Gene expression plasmid-based

The elements of a nonviral gene delivery system include a gene coding for the therapeutic gene, a plasmid-based expression system, and a synthetic delivery system. 

DNA plasmid-based treatment ( gene therapy ) is considered an alternative to the one based on classical chemical drugs or proteins recovered from recombinant cells. Treatment of acquired and inherent genetic diseases as well as the use of DNA for the purpose of vaccination are potential applications of plasmid DNA (pDNA). The plasmid carries information that allows protein expression in the targeted human cells as well as eukaryotic regulatory elements and specific prokaryotic sequences that control replication in the host cell, see Fig. 10. Formulation is required for ex- or in-vivo administration. Selected systems for gene expression can be viral or non-viral. 

Abruzzese, R.V., D. Godin, M. Burcin, V. Mehta, M. French, Y. Li, B.W. O Malley, and J.L. Nordstrom, Ligand-dependent regulation of plasmid-based transgene expression in vivo. Hum Gene Ther, 1999. 10(9) 1499-507. 

Nonviral vector systems are usually either composed of a plasmid based expression cassette alone ( naked DNA), or are prepared with a synthetic amphipathic DNA-complexing agent (84, 88). Gene delivery systems based on nonviral vectors mainly comprise cationic liposomes, DNA-polymer-protein complexes, and mechanic administration of naked DNA. An idealized/optimized multifunctional nonviral gene delivery system is depicted in Figure 13.4. 

Kobayashi, N., Kuramoto, T., Yamaoka, K., Hashida, M. and Takakura, Y. (2001) Hepatic uptake and gene expression mechanisms following intravenous administration of plasmid DNA by conventional and hydrodynamics-based... 

Figure 21.3 Histochemical analysis of / -galactosidase gene expression in liver. Ten pa of pCMV-LacZ plasmid DNA were injected into a mouse via the tail vein using the hydrodynamics-based procedure, / -galactosidase gene expression was assayed eight hours post injection. HV, hepatic vein HA, hepatic artery PV, portal vein (50x). (see Color Plate 15)...

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