Therapeutic face of RNAi: in vivo challenges

Bibliography

• Napoli C, Lemieux C, Jorgensen R. Introduction of a chimeric chalcone synthase gene into petunia results in reversible co-suppression of homologous genes in trans. Plant Cell 1990;2:279-89
   PubMed Web of Science ®Google Scholar
• Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391(6669):806-11
   PubMed Web of Science ®Google Scholar
• Dorer DR, Henikoff S. Expansions of transgene repeats cause heterochromatin formation and gene silencing in Drosophila. Cell 1994;77:993-1002
   PubMedGoogle Scholar
• Grishok A. RNAi mechanisms in Caenorhabditis elegans. FEBS Lett 2005;579(26):5932-9
   PubMed Web of Science ®Google Scholar
• Ketting RF, Haverkamp TH, van Luenen HG, Plasterk RH. Mut-7 of C. elegans, required for transposon silencing and RNA interference, is a homolog of Werner syndrome helicase and RNaseD. Cell 1999;99(2):133-41
   PubMed Web of Science ®Google Scholar
• Lu R, Maduro M, Li F, et al. Animal virus replication and RNAi-mediated antiviral silencing in Caenorhabditis elegans. Nature 2005;436(7053):1040-3
   PubMed Web of Science ®Google Scholar
• Tabara H, Sarkissian M, Kelly WG, et al. The rde-1 gene, RNA interference, and transposon silencing in C. elegans. Cell 1999;99(2):123-32
   PubMed Web of Science ®Google Scholar
• Wassenegger M. The role of the RNAi machinery in heterochromatin formation. Cell 2005;122:13-16
   PubMedGoogle Scholar
• Kim VN. MicroRNA biogenesis: coordinated cropping and dicing. Nat Rev Mol Cell Biol 2005;6:376-85
   PubMed Web of Science ®Google Scholar
• Klenov MS, Lavrov SA, Stolyarenko AD, et al. Repeat-associated siRNAs cause chromatin silencing of retrotransposons in the Drosophila melanogaster germline. Nucleic Acids Res 2007;35(16):5430-8
   PubMed Web of Science ®Google Scholar
• Azimzadeh Jamalkandi S, Azadian E, Masoudi-Nejad A. Human RNAi pathway: crosstalk with organelles and cells. Funct Integr Genomics 2014;14:31-46
   PubMed Web of Science ®Google Scholar
• Lee RC, Hammell CM, Ambros V. Interacting endogenous and exogenous RNAi pathways in Caenorhabditis elegans. RNA 2006;12(4):589-97
   PubMed Web of Science ®Google Scholar
• Azimzadeh Jamalkandi S, Masoudi-Nejad A. RNAi pathway integration in Caenorhabditis elegans development. Funct Integr Genomics 2011;11(3):389-405
   PubMed Web of Science ®Google Scholar
• Azimzadeh Jamalkandi S, Masoudi-Nejad A. Reconstruction of Arabidopsis thaliana fully integrated small RNA pathway. Funct Integr Genomics 2009;9(4):419-32
   PubMed Web of Science ®Google Scholar
• Chen X, Gao C, Li H, et al. Identification and characterization of microRNAs in raw milk during different periods of lactation, commercial fluid, and powdered milk products. Cell Res 2010;20(10):1128-37
   PubMed Web of Science ®Google Scholar
• Ries J, Vairaktaris E, Agaimy A, et al. miR-186, miR-3651 and miR-494: potential biomarkers for oral squamous cell carcinoma extracted from whole blood. Oncol Rep 2014;31(3):1429-36
   PubMed Web of Science ®Google Scholar
• Mlcochova H, Hezova R, Stanik M, Slaby O. Urine microRNAs as potential noninvasive biomarkers in urologic cancers. Urol Oncol 2014;32:41. e1-9
   PubMed Web of Science ®Google Scholar
• Wang J, Zhang K-Y, Liu S-M, Sen S. Tumor-associated circulating MicroRNAs as biomarkers of cancer. Molecules 2014;19(2):1912-38
   PubMed Web of Science ®Google Scholar
• Kosik KS. The neuronal microRNA system. Nat Rev Neurosci 2006;7(12):911-20
   PubMed Web of Science ®Google Scholar
• Soo CY, Song Y, Zheng Y, et al. Nanoparticle tracking analysis monitors microvesicle and exosome secretion from immune cells. Immunology 2012;136(2):192-7
   PubMed Web of Science ®Google Scholar
• Dykxhoorn DM, Novina CD, Sharp PA. Killing the messenger: short RNAs that silence gene expression. Nat Rev 2003;4:457-67
   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
• Lavery KS, King TH. Antisense and RNAi: powerful tools in drug target discovery and validation. Curr Opin Drug Discov Devel 2003;4:561-9
   Google Scholar
• Lu PY, Xie FY, Woodle MC. siRNA-mediated antitumorigenesis for drug target validation and therapeutics. Curr Opin Mol Ther 2003;5:225-34
   PubMed Web of Science ®Google Scholar
• Cristofaro P, Ramratnam B. RNAi tackles a sexually transmitted disease. Nat Biotechnol 2006;24:48-9
