Delivery of RNA interference therapeutics using polycation-based nanoparticles

Advanced Drug Delivery Reviews - Tập 61 - Trang 710-720 - 2009
Kenneth Alan Howard1
1Interdisciplinary Nanoscience Center (iNANO), Department of Molecular Biology, University of Aarhus, 8000 Aarhus C, Denmark

Tài liệu tham khảo

Behlke, 2006, Progress towards in vivo use of siRNAs, Mol. Ther., 13, 644, 10.1016/j.ymthe.2006.01.001 Xie, 2006, Harnessing in vivo siRNA delivery for drug discovery and therapeutic development, Drug Discov. Today, 11, 67, 10.1016/S1359-6446(05)03668-8 De Fougerolles, 2007, Interfering with disease: a progress report on siRNA-based therapeutics, Nat. Rev. Drug. Discov., 6, 443, 10.1038/nrd2310 Pouton, 2001, Key issues in non-viral gene delivery, Adv. Drug Deliv. Rev., 46, 187, 10.1016/S0169-409X(00)00133-2 Howard, 2007, Polycation-based nanoparticle delivery for improved RNA interference therapeutics, Expert Opin. Biol. Ther., 7, 1811, 10.1517/14712598.7.12.1811 Pille, 2006, Intravenous delivery of anti-RhoA small interfering RNA loaded in nanoparticles of chitosan in mice: safety and efficacy in xenografted aggressive breast cancer, Hum. Gene Ther., 17, 1019, 10.1089/hum.2006.17.1019 Elouahabi, 2005, Formation and intracellular trafficking of lipoplexes and polyplexes, Mol. Ther., 11, 336, 10.1016/j.ymthe.2004.12.006 Torchilin, 2000, Drug targeting, Eur. J. Pharm. Sci., 11, S81, 10.1016/S0928-0987(00)00166-4 Liu, 2007, The influence of polymeric properties on chitosan/siRNA nanoparticle formulation and gene silencing, Biomaterials, 28, 1280, 10.1016/j.biomaterials.2006.11.004 Koping-Hoggard, 2003, Relationship between the physical shape and the efficiency of oligomeric chitosan as a gene delivery system in vitro and in vivo, J. Gene Med., 5, 130, 10.1002/jgm.327 Koping-Hoggard, 2004, Improved chitosan-mediated gene delivery based on easily dissociated chitosan polyplexes of highly defined chitosan oligomers, Gene. Ther., 11, 1441, 10.1038/sj.gt.3302312 Shen, 2007, Importance of size-to-charge ratio in construction of stable and uniform nanoscale RNA/dendrimer complexes, Org. Biomol. Chem., 5, 3674, 10.1039/b711242d Grayson, 2006, Biophysical and structural characterization of polyethylenimine-mediated siRNA delivery in vitro, Pharm. Res., 23, 1868, 10.1007/s11095-006-9009-2 Thomas, 2005, Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung, Proc. Natl. Acad. Sci. U. S. A., 102, 5679, 10.1073/pnas.0502067102 Howard, 2009, Chitosan/siRNA nanoparticle-mediated TNF-alpha knockdown in peritoneal macrophages for anti-inflammatory treatment in a murine arthritis model, Mol. Ther., 17, 162, 10.1038/mt.2008.220 Dash, 2000, Decreased binding to proteins and cells of polymeric gene delivery vectors surface modified with a multivalent hydrophilic polymer and retargeting through attachment of transferrin, J. Biol. Chem., 275, 3793, 10.1074/jbc.275.6.3793 Owens, 2006, Opsonization, biodistribution, and pharmacokinetics of polymeric nanoparticles, Int. J. Pharm., 307, 93, 10.1016/j.ijpharm.2005.10.010 De Wolf, 2007, Effect of cationic carriers on the pharmacokinetics and tumor localization of nucleic acids after intravenous administration, Int. J. Pharm., 331, 167, 10.1016/j.ijpharm.2006.10.029 Takeshita, 