Non-viral siRNA Delivery Systems |
Won, Young-Wook
(Department of Bioengineering, Institute for Bioengineering and Biopharmaceutical Research, Hanyang University)
Jang, Yeon-Lim (College of Pharmacy, Sungkyunkwan University) Kim, Jin-Seok (College of Pharmacy, Sookmyung Women's University) Jeong, Ji-Hoon (College of Pharmacy, Sungkyunkwan University) Kim, Yong-Hee (Department of Bioengineering, Institute for Bioengineering and Biopharmaceutical Research, Hanyang University) |
1 | Zimmermann, T.S., Lee, A.C., Akinc, A., Bramlage, B., Bumcrot, D., Fedoruk, M.N., Harborth, J., Heyes, J.A., Jeffs, L.B., John, M., Judge, A.D., Lam, K., McClintock, K., Nechev, L.V., Palmer, L.R., Racie, T., Rohl, I., Seiffert, S., Shanmugam, S., Sood, V., Soutschek, J., Toudjarska, I., Wheat, A.J., Yaworski, E., Zedalis, W., Koteliansky, V., Manoharan, M., Vornlocher, H.P., MacLachlan, I., 2006. RNAi-mediated gene silencing in non-human primates. Nature 441, 111-114. DOI ScienceOn |
2 | Villares, G.J., Zigler, M., Wang, H., Melnikova, V.O., Wu, H., Friedman, R., Leslie, M.C., Vivas-Mejia, P.E., Lopez-Berestein, G., Sood, A.K., Bar-Eli, M., 2008. Targeting melanoma growth and metastasis with systemic delivery of liposomeincorporated protease-activated receptor-1 small interfering RNA. Cancer Res 68, 9078-9086. DOI |
3 | Wadia, J.S., Dowdy, S.F., 2005. Transmembrane delivery of protein and peptide drugs by TAT-mediated transduction in the treatment of cancer. Advanced Drug Delivery Reviews 57, 579-596. DOI |
4 | Wadia, J.S., Stan, R.V., Dowdy, S.F., 2004. Transducible TAT-HA fusogenic peptide enhances escape of TAT-fusion proteins after lipid raft macropinocytosis. Nat Med 10, 310-315. DOI |
5 | Wang, Y.-H., Hou, Y.-W., Lee, H.-J., 2007. An intracellular delivery method for siRNA by an arginine-rich peptide. Journal of Biochemical and Biophysical Methods 70, 579-586. DOI |
6 | Watanabe, K., Harada-Shiba, M., Suzuki, A., Gokuden, R., Kurihara, R., Sugao, Y., Mori, T., Katayama, Y., Niidome, T., 2009. In vivo siRNA delivery with dendritic poly(l-lysine) for the treatment of hypercholesterolemia. Molecular BioSystems 5, 1306-1310. DOI |
7 | Won, Y.-W., Kim, H.A., Lee, M., Kim, Y.-H., 2010a. Reducible Poly(oligo-D-arginine) for Enhanced Gene Expression in Mouse Lung by Intratracheal Injection. Mol Ther 18, 734-742. DOI |
8 | Won, Y.-W., Kim, J.-K., Cha, M.-J., Hwang, K.-C., Choi, D., Kim, Y.-H., 2010b. Prolongation and enhancement of the anti-apoptotic effects of PTD-Hsp27 fusion proteins using an injectable thermo-reversible gel in a rat myocardial infarction model. Journal of Controlled Release 144, 181-189. DOI |
9 | Yano, J., Hirabayashi, K., Nakagawa, S., Yamaguchi, T., Nogawa, M., Kashimori, I., Naito, H., Kitagawa, H., Ishiyama, K., Ohgi, T., Irimura, T., 2004. Antitumor activity of small interfering RNA/cationic liposome complex in mouse models of cancer. Clin Cancer Res 10, 7721-7726. DOI |
10 | Stevenson, M., Ramos-Perez, V., Singh, S., Soliman, M., Preece, J.A., Briggs, S.S., Read, M.L., Seymour, L.W., 2008. Delivery of siRNA mediated by histidine-containing reducible polycations. Journal of Controlled Release 130, 46-56. DOI |
11 | Tanaka, K., Kanazawa, T., Ogawa, T., Takashima, Y., Fukuda, T., Okada, H., Disulfide crosslinked stearoyl carrier peptides containing arginine and histidine enhance siRNA uptake and gene silencing. International Journal of Pharmaceutics In Press, Corrected Proof. |
