Influence of cationic lipid composition on gene silencing properties of lipid nanoparticle formulations of siRNA in antigen-presenting cells

Molecular Therapy

Volumn 19, Issue 12, 2011, Pages 2186-2200

  Basha, Genc ^a   Novobrantseva, Tatiana I ^b   Rosin, Nicole ^a   Tam, Yuen Yi C ^a   Hafez, Ismail M ^a   Wong, Matthew K ^a   Sugo, Tsukasa ^b   Ruda, Vera M ^c   Qin, June ^b   Klebanov, Boris ^b   Ciufolini, Marco ^a   Akinc, Akin ^b   Tam, Ying K ^d   Hope, Michael J ^d   Cullis, Pieter R ^a  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b ALNYLAM PHARMACEUTICALS   (United States)

^c MASSACHUSETTS GENERAL HOSPITAL   (United States)

^d AlCana Technologies Inc   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

1,2 DILINEOYL 3 DIMETHLAMMONIUM PROPANE; 1,2 DILINEOYL 4 (2 DIMETHYLAMINOETHYL) [1,3] DIOXOLANE; 1,2 DILINOLEYLOXY 3 N,N DIMETHYLAMINOPROPANE; 1,2 DILINOLEYLOXY KETO N,N DIMETHYL 3 AMINOPROPANE; CATION; CATIONIC LIPID; LIPID; LIPID NANOPARTICLE; NANOPARTICLE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL TISSUE; ANTIGEN PRESENTING CELL; ARTICLE; CHEMICAL LABELING; CONTROLLED STUDY; CYTOPLASM; DENDRITIC CELL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG FORMULATION; DRUG POTENCY; DRUG TRANSPORT; DRUG UPTAKE; ENDOSOME; FLUORESCENCE; GENE SILENCING; IN VIVO STUDY; INTRACELLULAR TRANSPORT; LIPID COMPOSITION; LIVER CELL; MACROPHAGE; MOUSE; NONHUMAN; PERITONEUM; PROTEIN SYNTHESIS INHIBITION; SPLEEN;

EID: 82955233888     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.190     Document Type: Article

References (50)

