N-acetylgalactosamine functionalized mixed micellar nanoparticles for targeted delivery of siRNA to liver

Journal of Controlled Release

Volumn 166, Issue 2, 2013, Pages 106-114

  Wang, Hong Xia ^a   Xiong, Meng Hua ^b   Wang, Yu Cai ^b   Zhu, Jing ^a   Wang, Jun ^a  

^a UNIVERSITY OF SCIENCE AND TECHNOLOGY OF CHINA   (China)

^b UNIVERSITY OF SCIENCE AND TECHNOLOGY OF CHINA   (China)

Author keywords

Hepatocytes targeting; Micellar nanoparticles; N acetylgalactosamine; Polyphosphoester; siRNA delivery

Indexed keywords

HEPATOCYTES; MICELLAR NANOPARTICLES; N-ACETYLGALACTOSAMINE; POLYPHOSPHOESTERS; SIRNA DELIVERY;

ETHYLENE; GENE EXPRESSION; GENETIC ENGINEERING; POLYETHYLENE GLYCOLS; RNA; ZETA POTENTIAL;

NANOPARTICLES;

APOLIPOPROTEIN B; MACROGOL; N ACETYLGALACTOSAMINE; NANOPARTICLE; POLYCAPROLACTONE; SMALL INTERFERING RNA;

ANIMAL CELL; AQUEOUS SOLUTION; ARTICLE; CONTROLLED STUDY; DRUG CYTOTOXICITY; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE SILENCING; GENE TARGETING; IN VITRO GENE TRANSFER; IN VIVO GENE TRANSFER; LIVER CELL; MALE; MICELLE; MOUSE; NONHUMAN; PRIORITY JOURNAL; RECEPTOR DOWN REGULATION; ZETA POTENTIAL;

3T3 CELLS; ACETYLGALACTOSAMINE; ANIMALS; APOLIPOPROTEINS B; CELL SURVIVAL; DRUG CARRIERS; DRUG DELIVERY SYSTEMS; ELECTROCHEMISTRY; ENDOCYTOSIS; ETHYLENE GLYCOLS; GENE SILENCING; HEPATOCYTES; LIVER; MALE; MICE; MICE, INBRED BALB C; MICELLES; NANOPARTICLES; PARTICLE SIZE; POLYESTERS; RNA, SMALL INTERFERING;

EID: 84873960174     PISSN: 01683659     EISSN: 18734995     Source Type: Journal    
DOI: 10.1016/j.jconrel.2012.12.017     Document Type: Article

References (57)

