Cationic lipids and transfection methods
The present invention relates in part to novel cationic lipids and their use, e.g., in delivering nucleic acids to cells.
1. A compound of the formula:
2. A pharmaceutical composition comprising the compound of claim 1 .
3. A lipid aggregate comprising the compound of claim 1 .
4. A lipid nanoparticle comprising the compound of claim 1 .
5. A liposome comprising the compound of claim 1 .
6. A lipid carrier comprising the compound of claim 1 .
7. A composition comprising the compound of claim 1 , and a nucleic acid.
8. The composition of claim 7 , wherein the nucleic acid comprises DNA.
9. The composition of claim 7 , wherein the nucleic acid comprises RNA.
10. The composition of claim 7 , wherein the nucleic acid is an in vitro transcribed mRNA.
11. The composition of claim 10 , wherein the in vitro transcribed mRNA comprises a non-canonical nucleotide.
12. The composition of claim 7 , wherein the nucleic acid is an siRNA.
13. The composition of claim 7 , wherein the nucleic acid is an antisense oligonucleotide.
14. The pharmaceutical composition of claim 2 , further comprising an additional lipid.
15. The lipid aggregate of claim 3 , further comprising an additional lipid.
16. The lipid nanoparticle of claim 4 , further comprising an additional lipid.
17. The liposome of claim 5 , further comprising an additional lipid.
18. The lipid carrier of claim 6 , further comprising an additional lipid.
19. The composition of claim 7 , further comprising an additional lipid.
20. The composition of claim 9 , further comprising an additional lipid.
21. The composition of claim 10 , further comprising an additional lipid.
22. The pharmaceutical composition of claim 14 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
23. The lipid aggregate of claim 15 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
24. The lipid nanoparticle of claim 16 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
25. The liposome of claim 17 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
26. The lipid carrier of claim 18 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
27. The composition of claim 19 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
28. The composition of claim 20 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
29. The composition of claim 21 , wherein the additional lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.
30. The composition of claim 11 , further comprising an additional lipid or helper, wherein the additional or helper lipid is selected from dioleoylphosphatidylethanolamine (DOPE), 1,2-Dioleoyl-sn-glycero-3-phosphocholine (DOPC), cholesterol, and a polyethylene glycol (PEG)-modified lipid.