   PubMed Web of Science ®Google Scholar
• Taylor DW, Ma E, Shigematsu H, et al. Substrate-specific structural rearrangements of human Dicer. Nat Struct Mol Biol 2013;20(6):662-70
   PubMed Web of Science ®Google Scholar
• Chiu Y, Rana TM. RNAi in human cells: basic structural and functional features of small interfering RNA. Mol Cell 2002;10:549-61
   PubMed Web of Science ®Google Scholar
• Wang X, Wang J, Huang V, et al. Induction of NANOG expression by targeting promoter sequence with small activating RNA antagonizes retinoic acid-induced differentiation. Biochem J 2012;443(3):821-8
   PubMedGoogle Scholar
• Gallo A, Tandon M, Alevizos I, Illei GG. The majority of microRNAs detectable in serum and saliva is concentrated in exosomes. PLoS One 2012;7(3):e30679
   PubMed Web of Science ®Google Scholar
• Lee HY, Zhou K, Smith AM, et al. Differential roles of human Dicer-binding proteins TRBP and PACT in small RNA processing. Nucleic Acids Res 2013;41(13):6568-76
   PubMed Web of Science ®Google Scholar
• Chiu YL, Rana TM. RNAi in human cells: basic structural and functional features of small interfering RNA. Mol Cell 2002;10(3):549-61
   PubMed Web of Science ®Google Scholar
• Franks TM, Lykke-Andersen J. The control of mRNA decapping and P-body formation. Mol Cell 2008;32(5):605-15
   PubMed Web of Science ®Google Scholar
• Gregory RI, Yan KP, Amuthan G, et al. The Microprocessor complex mediates the genesis of microRNAs. Nature 2004;432(7014):235-40
   PubMed Web of Science ®Google Scholar
• Sand M, Skrygan M, Georgas D, et al. Expression levels of the microRNA maturing microprocessor complex component DGCR8 and the RNA-induced silencing complex (RISC) components Argonaute-1, Argonaute-2, PACT, TARBP1, and TARBP2 in epithelial skin cancer. Mol Carcinog 2012;51(11):916-22
   PubMed Web of Science ®Google Scholar
• Calado A, Treichel N, Muller EC, et al. Exportin-5-mediated nuclear export of eukaryotic elongation factor 1A and tRNA. EMBO J 2002;21(22):6216-24
   PubMed Web of Science ®Google Scholar
• Curtis HJ, Sibley CR, Wood MJ. Mirtrons, an emerging class of atypical miRNA. Wiley Interdiscip Rev RNA 2012;3(5):617-32
   PubMed Web of Science ®Google Scholar
• Schopman NC, Heynen S, Haasnoot J, Berkhout B. A miRNA-tRNA mix-up: tRNA origin of proposed miRNA. RNA Biol 2010;7(5):573-6
   PubMed Web of Science ®Google Scholar
• Lin YT, Sullivan CS. Expanding the role of Drosha to the regulation of viral gene expression. Proc Natl Acad Sci USA 2011;108(27):11229-34
   PubMed Web of Science ®Google Scholar
• Stern-Ginossar N, Elefant N, Zimmermann A, et al. Host immune system gene targeting by a viral miRNA. Science 2007;317(5836):376-81
   PubMed Web of Science ®Google Scholar
• Sigurdsson MI, Smith AV, Bjornsson HT, Jonsson JJ. The distribution of a germline methylation marker suggests a regional mechanism of LINE-1 silencing by the piRNA-PIWI system. BMC Genet 2012;13:31
   PubMedGoogle Scholar
• Czauderna F, Fechtner M, Dames S, et al. Structural variations and stabilizing modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res 2003;31:2705-16
   PubMed Web of Science ®Google Scholar
• Broering R, Real CI, John MJ, et al. Chemical modifications on siRNAs avoid Toll-like-receptor-mediated activation of the hepatic immune system in vivo and in vitro. Int Immunol 2014;26(1):35-46
   PubMed Web of Science ®Google Scholar
• Deng Y, Wang CC, Choy KW, et al. Therapeutic potentials of gene silencing by RNA interference: principles, challenges, and new strategies. Gene 2014;538(2):217-27
   PubMed Web of Science ®Google Scholar
• Fellmann C, Lowe SW. Stable RNA interference rules for silencing. Nat Cell Biol 2014;16(1):10-18
   PubMed Web of Science ®Google Scholar
• Kirui DK, Mai J, Palange A-L, et al. Transient Mild Hyperthermia Induces E-selectin Mediated Localization of Mesoporous Silicon Vectors in Solid Tumors. PLoS One 2014;9(2):e86489
   PubMedGoogle Scholar
• Du Q, Thonberg H, Wang J, et al. A systematic analysis of the silencing effects of an active siRNA at all singlenucleotide mismatched target sites. Nucleic Acids Res 2005;33:1671-7
   PubMed Web of Science ®Google Scholar
• Snove OJr, Holen T. Many commonly used siRNAs risk offtarget activity. Biochem Biophys Res Commun 2004;319:256-63
   PubMed Web of Science ®Google Scholar