2005, Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo, Proc. Natl. Acad. Sci. U. S. A., 102, 12177, 10.1073/pnas.0501753102 Hwang, 2008, Tumor targetability and antitumor effect of docetaxel-loaded hydrophobically modified glycol chitosan nanoparticles, J. Control. Release, 128, 23, 10.1016/j.jconrel.2008.02.003 Mao, 2001, Chitosan–DNA nanoparticles as gene carriers: synthesis, characterization and transfection efficiency, J. Control. Release, 70, 399, 10.1016/S0168-3659(00)00361-8 Schiffelers, 2004, Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle, Nucleic Acids Res., 32, e149, 10.1093/nar/gnh140 Kim, 2008, Local and systemic delivery of VEGF siRNA using polyelectrolyte complex micelles for effective treatment of cancer, J. Control. Release, 129, 107, 10.1016/j.jconrel.2008.03.008 Hu-Lieskovan, 2005, Sequence-specific knockdown of EWS-FLI1 by targeted, nonviral delivery of small interfering RNA inhibits tumor growth in a murine model of metastatic Ewing's sarcoma, Cancer Res., 65, 8984, 10.1158/0008-5472.CAN-05-0565 Heidel, 2007, Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA, Proc. Natl. Acad. Sci. U. S. A., 104, 5715, 10.1073/pnas.0701458104 Bartlett, 2008, Impact of tumor-specific targeting and dosing schedule on tumor growth inhibition after intravenous administration of siRNA-containing nanoparticles, Biotechnol. Bioeng., 99, 975, 10.1002/bit.21668 Bartlett, 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, 10.1073/pnas.0707461104 Kleinman, 2008, Sequence- and target-independent angiogenesis suppression by siRNA via TLR3, Nature, 452, 591, 10.1038/nature06765 Bartlett, 2007, Physicochemical and biological characterization of targeted, nucleic acid-containing nanoparticles, Bioconjug. Chem., 18, 456, 10.1021/bc0603539 Takeshita, 2006, Therapeutic potential of RNA interference against cancer, Cancer Sci., 97, 689, 10.1111/j.1349-7006.2006.00234.x Kawata, 2008, Administration of PLK-1 small interfering RNA with atelocollagen prevents the growth of liver metastases of lung cancer, Mol. Cancer Ther., 7, 2904, 10.1158/1535-7163.MCT-08-0473 Hanai, 2006, Atelocollagen-mediated systemic DDS for nucleic acid medicines, Ann. N. Y. Acad. Sci., 1082, 9, 10.1196/annals.1348.010 Ishimoto, 2008, Downregulation of monocyte chemoattractant protein-1 involving short interfering RNA attenuates hapten-induced contact hypersensitivity, Mol. Ther., 16, 387, 10.1038/sj.mt.6300360 Kinouchi, 2008, Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass, Gene Ther., 15, 1126, 10.1038/gt.2008.24 Song, 2005, Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors, Nat. Biotechnol., 23, 709, 10.1038/nbt1101 Peer, 2007, Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1, Proc. Natl. Acad. Sci. U. S. A., 104, 4095, 10.1073/pnas.0608491104 Peer, 2008, Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target, Science, 319, 627, 10.1126/science.1149859 Baigude, 2007, Design and creation of new nanomaterials for therapeutic RNAi, ACS Chem. Biol., 2, 237, 10.1021/cb7000582 Soutschek, 2004, Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs, Nature, 432, 173, 10.1038/nature03121 Zimmermann, 2006, RNAi-mediated gene silencing in non-human primates, Nature, 441, 111, 10.1038/nature04688 Mori, 2005, Mechanism of macrophage activation by chitin derivatives, J. Vet. Med. Sci., 67, 51, 10.1292/jvms.67.51 