Agents for improved delivery of nucleic acids to eukaryotic cells
AssigneeMolecular Transfer, Inc.
InventorsJoel Jessee, Gulilat Gebeyehu
Life of this patent
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Abstract
New cationic lipids are provided that are useful for delivering macromolecules, such as nucleic acids, into eukaryotic cells. The lipids can be used alone, in combination with other lipids and/or in combination with other transfection enhancing reagents to prepare transfection complexes.
Claims30
A compound having the structure
- wherein each R1 independently is C1-C23 alkyl, C1-C23 alkenyl, —(CO)C1-C23 alkyl, or —(CO)C1-C23 alkenyl;
- and
- each R2 independently is —CH2—(CHR3)1-6—CH2—NHR4 or —CH2—(CHR3)0-6—CH2—OH, wherein each R3 independently is H, OH, or NH2,
- R4 is H or CH3;
- and
- R10 is H or C1-C8 alkyl.
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A composition comprising an amphipathic peptide optionally comprising a polycationic nucleic acid binding moiety and a compound having the structure (II):
- wherein each R1 independently is C1-C23 alkyl, C1-C23 alkenyl, —(CO)C1-C23 alkyl, or —(CO)C1-C23 alkenyl;
- each R2 independently is C1-C6 alkyl, or C1-C6 alkenyl, optionally interrupted by O;
- each R3 independently is H, C1-C6 alkyl, C1-C6 alkenyl, C2-C6 alkynyl, C3-C7 cycloalkyl, C3-C7 cycloalkyl-C1-C6 alkyl, C5-C7-cycloalkenyl, C5-C7-cycloalkenyl-C1-C6 alkyl, —(CH2)mNR6(CH2)nNHR7, —(CH2)2-6NHR7, —(CH2)3-6NHC(═NH)NH2, ((CH2)mOx)y(CH2)zOxR8, or —(CH2)0-3Het;
- each R4 independently is H, C1-C6 alkyl, or C1-C6 alkenyl;
- each R5 independently is H, an amine protecting group, —(CH2)mNR6(CH2)nNHR7, —(CH2)2-6NHR7, —(CH2)3-6NHC(═NH)NH2, —(CO)C1-C23 alkyl, —(CO)C1-C23 alkenyl, or a peptide containing 1-20 amino acid residues;
- each m independently is 2-5,
- each n independently is 2-5,
- each x independently is 0 or 1,
- y is 0-2,
- z is 1-6
- R6 and R7 independently are H, an amine protecting group, —(CO)C1-C23 alkyl, or —(CO)C1-C23 alkenyl;
- R8 is H, C1-C6 alkyl, or C1-C6 alkenyl
- and
- Het is a 5-7 membered monocyclic basic heterocycle, or an 8-11 membered bicyclic basic heterocycle.
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Dependencies are inferred from the claim text.