12 | Torchilin, V.P., Levchenko, T.S., Rammohan, R., Volodina, N., Papahadjopoulos-Sternberg, B., D'Souza, G.G.M., 2003. Cell transfection in vitro and in vivo with nontoxic TAT peptideliposome-DNA complexes. Proceedings of the National Academy of Sciences of the United States of America 100, 1972-1977. DOI |
13 | Torchilin, V.P., Rammohan, R., Weissig, V., Levchenko, T.S., 2001. TAT peptide on the surface of liposomes affords their efficient intracellular delivery even at low temperature and in the presence of metabolic inhibitors. Proceedings of the National Academy of Sciences of the United States of America 98, 8786-8791. DOI |
14 | Troussard, A.A., Mawji, N.M., Ong, C., Mui, A., St -Arnaud, R., Dedhar, S., 2003. Conditional knock-out of integrin-linked kinase demonstrates an essential role in protein kinase B/Akt activation. J Biol Chem 278, 22374-22378. DOI |
15 | Urban-Klein, B., Werth, S., Abuharbeid, S., Czubayko, F., Aigner, A., 2005. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther 12, 461-466. DOI |
16 | Schiffelers, R.M., Woodle, M.C., Scaria, P., 2004. Pharmaceutical prospects for RNA interference. Pharm Res 21, 1-7. DOI |
17 | Veldhoen, S., Laufer, S.D., Trampe, A., Restle, T., 2006. Cellular delivery of small interfering RNA by a non-covalently attached cell-penetrating peptide: quantitative analysis of uptake and biological effect. Nucl. Acids Res. 34, 6561-6573. DOI |
18 | Rahbek, U.L., Howard, K.A., Oupicky, D., Manickam, D.S., Dong, M., Nielsen, A.F., Hansen, T.B., Besenbacher, F., Kjems, J., 2008. Intracellular siRNA and precursor miRNA trafficking using bioresponsive copolypeptides. The Journal of Gene Medicine 10, 81-93. DOI |
19 | Santel, A., Aleku, M., Keil, O., Endruschat, J., Esche, V., Durieux, B., Loffler, K., Fechtner, M., Rohl, T., Fisch, G., Dames, S., Arnold, W., Giese, K., Klippel, A., Kaufmann, J., 2006. RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy. Gene Ther 13, 1360-1370. DOI ScienceOn |
20 | Simeoni, F., Morris, M.C., Heitz, F., Divita, G., 2003. Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells. Nucl. Acids Res. 31, 2717-2724. DOI |
21 | Sonawane, N.D., Szoka, F.C., Jr., Verkman, A.S., 2003. Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes. J Biol Chem 278, 44826-44831. DOI ScienceOn |
22 | Soriano, P., Dijkstra, J., Legrand, A., Spanjer, H., Londos-Gagliardi, D., Roerdink, F., Scherphof, G., Nicolau, C., 1983. Targeted and nontargeted liposomes for in vivo transfer to rat liver cells of a plasmid containing the preproinsulin I gene. Proc Natl Acad Sci U S A 80, 7128-7131. DOI |
23 | Morrissey, D.V., Lockridge, J.A., Shaw, L., Blanchard, K., Jensen, K., Breen, W., Hartsough, K., Machemer, L., Radka, S., Jadhav, V., Vaish, N., Zinnen, S., Vargeese, C., Bowman, K., Shaffer, C.S., Jeffs, L.B., Judge, A., MacLachlan, I., Polisky, B., 2005. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat Biotechnol 23, 1002-1007. DOI |
24 | Soutschek, J., Akinc, A., Bramlage, B., Charisse, K., Constien, R., Donoghue, M., Elbashir, S., Geick, A., Hadwiger, P., Harborth, J., John, M., Kesavan, V., Lavine, G., Pandey, R.K., Racie, T., Rajeev, K.G., Rohl, I., Toudjarska, I., Wang, G., Wuschko, S., Bumcrot, D., Koteliansky, V., Limmer, S., Manoharan, M., Vornlocher, H.P., 2004. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432, 173-178. DOI |
