Overcoming obstacles to develop effective and safe siRNA therapeutics

Bibliography

• Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391:806-11
   PubMed Web of Science ®Google Scholar
• Zamore PD, Tuschl T, Sharp PA, Bartel DP. RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 2000;101:25-33
   PubMed Web of Science ®Google Scholar
• Elbashir SM, Lendeckel W, Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev 2001;15:188-200
   PubMed Web of Science ®Google Scholar
• Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001;409:363-6
   PubMed Web of Science ®Google Scholar
• Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001;411:494-8
   PubMed Web of Science ®Google Scholar
• Tomari Y, Zamore PD. Perspective: machines for RNAi. Genes Dev 2005;19:517-29
   PubMed Web of Science ®Google Scholar
• Meister G, Tuschl T. Mechanisms of gene silencing by double-stranded RNA. Nature 2004;431:343-9
   PubMed Web of Science ®Google Scholar
• Nykanen A, Haley B, Zamore PD. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell 2001;107:309-21
   PubMed Web of Science ®Google Scholar
• Wang Y, Juranek S, Li H, et al. Structure of an argonaute silencing complex with a seed-containing guide DNA and target RNA duplex. Nature 2008;456:921-6
   PubMed Web of Science ®Google Scholar
• Wang Y, Sheng G, Juranek S, et al. Structure of the guide-strand-containing argonaute silencing complex. Nature 2008;456:209-13
   PubMed Web of Science ®Google Scholar
• Kim DH, Behlke MA, Rose SD, et al. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat Biotechnol 2005;23:222-6
   PubMed Web of Science ®Google Scholar
• Rose SD, Kim DH, Amarzguioui M, et al. Functional polarity is introduced by Dicer processing of short substrate RNAs. Nucleic Acids Res 2005;33:4140-56
   PubMed Web of Science ®Google Scholar
• Sun X, Rogoff HA, Li CJ. Asymmetric RNA duplexes mediate RNA interference in mammalian cells. Nat Biotechnol 2008;26:1379-82
   PubMed Web of Science ®Google Scholar
• Bramsen JB, Laursen MB, Damgaard CK, et al. Improved silencing properties using small internally segmented interfering RNAs. Nucleic Acids Res 2007;35:5886-97
   PubMed Web of Science ®Google Scholar
• Lima WF, Murray H, Nichols JG, et al. Human Dicer binds short single-strand and double-strand RNA with high affinity and interacts with different regions of the nucleic acids. J Biol Chem 2009;284:2535-48
   PubMed Web of Science ®Google Scholar
• Reich SJ, Fosnot J, Kuroki A, et al. Small interfering RNA (siRNA) targeting VEGF effectively inhibits ocular neovascularization in a mouse model. Mol Vis 2003;9:210-6
   PubMed Web of Science ®Google Scholar
• Xia XB, Xiong SQ, Song WT, et al. Inhibition of retinal neovascularization by siRNA targeting VEGF(165). Mol Vis 2008;14:1965-73
   PubMed Web of Science ®Google Scholar
• Zhang W, Yang H, Kong X, et al. Inhibition of respiratory syncytial virus infection with intranasal siRNA nanoparticles targeting the viral NS1 gene. Nat Med 2005;11:56-62
   PubMed Web of Science ®Google Scholar
• Bitko V, Musiyenko A, Shulyayeva O, Barik S. Inhibition of respiratory viruses by nasally administered siRNA. Nat Med 2005;11:50-5
   PubMed Web of Science ®Google Scholar
• Soutschek J, Akinc A, Bramlage B, et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004;432:173-8
   PubMed Web of Science ®Google Scholar
• Wolfrum C, Shi S, Jayaprakash KN, et al. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat Biotechnol 2007;25:1149-57
   PubMed Web of Science ®Google Scholar
• Nishina K, Unno T, Uno Y, et al. Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol. Mol Ther 2008;16:734-40
   PubMed Web of Science ®Google Scholar
• Xia CF, Zhang Y, Zhan Y, et al. Intravenous siRNA of brain cancer with receptor targeting and avidin-biotin technology. Pharm Res 2007;24:2309-16
   PubMed Web of Science ®Google Scholar
• Hu-Lieskovan S, Heidel JD, Bartlett DW, et al. 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 2005;65:8984-92
   PubMed Web of Science ®Google Scholar
• Rozema DB, Lewis DL, Wakefield DH, et al. Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc Natl Acad Sci USA 2007;104:12982-7
   PubMed Web of Science ®Google Scholar
• Howard KA, Paludan SR, Behlke MA, et al. Chitosan/siRNA nanoparticle-mediated TNF-α knockdown in peritoneal macrophages for anti-inflammatory treatment in a murine arthritis model. Mol Ther 2009;17:162-8
   PubMed Web of Science ®Google Scholar
• Pille JY, Li H, Blot E, et al. 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 2006;17:1019-26
   PubMed Web of Science ®Google Scholar
• Song E, Zhu P, Lee SK, et al. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat Biotechnol 2005;23:709-17
   PubMed Web of Science ®Google Scholar
• Takeshita F, Minakuchi Y, Nagahara S, et al. Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc Natl Acad Sci USA 2005;102:12177-82
   PubMed Web of Science ®Google Scholar
• Lively TN, Kossen K, Balhorn A, et al. Effect of chemically modified IL-13 short interfering RNA on development of airway hyperresponsiveness in mice. J Allergy Clin Immunol 2008;121:88-94
   PubMed Web of Science ®Google Scholar
• Thomas M, Lu JJ, Ge Q, et al. Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. Proc Natl Acad Sci USA 2005;102:5679-84
   PubMed Web of Science ®Google Scholar
