Journal
GENE THERAPY
Volume 14, Issue 8, Pages 704-708Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/sj.gt.3302917
Keywords
bifunctional compounds; nonviral vectors; gene delivery; targeted gene delivery
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Funding
- NCI NIH HHS [CA76541] Funding Source: Medline
- NIBIB NIH HHS [R01 EB002946] Funding Source: Medline
- NATIONAL CANCER INSTITUTE [R01CA076541, R29CA076541] Funding Source: NIH RePORTER
- NATIONAL INSTITUTE OF BIOMEDICAL IMAGING AND BIOENGINEERING [R01EB002946] Funding Source: NIH RePORTER
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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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