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filingDate 1994-04-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationDate 1996-09-17-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-H08508645-A
titleOfInvention Targeted delivery of genes encoding antisense polyribonucleotides
abstract (57) [Summary]nTargeting a specific cell in vivo to a gene encoding an antisense polyribonucleotide, resulting in the production of polyribonucleotide in the target cell, and specific expression of a cellular or non-cellular gene. Disclosed are molecular complexes for achieving inhibition. An expressible gene encoding the desired antisense polyribonucleotide is complexed with a conjugate of a cell-specific binding agent and a gene binding agent. Cell-specific binding agents are specific to cell surface structures that mediate ligand internalization by endocytosis. An example is the hepatocyte asialoglycoprotein receptor. A gene binding agent is a compound such as a polycation that stably complexes with a gene under extracellular conditions and releases the gene under intracellular conditions so that it can be functional intracellularly. This molecular complex is stable and soluble in physiological fluids and is used in antisense gene therapy to selectively transfect cells in vivo or in vitro to target cells within target cells. Inhibition of antisense ribonucleotide production and expression of cellular or non-cellular genes can be provided.
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