TARGETED DELIVERY OF GENES ENCODING CELL SURFACE RECEPTORS

摘要

Molecular complexes for targeting a gene encoding a cell surface receptor to a specific cell in vivo and obtaining expression of the gene and insertion of the gene-encoded receptor in the cell membrane. An expressible gene encoding a desired cell surface receptor is complexed with a carrier of a cell-specific binding agent and a gene-binding agent. The cell-specific binding agent is specific for a cellular surface structure which mediates internalization of ligands by endocytosis. An example is the asialoglycoprotein receptor of hepatocytes. The gene-binding agent is a compound such as a polycation which stably complexes the gene under extracellular conditions and releases the gene under intracellular conditions so that it can function within a cell. The molecular complex is stable and soluble in physiological fluids and can be used in gene therapy to selectively transfect cells in vivo to provide for production, membrane insertion and function of a cellular surface receptor.