• Reynolds A, Leake D, Boese Q, et al. Rational siRNA design for RNA interference. Nat Biotechnol 2004;22:326-30
   PubMed Web of Science ®Google Scholar
• Jackson AL, Bartz SR, Schelter J, et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 2003;21:635-7
   PubMed Web of Science ®Google Scholar
• Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 2001;12(8):861-70
   PubMed Web of Science ®Google Scholar
• Naito Y, Yamada T, Ui-Tei K, et al. siDirect: highly effective, target-specific siRNA design software for mammalian RNA interference. Nucleic Acids Res 2004;32:W124-9
   PubMed Web of Science ®Google Scholar
• Zufferey R, Dull T, Mandel RJ, et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 1998;72(12):9873-80
   PubMed Web of Science ®Google Scholar
• Bantounas I. RNA interference and the use of small interfering RNA to study gene function in mammalian systems. J Mol Endocrinol 2004;33:545-57
   PubMed Web of Science ®Google Scholar
• Xie FY. Delivering siRNA to animal disease models for validation of novel drug targets in vivo. PharmaGenomics 2004;28-38
   Google Scholar
• Kim B. Inhibition of ocular angiogenesis by siRNA targeting vascular endothelial growth factor pathway genes: therapeutic strategy for herpetic stromal keratitis. Am J Pathol 2004;165:2177-85
   PubMed Web of Science ®Google Scholar
• Song E. 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
• Kennedy S, Wang D, Ruvkun G. A conserved siRNA-degrading RNase negatively regulates RNA interference in C. elegans. Nature 2004;427(6975):645-9
   PubMed Web of Science ®Google Scholar
• Chiu YL, Rana TM. siRNA function in RNAi: a chemical modification analysis. RNA 2003;9(9):1034-48
   PubMed Web of Science ®Google Scholar
• Braasch DA, Jensen S, Liu Y, et al. RNA interference in mammalian cells by chemically-modified RNA. Biochemistry 2003;42(26):7967-75
   PubMed Web of Science ®Google Scholar
• Amarzguioui M, Holen T, Babaie E, Prydz H. Tolerance for mutations and chemical modifications in a siRNA. Nucleic Acids Res 2003;31(2):589-95
   PubMed Web of Science ®Google Scholar
• Harborth J, Elbashir SM, Vandenburgh K, et al. Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev 2003;13(2):83-105
   PubMedGoogle Scholar
• Choung S, Kim YJ, Kim S, et al. Chemical modification of siRNAs to improve serum stability without loss of efficacy. Biochem Biophys Res Commun 2006;342(3):919-27
   PubMed Web of Science ®Google Scholar
• Hall AH, Wan J, Shaughnessy EE, et al. RNA interference using boranophosphate siRNAs: structure-activity relationships. Nucleic Acids Res 2004;32(20):5991-6000
   PubMed Web of Science ®Google Scholar
• Sheehan D, Lunstad B, Yamada CM, et al. Biochemical properties of phosphonoacetate and thiophosphonoacetate oligodeoxyribonucleotides. Nucleic Acids Res 2003;31(14):4109-18
   PubMed Web of Science ®Google Scholar
• Yamada CM, Dellinger DJ, Caruthers MH. Synthesis and biological activity of phosphonocarboxylate DNA. Nucleosides Nucleotides Nucleic Acids 2007;26(6-7):539-46
   PubMed Web of Science ®Google Scholar
• Perreault DM, Anslyn EV. Unifying the current data on the mechanism of cleavage–transesterification of RNA. Angew Chem Int Ed Engl 1997;36(5):432-50
   Web of Science ®Google Scholar
• Jackson AL, Burchard J, Leake D, et al. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA 2006;12(7):1197-205
   PubMed Web of Science ®Google Scholar
• Odadzic D, Bramsen JB, Smicius R, et al. Synthesis of 2’-O-modified adenosine building blocks and application for RNA interference. Bioorg Med Chem 2008;16(1):518-29
   PubMed Web of Science ®Google Scholar
• Bramsen JB, Laursen MB, Nielsen AF, et al. A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity. Nucleic Acids Res 2009;37(9):2867-81
   PubMed Web of Science ®Google Scholar
• Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference-based therapeutics. Nature 2009;7228):426-33
   PubMed Web of Science ®Google Scholar
• Judge AD, Bola G, Lee AC, MacLachlan I. Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Mol Ther 2006;13(3):494-505
   PubMed Web of Science ®Google Scholar
• Wengel J, Petersen M, Nielsen KE, et al. LNA (locked nucleic acid) and the diastereoisomeric alpha-L-LNA: conformational tuning and high-affinity recognition of DNA/RNA targets. Nucleosides Nucleotides Nucleic Acids 2001;20(4-7):389-96
