Method for the controlled intracellular delivery of nucleic acids

摘要

The present invention relates to a method for the controlled intracellular delivery of nucleic acid molecules into one or more target cells, in particular tumor cells, the method comprising: providing a polymeric complex formed between one or more nucleic acid molecules to be delivered and one or more cationic carrier molecules, wherein at least a part of the one or more carrier molecules in the polymeric complex are covalently attached to hydroxyalkyl starch, and wherein the hydroxyalkyl starch is shielding the polymeric complex; allowing the shielded polymeric complex to get into contact with the one or more target cells; deshielding the polymeric complex by removing the hydroxyalkyl starch; and allowing the deshielded polymeric complex to internalize into the one or more target cells. Removal of the hydroxyalkyl starch can be accomplished enzymatically by exposing the polymeric complex to amylase. The invention also concerns the use of such method for the prevention and/or treatment of a condition selected from the group consisting of cancer, immune diseases, cardiovascular diseases, neuronal diseases, infections, and inflammatory diseases.

主权项

1. Method for the controlled intracellular delivery of nucleic acid molecules into one or more target cells, the method comprising:
(a) providing a shielded polymeric complex formed between one or more nucleic acid molecules to be delivered and one or more cationic carrier molecules, wherein at least a part of the one or more carrier molecules in the polymeric complex are covalently attached to hydroxyalkyl starch, and wherein the hydroxyalkyl starch is shielding the polymeric complex; (b) allowing the shielded polymeric complex to get into contact with the one or more target cells; (c) deshielding the polymeric complex by removing the hydroxyalkyl starch; and (d) allowing the deshielded polymeric complex to internalize into the one or more target cells.