25 | Spagnou, S., Miller, A.D., Keller, M., 2004. Lipidic carriers of siRNA: differences in the formulation, cellular uptake, and delivery with plasmid DNA. Biochemistry 43, 13348-13356. DOI ScienceOn |
26 | Mok, H., Park, T.G., 2008b. Self-crosslinked and reducible fusogenic peptides for intracellular delivery of siRNA. Biopolymers 89, 881-888. DOI |
27 | Moschos, S.A., Jones, S.W., Perry, M.M., Williams, A.E., Erjefalt, J.S., Turner, J.J., Barnes, P.J., Sproat, B.S., Gait, M.J., Lindsay, M.A., 2007. Lung Delivery Studies Using siRNA Conjugated to TAT(4860) and Penetratin Reveal Peptide Induced Reduction in Gene Expression and Induction of Innate Immunity. Bioconjugate Chemistry 18, 1450-1459. DOI |
28 | Nakamura, Y., Kogure, K., Futaki, S., Harashima, H., 2007. Octaarginine-modified multifunctional envelope-type nano device for siRNA. Journal of Controlled Release 119, 360-367. DOI |
29 | Okamoto, Y., Watanabe, M., Miyatake, K., Morimoto, M., Shigemasa, Y., Minami, S., 2002. Effects of chitin/chitosan and their oligomers/monomers on migrations of fibroblasts and vascular endothelium. Biomaterials 23, 1975-1979. DOI |
30 | Peer, D., Park, E.J., Morishita, Y., Carman, C.V., Shimaoka, M., 2008. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 319, 627-630. DOI ScienceOn |
31 | Pirollo, K.F., Rait, A., Zhou, Q., Hwang, S.H., Dagata, J.A., Zon, G., Hogrefe, R.I., Palchik, G., Chang, E.H., 2007. Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system. Cancer Res 67, 2938-2943. DOI ScienceOn |
32 | Potente, M., Fisslthaler, B., Busse, R., Fleming, I., 2003. 11,12-Epoxyeicosatrienoic acid-induced inhibition of FOXO factors promotes endothelial proliferation by down-regulating p27Kip1. J Biol Chem 278, 29619-29625. DOI |
33 | Lu, J.J., Langer, R., Chen, J., 2009. A novel mechanism is involved in cationic lipid-mediated functional siRNA delivery. Mol Pharm 6, 763-771. DOI |
34 | Lundberg, P., El-Andaloussi, S., Sutlu, T., Johansson, H., Langel, U., 2007. Delivery of short interfering RNA using endosomolytic cell-penetrating peptides. FASEB J. 21, 2664-2671. DOI |
35 | Malek, A., Czubayko, F., Aigner, A., 2008. PEG grafting of polyethylenimine (PEI) exerts different effects on DNA transfection and siRNA-induced gene targeting efficacy. J Drug Target 16, 124-139. DOI |
36 | Malek, A., Merkel, O., Fink, L., Czubayko, F., Kissel, T., Aigner, A., 2009. In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(-PEG)/siRNA complexes. Toxicol Appl Pharmacol 236, 97-108. DOI |
37 | McManus, M.T., Sharp, P.A., 2002. Gene silencing in mammals by small interfering RNAs. Nat Rev Genet 3, 737-747. DOI |
38 | Meade, B.R., Dowdy, S.F., 2007. Exogenous siRNA delivery using peptide transduction domains/cell penetrating peptides. Advanced Drug Delivery Reviews 59, 134-140. DOI |
39 | Merritt, W.M., Lin, Y.G., Spannuth, W.A., Fletcher, M.S., Kamat, A.A., Han, L.Y., Landen, C.N., Jennings, N., De Geest, K., Langley, R.R., Villares, G., Sanguino, A., Lutgendorf, S.K., Lopez-Berestein, G., Bar-Eli, M.M., Sood, A.K., 2008. Effect of interleukin-8 gene silencing with liposome-encapsulated small interfering RNA on ovarian cancer cell growth. J Natl Cancer Inst 100, 359-372. DOI ScienceOn |
40 | Merkel, O.M., Beyerle, A., Librizzi, D., Pfestroff, A., Behr, T.M., Sproat, B., Barth, P.J., Kissel, T., 2009. Nonviral siRNA delivery to the lung: investigation of PEG-PEI polyplexes and their in vivo performance. Mol Pharm 6, 1246-1260. DOI |