   PubMed Web of Science ®Google Scholar
• Trang P, Medina PP, Wiggins JF, et al. Regression of murine lung tumors by the let-7 microRNA. Oncogene 2010;29(11):1580-7
   PubMed Web of Science ®Google Scholar
• Glud SZ, Bramsen JB, Dagnaes-Hansen F, et al. Naked siLNA-mediated gene silencing of lung bronchoepithelium EGFP expression after intravenous administration. Oligonucleotides 2009;19(2):163-8
   PubMed Web of Science ®Google Scholar
• Mook OR, Baas F, de Wissel MB, Fluiter K. Evaluation of locked nucleic acid-modified small interfering RNA in vitro and in vivo. Mol Cancer Ther 2007;6(3):833-43
   PubMed Web of Science ®Google Scholar
• Elmen J, Thonberg H, Ljungberg K, et al. Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality. Nucleic Acids Res 2005;33(1):439-47
   PubMed Web of Science ®Google Scholar
• Laursen MB, Pakula MM, Gao S, et al. Utilization of unlocked nucleic acid (UNA) to enhance siRNA performance in vitro and in vivo. Mol Biosyst 2010;6(5):862-70
   PubMed Web of Science ®Google Scholar
• Vaish N, Chen F, Seth S, et al. Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs. Nucleic Acids Res 2011;39(5):1823-32
   PubMed Web of Science ®Google Scholar
• Nauwelaerts K, Fisher M, Froeyen M, et al. Structural characterization and biological evaluation of small interfering RNAs containing cyclohexenyl nucleosides. J Am Chem Soc 2007;129(30):9340-8
   PubMed Web of Science ®Google Scholar
• Hoshika S, Minakawa N, Shionoya A, et al. Study of modification pattern-RNAi activity relationships by using siRNAs modified with 4’-thioribonucleosides. Chembiochem 2007;8(17):2133-8
   PubMed Web of Science ®Google Scholar
• Fisher M, Abramov M, Van Aerschot A, et al. Inhibition of MDR1 expression with altritol-modified siRNAs. Nucleic Acids Res 2007;35(4):1064-74
   PubMed Web of Science ®Google Scholar
• Peacock H, Kannan A, Beal PA, Burrows CJ. Chemical modification of siRNA bases to probe and enhance RNA interference. J Org Chem 2011;76(18):7295-300
   PubMed Web of Science ®Google Scholar
• Dowler T, Bergeron D, Tedeschi AL, et al. Improvements in siRNA properties mediated by 2’-deoxy-2’-fluoro-beta-D-arabinonucleic acid (FANA). Nucleic Acids Res 2006;34(6):1669-75
   PubMed Web of Science ®Google Scholar
• Whitehead KA, Langer R, Anderson DG. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 2009;8(2):129-38
   PubMed Web of Science ®Google Scholar
• Lavan DA, McGuire T, Langer R. Small-scale systems for in vivo drug delivery. Nat Biotechnol 2003;21(10):1184-91
   PubMed Web of Science ®Google Scholar
• Gary DJ, Puri N, Won Y-Y. Polymer-based siRNA delivery: perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery. J Control Release 2007;121(1):64-73
   PubMed Web of Science ®Google Scholar
• Lingor P, Michel U, Scholl U, et al. Transfection of "naked" siRNA results in endosomal uptake and metabolic impairment in cultured neurons. Biochem Biophys Res Commun 2004;315(4):1126-33
   PubMed Web of Science ®Google Scholar
• McManus MT, Sharp PA. Gene silencing in mammals by small interfering RNAs. Nat Rev Genet 2002;3(10):737-47
   PubMed Web of Science ®Google Scholar
• Pardridge WM. shRNA and siRNA delivery to the brain. Adv Drug Deliv Rev 2007;59(2-3):141-52
   PubMed Web of Science ®Google Scholar
• Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002;296(5567):550-3
   PubMed Web of Science ®Google Scholar
• Patil SD, Rhodes DG, Burgess DJ. DNA-based therapeutics and DNA delivery systems: a comprehensive review. AAPS J 2005;7(1):E61-77
   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(7089):111-14
   PubMed Web of Science ®Google Scholar
• Kim SH, Jeong JH, Lee SH, et al. Local and systemic delivery of VEGF siRNA using polyelectrolyte complex micelles for effective treatment of cancer. J Control Release 2008;129(2):107-16
   PubMed Web of Science ®Google Scholar
• Takakura Y, Nishikawa M, Yamashita F, Hashida M. Influence of physicochemical properties on pharmacokinetics of non-viral vectors for gene delivery. J Drug Target 2002;10(2):99-104
   PubMed Web of Science ®Google Scholar
• Shen Y. Advances in the development of siRNA-based therapeutics for cancer. IDrugs 2008;11(8):572-8
   PubMed Web of Science ®Google Scholar
• Shim MS, Kwon YJ. Efficient and targeted delivery of siRNA in vivo. FEBS J 2010;277(23):4814-27
