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An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes

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Book cover EJB Reviews 1994

Part of the book series: EJB Reviews 1994 ((EJB REVIEWS,volume 1994))

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Abstract

Receptor-mediated gene transfer is an attractive method for therapeutically correcting human genetic diseases since it permits the targeting of DNA to cellular receptors in specific tissues of adult animals. Genes introduced by this technique have been shown to be expressed in the target tissue for varying periods. However, to be useful for gene therapy, it is critical that both the chemical properties and physical interactions of the reagents involved in the design of the DNA delivery vehicle be rigorously characterized. In this review, we discuss the critical steps in the preparation of the DNA-ligand complex and the factors involved in the delivery and regulated expression of a transgene in animal tissues. The feasibility of using this technique for the therapeutic delivery of genes to mammalian tissues will also be evaluated.

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Abbreviations

PPyrCK:

phosphoenolpyruvate carboxykinase

EM:

electron microscopy

SV-40:

simian virus 40

hFIX:

human factor IX

CAT:

chloramphenicol acetyltransferase

LDL:

low-density lipoprotein

NLS:

nuclear localization signal

HIV-1:

human immunodeficiency virus type 1.

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Perales, J.C., Ferkol, T., Molas, M., Hanson, R.W. (1994). An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. In: EJB Reviews 1994. EJB Reviews 1994, vol 1994. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-79502-2_16

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  • DOI: https://doi.org/10.1007/978-3-642-79502-2_16

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