Hepatocyte-targeted in vivo gene expression by intravenous injection of plasmid DNA complexed with synthetic multi-functional gene delivery system

@article{Nishikawa2000HepatocytetargetedIV,
  title={Hepatocyte-targeted in vivo gene expression by intravenous injection of plasmid DNA complexed with synthetic multi-functional gene delivery system},
  author={Makiya Nishikawa and Masamichi Yamauchi and Kazuhiro Morimoto and Emi Ishida and Yoshinobu Takakura and Mitsuru Hashida},
  journal={Gene Therapy},
  year={2000},
  volume={7},
  pages={548-555}
}
To achieve hepatocyte-targeted in vivo gene expression, a carrier that controls both the tissue and intracellular distribution of DNA was designed and synthesized. A cationic polymer, poly(L-ornithine) (pOrn), was modified first with galactose, then with a fusigenic peptide (mHA2) to obtain Gal-pOrn-mHA2. When applied with Gal-pOrn-mHA2 to asialoglycoprotein receptor-positive cells, fluorescein-labeled DNA showed a diffuse profile, suggesting the release of DNA from endosomes and/or lysosomes… CONTINUE READING
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