   PubMed Web of Science ®Google Scholar
• Dykxhoorn DM, Novina CD, Sharp PA. Killing the messenger: short RNAs that silence gene expression. Nat Rev Mol Cell Biol 2003;4(6):457-67
   PubMed Web of Science ®Google Scholar
• Zhang C, Wang YS, Wu H, et al. Inhibitory efficacy of hypoxia-inducible factor 1alpha short hairpin RNA plasmid DNA-loaded poly (D, L-lactide-co-glycolide) nanoparticles on choroidal neovascularization in a laser-induced rat model. Gene Ther 2010;17(3):338-51
   PubMed Web of Science ®Google Scholar
• Cotrim AP, Baum BJ. Gene therapy: some history, applications, problems, and prospects. Toxicol Pathol 2008;36(1):97-103
   PubMed Web of Science ®Google Scholar
• Li YJ, Zhang YX, Wang PY, et al. Regression of A549 lung cancer tumors by anti-miR-150 vector. Oncol Rep 2012;27(1):129-34
   PubMed Web of Science ®Google Scholar
• Torchilin VP. Multifunctional nanocarriers. Adv Drug Deliv Rev 2006;58(14):1532-55
   PubMed Web of Science ®Google Scholar
• Lee M, Kim SW. Polyethylene glycol-conjugated copolymers for plasmid DNA delivery. Pharm Res 2005;22(1):1-10
   PubMed Web of Science ®Google Scholar
• Park TG, Jeong JH, Kim SW. Current status of polymeric gene delivery systems. Adv Drug Deliv Rev 2006;58(4):467-86
   PubMed Web of Science ®Google Scholar
• Takakura Y, Hashida M. Macromolecular drug carrier systems in cancer chemotherapy: macromolecular prodrugs. Crit Rev Oncol Hematol 1995;18(3):207-31
   PubMed Web of Science ®Google Scholar
• Ganta S, Devalapally H, Shahiwala A, Amiji M. A review of stimuli-responsive nanocarriers for drug and gene delivery. J Control Release 2008;126(3):187-204
   PubMed Web of Science ®Google Scholar
• Wadia JS, Dowdy SF. Transmembrane delivery of protein and peptide drugs by TAT-mediated transduction in the treatment of cancer. Adv Drug Deliv Rev 2005;57(4):579-96
   PubMed Web of Science ®Google Scholar
• Frankel AD, Pabo CO. Cellular uptake of the tat protein from human immunodeficiency virus. Cell 1988;55(6):1189-93
   PubMed Web of Science ®Google Scholar
• Elliott G, O’Hare P. Intercellular trafficking and protein delivery by a herpesvirus structural protein. Cell 1997;88(2):223-33
   PubMed Web of Science ®Google Scholar
• Morris M, Vidal P, Chaloin L, et al. A new peptide vector for efficient delivery of oligonucleotides into mammalian cells. Nucleic Acids Res 1997;25(14):2730-6
   PubMed Web of Science ®Google Scholar
• Oehlke J, Scheller A, Wiesner B, et al. Cellular uptake of an alpha-helical amphipathic model peptide with the potential to deliver polar compounds into the cell interior non-endocytically. Biochim Biophys Acta 1998;1414(1):127-39
   PubMed Web of Science ®Google Scholar
• Futaki S, Suzuki T, Ohashi W, et al. Arginine-rich peptides An abundant source of membrane-permeable peptides having potential as carriers for intracellular protein delivery. J Biol Chem 2001;276(8):5836-40
   PubMed Web of Science ®Google Scholar
• El-Aneed A. An overview of current delivery systems in cancer gene therapy. J Control Release 2004;94(1):1-14
   PubMed Web of Science ®Google Scholar
• Wang J, Lu Z, Wientjes MG, Au JL-S. Delivery of siRNA therapeutics: barriers and carriers. AAPS J 2010;12(4):492-503
   PubMed Web of Science ®Google Scholar
• Davidson TJ, Harel S, Arboleda VA, et al. Highly efficient small interfering RNA delivery to primary mammalian neurons induces MicroRNA-like effects before mRNA degradation. J Neurosci 2004;24(45):10040-6
   PubMed Web of Science ®Google Scholar
• Kumar P, Wu H, McBride JL, et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 2007;448(7149):39-43
   PubMed Web of Science ®Google Scholar
• Panyam J, Zhou W-Z, Prabha S, et al. Rapid endo-lysosomal escape of poly (DL-lactide-co-glycolide) nanoparticles: implications for drug and gene delivery. FASEB J 2002;16(10):1217-26
   PubMed Web of Science ®Google Scholar
• Samal SK, Dash M, Van Vlierberghe S, et al. Cationic polymers and their therapeutic potential. Chem Soc Rev 2012;41(21):7147-94
   PubMed Web of Science ®Google Scholar
• Dokka S, Rojanasakul Y. Novel non-endocytic delivery of antisense oligonucleotides. Adv Drug Deliv Rev 2000;44(1):35-49
   PubMed Web of Science ®Google Scholar
• Oishi M, Nagasaki Y, Itaka K, et al. 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 2005;127(6):1624-5
