Strategies for targeted nonviral delivery of siRNAs in vivo

Trends in Molecular Medicine - Tập 15 - Trang 491-500 - 2009
Sang-Soo Kim1, Himanshu Garg1, Anjali Joshi1, N. Manjunath1
1Department of Biomedical Sciences, Center of Excellence for Infectious Diseases, Paul L. Foster School of Medicine, Texas Tech University Health Sciences Center, El Paso, TX 79905, USA

Tài liệu tham khảo

Kim, 2009, Overview of gene silencing by RNA interference, Curr. Protoc. Nucleic. Acid Chem., 11 Kim, 2008, RNAi mechanisms and applications, Biotechniques, 44, 613, 10.2144/000112792 de Fougerolles, 2007, Interfering with disease: a progress report on siRNA-based therapeutics, Nat. Rev. Drug Discov., 6, 443, 10.1038/nrd2310 Watanabe, 2008, Endogenous siRNAs from naturally formed dsRNAs regulate transcripts in mouse oocytes, Nature, 453, 539, 10.1038/nature06908 Babiarz, 2008, Mouse ES cells express endogenous shRNAs, siRNAs and other microprocessor-independent, Dicer-dependent small RNAs, Genes Dev., 22, 2773, 10.1101/gad.1705308 Castanotto, 2009, The promises and pitfalls of RNA-interference-based therapeutics, Nature, 457, 426, 10.1038/nature07758 Manjunath, 2006, Interfering antiviral immunity: application, subversion, hope?, Trends Immunol., 27, 328, 10.1016/j.it.2006.05.006 Check, 2005, A crucial test, Nat. Med., 11, 243, 10.1038/nm0305-243 DeVincenzo, 2008, Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV), Antiviral Res., 77, 225, 10.1016/j.antiviral.2007.11.009 Chappelow, 2008, Neovascular age-related macular degeneration: potential therapies, Drugs, 68, 1029, 10.2165/00003495-200868080-00002 Kleinman, 2008, Sequence- and target-independent angiogenesis suppression by siRNA via TLR3, Nature, 452, 591, 10.1038/nature06765 Kirchhoff, 2008, Silencing HIV-1 in vivo, Cell, 134, 566, 10.1016/j.cell.2008.08.004 Grimm, 2007, Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?, J. Clin. Invest., 117, 3633, 10.1172/JCI34129 Manjunath, N. et al. (2009) Lentiviral delivery of short hairpin RNAs. Adv Drug Deliv Rev. (in press) Akhtar, 2007, Nonviral delivery of synthetic siRNAs in vivo, J. Clin. Invest., 117, 3623, 10.1172/JCI33494 Aagaard, 2007, RNAi therapeutics: principles, prospects and challenges, Adv. Drug Deliv. Rev., 59, 75, 10.1016/j.addr.2007.03.005 Detzer, 2008, Phosphorothioate-stimulated cellular uptake of siRNA: a cell culture model for mechanistic studies, Curr. Pharm. Des., 14, 3666, 10.2174/138161208786898770 Juliano, 2008, Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides, Nucleic. Acids Res., 36, 4158, 10.1093/nar/gkn342 Dykxhoorn, 2006, The silent treatment: siRNAs as small molecule drugs, Gene Ther., 13, 541, 10.1038/sj.gt.3302703 Lewis, 2002, Efficient delivery of siRNA for inhibition of gene expression in postnatal mice, Nat. Genet, 32, 107, 10.1038/ng944 Rippe, 2002, Transendothelial transport: the vesicle controversy, J. Vasc. Res., 39, 375, 10.1159/000064521 Greish, 2007, Enhanced permeability and retention of macromolecular drugs in solid tumors: a royal gate for targeted anticancer nanomedicines, J. Drug Target., 15, 457, 10.1080/10611860701539584 Zamecnik, 2004, Extracellular matrix glycoproteins and diffusion barriers in human astrocytic tumors, Neuropathol. Appl. Neurobiol., 30, 338, 10.1046/j.0305-1846.2003.00541.x Oliveira, 2007, Fusogenic peptides enhance endosomal escape improving siRNA-induced silencing of oncogenes, Int. J. Pharm., 331, 211, 10.1016/j.ijpharm.2006.11.050 Song, 2003, RNA interference targeting Fas protects mice from fulminant