Cell-specifically effective molecules on the basis of siRNA and application kits for the production thereof and use thereof

摘要

A biologically inactivated cell-specifically effective molecule for biologically inactive transfection into a target cell to inhibit expression of genes in the target cell after biological activation of the molecule, by bonding to mRNA and with the formation of a RISC complex, the biologically inactivated cell-specifically effective molecule comprising siRNA coupled with at least one peptide via a linker which remains at the siRNA after biological activation of the molecule, the linker comprising an amino Cn linker wherein n is an integer of 1-6. Kits include the molecule or the constituents thereof and transfection reagents in ampoules and injection equipment for injecting mixtures of the ampoule contents into a medium containing a target cell.

主权项

1. A biologically inactivated molecule for biologically inactive transfection into a target cell that becomes specifically effective in the target cell upon biological activation therein in order to inhibit expression of genes in the target cell by bonding to mRNA to form a RISC complex, the biologically inactivated molecule comprising a linker consisting of an amino-Cn linker directly coupled at a first linker end to at least one peptide and directly coupled at a second linker end to siRNA, n being an integer of 1-6.