1. Aagaard, L and Rossi, JJ (2007). RNAi therapeutics: principles, prospects and challenges. Adv Drug Deliv Rev 59:75-86. (Pubitemid 46770900)
2. Nabel, EG, Gordon, D, Yang, ZY, Xu, L, San, H, Plautz, GE et al. (1992). Gene transfer in vivo with DNA-liposome complexes: lack of autoimmunity and gonadal localization. Hum Gene Ther 3:649-656.
3. Liu, Y, Liggitt, D, Zhong, W, Tu, G, Gaensler, K and Debs, R (1995). Cationic liposomemediated intravenous gene delivery. J Biol Chem 270:24864-24870.
4. Deshpande, D, Blezinger, P, Pillai, R, Duguid, J, Freimark, B and Rolland, A (1998). Target specific optimization of cationic lipid-based systems for pulmonary gene therapy. Pharm Res 15:1340-1347. (Pubitemid 28427919)
5. Wheeler, JJ, Palmer, L, Ossanlou, M, MacLachlan, I, Graham, RW, Zhang, YP et al. (1999). Stabilized plasmid-lipid particles: construction and characterization. Gene Ther 6:271-281. (Pubitemid 29089926)
6. Maurer, N, Wong, KF, Stark, H, Louie, L, McIntosh, D, Wong, T et al. (2001). Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes. Biophys J 80:2310-2326. (Pubitemid 32401995)
7. Semple, SC, Klimuk, SK, Harasym, TO, Dos Santos, N, Ansell, SM, Wong, KF et al. (2001). Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures. Biochim Biophys Acta 1510:152-166. (Pubitemid 32149893)
8. Morrissey, DV, Lockridge, JA, Shaw, L, Blanchard, K, Jensen, K, Breen, W et al. (2005). Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat Biotechnol 23:1002-1007. (Pubitemid 41114035)
9. de Fougerolles, AR (2008). Delivery vehicles for small interfering RNA in vivo. Hum Gene Ther 19:125-132. (Pubitemid 351278372)
10. Whitehead, KA, Langer, R and Anderson, DG (2009). Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 8:129-138.
11. Akinc, A, Zumbuehl, A, Goldberg, M, Leshchiner, ES, Busini, V, Hossain, N et al. (2008). A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 26:561-569. (Pubitemid 351668042)
12. Zimmermann, TS, Lee, AC, Akinc, A, Bramlage, B, Bumcrot, D, Fedoruk, MN et al. (2006). RNAi-mediated gene silencing in non-human primates. Nature 441:111-114.
13. Semple, SC, Akinc, A, Chen, J, Sandhu, AP, Mui, BL, Cho, CK et al. (2010). Rational design of cationic lipids for siRNA delivery. Nat Biotechnol 28:172-176.
14. Felgner, PL, Gadek, TR, Holm, M, Roman, R, Chan, HW, Wenz, Met al. (1987) Lipofection - a highly efficient, lipid-mediated dna-transfection procedure. Proc Natl Acad Sci USA 84(21):7413-7417.
15. Behr, JP, Demeneix, B, Loeffler, JP and Perez-Mutul, J (1989). Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc Natl Acad Sci USA 86:6982-6986. (Pubitemid 19240851)
16. Ferrari, ME, Nguyen, CM, Zelphati, O, Tsai, Y and Felgner, PL (1998). Analytical methods for the characterization of cationic lipid-nucleic acid complexes. Hum Gene Ther 9:341-351. (Pubitemid 28085022)
17. Mislick, KA and Baldeschwieler, JD (1996). Evidence for the role of proteoglycans in cation-mediated gene transfer. Proc Natl Acad Sci USA 93:12349-12354. (Pubitemid 26367146)
18. Mounkes, LC, Zhong, W, Cipres-Palacin, G, Heath, TD and Debs, RJ (1998). Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo. J Biol Chem 273:26164-26170. (Pubitemid 28475853)
19. Kopatz, I, Remy, JS and Behr, JP (2004). A model for non-viral gene delivery: through syndecan adhesion molecules and powered by actin. J Gene Med 6:769-776. (Pubitemid 40228093)
20. Hafez, IM, Ansell, S and Cullis, PR (2000). Tunable pH-sensitive liposomes composed of mixtures of cationic and anionic lipids. Biophys J 79:1438-1446.
21. Oja, CD, Semple, SC, Chonn, A and Cullis, PR (1996). Influence of dose on liposome clearance: critical role of blood proteins. Biochim Biophys Acta 1281:31-37. (Pubitemid 26178845)
22. Levchenko, TS, Rammohan, R, Lukyanov, AN, Whiteman, KR and Torchilin, VP (2002). Liposome clearance in mice: the effect of a separate and combined presence of surface charge and polymer coating. Int J Pharm 240:95-102. (Pubitemid 34607706)
23. Semple, SC, Harasym, TO, Clow, KA, Ansell, SM, Klimuk, SK and Hope, MJ (2005). Immunogenicity and rapid blood clearance of liposomes containing polyethylene glycol-lipid conjugates and nucleic Acid. J Pharmacol Exp Ther 312:1020-1026. (Pubitemid 40293760)
24. Parr, MJ, Bally, MB and Cullis, PR (1993). The presence of GM1 in liposomes with entrapped doxorubicin does not prevent RES blockade. Biochim Biophys Acta 1168:249-252. (Pubitemid 23175029)
25. Litzinger, DC, Brown, JM, Wala, I, Kaufman, SA, Van, GY, Farrell, CL et al. (1996). Fate of cationic liposomes and their complex with oligonucleotide in vivo. Biochim Biophys Acta 1281:139-149. (Pubitemid 26194106)
