Strategies for targeted nonviral delivery of siRNAs in vivo

Trends in Molecular Medicine

Volumn 15, Issue 11, 2009, Pages 491-500

  Kim, Sang Soo ^a   Garg, Himanshu ^a   Joshi, Anjali ^a   Manjunath, N ^a  

^a TEXAS TECH UNIVERSITY HEALTH SCIENCES CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AEG 35156; AGN 211745; AKII 5; ALN RSV 01; APTAMER; ATN RNA; ATU 027; BEVASIRANIB; CALAA 01; CHITOSAN; CYCLODEXTRIN; GLUCAN; LENTIVIRUS VECTOR; LIPID; LIPOSOME; NUC B1000; NUCLEIC ACID; OLIGOSACCHARIDE; POLYMER; PRO 020401; RTP 801I 14; SHORT HAIRPIN RNA; SIRNA 027; SMALL INTERFERING RNA; SPC 3649; STABLE NUCLEIC ACID LIPID PARTICLE; TD 101; UNCLASSIFIED DRUG;

ACUTE KIDNEY FAILURE; ADVANCED CANCER; ANTINEOPLASTIC ACTIVITY; ANTIVIRAL ACTIVITY; BRAIN CANCER; BREAST CANCER; CELL TRANSPORT; CELL TYPE; CHRONIC LYMPHATIC LEUKEMIA; CLINICAL TRIAL; DIABETIC MACULAR EDEMA; ENTERITIS; EWING SARCOMA; EXTRACELLULAR MATRIX; GENE EXPRESSION; GENE FUNCTION; GENE SILENCING; GENE TARGETING; GENETIC ANALYSIS; GLIOBLASTOMA; HEPATITIS B; HEPATITIS C; HERPES SIMPLEX; HUMAN; HYPERCHOLESTEROLEMIA; IMMUNOSTIMULATION; IN VIVO STUDY; INFLUENZA; INNATE IMMUNITY; LIPOSOMAL DELIVERY; LUNG NON SMALL CELL CANCER; MELANOMA; NASOPHARYNX CARCINOMA; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PANCREAS CANCER; PARONYCHIA; PROTEIN AGGREGATION; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; RNA INTERFERENCE; SOLID TUMOR; VIRUS ENCEPHALITIS; VIRUS HEMORRHAGIC FEVER; VIRUS INFECTION;

ANIMALS; APTAMERS, NUCLEOTIDE; GENE SILENCING; GENETIC VECTORS; MICE; RNA INTERFERENCE; RNA, SMALL INTERFERING;

EID: 70449089802     PISSN: 14714914     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.molmed.2009.09.001     Document Type: Review

References (81)