41 | Mok, H., Park, T.G., 2008a. Self-crosslinked and reducible fusogenic peptides for intracellular delivery of siRNA. Biopolymers. |
42 | Konate, K., Crombez, L., Deshayes, S.b., Decaffmeyer, M., Thomas, A., Brasseur, R., Aldrian, G., Heitz, F., Divita, G., 2010. Insight into the Cellular Uptake Mechanism of a Secondary Amphipathic Cell-Penetrating Peptide for siRNA Delivery. Biochemistry 49, 3393-3402. DOI |
43 | Landen, C.N., Jr., Chavez-Reyes, A., Bucana, C., Schmandt, R., Deavers, M.T., Lopez-Berestein, G., Sood, A.K., 2005. Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery. Cancer Res 65, 6910-6918. DOI |
44 | Law, M., Jafari, M., Chen, P., 2008. Physicochemical characterization of siRNA-peptide complexes. Biotechnology Progress 24, 957-963. DOI |
45 | Leirdal, M., Sioud, M., 2002. Gene silencing in mammalian cells by preformed small RNA duplexes. Biochem Biophys Res Commun 295, 744-748. DOI |
46 | Leng, Q., Scaria, P., Zhu, J., Ambulos, N., Campbell, P., Mixson, A.J., 2005. Highly branched HK peptides are effective carriers of siRNA. The Journal of Gene Medicine 7, 977-986. DOI |
47 | Lorenz, C., Hadwiger, P., John, M., Vornlocher, H.P., Unverzagt, C., 2004. Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Bioorg Med Chem Lett 14, 4975-4977. DOI |
48 | Leu, Y.W., Rahmatpanah, F., Shi, H., Wei, S.H., Liu, J.C., Yan, P.S., Huang, T.H., 2003. Double RNA interference of DNMT3b and DNMT1 enhances DNA demethylation and gene reactivation. Cancer Res 63, 6110-6115. |
49 | Li, S.D., Chono, S., Huang, L., 2008. Efficient gene silencing in metastatic tumor by siRNA formulated in surface-modified nanoparticles. J Control Release 126, 77-84. DOI |
50 | Lim, K.S., Won, Y.-W., Park, Y.S., Kim, Y.-H., 2010. Preparation and functional analysis of recombinant protein transduction domain-metallothionein fusion proteins. Biochimie 92, 964-970. DOI |
51 | Khoury, M., Louis-Plence, P., Escriou, V., Noel, D., Largeau, C., Cantos, C., Scherman, D., Jorgensen, C., Apparailly, F., 2006. Efficient new cationic liposome formulation for systemic delivery of small interfering RNA silencing tumor necrosis factor alpha in experimental arthritis. Arthritis Rheum 54, 1867-1877. DOI |
52 | Kim, S.H., Jeong, J.H., Kim, T.I., Kim, S.W., Bull, D.A., 2009. VEGF siRNA delivery system using arginine-grafted bioreducible poly(disulfide amine). Mol Pharm 6, 718-726. DOI |
53 | Kim, S.H., Jeong, J.H., Lee, S.H., Kim, S.W., Park, T.G., 2006a. PEG conjugated VEGF siRNA for anti-angiogenic gene therapy. J Control Release 116, 123-129. DOI |
54 | Kim, S.H., Jeong, J. H., Lee, S. H., Kim, S. W., Park, T. G. , 2008. Local and systemic delivery of VEGF siRNA using polyelectrolyte complex micelles for effective treatment of cancer. J Control Release in press. |
55 | Kinouchi, N., Ohsawa, Y., Ishimaru, N., Ohuchi, H., Sunada, Y., Hayashi, Y., Tanimoto, Y., Moriyama, K., Noji, S., 2008. Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass. Gene Ther 15, 1126-1130. DOI |
56 | Kim, S.H., Mok, H., Jeong, J.H., Kim, S.W., Park, T.G., 2006b. Comparative evaluation of target-specific GFP gene silencing efficiencies for antisense ODN, synthetic siRNA, and siRNA plasmid complexed with PEI-PEG-FOL conjugate. Bioconjug Chem 17, 241-244. DOI |
57 | Kim, S.W., Kim, N.Y., Choi, Y.B., Park, S.H., Yang, J.M., Shin, S., 2010. RNA interference in vitro and in vivo using an arginine peptide/siRNA complex system. Journal of Controlled Release 143, 335-343. DOI |