hepatitis, Nat. Med., 9, 347, 10.1038/nm828 Fattal, 2008, State of the art and perspectives for the delivery of antisense oligonucleotides and siRNA by polymeric nanocarriers, Int. J. Pharm., 364, 237, 10.1016/j.ijpharm.2008.06.011 Hornung, 2005, Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7, Nat. Med., 11, 263, 10.1038/nm1191 Kim, 2007, Strategies for silencing human disease using RNA interference, Nat. Rev. Genet., 8, 173, 10.1038/nrg2006 Liu, 2007, Exploring cell type-specific internalizing antibodies for targeted delivery of siRNA, Brief Funct. Genomic Proteomic, 6, 112, 10.1093/bfgp/elm015 Chiu, 2004, Visualizing a correlation between siRNA localization, cellular uptake and RNAi in living cells, Chem. Biol., 11, 1165, 10.1016/j.chembiol.2004.06.006 Rossi, 2005, Receptor-targeted siRNAs, Nat. Biotechnol., 23, 682, 10.1038/nbt0605-682 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 Kumar, 2007, Transvascular delivery of small interfering RNA to the central nervous system, Nature, 448, 39, 10.1038/nature05901 Kumar, 2008, T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice, Cell, 134, 577, 10.1016/j.cell.2008.06.034 Zimmermann, 2006, RNAi-mediated gene silencing in non-human primates, Nature, 441, 111, 10.1038/nature04688 Kawakami, 2007, Targeted delivery systems of small interfering RNA by systemic administration, Drug Metab. Pharmacokinet., 22, 142, 10.2133/dmpk.22.142 Jeong, 2009, siRNA conjugate delivery systems, Bioconjug. Chem., 20, 5, 10.1021/bc800278e Soutschek, 2004, Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs, Nature, 432, 173, 10.1038/nature03121 Peer, 2008, Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target, Science, 319, 627, 10.1126/science.1149859 Tompkins, 2004, Protection against lethal influenza virus challenge by RNA interference in vivo, Proc. Natl. Acad. Sci. U S A, 101, 8682, 10.1073/pnas.0402630101 Palliser, 2006, An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection, Nature, 439, 89, 10.1038/nature04263 Kumar, 2006, A single siRNA suppresses fatal encephalitis induced by two different flaviviruses, PLoS Med., 3, e96, 10.1371/journal.pmed.0030096 Pardridge, 2007, shRNA and siRNA delivery to the brain, Adv. Drug Deliv. Rev., 59, 141, 10.1016/j.addr.2007.03.008 Zhang, 2004, Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer, Clin. Cancer Res., 10, 3667, 10.1158/1078-0432.CCR-03-0740 Pirollo, 2006, Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery, Hum. Gene. Ther., 17, 117, 10.1089/hum.2006.17.117 Hogrefe, 2006, Chemically modified short interfering hybrids (siHYBRIDS): nanoimmunoliposome delivery in vitro and in vivo for RNAi of HER-2, Nucleosides Nucleotides Nucleic. Acids, 25, 889, 10.1080/15257770600793885 Morrissey, 2005, Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs, Nat. Biotechnol., 23, 1002, 10.1038/nbt1122 Ma, 2005, Cationic lipids enhance siRNA-mediated interferon response in mice, Biochem. Biophys. Res. Commun., 330, 755, 10.1016/j.bbrc.2005.03.041 Akinc, 2008, A combinatorial library of lipid-like materials for delivery of RNAi therapeutics, Nat. Biotechnol., 26, 561, 10.1038/nbt1402 Shim, 2008, Controlled delivery of plasmid DNA and siRNA to intracellular targets using ketalized polyethylenimine, Biomacromolecules, 9, 444, 10.1021/bm7007313 Tan, 2005, Gene knockdown with intrathecal siRNA of NMDA receptor NR2B subunit reduces formalin-induced nociception in the rat, Gene. Ther., 12, 59, 10.1038/sj.gt.3302376 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 Chiu, 