Goula, 2000, Rapid crossing of the pulmonary endothelial barrier by polyethylenimine/DNA complexes, Gene Ther., 7, 499, 10.1038/sj.gt.3301113 Ge, 2004, Inhibition of influenza virus production in virus-infected mice by RNA interference, Proc. Natl. Acad. Sci. U. S. A., 101, 8676, 10.1073/pnas.0402486101 Ochiya, 2001, Biomaterials for gene delivery: atelocollagen-mediated controlled release of molecular medicines, Curr. Gene Ther., 1, 31, 10.2174/1566523013348887 Minakuchi, 2004, Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo, Nucleic Acids Res., 32, e109, 10.1093/nar/gnh093 Yamato, 2008, New highly potent and specific E6 and E7 siRNAs for treatment of HPV16 positive cervical cancer, Cancer Gene Ther., 15, 140, 10.1038/sj.cgt.7701118 Iwaki, 2008, A small interfering RNA targeting proteinase-activated receptor-2 is effective in suppression of tumor growth in a Panc1 xenograft model, Int. J. Cancer., 122, 658, 10.1002/ijc.23123 Matoba, 2008, An siRNA against JC virus (JCV) agnoprotein inhibits JCV infection in JCV-producing cells inoculated in nude mice, Neuropathology, 28, 286, 10.1111/j.1440-1789.2007.00878.x Grzelinski, 2006, RNA interference-mediated gene silencing of pleiotrophin through polyethylenimine-complexed small interfering RNAs in vivo exerts antitumoral effects in glioblastoma xenografts, Hum. Gene Ther., 17, 751, 10.1089/hum.2006.17.751 De Martimprey, 2008, siRNA nanoformulation against the ret/PTC1 junction oncogene is efficient in an in vivo model of papillary thyroid carcinoma, Nucleic Acids Res., 36, e2, 10.1093/nar/gkm1094 Wang, 2008, Prevention of airway inflammation with topical cream containing imiquimod and small interfering RNA for natriuretic peptide receptor, Genet. Vaccines Ther., 6, 7, 10.1186/1479-0556-6-7 Urban-Klein, 2005, RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo, Gene. Ther., 12, 461, 10.1038/sj.gt.3302425 Howard, 2000, Influence of hydrophilicity of cationic polymers on the biophysical properties of polyelectrolyte complexes formed by self-assembly with DNA, Biochim. Biophys. Acta., 1475, 245, 10.1016/S0304-4165(00)00076-3 Ikehara, 2006, A carbohydrate recognition-based drug delivery and controlled release system using intraperitoneal macrophages as a cellular vehicle, Cancer Res., 66, 8740, 10.1158/0008-5472.CAN-06-0470 Howard, 2006, RNA interference in vitro and in vivo using a novel chitosan/siRNA nanoparticle system, Mol. Ther., 14, 476, 10.1016/j.ymthe.2006.04.010 Ogra, 1994 Tamura, 2004, Defense mechanisms against influenza virus infection in the respiratory tract mucosa, Jpn. J. Infect. Dis., 57, 236 Bitko, 2005, Inhibition of respiratory viruses by nasally administered siRNA, Nat. Med., 11, 50, 10.1038/nm1164 Zhang, 2004, Small interfering RNA targeting heme oxygenase-1 enhances ischemia–reperfusion-induced lung apoptosis, J. Biol. Chem., 279, 10677, 10.1074/jbc.M312941200 Massaro, 2004, Noninvasive delivery of small inhibitory RNA and other reagents to pulmonary alveoli in mice, Am. J. Physiol. Lung. Cell. Mol. Physiol., 287, L1066, 10.1152/ajplung.00067.2004 Soane, 1999, Evaluation of the clearance characteristics of bioadhesive systems in humans, Int. J. Pharm., 178, 55, 10.1016/S0378-5173(98)00367-6 Artursson, 1994, Effect of chitosan on the permeability of monolayers of