1. L.J. Li, H.Y. Wang, Z.Y. Ong, K.J. Xu, P.L.R. Ee, S.S. Zheng, J.L. Hedrick, and Y.Y. Yang Polymer-and lipid-based nanoparticle therapeutics for the treatment of liver diseases Nano Today 5 2010 296 312
2. A.P. McCaffrey, H. Nakai, K. Pandey, Z. Huang, F.H. Salazar, H. Xu, S.F. Wieland, P.L. Marion, and M.A. Kay Inhibition of hepatitis B virus in mice by RNA interference Nat. Biotechnol. 21 2003 639 644
3. T.S. Zimmermann, A.C.H. Lee, A. Akinc, B. Bramlage, D. Bumcrot, M.N. Fedoruk, J. Harborth, J.A. Heyes, L.B. Jeffs, and M. John RNAi-mediated gene silencing in non-human primates Nature 441 2006 111 114
4. Y. Sato, K. Murase, J. Kato, M. Kobune, T. Sato, Y. Kawano, R. Takimoto, K. Takada, K. Miyanishi, and T. Matsunaga Resolution of liver cirrhosis using vitamin A-coupled liposomes to deliver siRNA against a collagen-specific chaperone Nat. Biotechnol. 26 2008 431 442
5. M. Frank-Kamenetsky, A. Grefhorst, N.N. Anderson, T.S. Racie, B. Bramlage, A. Akinc, D. Butler, K. Charisse, R. Dorkin, and Y. Fan Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates Proc. Natl. Acad. Sci. U. S. A. 105 2008 11915 11920
6. D.V. Morrissey, J.A. Lockridge, L. Shaw, K. Blanchard, K. Jensen, W. Breen, K. Hartsough, L. Machemer, S. Radka, and V. Jadhav Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs Nat. Biotechnol. 23 2005 1002 1007
7. C.R. Jung, J. Yoo, Y.J. Jang, S. Kim, I.S. Chu, Y.I. Yeom, J.Y. Choi, and D.S. Iml Adenovirus-mediated transfer of siRNA against PTTG1 inhibits liver cancer cell growth in vitro and in vivo Hepatology 43 2006 1042 1052
8. Y.C. Chen, M.C. Lin, H. Yao, H. Wang, A.Q. Zhang, J. Yu, C.K. Hui, G.K. Lau, M.L. He, J. Sung, and H.F. Kung Lentivirus-mediated RNA interference targeting enhancer of zeste homolog 2 inhibits hepatocellular carcinoma growth through down-regulation of stathmin Hepatology 46 2007 200 208
9. P. Romano, D. McCallus, and C. Pachuk RNA interference-mediated prevention and therapy for hepatocellular carcinoma Oncogene 25 2006 3857 3865
10. M. Mével, N. Kamaly, S. Carmona, M.H. Oliver, M.R. Jorgensen, C. Crowther, F.H. Salazar, P.L. Marion, M. Fujino, and Y. Natori DODAG; a versatile new cationic lipid that mediates efficient delivery of pDNA and siRNA J. Controlled Release 143 2010 222 232
11. D.V. Morrissey, K. Blanchard, L. Shaw, K. Jensen, J.A. Lockridge, B. Dickinson, J.A. McSwiggen, C. Vargeese, K. Bowman, and C.S. Shaffer Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication Hepatology 41 2005 1349 1356
12. J. Soutschek, A. Akinc, B. Bramlage, K. Charisse, R. Constien, M. Donoghue, S. Elbashir, A. Geick, P. Hadwiger, and J. Harborth Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs Nature 432 2004 173 178
13. J.M. Layzer, A.P. McCaffrey, A.K. Tanner, Z. Huang, M.A. Kay, and B.A. Sullenger In vivo activity of nuclease-resistant siRNAs RNA 10 2004 766 771
14. S. Lau, B. Graham, N. Cao, B.J. Boyd, C.W. Pouton, and P.J. White Enhanced extravasation, stability and in vivo cardiac gene silencing via in situ siRNA-albumin conjugation Mol. Pharmaceutics 9 2012 71 80
15. X.Z. Yang, S. Dou, T.M. Sun, C.Q. Mao, H.X. Wang, and J. Wang Systemic delivery of siRNA with cationic lipid assisted PEG-PLA nanoparticles for cancer therapy J. Controlled Release 165 2011 203 211
16. F. Jacobs, E. Wisse, and B. De Geest The role of liver sinusoidal cells in hepatocyte-directed gene transfer Am. J. Pathol. 176 2010 14
17. E. Wisse An electron microscopic study of the fenestrated endothelial lining of rat liver sinusoids J. Ultrastruct. Res. 31 1970 125 150
18. E. Wisse, R. De Zanger, K. Charels, P. Van Der Smissen, and R. McCuskey The liver sieve: considerations concerning the structure and function of endothelial fenestrae, the sinusoidal wall and the space of Disse Hepatology 5 1985 683 692
19. K.T. Love, K.P. Mahon, C.G. Levins, K.A. Whitehead, W. Querbes, J.R. Dorkin, J. Qin, W. Cantley, L.L. Qin, T. Racie, M. Frank-Kamenetsky, K.N. Yip, R. Alvarez, D.W. Sah, A. de Fougerolles, K. Fitzgerald, V. Koteliansky, A. Akinc, R. Langer, and D.G. Anderson Lipid-like materials for low-dose, in vivo gene silencing Proc. Natl. Acad. Sci. U. S. A. 107 2010 1864 1869
