发明名称 PERFLUORINATED COMPOUNDS FOR THE NON-VIRAL TRANSFER OF NUCLEIC ACIDS
摘要 The invention relates to a compound of general formula (I): A - B - C(F, G')- D - E - F - G - A' or a structure of general formula (II): A - B - C(F', G')- D - B - E - F - G - A' (II), wherein - A is at least one molecule selected from the group of the perfluorocarbons (PFCs), perfluorinated silicon compounds, and/or further perfluorinated compounds, - B is at least one predetermined breaking point in the form of a physically, chemically, or enzymatically severable bond, - C is absent or at least one linker molecule, - D is absent or at least one spacer molecule, - E is at least one molecule selected from the group containing nucleobases, nucleosides, nucleotides, oligonucleotides, nucleic acids, modified nucleobases, modified nucleosides, modified nucleotides, modified oligonucleotides, modified nucleic acids, monomers of peptide nucleic acids, oligomers of peptide nucleic acids, and peptide nucleic acids or other nucleic acid analogs, - F, F' is absent or at least one ligand, - G, G' is absent or at least one marker molecule, - A' is absent or has the meaning of A, and wherein the compounds i), ii), iii), iv), v), vi) are excluded. The invention further relates to the use of said compound for the non-viral transfer of molecule E into a cell, to a pharmaceutical composition containing said compound, and to the use of said pharmaceutical composition.
申请公布号 WO2012130941(A2) 申请公布日期 2012.10.04
申请号 WO2012EP55639 申请日期 2012.03.29
申请人 SCHAEFER, KONSTANZE 发明人 SCHAEFER, KONSTANZE
分类号 A61K47/48 主分类号 A61K47/48
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