intestinal epithelial cells (Caco-2), Pharm. Res., 11, 1358, 10.1023/A:1018967116988 Kong, 2007, Respiratory syncytial virus infection in Fischer 344 rats is attenuated by short interfering RNA against the RSV-NS1 gene, Genet. Vaccines Ther., 5, 4, 10.1186/1479-0556-5-4 Zhang, 2005, Inhibition of respiratory syncytial virus infection with intranasal siRNA nanoparticles targeting the viral NS1 gene, Nat. Med., 11, 56, 10.1038/nm1174 Andersen, 2008, Delivery of siRNA from lyophilized polymeric surfaces, Biomaterials, 29, 506, 10.1016/j.biomaterials.2007.10.003 Mislick, 1996, Evidence for the role of proteoglycans in cation-mediated gene transfer, Proc. Natl. Acad. Sci. U. S. A., 93, 12349, 10.1073/pnas.93.22.12349 Meade, 2008, Enhancing the cellular uptake of siRNA duplexes following noncovalent packaging with protein transduction domain peptides, Adv. Drug Deliv. Rev., 60, 530, 10.1016/j.addr.2007.10.004 Simeoni, 2003, Insight into the mechanism of the peptide-based gene delivery system MPG: implications for delivery of siRNA into mammalian cells, Nucleic Acids Res., 31, 2717, 10.1093/nar/gkg385 Crombez, 2007, A non-covalent peptide-based strategy for siRNA delivery, Biochem. Soc. Trans., 35, 44, 10.1042/BST0350044 Veldhoen, 2006, Cellular delivery of small interfering RNA by a non-covalently attached cell-penetrating peptide: quantitative analysis of uptake and biological effect, Nucleic Acids Res., 34, 6561, 10.1093/nar/gkl941 Kim, 2006, Cholesteryl oligoarginine delivering vascular endothelial growth factor siRNA effectively inhibits tumor growth in colon adenocarcinoma, Mol. Ther., 14, 343, 10.1016/j.ymthe.2006.03.022 Kim, 2006, 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, 10.1021/bc050289f Kim, 2008, LHRH receptor-mediated delivery of siRNA using polyelectrolyte complex micelles self-assembled from siRNA–PEG–LHRH conjugate and PEI, Bioconjug. Chem., 19, 2156, 10.1021/bc800249n Jiang, 2008, Hyaluronic acid-polyethyleneimine conjugate for target specific intracellular delivery of siRNA, Biopolymers, 89, 635, 10.1002/bip.20978 Oishi, 2005, Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile beta-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells, J. Am. Chem. Soc., 127, 1624, 10.1021/ja044941d Kim, 2005, Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy, Nat. Biotechnol., 23, 222, 10.1038/nbt1051 Lee, 2002, MicroRNA maturation: stepwise processing and subcellular localization, EMBO J., 21, 4663, 10.1093/emboj/cdf476 Morris, 2004, Small interfering RNA-induced transcriptional gene silencing in human cells, Science, 305, 1289, 10.1126/science.1101372 Boussif, 1995, A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine, Proc. Natl. Acad. Sci. U. S. A., 92, 7297, 10.1073/pnas.92.16.7297 Breunig, 2008, Mechanistic investigation of poly(ethylene imine)-based siRNA delivery: disulfide bonds boost intracellular release of the cargo, J. Control. Release, 130, 57, 10.1016/j.jconrel.2008.05.016 Huang, 2005, Transfection efficiency of chitosan vectors: effect of polymer molecular weight and degree of deacetylation, J. Control Release., 106, 391, 10.1016/j.jconrel.2005.05.004 Ishii, 2001, Mechanism of cell transfection with plasmid/chitosan complexes, Biochim. Biophys. Acta, 1514, 51, 10.1016/S0005-2736(01)00362-5 Zhou, 2006, PAMAM