20. S.C. Semple, A. Akinc, J.X. Chen, A.P. Sandhu, B.L. Mui, C.K. Cho, D.W.Y. Sah, D. Stebbing, E.J. Crosley, E. Yaworski, I.M. Hafez, J.R. Dorkin, J. Qin, K. Lam, K.G. Rajeev, K.F. Wong, L.B. Jeffs, L. Nechev, M.L. Eisenhardt, M. Jayaraman, M. Kazem, M.A. Maier, M. Srinivasulu, M.J. Weinstein, Q.M. Chen, R. Alvarez, S.A. Barros, S. De, S.K. Klimuk, T. Borland, V. Kosovrasti, W.L. Cantley, Y.K. Tam, M. Manoharan, M.A. Ciufolini, M.A. Tracy, A. de Fougerolles, I. MacLachlan, P.R. Cullis, T.D. Madden, and M.J. Hope Rational design of cationic lipids for siRNA delivery Nat. Biotechnol. 28 2010 172 178
21. A. Akinc, W. Querbes, S. De, J. Qin, M. Frank-Kamenetsky, K.N. Jayaprakash, M. Jayaraman, K.G. Rajeev, W.L. Cantley, J.R. Dorkin, J.S. Butler, L.L. Qin, T. Racie, A. Sprague, E. Fava, A. Zeigerer, M.J. Hope, M. Zerial, D.W. Sah, K. Fitzgerald, M.A. Tracy, M. Manoharan, V. Koteliansky, A. de Fougerolles, and M.A. Maier Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms Mol. Ther. 18 2010 1357 1364
22. M.E. Davis The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic Mol. Pharmaceutics 6 2009 659 668
23. D.B. Rozema, D.L. Lewis, D.H. Wakefield, S.C. Wong, J.J. Klein, P.L. Roesch, S.L. Bertin, T.W. Reppen, Q. Chu, A.V. Blokhin, J.E. Hagstrom, and J.A. Wolff Dynamic polyconjugates for targeted in vivo delivery of siRNA to hepatocytes Proc. Natl. Acad. Sci. U. S. A. 104 2007 12982 12987
24. T.M. Sun, J.Z. Du, L.F. Yan, H.Q. Mao, and J. Wang Self-assembled biodegradable micellar nanoparticles of amphiphilic and cationic block copolymer for siRNA delivery Biomaterials 29 2008 4348 4355
25. C.W. Beh, W.Y. Seow, Y. Wang, Y. Zhang, Z.Y. Ong, P.L.R. Ee, and Y.Y. Yang Efficient delivery of Bcl-2-targeted siRNA using cationic polymer nanoparticles: downregulating mRNA expression level and sensitizing cancer cells to anticancer drug Biomacromolecules 10 2009 41 48
26. X.B. Xiong, H. Uludag, and A. Lavasanifar Biodegradable amphiphilic poly (ethylene oxide)-block-polyesters with grafted polyamines as supramolecular nanocarriers for efficient siRNA delivery Biomaterials 30 2009 242 253
27. C.Q. Mao, J.Z. Du, T.M. Sun, Y.D. Yao, P.Z. Zhang, E.W. Song, and J. Wang A biodegradable amphiphilic and cationic triblock copolymer for the delivery of siRNA targeting the acid ceramidase gene for cancer therapy Biomaterials 32 2011 3124 3133
28. H.J. Yu, Y.L. Zou, Y.G. Wang, X.N. Huang, G. Huang, B.D. Sumer, D.A. Boothman, and J.M. Gao Overcoming endosomal barrier by amphotericin B-loaded dual pH-responsive PDMA-b-PDPA micelleplexes for siRNA Delivery ACS Nano 5 2011 9246 9255
29. T. Segura, and J.A. Hubbell Synthesis and in vitro characterization of an ABC triblock copolymer for siRNA delivery Bioconjugate Chem. 18 2007 736 745
30. T.M. Sun, J.Z. Du, Y.D. Yao, C.Q. Mao, S. Dou, S.Y. Huang, P.Z. Zhang, K.W. Leong, E.W. Song, and J. Wang Simultaneous delivery of siRNA and paclitaxel via a "two-in-one" micelleplex promotes synergistic tumor suppression ACS Nano 5 2011 1483 1494
31. H. Otsuka, Y. Nagasaki, and K. Kataoka PEGylated nanoparticles for biological and pharmaceutical applications Adv. Drug Delivery Rev. 55 2003 403 419
32. A. Sato, S.W. Choi, M. Hirai, A. Yamayoshi, R. Moriyama, T. Yamano, M. Takagi, A. Kano, A. Shimamoto, and A. Maruyama Polymer brush-stabilized polyplex for a siRNA carrier with long circulatory half-life J. Controlled Release 122 2007 209 216
33. Z. Zhao, J. Wang, H.Q. Mao, and K.W. Leong Polyphosphoesters in drug and gene delivery Adv. Drug Delivery Rev. 55 2003 483 499
34. H.Q. Mao, and K.W. Leong Design of polyphosphoester-DNA nanoparticles for non-viral gene delivery Adv. Genet. 53 2005 275 306
35. L. Mu, T. Elbayoumi, and V. Torchilin Mixed micelles made of poly (ethylene glycol)-phosphatidylethanolamine conjugate and d-alpha-tocopheryl polyethylene glycol 1000 succinate as pharmaceutical nanocarriers for camptothecin Int. J. Pharm. 306 2005 142 149
36. Y. Wang, L. Yu, L. Han, X. Sha, and X. Fang Difunctional Pluronic copolymer micelles for paclitaxel delivery: synergistic effect of folate-mediated targeting and Pluronic-mediated overcoming multidrug resistance in tumor cell lines Int. J. Pharm. 337 2007 63 73
