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Liver-selective nucleic acid targeting using the asialoglycoprotein receptor

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image of Gene Therapy and Regulation

Gene transfer to the liver by receptor-mediated endocytosis represents a promising method for therapeutic intervention of genetic disorders and acquired diseases affecting the liver. Based on the observation that asialoglycoprotein receptors are abundantly expressed on the surface of human hepatocytes in vivo, gene delivery and expression by DNA-ligand complexes targeted to liver have been developed in several different experimental models. This review will cover general aspects related to targeting to the liver via the asialoglycoprotein receptor, and the relationship between gene expression, persistence, the structure and size of DNA complexes, as well as the current strategies aimed to improve the overall efficiency of receptor-mediated gene therapy.

Affiliations: 1: Division of Gastroenterology-Hepatology, Department of Medicine, University of Connecticut Health Center, Farmington, CT 06030-1845, USA


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