• Schiffelers RM, Ansari A, Xu J, et al. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res 2004;32:e149. Published online 1 November 2004
   PubMed Web of Science ®Google Scholar
• Available from: http://www.calandopharma.com/rnai.html [last accessed 26 March 2009]
   Google Scholar
• Kumar P, Wu H, Mcbride JL, et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 2007;448:39-43
   PubMed Web of Science ®Google Scholar
• Khoury M, Louis-Plence P, Escriou V, et al. Efficient new cationic liposome formulation for systemic delivery of small interfering RNA silencing tumor necrosis factor α in experimental arthritis. Arthritis Rheum 2006;54:1867-77
   PubMed Web of Science ®Google Scholar
• Sonoke S, Ueda T, Fujiwara K, et al. Tumor regression in mice by delivery of Bcl-2 small interfering RNA with pegylated cationic liposomes. Cancer Res 2008;68:8843-51
   PubMed Web of Science ®Google Scholar
• Miyawaki-Shimizu K, Predescu D, Shimizu J, et al. siRNA-induced caveolin-1 knockdown in mice increases lung vascular permeability via the junctional pathway. Am J Physiol Lung Cell Mol Physiol 2006;290:L405-13
   PubMed Web of Science ®Google Scholar
• Zimmermann TS, Lee AC, Akinc A, et al. RNAi-mediated gene silencing in non-human primates. Nature 2006;441:111-4
   PubMed Web of Science ®Google Scholar
• Morrissey DV, Lockridge JA, Shaw L, et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat Biotechnol 2005;23:1002-7
   PubMed Web of Science ®Google Scholar
• Landen CN Jr, Chavez-Reyes A, Bucana C, et al. Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery. Cancer Res 2005;65:6910-8
   PubMed Web of Science ®Google Scholar
• Sato Y, Murase K, Kato J, et al. Resolution of liver cirrhosis using vitamin A-coupled liposomes to deliver siRNA against a collagen-specific chaperone. Nat Biotechnol 2008;26:431-42
   PubMed Web of Science ®Google Scholar
• Chien PY, Wang J, Carbonaro D, et al. Novel cationic cardiolipin analogue-based liposome for efficient DNA and small interfering RNA delivery in vitro and in vivo. Cancer Gene Ther 2005;12:321-8
   PubMed Web of Science ®Google Scholar
• Pirollo KF, Rait A, Zhou Q, et al. Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system. Cancer Res 2007;67:2938-43
   PubMed Web of Science ®Google Scholar
• Santel A, Aleku M, Keil O, et al. RNA interference in the mouse vascular endothelium by systemic administration of siRNA-lipoplexes for cancer therapy. Gene Ther 2006;13:1360-70
   PubMed Web of Science ®Google Scholar
• Santel A, Aleku M, Keil O, et al. A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium. Gene Ther 2006;13:1222-34
   PubMed Web of Science ®Google Scholar
• Akinc A, Zumbuehl A, Goldberg M, et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat Biotechnol 2008;26:561-9
   PubMed Web of Science ®Google Scholar
• Heyes J, Palmer L, Bremner K, MacLachlan I. Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids. J Control Release 2005;107:276-87
   PubMed Web of Science ®Google Scholar
• MacLachlan I, Cullis P. Diffusible-PEG-lipid stabilized plasmid lipid particles. Adv Genet 2005;53:157-88
   PubMed Web of Science ®Google Scholar
• Aleku M, Schulz P, Keil O, et al. Atu027, a liposomal small interfering RNA formulation targeting protein kinase N3, inhibits cancer progression. Cancer Res 2008;68:9788-98
   PubMed Web of Science ®Google Scholar
• Frank-Kamenetsky M, Grefhorst A, Anderson NN, et al. Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Proc Natl Acad Sci USA 2008;105:11915-20
   PubMed Web of Science ®Google Scholar
• Li SD, Chono S, Huang L. Efficient oncogene silencing and metastasis inhibition via systemic delivery of siRNA. Mol Ther 2008;16:942-6
   PubMed Web of Science ®Google Scholar
• Li SD, Chono S, Huang L. Efficient gene silencing in metastatic tumor by siRNA formulated in surface-modified nanoparticles. J Control Release 2008;126:77-84
   PubMed Web of Science ®Google Scholar
• Peer D, Park EJ, Morishita Y, et al. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 2008;319:627-30
   PubMed Web of Science ®Google Scholar
• Peer D, Shimaoka M. Systemic siRNA delivery to leukocyte-implicated diseases. Cell Cycle 2009;8:853-9
   PubMed Web of Science ®Google Scholar
• Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006;441:537-41
   PubMed Web of Science ®Google Scholar
• John M, Constien R, Akinc A, et al. Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway. Nature 2007;449:745-7
   PubMed Web of Science ®Google Scholar
• Kleinman ME, Yamada K, Takeda A, et al. Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Nature 2008;452:591-7
   PubMed Web of Science ®Google Scholar
• Robbins M, Judge A, Ambegia E, et al. Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation. Hum Gene Ther 2008;19:991-9
   PubMed Web of Science ®Google Scholar
• Burchard J, Jackson AL, Malkov V, et al. MicroRNA-like off-target transcript regulation by siRNAs is species. specific RNA 2009;15:308-15
   PubMed Web of Science ®Google Scholar
• Abbott Laboratories, Yu Shen, Xiaoyu Lin, Stephen W Fesik, Leiming Li. Methods for assessing the delivery of small inhibitory RNA molecules. WO/2008/137814; 2008
   Google Scholar
• Kumar P, Ban HS, Kim SS, et al. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 2008;134:577-86
   PubMed Web of Science ®Google Scholar