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Bifunctional compounds for targeted hepatic gene delivery

Gene Therapy volume 14, pages 704–708 (2007)Cite this article

Abstract

A series of bifunctional compounds with galactosyl residues as targeting ligand for asialoglycoprotein receptors on hepatocytes and various dendrimers as the DNA-binding domain was synthesized. When mixed with plasmid DNA, these compounds self assembled into particles that exhibited high transfection activity both in vitro and in vivo. Optimal activity in liver cells was observed with compounds containing three galactosyl residues and 16 dendrimer arms. These results suggest that domain-based design is an effective strategy for development of a new generation of synthetic gene carriers.

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Acknowledgements

We thank Dr Joseph E Knapp for critical reading of this manuscript. This work was supported by NIH grants RO1 EB002946 to D Liu and CA76541 to DB stolz.

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Author notes

  1. Y Lei

    Present address: 3Current Address: Wilmington PharmTech, 1 Innovation Way, Newark, DE 19713, USA.,

Authors and Affiliations

  1. Department of Pharmaceutical Sciences, School of Pharmacy, University of Pittsburgh, Pittsburgh, PA, USA

    K S Kim, Y Lei & D Liu

  2. Department of Cell Biology and Physiology Center for Biological Imaging, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA

    D B Stolz

Authors
  1. K S Kim
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  2. Y Lei
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  3. D B Stolz
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  4. D Liu
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Correspondence to D Liu.

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Kim, K., Lei, Y., Stolz, D. et al. Bifunctional compounds for targeted hepatic gene delivery. Gene Ther 14, 704–708 (2007). https://doi.org/10.1038/sj.gt.3302917

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