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Carrier system and method for the introduction of genes into mammalian cells

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TLDR
In this paper, a targetable gene delivery system is provided for introducing foreign genes into mammalian cells, which employs a soluble targetable DNA complex and utilizes receptor-mediated endocytosis to endow cell specificity.
Abstract
A targetable gene delivery system is provided for introducing foreign genes into mammalian cells. The system employs a soluble targetable DNA complex and utilizes receptor-mediated endocytosis to endow cell specificity. The soluble DNA-carrying complex is formed by non-covalently binding a ligand conjugate with the foreign gene. The conjugate, in turn, is formed by bonding receptor-specific ligands such as asialoglycoproteins to polycations such as polylysine through covalent bonds such as disulfide bonds. The system exhibits a high degree of cell specificity and offers potential for the treatment of inherited genetic disorders.

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References
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Journal ArticleDOI

Receptor-mediated in vitro gene transformation by a soluble DNA carrier system.

TL;DR: There is evidence that foreign DNA can be specifically delivered to cells by a soluble carrier system that takes advantage of receptor-mediated endocytosis, and that competition by a 10-fold excess of ASOR prevented gene transformation by the ASOR X poly-L-lysine X DNA complex.
Journal ArticleDOI

The galactose-specific recognition system of mammalian liver: The route of ligand internalization in rat hepatocytes

TL;DR: Serial sectioning indicated that tubular structures in Golgi-lysosome regions were often interconnected to the larger vesicles, but that tubules in the peripheral cytoplasm were only occasionally connected to larger structures.
Journal ArticleDOI

Evidence for targeted gene delivery to Hep G2 hepatoma cells in vitro.

TL;DR: A system for targeting foreign DNA to hepatocytes in vitro using a soluble DNA carrier that takes advantage of receptor-mediated endocytosis to achieve internalization is developed based on the fact that hepatocytes possess a unique receptor that binds and internalizes galactose-terminal (asialo)glycoproteins.
Journal ArticleDOI

Disulfide spacer between methotrexate and poly(D-lysine). A probe for exploring the reductive process in endocytosis.

TL;DR: It is concluded that the reductive process through which methotrexate is released from the disulfide spacer occurs inside cells and not at the cell surface, requires neither acid pH nor lysosomal enzymes, and is not mediated by a glutathione-disulfide exchange reaction requiring high glutathion concentrations.
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