2004, Tumor-targeted gene delivery via anti-HER2 antibody (trastuzumab, Herceptin) conjugated polyethylenimine, J. Control Release, 97, 357, 10.1016/j.jconrel.2004.03.019 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 Guo, 2006, Construction of folate-conjugated pRNA of bacteriophage phi29 DNA packaging motor for delivery of chimeric siRNA to nasopharyngeal carcinoma cells, Gene. Ther., 13, 814, 10.1038/sj.gt.3302856 Pirollo, 2007, Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system, Cancer Res., 67, 2938, 10.1158/0008-5472.CAN-06-4535 Park, 2002, Anti-HER2 immunoliposomes: enhanced efficacy attributable to targeted delivery, Clin. Cancer Res., 8, 1172 Mamot, 2005, Epidermal growth factor receptor-targeted immunoliposomes significantly enhance the efficacy of multiple anticancer drugs in vivo, Cancer Res., 65, 11631, 10.1158/0008-5472.CAN-05-1093 Schiffelers, 2004, Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle, Nucleic. Acids Res., 32, e149, 10.1093/nar/gnh140 Alshamsan, 2009, Formulation and delivery of siRNA by oleic acid and stearic acid modified polyethylenimine, Mol. Pharm., 6, 121, 10.1021/mp8000815 Zintchenko, 2008, Simple modifications of branched PEI lead to highly efficient siRNA carriers with low toxicity, Bioconjug. Chem., 19, 1448, 10.1021/bc800065f 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 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 Zhang, 2005, Inhibition of respiratory syncytial virus infection with intranasal siRNA nanoparticles targeting the viral NS1 gene, Nat. Med., 11, 56, 10.1038/nm1174 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 Aouadi, 2009, Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation, Nature, 458, 1180, 10.1038/nature07774 Vangasseri, 2005, Lipid-protamine-DNA-mediated antigen delivery, Curr. Drug Deliv., 2, 401, 10.2174/156720105774370168 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 Schwarze, 1999, In vivo protein transduction: delivery of a biologically active protein into the mouse, Science, 285, 1569, 10.1126/science.285.5433.1569 Torchilin, 2001, TAT peptide on the surface of liposomes affords their efficient intracellular delivery even at low temperature and in the presence of metabolic inhibitors, Proc. Natl. Acad Sci. U S A, 98, 8786, 10.1073/pnas.151247498 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 McNamara, 2006, Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras, Nat. Biotechnol., 24, 1005, 10.1038/nbt1223 Chu, 2006, Aptamer mediated siRNA delivery, Nucleic. Acids Res., 34, e73, 10.1093/nar/gkl388 Zhou, 2008, Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy, Mol. Ther., 16, 1481, 10.1038/mt.2008.92 Zhou, 2009, Selection, characterization and application of new RNA HIV gp120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells, Nucleic. Acids Res., 37, 3094, 10.1093/nar/gkp185 Marshall, 2003, Gene therapy. Second child in French trial is found to have leukemia, Science, 299, 320, 10.1126/science.299.5605.320 Hacein-Bey-Abina, 2002, Gene therapy of X-linked severe combined immunodeficiency, Int. J. Hematol., 76, 295, 10.1007/BF02982686 Song, 2003, Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages, J. Virol., 77, 7174, 10.1128/JVI.77.13.7174-7181.2003 Omi, 2004, Long-lasting RNAi activity in mammalian neurons, FEBS Lett., 558, 89, 10.1016/S0014-5793(04)00017-1 Cardoso, 2008, Tf-lipoplexes for neuronal siRNA delivery: a promising system to mediate gene silencing in the CNS, J. Control Release, 132, 113, 10.1016/j.jconrel.2008.08.014