58 | Kim, W.J., Christensen, L.V., Jo, S., Yockman, J.W., Jeong, J.H., Kim, Y.-H., Kim, S.W., 2006c. Cholesteryl Oligoarginine Delivering Vascular Endothelial Growth Factor siRNA Effectively Inhibits Tumor Growth in Colon Adenocarcinoma. Mol Ther 14, 343-350. DOI |
59 | Ifediba, M.A., Medarova, Z., Ng, S.-w., Yang, J., Moore, A., 2010. siRNA Delivery to CNS Cells using a Membrane Translocation Peptide. Bioconjugate Chemistry 21, 803-806. DOI |
60 | Inoue, Y., Kurihara, R., Tsuchida, A., Hasegawa, M., Nagashima, T., Mori, T., Niidome, T., Katayama, Y., Okitsu, O., 2008. Efficient delivery of siRNA using dendritic poly(l-lysine) for lossof-function analysis. Journal of Controlled Release 126, 59-66. DOI |
61 | Jeong, J.H., Christensen, L.V., Yockman, J.W., Zhong, Z., Engbersen, J.F.J., Kim, W.J., Feijen, J., Kim, S.W., 2007a. Reducible poly(amido ethylenimine) directed to enhance RNA interference. Biomaterials 28 1912-1917. DOI |
62 | Jeong, J.H., Kim, S.H., Lee, M., Kim, W.J., Park, T.G., Ko, K.S., Kim, S.W., Non-viral systemic delivery of Fas siRNA suppresses cyclophosphamide-induced diabetes in NOD mice. J Control Release 143, 88-94. DOI |
63 | Kang, H., DeLong, R., Fisher, M., Juliano, R., 2005. Tat-Conjugated PAMAM Dendrimers as Delivery Agents for Antisense and siRNA Oligonucleotides. Pharmaceutical Research 22, 2099-2106. DOI |
64 | Jeong, J.H., Kim, S.W., Park, T.G., 2007b. Molecular design of functional polymers for gene therapy. Progress in Polymer Science 32, 1239-1274. DOI |
65 | Jiang, G., Park, K., Kim, J., Kim, K.S., Hahn, S.K., 2009. Target specific intracellular delivery of siRNA/PEI-HA complex by receptor mediated endocytosis. Mol Pharm 6, 727-737. DOI |
66 | Kaiser, P.K., Symons, R.C., Shah, S.M., Quinlan, E.J., Tabandeh, H., Do, D.V., Reisen, G., Lockridge, J.A., Short, B., Guerciolini, R., Nguyen, Q.D., RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027. Am J Ophthalmol 150, 33-39 e32. DOI |
67 | Dorsett, Y., Tuschl, T., 2004. siRNAs: applications in functional genomics and potential as therapeutics. Nat Rev Drug Discov 3, 318-329. DOI |
68 | Eguchi, A., Meade, B.R., Chang, Y.-C., Fredrickson, C.T., Willert, K., Puri, N., Dowdy, S.F., 2009. Efficient siRNA delivery into primary cells by a peptide transduction domain-dsRNA binding domain fusion protein. Nat Biotech 27, 567-571. DOI |
69 | Felgner, P.L., Ringold, G.M., 1989. Cationic liposome-mediated transfection. Nature 337, 387-388. DOI |
70 | Frankel, A.D., Pabo, C.O., 1988. Cellular uptake of the tat protein from human immunodeficiency virus. Cell 55, 1189-1193. DOI |
71 | Grayson, A.C., Ma, J., Putnam, D., 2006. Kinetic and efficacy analysis of RNA interference in stably and transiently expressing cell lines. Mol Pharm 3, 601-613. DOI |
72 | Howard, K.A., Rahbek, U.L., Liu, X., Damgaard, C.K., Glud, S.Z., Andersen, M.O., Hovgaard, M.B., Schmitz, A., Nyengaard, J.R., Besenbacher, F., Kjems, J., 2006. RNA interference in vitro and in vivo using a novel chitosan/siRNA nanoparticle system. Mol Ther 14, 476-484. DOI ScienceOn |
73 | Halder, J., Kamat, A.A., Landen, C.N., Jr., Han, L.Y., Lutgendorf, S.K., Lin, Y.G., Merritt, W.M., Jennings, N.B., Chavez-Reyes, A., Coleman, R.L., Gershenson, D.M., Schmandt, R., Cole, S.W., Lopez-Berestein, G., Sood, A.K., 2006. Focal adhesion kinase targeting using in vivo short interfering RNA delivery in neutral liposomes for ovarian carcinoma therapy. Clin Cancer Res 12, 4916-4924. DOI |
