C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/87 (2006.01) A61K 47/48 (2006.01) A61K 48/00 (2006.01)
Patent
CA 2103371
Molecular complexes for targeting a gene encoding a secretory protein to a specific cell en vivo and obtaining secretion of the protein by the targeted cell are disclosed. An expressible gene encoding a desired secretory protein is complexed to a conju- gate 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 hepatacytes. 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 and secre- tion of a desired secretory protein.
Wilson James M.
Wu Catherine H.
Wu George Y.
Borden Ladner Gervais Llp
The Board Of Regents Acting For And On Behalf Of The University
The University Of Connecticut
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