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US 2003/0203871 A1Published application
Novel cationic cholesteryl derivatives containing cyclic polar groups
AssigneeUniversity of Pittsburgh
InventorsHemant M. Deshmukh, Leaf Huang
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Abstract
The present invention discloses compounds which are cationic cholesteryl derivatives having a nitrogen-containing ring structure as their polar head group. These compounds are useful for delivering biologically active substances to cells and for transfecting nucleic acids into cells.
Claims30
A compound having a structure X—R2—R1—O-Cholesteryl where R1 is a linker bond
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- is a direct bond a branched or linear alkylene or alkenylene chain of 1 to 10 carbons in length, a C3-C7 cycloalkylene, preferably C5 and C6, or a phenylene;
- X is a 4-7-membered nitrogen-containing cyclic structure wherein said cyclic structure can optionally include further heteroatoms such as S, O, or NR3 where R3 is —H, —CH3, —C2H5, —CH2(CH2)ZOH or —CH2(CH2)ZSH where Z is 0-3, and X is linked to the R2 spacer via a carbon atom of the nitrogen-containing cyclic structure or via a nitrogen atom of the cyclic structure.
A method for transfecting nucleic acids into cells, said method comprising:
- (a) mixing a nucleic acid which encodes a protein or peptide or, which effects gene expression, with a lipid dispersion to form a nucleic acid:lipid complex wherein the lipid dispersion comprises a compound having a structure X—R2—R1 cholesteryl where R1 is a direct bond to the 3-carbon of the cholesteryl moiety or a Linker bond selected from
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- or NH;
- R2 is a direct bond, a linear or branched chain alkylene or alkenylene group;
- a C3-C7 cycloalkylene, preferably C5-C6, or a phenylene;
- and X is a 4-7-membered nitrogen-containing cyclic structure wherein said cyclic structure can optionally include further heteroatoms such as S, O, or NR3 where R3 is —H, —CH3, —C2H5, —CH2(CH2)2OH or —CH2(CH2)2SH where Z is 0-3, and X is linked to the R2 spacer either via a carbon atom on the nitrogen-containing cyclic structure or via a nitrogen atom of the cyclic structure;
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- (b) contacting said cells with said complex.
6 more elements
Dependencies are inferred from the claim text.