Perfluorinated compounds for non-viral transfer of nucleic acids

Abstract

The present invention relates to the compound of general formula (I): A-B-C (F, G')-D-E-F-G-A' or the compound of general formula (II): A-B-C (F ', G')-D-B-E-F-G-A' (II), wherein -A is at least one selected from perfluorocarbons (PFCs), perfluorinated silicon compounds and / or Molecules in the group of further perfluorinated compounds, -B is at least one predetermined breaking point in the form of a physically, chemically or enzymatically cleavable bond, -C is absent or is at least one linking molecule, -D is absent or is at least one spacer Molecule, -E is at least one member selected from the group consisting of nucleobases, nucleosides, nucleotides, oligonucleotides, nucleic acids, modified nucleobases, modified nucleosides, modified nucleotides, modified oligonucleotides , modified nucleic acids, peptide nucleic acid monomers, peptide nucleic acid oligomers and peptide nucleic acids or other nucleic acid analogues in the group of molecules, -F, F' is absent or is at least one ligand, -G, G' is absent Or be at least one marker molecule, -A' does not exist or is the meaning of A, and wherein compounds i), ii), iii), iv), v), vi) are excepted. The present invention also relates to the use of said compound for the non-viral transfer of molecule E into cells, the pharmaceutical composition containing said compound, and the use of said pharmaceutical composition.

Description

technical field

[0001] The present invention relates to the compound of general formula (I): A-B-C(F', G')-D-E-F-G-A' or general formula (II): A-B-C(F', G')-D-B-E-F-G-A', the use of the compound, A pharmaceutical composition comprising the compound and uses of the composition. Background technique

[0002] Non-viral gene transfer is an important area of ​​interest in basic research and medicine. Possible applications relate in particular to traditional and acquired genetic diseases such as HIV, chronic infectious diseases, tumors, cardiac and circulatory diseases. In the past, attempts to establish gene therapy in medicine have focused on viral vectors. However, they have substantial drawbacks. These applications are not safe enough, it triggers an immune response more after one application in the body, which makes a second application impossible. In addition to this, there have been numerous reports of accidents in which the treatment made the patient seriously ill or e...

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