26. Kamps, JA, Morselt, HW and Scherphof, GL (1999). Uptake of liposomes containing phosphatidylserine by liver cells in vivo and by sinusoidal liver cells in primary culture: in vivo-in vitro differences. Biochem Biophys Res Commun 256:57-62. (Pubitemid 29298916)
27. Rothkopf, C, Fahr, A, Fricker, G, Scherphof, GL and Kamps, JA (2005). Uptake of phosphatidylserine-containing liposomes by liver sinusoidal endothelial cells in the serum-free perfused rat liver. Biochim Biophys Acta 1668:10-16. (Pubitemid 40164728)
28. Ichim, TE, Zhong, R and Min, WP (2003). Prevention of allograft rejection by in vitro generated tolerogenic dendritic cells. Transpl Immunol 11:295-306. (Pubitemid 37120549)
29. Hill, JA, Ichim, TE, Kusznieruk, KP, Li, M, Huang, X, Yan, X et al. (2003). Immune modulation by silencing IL-12 production in dendritic cells using small interfering RNA. J Immunol 171:691-696. (Pubitemid 36871378)
30. Gu, X, Xiang, J, Yao, Y and Chen, Z (2006). Effects of RNA interference on CD80 and CD86 expression in bone marrow-derived murine dendritic cells. Scand J Immunol 64:588-594. (Pubitemid 44704307)
31. Geisbert, TW, Hensley, LE, Kagan, E, Yu, EZ, Geisbert, JB, Daddario-DiCaprio, K et al. (2006). Postexposure protection of guinea pigs against a lethal Ebola virus challenge is conferred by RNA interference. J Infect Dis 193(12):1650-1657. (Pubitemid 43839469)
32. Soutschek, J, Akinc, A, Bramlage, B, Charisse, K, Constien, R, Donoghue, M et al. (2004). Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432:173-178. (Pubitemid 39545841)
33. Desjardins, M and Griffiths, G (2003). Phagocytosis: latex leads the way. Curr Opin Cell Biol 15:498-503. (Pubitemid 36928054)
34. Wisse, E, Jacobs, F, Topal, B, Frederik, P and De Geest, B (2008). The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer. Gene Ther 15:1193-1199.
35. Sanderson, S and Shastri, N (1994). LacZ inducible, antigen/MHC-specific T cell hybrids. Int Immunol 6(3):369-376. (Pubitemid 24087229)
36. Ghosh, YK, Visweswariah, SS and Bhattacharya, S (2000). Nature of linkage between the cationic headgroup and cholesteryl skeleton controls gene transfection efficiency. FEBS Lett 473:341-344. (Pubitemid 30249151)
37. McCaffrey, AP, Meuse, L, Pham, TT, Conklin, DS, Hannon, GJ and Kay, MA (2002). RNA interference in adult mice. Nature 418:38-39. (Pubitemid 34742563)
38. Lewis, DL, Hagstrom, JE, Loomis, AG, Wolff, JA and Herweijer, H (2002). Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat Genet 32:107-108. (Pubitemid 34977204)
39. Zender, L, Hutker, S, Liedtke, C, Tillmann, HL, Zender, S, Mundt, B et al. (2003). Caspase 8 small interfering RNA prevents acute liver failure in mice. Proc Natl Acad Sci USA 100:7797-7802. (Pubitemid 36760049)
40. Aouadi, M, Tesz, GJ, Nicoloro, SM, Wang, M, Chouinard, M, Soto, E et al. (2009). Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation. Nature 458:1180-1184.
41. Peer, D, Park, EJ, Morishita, Y, Carman, CV and Shimaoka, M (2008). Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 319:627-630. (Pubitemid 351197066)
42. Zheng, X, Vladau, C, Zhang, X, Suzuki, M, Ichim, TE, Zhang, ZX et al. (2009). A novel in vivo siRNA delivery system specifically targeting dendritic cells and silencing CD40 genes for immunomodulation. Blood 113:2646-2654.
43. Vial, T, Choquet-Kastylevsky, G and Descotes, J (2002). Adverse effects of immunotherapeutics involving the immune system. Toxicology 174:3-11. (Pubitemid 34327821)
44. Descotes, J (2009). Immunotoxicity of monoclonal antibodies. MAbs 1:104-111.
45. Akinc, A, Querbes, W, De, S, Qin, J, Frank-Kamenetsky, M, Jayaprakash, KN et al. (2010). Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol Ther 18:1357-1364.
46. Lazarovits, AI, Poppema, S, Zhang, Z, Khandaker, M, Le Feuvre, CE, Singhal, SK et al. (1996). Prevention and reversal of renal allograft rejection by antibody against CD45RB. Nature 380:717-720. (Pubitemid 26126094)
47. Tan, J, Town, T, Mori, T, Wu, Y, Saxe, M, Crawford, F et al. (2000). CD45 opposes beta-amyloid peptide-induced microglial activation via inhibition of p44/42 mitogenactivated protein kinase. J Neurosci 20:7587-7594.
48. Basadonna, GP, Auersvald, L, Khuong, CQ, Zheng, XX, Kashio, N, Zekzer, D et al. (1998). Antibody-mediated targeting of CD45 isoforms: a novel immunotherapeutic strategy. Proc Natl Acad Sci USA 95:3821-3826. (Pubitemid 28173213)
49. Kortylewski, M, Swiderski, P, Herrmann, A, Wang, L, Kowolik, C, Kujawski, M et al. (2009). In vivo delivery of siRNA to immune cells by conjugation to a TLR9 agonist enhances antitumor immune responses. Nat Biotechnol 27:925-932.
50. de Jong, SD, Basha, G, Wilson, KD, Kazem, M, Cullis, P, Jefferies, W et al. (2010). The immunostimulatory activity of unmethylated and methylated CpG oligodeoxynucleotide is dependent on their ability to colocalize with TLR9 in late endosomes. J Immunol 184:6092-6102.