BIOLOGICALLY ACTIVE MOLECULES, PARTICULARLY BASED ON PNA AND SIRNA, METHOD FOR THE CELL-SPECIFIC ACTIVATION THEREOF, AND APPLICATION KIT TO BE ADMINISTERED

摘要

The aim of the invention is to produce biological active substances which can be introduced into cells both in vitro andin vivo and controllably inhibit gene expression exclusively in the desired target cells without influencing protein formation in other cells of the organism. Said aim is achieved by active substances (1) comprising at least one covalent bond to one or more peptides (2, 3), each of which has one or more specific amino acid sequences that are selected in respect of enzymes typical of the target cells. Said bond, which is broken exclusively by said enzymes typical of the target cells in order to biologically activate the active substance, allows the molecules to remain biologically inactive in cells other than the target cells. Said molecules are used for influencing gene expression of preferably sick and infected organs or cells, for example.