal mucosa of cystic fibrosis subjects are safe and demonstrate partial to complete CFTR reconstitution. Hum Gene Ther 2004; 15: 1255-1269.
5. Dabauvalle MC, Schulz B, Scheer U, Peters R. Inhibition of nuclear accumulation of karyophilic proteins in living cells by microinjection of the lectin wheat germ agglutinin. Exp Cell Res 1988; 174: 291-296.
6. Wolff B, Willingham MC, Hanover JA. Nuclear protein import: Specificity for transport across the nuclear pore. Exp Cell Res 1988; 178: 318-334.
7. Lukacs GL, Haggie P, Seksek O, Lechardeur D, Freedman N, Verkman AS. Size-dependent DNA mobility in cytoplasm and nucleus. J Biol Chem 2000; 275: 1625-1629.
8. Kube D, Davis PB. Intracellular trafficking of non-targeted stabilized molecular conjugates in human airway epithelia cells. Mol Ther 2003; 7: S371 (Abstract 965).