dendrimers for efficient siRNA delivery and potent gene silencing, Chem. Commun. (Camb)., 2362, 10.1039/b601381c Convertine, 2009, Development of a novel endosomolytic diblock copolymer for siRNA delivery, J. Control. Release, 133, 221, 10.1016/j.jconrel.2008.10.004 Kwon, 2008, Application of an HIV gp41-derived peptide for enhanced intracellular trafficking of synthetic gene and siRNA delivery vehicles, Bioconjug. Chem., 19, 920, 10.1021/bc700448h Meyer, 2008, Breathing life into polycations: functionalization with pH-responsive endosomolytic peptides and polyethylene glycol enables siRNA delivery, J. Am. Chem. Soc., 130, 3272, 10.1021/ja710344v Lee, 2008, Thermally sensitive cationic polymer nanocapsules for specific cytosolic delivery and efficient gene silencing of siRNA: swelling induced physical disruption of endosome by cold shock, J. Control. Release, 125, 25, 10.1016/j.jconrel.2007.09.011 He, 2004, MicroRNAs: small RNAs with a big role in gene regulation, Nat. Rev. Genet., 5, 522, 10.1038/nrg1379 Rahbek, 2008, Intracellular siRNA and precursor miRNA trafficking using bioresponsive copolypeptides, J. Gene. Med., 10, 81, 10.1002/jgm.1120 Boden, 2004, Enhanced gene silencing of HIV-1 specific siRNA using microRNA designed hairpins, Nucleic Acids Res., 32, 1154, 10.1093/nar/gkh278 Read, 2005, Barriers to gene delivery using synthetic vectors, Adv. Genet., 53, 19, 10.1016/S0065-2660(05)53002-5 Bellomo, 1992, Demonstration of nuclear compartmentalization of glutathione in hepatocytes, Proc. Natl. Acad. Sci. U. S. A., 89, 4412, 10.1073/pnas.89.10.4412 Hoon Jeong, 2007, Reducible poly(amido ethylenimine) directed to enhance RNA interference, Biomaterials, 28, 1912, 10.1016/j.biomaterials.2006.12.019 Shim, 2008, Controlled delivery of plasmid DNA and siRNA to intracellular targets using ketalized polyethylenimine, Biomacromolecules., 9, 444, 10.1021/bm7007313 Shim, 2009, Controlled cytoplasmic and nuclear localization of plasmid DNA and siRNA by differentially tailored polyethylenimine, J. Control. Release, 133, 206, 10.1016/j.jconrel.2008.10.007 Howard, 2007, Nanocarrier stimuli-activated gene delivery, Small, 3, 54, 10.1002/smll.200600328 Lv, 2006, Toxicity of cationic lipids and cationic polymers in gene delivery, J. Control. Release, 114, 100, 10.1016/j.jconrel.2006.04.014 Patil, 2008, Surface-modified and internally cationic polyamidoamine dendrimers for efficient siRNA delivery, Bioconjug. Chem., 19, 1396, 10.1021/bc8000722 Akhtar, 2007, Toxicogenomics of non-viral drug delivery systems for RNAi: potential impact on siRNA-mediated gene silencing activity and specificity, Adv. Drug. Deliv. Rev., 59, 164, 10.1016/j.addr.2007.03.010 Marques, 2006, A structural basis for discriminating between self and nonself double-stranded RNAs in mammalian cells, Nat. Biotechnol., 24, 559, 10.1038/nbt1205 Judge, 2006, Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo, Mol. Ther., 13, 494, 10.1016/j.ymthe.2005.11.002 Collingwood, 2008, Chemical modification patterns compatible with high potency Dicer-substrate small interfering RNAs, Oligonucleotides, 10.1089/oli.2008.0123 Bonnet, 2008, Systemic delivery of DNA or siRNA mediated by linear polyethylenimine (L-PEI) does not induce an inflammatory response, Pharm. Res., 25, 2972, 10.1007/s11095-008-9693-1