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Non-viral transfection agent

27.05.2011
The new non-viral transfection agent comprises polymer/nucleic acid complexes, wherein polymer/nucleic acid complexes comprise at least one nucleic acid and at least one cationic polymer. Nanofibers carry these polymer/nucleic acid complexes. The cationic polymer of the complex is a polyimine or polyethyleneimine. It can be modified with hydrophilic polymers or coupled with carbohydrates and/or receptor-specific ligands. The nucleic acid is a DNA or RNA, or a derivative thereof, advantageously a therapeutically effective nucleic acid. The nanofibers comprise biodegradable, biocompatible polymers. The fibers, or rather the entire transfection agent, can be provided with a polymeric coating.

For the first time, the new product allows for the release of nucleic acids in the transfection of eukaryotic cells to be controlled in a loco-regional and temporal manner. The new complexes are capable of being stored for several weeks. As the complexes are carried by nanofibers, this does not cause aggregate formation. Because the complexes are not based on viruses, the danger of immune reactions is significantly reduced in comparison to conventional transfection agents.

The non-viral transfection agent according to the present invention is therefore suitable for the transfection of eukaryotic cells both in cell culture and in the organism. With regard to the latter, the non-viral transfection agent is suitable for being used for the production of a pharmaceutical for gene therapeutic applications, e.g. for the therapeutic knockdown of a gene. It is particularly advantageous to use the non-viral transfection agents with difficult-to-transfect cell lines and the in vivo transfection.

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Dr. Peter Stumpf

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