Methods for Targeted Gene Transfer to Liver Using DNA-Protein Complexes.
The potential of therapeutic gene transfer to treat human disease has prompted a diverse and growing range of basic and applied research efforts to explore and develop gene therapy strategies (1-5). Reported approaches to gene therapy mclude the uses of retroviruses (6,7), adenovirus (8,9), receptor-mediated endocytosis (10,11), direct injection (12), and liposomes (13,14), among others. Targeted delivery of DNA via receptors has been successfully applied using protein ligands to the hepatic asialoglycoprotein receptor (ASGr) (10,15-18), and, subsequently, the transferrin receptor (11). The ASGr is a cell-surface receptor that is highly represented on hepatocytes. Thus, genes targeted to this receptor can be delivered in a highly selective manner to the liver.
Pharmaceutical Peptides, Inc., Cambridge, MA.
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