   PubMed Web of Science ®Google Scholar
• Liu Y, Tao J, Li Y, et al. Targeting hypoxia-inducible factor-1alpha With Tf–PEI–shRNA complex via transferrin receptor–mediated endocytosis inhibits melanoma growth. Mol Ther 2009;17(2):269-77
   PubMed Web of Science ®Google Scholar
• Schmaljohann D. Thermo-and pH-responsive polymers in drug delivery. Adv Drug Deliv Rev 2006;58(15):1655-70
   PubMed Web of Science ®Google Scholar
• Han G, You CC, Kim BJ, et al. Light-regulated release of DNA and its delivery to nuclei by means of photolabile gold nanoparticles. Angew Chem 2006;118(19):3237-41
   Google Scholar
• Wolff JA, Rozema DB. Breaking the bonds: non-viral vectors become chemically dynamic. Mol Ther 2008;16(1):8-15
   PubMed Web of Science ®Google Scholar
• Tsai CJ, Zheng J, Nussinov R. Designing a nanotube using naturally occurring protein building blocks. PLoS Comput Biol 2006;2(4):e42
   PubMedGoogle Scholar
• Lasic DD. Novel applications of liposomes. Trends Biotechnol 1998;16(7):307-21
   PubMed Web of Science ®Google Scholar
• Lavigne C, Slater K, Gajanayaka N, et al. Influence of lipoplex surface charge on siRNA delivery: application to the in vitro downregulation of CXCR4 HIV-1 co-receptor. Expert Opin Biol Ther 2013;13(7):973-85
   PubMed Web of Science ®Google Scholar
• Hashida M, Kawakami S, Yamashita F. Lipid carrier systems for targeted drug and gene delivery. Chem Pharm Bull(Tokyo) 2005;53(8):871-80
   PubMed Web of Science ®Google Scholar
• Sakurai F, Nishioka T, Saito H, et al. Interaction between DNA-cationic liposome complexes and erythrocytes is an important factor in systemic gene transfer via the intravenous route in mice: the role of the neutral helper lipid. Gene Ther 2001;8(9):677-86
   PubMed Web of Science ®Google Scholar
• Mahato RI, Kawabata K, Nomura T, et al. Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes. J Pharm Sci 1995;84(11):1267-71
   PubMed Web of Science ®Google Scholar
• Zhu N, Liggitt D, Liu Y, Debs R. Systemic gene expression after intravenous DNA delivery into adult mice. Science 1993;261(5118):209-11
   PubMed Web of Science ®Google Scholar
• Uyechi LS, Gagne L, Thurston G, Szoka FCJr. Mechanism of lipoplex gene delivery in mouse lung: binding and internalization of fluorescent lipid and DNA components. Gene Ther 2001;8(11):828-36
   PubMed Web of Science ®Google Scholar
• Seow Y, Wood MJ. Biological gene delivery vehicles: beyond viral vectors. Mol Ther 2009;17(5):767-77
   PubMed Web of Science ®Google Scholar
• Kawakami S, Sato A, Nishikawa M, et al. Mannose receptor-mediated gene transfer into macrophages using novel mannosylated cationic liposomes. Gene Ther 2000;7(4):292-9
   PubMed Web of Science ®Google Scholar
• Yamada M, Nishikawa M, Kawakami S, et al. Tissue and intrahepatic distribution and subcellular localization of a mannosylated lipoplex after intravenous administration in mice. J Control Release 2004;98(1):157-67
   PubMed Web of Science ®Google Scholar
• Kawakami S, Fumoto S, Nishikawa M, et al. In vivo gene delivery to the liver using novel galactosylated cationic liposomes. Pharm Res 2000;17(3):306-13
   PubMed Web of Science ®Google Scholar
• Cardoso A, Simoes S, De Almeida L, et al. siRNA delivery by a transferrin-associated lipid-based vector: a non-viral strategy to mediate gene silencing. J Gene Med 2007;9(3):170-83
   PubMed Web of Science ®Google Scholar
• Lee CC, MacKay JA, Fréchet JM, Szoka FC. Designing dendrimers for biological applications. Nat Biotechnol 2005;23(12):1517-26
   PubMed Web of Science ®Google Scholar
• Liu M, Fréchet JM. Designing dendrimers for drug delivery. Pharm Sci Technol Today 1999;2(10):393-401
   PubMedGoogle Scholar
• Kobayashi H, Brechbiel MW. Dendrimer-based nanosized MRI contrast agents. Curr Pharm Biotechnol 2004;5(6):539-49
   PubMed Web of Science ®Google Scholar
• Kang H, DeLong R, Fisher MH, Juliano RL. Tat-conjugated PAMAM dendrimers as delivery agents for antisense and siRNA oligonucleotides. Pharm Res 2005;22(12):2099-106
   PubMed Web of Science ®Google Scholar
• Li S, Huang L. Gene therapy progress and prospects: non-viral gene therapy by systemic delivery. Gene Ther 2006;13(18):1313-19
   PubMed Web of Science ®Google Scholar
• Braun CS, Vetro JA, Tomalia DA, et al. Structure/function relationships of polyamidoamine/DNA dendrimers as gene delivery vehicles. J Pharm Sci 2005;94(2):423-36