74 | Hannon, G.J., Rossi, J.J., 2004. Unlocking the potential of the human genome with RNA interference. Nature 431, 371-378. DOI |
75 | Hatakeyama, H., Ito, E., Akita, H., Oishi, M., Nagasaki, Y., Futaki, S., Harashima, H., 2009. A pH-sensitive fusogenic peptide facilitates endosomal escape and greatly enhances the gene silencing of siRNA-containing nanoparticles in vitro and in vivo. Journal of Controlled Release 139, 127-132. DOI |
76 | Caplen, N.J., Parrish, S., Imani, F., Fire, A., Morgan, R.A., 2001. Specific inhibition of gene expression by small doublestranded RNAs in invertebrate and vertebrate systems. Proc Natl Acad Sci U S A 98, 9742-9747. DOI |
77 | Chien, P.Y., Wang, J., Carbonaro, D., Lei, S., Miller, B., Sheikh, S., Ali, S.M., Ahmad, M.U., Ahmad, I., 2005. Novel cationic cardiolipin analogue-based liposome for efficient DNA and small interfering RNA delivery in vitro and in vivo. Cancer Gene Ther 12, 321-328. DOI |
78 | Choi, Y.-S., Lee, J.Y., Suh, J.S., Kwon, Y.-M., Lee, S.-J., Chung, J.-K., Lee, D.-S., Yang, V.C., Chung, C.-P., Park, Y.-J., 2010. The systemic delivery of siRNAs by a cell penetrating peptide, low molecular weight protamine. Biomaterials 31, 1429-1443. DOI |
79 | Crombez, L., Aldrian-Herrada, G., Konate, K., Nguyen, Q.N., McMaster, G.K., Brasseur, R., Heitz, F., Divita, G., 2008. A New Potent Secondary Amphipathic Cell-penetrating Peptide for siRNA Delivery Into Mammalian Cells. Mol Ther 17, 95-103. DOI |
80 | Crombez, L., Morris, M.C., Dufort, S., Aldrian-Herrada, G., Nguyen, Q., Mc Master, G., Coll, J.-L., Heitz, F., Divita, G., 2009. Targeting cyclin B1 through peptide-based delivery of siRNA prevents tumour growth. Nucl. Acids Res. 37, 4559-4569. DOI ScienceOn |
81 | Dalby, B., Cates, S., Harris, A., Ohki, E.C., Tilkins, M.L., Price, P.J., Ciccarone, V.C., 2004. Advanced transfection with Lipofectamine 2000 reagent: primary neurons, siRNA, and high-throughput applications. Methods 33, 95-103. DOI |
82 | Davis, M.E., 2009. The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic. Mol Pharm 6, 659-668. DOI |
83 | Davis, M.E., Zuckerman, J.E., Choi, C.H., Seligson, D., Tolcher, A., Alabi, C.A., Yen, Y., Heidel, J.D., Ribas, A., Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464, 1067-1070. DOI |
84 | Akinc, A., Zumbuehl, A., Goldberg, M., Leshchiner, E.S., Busini, V., Hossain, N., Bacallado, S.A., Nguyen, D.N., Fuller, J., Alvarez, R., Borodovsky, A., Borland, T., Constien, R., de Fougerolles, A., Dorkin, J.R., Narayanannair Jayaprakash, K., Jayaraman, M., John, M., Koteliansky, V., Manoharan, M., Nechev, L., Qin, J., Racie, T., Raitcheva, D., Rajeev, K.G., Sah, D.W., Soutschek, J., Toudjarska, I., Vornlocher, H.P., Zimmermann, T.S., Langer, R., Anderson, D.G., 2008. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 26, 561-569. DOI |
85 | Bartlett, D.W., Davis, M.E., 2008. Impact of tumor-specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA-containing nanoparticles. Biotechnol Bioeng 99, 975-985. DOI |
86 | Bartlett, D.W., Su, H., Hildebrandt, I.J., Weber, W.A., Davis, M.E., 2007. Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging. Proc Natl Acad Sci U S A 104, 15549-15554. DOI |
87 | Zhang, C., Tang, N., Liu, X., Liang, W., Xu, W., Torchilin, V.P., 2006. siRNA-containing liposomes modified with polyarginine effectively silence the targeted gene. Journal of Controlled Release 112, 229-239. DOI |
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