37. L. Li, and Y.B. Tan Preparation and properties of mixed micelles made of Pluronic polymer and PEG-PE J. Colloid Interface Sci. 317 2008 326 331
38. H. Yin, E.S. Lee, D. Kim, K.H. Lee, K.T. Oh, and Y.H. Bae Physicochemical characteristics of pH-sensitive poly (L-histidine)-b-poly (ethylene glycol)/poly (L-lactide)-b-poly (ethylene glycol) mixed micelles J. Controlled Release 126 2008 130 138
39. Y. Bae, T.A. Diezi, A. Zhao, and G.S. Kwon Mixed polymeric micelles for combination cancer chemotherapy through the concurrent delivery of multiple chemotherapeutic agents J. Controlled Release 122 2007 324 330
40. C.P. O'Neil, A.J. van der Vlies, D. Velluto, C. Wandrey, D. Demurtas, J. Dubochet, and J.A. Hubbell Extracellular matrix binding mixed micelles for drug delivery applications J. Controlled Release 137 2009 146 151
41. E.S. Lee, K. Na, and Y.H. Bae Polymeric micelle for tumor pH and folate-mediated targeting J. Controlled Release 91 2003 103 113
42. S.S. Kulthe, N.N. Inamdar, Y.M. Choudhari, S.M. Shirolikar, L.C. Borde, and V.K. Mourya Mixed micelle formation with hydrophobic and hydrophilic Pluronic block copolymers: implications for controlled and targeted drug delivery, Bahadur Colloids Surf. B Biointerfaces 88 2011 691 696
43. O. Khorev, D. Stokmaier, O. Schwardt, B. Cutting, and B. Ernst Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor Bioorg. Med. Chem. 16 2008 5216 5231
44. Y.C. Wang, L.Y. Tang, T.M. Sun, C.H. Li, M.H. Xiong, and J. Wang Self-assembled micelles of biodegradable triblock copolymers based on poly (ethyl ethylene phosphate) and poly (ε-caprolactone) as drug carriers Biomacromolecules 9 2007 388 395
45. Y.C. Wang, X.Q. Liu, T.M. Sun, M.H. Xiong, and J. Wang Functionalized micelles from block copolymer of polyphosphoester and poly (ε-caprolactone) for receptor-mediated drug delivery J. Controlled Release 128 2008 32 40
46. F. Suriano, R. Pratt, J.P.K. Tan, N. Wiradharma, A. Nelson, Y.Y. Yang, P. Dubois, and J.L. Hedrick Synthesis of a family of amphiphilic glycopolymers via controlled ring-opening polymerization of functionalized cyclic carbonates and their application in drug delivery Biomaterials 31 2010 2637 2645
47. K. Babiuch, D. Pretzel, T. Tolstik, A. Vollrath, S. Stanca, F. Foertsch, C.R. Becer, M. Gottschaldt, C. Biskup, and U.S. Schubert Uptake of well-defined, highly glycosylated, pentafluorostyrene-based polymers and nanoparticles by human hepatocellular carcinoma cells Macromol. Biosci. 12 2012 1190 1199
48. M. Spiess The asialoglycoprotein receptor: a model for endocytic transport receptors Biochemistry 29 1990 10009 10018
49. F. Boess, M. Kamber, S. Romer, R. Gasser, D. Muller, S. Albertini, and L. Suter Gene expression in two hepatic cell lines, cultured primary hepatocytes, and liver slices compared to the in vivo liver gene expression in rats: possible implications for toxicogenomics use of in vitro systems Toxicol. Sci. 73 2003 386
50. E.Z. Jonathan, H.J.C. Chung, H. Han, and E.D. Mark Polycation-siRNA nanoparticles can disassemble at the kidney glomerular basement membrane Proc. Natl. Acad. Sci. U. S. A. 109 2012 3137 3142
51. J.J. Widmann, R.S. Cotran, and H.D. Fahimi Mononuclear phagocytes (Kupffer cells) and endothelial cells J. Cell Biol. 52 1972 159
52. E. Wisse, F. Braet, D. Luo, R. De Zanger, D. Jans, E. Crabbe, and A. Vermoesen Structure and function of sinusoidal lining cells in the liver Toxicol. Pathol. 24 1996 100 111
53. Z. Kmieć Cooperation of Liver Cells in Health and Disease 2001 Springer Verlag
54. R.M. Crooke, M.J. Graham, K.M. Lemonidis, C.P. Whipple, S. Koo, and R.J. Perera An apolipoprotein B antisense oligonucleotide lowers LDL cholesterol in hyperlipidemic mice without causing hepatic steatosis J. Lipid Res. 46 2005 872 884
55. J.T. Marques, and B.R.G. Williams Activation of the mammalian immune system by siRNAs Nat. Biotechnol. 23 2005 1399 1405
56. A.D. Judge, V. Sood, J.R. Shaw, D. Fang, K. McClintock, and I. MacLachlan Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA Nat. Biotechnol. 23 2005 457 462
57. W.S. Cho, M. Cho, J. Jeong, M. Choi, H.Y. Cho, B.S. Han, S.H. Kim, H.O. Kim, Y.T. Lim, and B.H. Chung Acute toxicity and pharmacokinetics of 13 nm-sized PEG-coated gold nanoparticles Toxicol. Appl. Pharmacol. 236 2009 16 24