   PubMed Web of Science ®Google Scholar
• Tomalia D, Reyna L, Svenson S. Dendrimers as multi-purpose nanodevices for oncology drug delivery and diagnostic imaging. Biochem Soc Trans 2007;35(1):61
   PubMed Web of Science ®Google Scholar
• Fox ME, Guillaudeu S, Fréchet JM, et al. Synthesis and in vivo antitumor efficacy of PEGylated poly(l-lysine) dendrimer−camptothecin conjugates. Mol Pharm 2009;6(5):1562-72
   PubMedGoogle Scholar
• Kaminskas LM, Kelly BD, McLeod VM, et al. Capping methotrexate alpha-carboxyl groups enhances systemic exposure and retains the cytotoxicity of drug conjugated PEGylated polylysine dendrimers. Mol Pharm 2011;8(2):338-49
   PubMedGoogle Scholar
• Kaminskas LM, McLeod VM, Kelly BD, et al. A comparison of changes to doxorubicin pharmacokinetics, antitumor activity, and toxicity mediated by PEGylated dendrimer and PEGylated liposome drug delivery systems. Nanomedicine 2012;8(1):103-11
   PubMed Web of Science ®Google Scholar
• Pack DW, Hoffman AS, Pun S, Stayton PS. Design and development of polymers for gene delivery. Nat Rev Drug Discov 2005;4(7):581-93
   PubMed Web of Science ®Google Scholar
• Gao S, Dagnaes-Hansen F, Nielsen EJ, et al. The effect of chemical modification and nanoparticle formulation on stability and biodistribution of siRNA in mice. Mol Ther 2009;17(7):1225-33
   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(19):e149
   PubMed Web of Science ®Google Scholar
• Feng Y, Hu J, Ma J, et al. RNAi-mediated silencing of VEGF-C inhibits non-small cell lung cancer progression by simultaneously down-regulating the CXCR4, CCR7, VEGFR-2 and VEGFR-3-dependent axes-induced ERK, p38 and AKT signalling pathways. Eur J Cancer 2011;47(15):2353-63
   PubMed Web of Science ®Google Scholar
• Lee SH, Kim SH, Park TG. Intracellular siRNA delivery system using polyelectrolyte complex micelles prepared from VEGF siRNA-PEG conjugate and cationic fusogenic peptide. Biochem Biophys Res Commun 2007;357(2):511-16
   PubMed Web of Science ®Google Scholar
• Merdan T, Callahan J, Petersen H, et al. Pegylated polyethylenimine-Fab’antibody fragment conjugates for targeted gene delivery to human ovarian carcinoma cells. Bioconjug Chem 2003;14(5):989-96
   PubMed Web of Science ®Google Scholar
• Patil ML, Zhang M, Minko T. Multifunctional triblock nanocarrier (PAMAM-PEG-PLL) for the efficient intracellular siRNA delivery and gene silencing. ACS Nano 2011;5(3):1877-87
   PubMed Web of Science ®Google Scholar
• Jeong JH, Mok H, Oh Y-K, Park TG. siRNA conjugate delivery systems. Bioconjug Chem 2008;20(1):5-14
   Web of Science ®Google Scholar
• Lee H, Lytton-Jean AK, Chen Y, et al. Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery. Nat Nanotechnol 2012;7(6):389-93
   PubMed Web of Science ®Google Scholar
• Lai SK, Wang YY, Hanes J. Mucus-penetrating nanoparticles for drug and gene delivery to mucosal tissues. Adv Drug Deliv Rev 2009;61(2):158-71
   PubMed Web of Science ®Google Scholar
• Suk JS, Lai SK, Boylan NJ, et al. Rapid transport of muco-inert nanoparticles in cystic fibrosis sputum treated with N-acetyl cysteine. Nanomedicine 2011;6(2):365-75
   PubMed Web of Science ®Google Scholar
• Ming X. Cellular delivery of siRNA and antisense oligonucleotides via receptor-mediated endocytosis. Expert Opin Drug Deliv 2011;8(4):435-49
   PubMed Web of Science ®Google Scholar
• McNamara JOII, Andrechek ER, Wang Y, et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat Biotechnol 2006;24(8):1005-15
   PubMed Web of Science ®Google Scholar
• Kelly C, Jefferies C, Cryan S-A. Targeted liposomal drug delivery to monocytes and macrophages. J Drug Deliv 2011;2011:727241
   PubMedGoogle Scholar
• Wang Z, Chui W-K, Ho PC. Integrin targeted drug and gene delivery. Expert Opin Drug Deliv 2010;7(2):159-71
   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(7014):173-8
   PubMed Web of Science ®Google Scholar
• Chiu S-J, Ueno NT, Lee RJ. Tumor-targeted gene delivery via anti-HER2 antibody (trastuzumab, Herceptin®) conjugated polyethylenimine. J Control Release 2004;97(2):357-69
   PubMed Web of Science ®Google Scholar
• Trubetskoy VS, Torchilin VP, Kennel SJ, Huang L. Use of N-terminal modified poly (L-lysine)-antibody conjugate as a carrier for targeted gene delivery in mouse lung endothelial cells. Bioconjug Chem 1992;3(4):323-7
   PubMed Web of Science ®Google Scholar
• Meade BR, Dowdy SF. Exogenous siRNA delivery using peptide transduction domains/cell penetrating peptides. Adv Drug Deliv Rev 2007;59(2):134-40
   PubMed Web of Science ®Google Scholar
• Zhuo RX, Du B, Lu ZR. In vitro release of 5-fluorouracil with cyclic core dendritic polymer. J Control Release 1999;57(3):249-57
   PubMed Web of Science ®Google Scholar
• Qiu LY, Bae YH. Polymer architecture and drug delivery. Pharm Res 2006;23(1):1-30
   PubMed Web of Science ®Google Scholar
• Rider TH, Zook CE, Boettcher TL, et al. Broad-spectrum antiviral therapeutics. PLoS One 2011;6(7):e22572
   PubMedGoogle Scholar
• Aigner A. Transkingdom RNA interference (tkRNAi) as a new delivery tool for therapeutic RNA. Expert Opin Biol Ther 2009;9(12):1533-42
   PubMed Web of Science ®Google Scholar
• Zender L, Hutker S, Liedtke C, et al. Caspase 8 small interfering RNA prevents acute liver failure in mice. Proc Natl Acad Sci USA 2003;100:7797-802
   PubMed Web of Science ®Google Scholar
• Song E, Lee S, Wang J, et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat Med 2003;9:347-51
   PubMed Web of Science ®Google Scholar
• McCaffrey AP, Meuse L, Pham TT, et al. RNA interference in adult mice. Nature 2002;418:38-9
   PubMed Web of Science ®Google Scholar
• Tompkins SM, Lo C, Tumpey TM, Epstein SL. Protection against lethal influenza virus challenge by RNA interference in vivo. Proc Natl Acad Sci USA 2004;101:8682-6
   PubMed Web of Science ®Google Scholar
• Ge Q, Filip L, Bai A, et al. Inhibition of influenza virus production in virus infected mice by RNA interference. Proc Natl Acad Sci USA 2004;101:8676-81
   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
• Palliser D, Chowdhury D, Wang Q, et al. An siRNA-based microbicide protects mice from lethal Herpes simplex virus 2 infection. Nature 2006;439:89-94
   PubMed Web of Science ®Google Scholar
• Li BJ, Tang Q, Cheng D, et al. Using siRNA in prophylactic and therapeutic regimens against SARS coronavirus in Rhesus macaque. Nat Med 2005;11(9):944-51
   PubMed Web of Science ®Google Scholar
• Haussecker D, Kay MA. miR-122 continues to blaze the trail for microRNA therapeutics. Mol Ther 2010;18(2):240-2
   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(1):50-5
   PubMed Web of Science ®Google Scholar
• Cohen H, Levy RJ, Gao J, et al. Sustained delivery and expression of DNA encapsulated in polymeric nanoparticles. Gene Ther 2000;7(22):1896-905
   PubMed Web of Science ®Google Scholar
• Wang JC, Lai S, Guo X, et al. Attenuation of fibrosis in vitro and in vivo with SPARC siRNA. Arthritis Res Ther 2010;12(2):R60
   PubMed Web of Science ®Google Scholar
• Rosas-Taraco AG, Higgins DM, Sanchez-Campillo J, et al. Local pulmonary immunotherapy with siRNA targeting TGFbeta1 enhances antimicrobial capacity in Mycobacterium tuberculosis infected mice. Tuberculosis 2011;91(1):98-106
   PubMed Web of Science ®Google Scholar
• Rosas-Taraco AG, Higgins DM, Sanchez-Campillo J, et al. Intrapulmonary delivery of XCL1-targeting small interfering RNA in mice chronically infected with Mycobacterium tuberculosis. Am J Respir Cell Mol Biol 2009;41(2):136-45
   PubMed Web of Science ®Google Scholar
• Moschos SA, Jones SW, Perry MM, et al. Lung delivery studies using siRNA conjugated to TAT (48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity. Bioconjug Chem 2007;18(5):1450-9
   PubMed Web of Science ®Google Scholar
• Gutbier B, Kube SM, Reppe K, et al. RNAi-mediated suppression of constitutive pulmonary gene expression by small interfering RNA in mice. Pulm Pharmacol Ther 2010;23(4):334-44
   PubMed Web of Science ®Google Scholar
• Merkel OM, Beyerle A, Librizzi D, et al. Nonviral siRNA delivery to the lung: Investigation of PEG − PEI polyplexes and their in vivo performance. Mol Pharm 2009;6(4):1246-60
   PubMedGoogle Scholar
• Garbuzenko OB, Saad M, Betigeri S, et al. Intratracheal versus intravenous liposomal delivery of siRNA, antisense oligonucleotides and anticancer drug. Pharm Res 2009;26(2):382-94
   PubMed Web of Science ®Google Scholar
• Beyerle A, Braun A, Merkel O, et al. Comparative in vivo study of poly (ethylene imine)/siRNA complexes for pulmonary delivery in mice. J Control Release 2011;151(1):51-6
   PubMed Web of Science ®Google Scholar