1. Kim D.H., and Rossi J.J. Overview of gene silencing by RNA interference. Curr. Protoc. Nucleic. Acid Chem. (2009) 11 Chapter 16, Unit 16
2. Kim D., and Rossi J. RNAi mechanisms and applications. Biotechniques 44 (2008) 613-616
3. de Fougerolles A., et al. Interfering with disease: a progress report on siRNA-based therapeutics. Nat. Rev. Drug Discov. 6 (2007) 443-453
4. Watanabe T., et al. Endogenous siRNAs from naturally formed dsRNAs regulate transcripts in mouse oocytes. Nature 453 (2008) 539-543
5. Babiarz J.E., et al. Mouse ES cells express endogenous shRNAs, siRNAs and other microprocessor-independent, Dicer-dependent small RNAs. Genes Dev. 22 (2008) 2773-2785
6. Castanotto D., and Rossi J.J. The promises and pitfalls of RNA-interference-based therapeutics. Nature 457 (2009) 426-433
7. Manjunath N., et al. Interfering antiviral immunity: application, subversion, hope?. Trends Immunol. 27 (2006) 328-335
8. Check E. A crucial test. Nat. Med. 11 (2005) 243-244
9. DeVincenzo J., et al. Evaluation of the safety, tolerability and pharmacokinetics of ALN-RSV01, a novel RNAi antiviral therapeutic directed against respiratory syncytial virus (RSV). Antiviral Res. 77 (2008) 225-231
10. Chappelow A.V., and Kaiser P.K. Neovascular age-related macular degeneration: potential therapies. Drugs 68 (2008) 1029-1036
11. Kleinman M.E., et al. Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Nature 452 (2008) 591-597
12. Kirchhoff F. Silencing HIV-1 in vivo. Cell 134 (2008) 566-568
13. Grimm D., and Kay M.A. Therapeutic application of RNAi: is mRNA targeting finally ready for prime time?. J. Clin. Invest. 117 (2007) 3633-3641
14. Manjunath, N. et al. (2009) Lentiviral delivery of short hairpin RNAs. Adv Drug Deliv Rev. (in press)
15. Akhtar S., and Benter I.F. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 117 (2007) 3623-3632
16. Aagaard L., and Rossi J.J. RNAi therapeutics: principles, prospects and challenges. Adv. Drug Deliv. Rev. 59 (2007) 75-86
17. Detzer A., et al. Phosphorothioate-stimulated cellular uptake of siRNA: a cell culture model for mechanistic studies. Curr. Pharm. Des. 14 (2008) 3666-3673
18. Juliano R., et al. Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic. Acids Res. 36 (2008) 4158-4171
19. Dykxhoorn D.M., et al. The silent treatment: siRNAs as small molecule drugs. Gene Ther. 13 (2006) 541-552
20. Lewis D.L., et al. Efficient delivery of siRNA for inhibition of gene expression in postnatal mice. Nat. Genet 32 (2002) 107-108
21. Rippe B., et al. Transendothelial transport: the vesicle controversy. J. Vasc. Res. 39 (2002) 375-390
22. Greish K. Enhanced permeability and retention of macromolecular drugs in solid tumors: a royal gate for targeted anticancer nanomedicines. J. Drug Target. 15 (2007) 457-464
23. Zamecnik J., et al. Extracellular matrix glycoproteins and diffusion barriers in human astrocytic tumors. Neuropathol. Appl. Neurobiol. 30 (2004) 338-350
24. Oliveira S., et al. Fusogenic peptides enhance endosomal escape improving siRNA-induced silencing of oncogenes. Int. J. Pharm. 331 (2007) 211-214
25. Song E., et al. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med. 9 (2003) 347-351
26. Fattal E., and Bochot A. State of the art and perspectives for the delivery of antisense oligonucleotides and siRNA by polymeric nanocarriers. Int. J. Pharm. 364 (2008) 237-248
27. Hornung V., et al. Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7. Nat. Med. 11 (2005) 263-270
28. Kim D.H., and Rossi J.J. Strategies for silencing human disease using RNA interference. Nat. Rev. Genet. 8 (2007) 173-184
29. Liu B. Exploring cell type-specific internalizing antibodies for targeted delivery of siRNA. Brief Funct. Genomic Proteomic 6 (2007) 112-119
30. Chiu Y.L., et al. Visualizing a correlation between siRNA localization, cellular uptake and RNAi in living cells. Chem. Biol. 11 (2004) 1165-1175
31. Rossi J.J. Receptor-targeted siRNAs. Nat. Biotechnol. 23 (2005) 682-684
32. Urban-Klein B., et al. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther. 12 (2005) 461-466
33. Kumar P., et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 448 (2007) 39-43
34. Kumar P., et al. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 134 (2008) 577-586
35. Zimmermann T.S., et al. RNAi-mediated gene silencing in non-human primates. Nature 441 (2006) 111-114
36. Kawakami S., and Hashida M. Targeted delivery systems of small interfering RNA by systemic administration. Drug Metab. Pharmacokinet. 22 (2007) 142-151
37. Jeong J.H., et al. siRNA conjugate delivery systems. Bioconjug. Chem. 20 (2009) 5-14
38. Soutschek J., et al. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 432 (2004) 173-178
39. Peer D., et al. Systemic leukocyte-directed siRNA delivery revealing cyclin D1 as an anti-inflammatory target. Science 319 (2008) 627-630
40. Tompkins S.M., et al. Protection against lethal influenza virus challenge by RNA interference in vivo. Proc. Natl. Acad. Sci. U S A 101 (2004) 8682-8686
41. Palliser D., et al. An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection. Nature 439 (2006) 89-94
42. Kumar P., et al. A single siRNA suppresses fatal encephalitis induced by two different flaviviruses. PLoS Med. 3 (2006) e96
43. Pardridge W.M. shRNA and siRNA delivery to the brain. Adv. Drug Deliv. Rev. 59 (2007) 141-152
44. Zhang Y., et al. Intravenous RNA interference gene therapy targeting the human epidermal growth factor receptor prolongs survival in intracranial brain cancer. Clin. Cancer Res. 10 (2004) 3667-3677
45. Pirollo K.F., et al. Tumor-targeting nanoimmunoliposome complex for short interfering RNA delivery. Hum. Gene. Ther. 17 (2006) 117-124
46. Hogrefe R.I., et al. Chemically modified short interfering hybrids (siHYBRIDS): nanoimmunoliposome delivery in vitro and in vivo for RNAi of HER-2. Nucleosides Nucleotides Nucleic. Acids 25 (2006) 889-907
47. Morrissey D.V., et al. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol. 23 (2005) 1002-1007
48. Ma Z., et al. Cationic lipids enhance siRNA-mediated interferon response in mice. Biochem. Biophys. Res. Commun. 330 (2005) 755-759
49. Akinc A., et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol. 26 (2008) 561-569
50. Shim M.S., and Kwon Y.J. Controlled delivery of plasmid DNA and siRNA to intracellular targets using ketalized polyethylenimine. Biomacromolecules 9 (2008) 444-455
51. Tan P.H., et al. Gene knockdown with intrathecal siRNA of NMDA receptor NR2B subunit reduces formalin-induced nociception in the rat. Gene. Ther. 12 (2005) 59-66
52. Thomas M., et al. Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. Proc. Natl. Acad Sci. U S A 102 (2005) 5679-5684
53. Chiu S.J., et al. Tumor-targeted gene delivery via anti-HER2 antibody (trastuzumab, Herceptin) conjugated polyethylenimine. J. Control Release 97 (2004) 357-369
54. Kim S.H., et al. 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 (2006) 241-244
55. Guo S., et al. Construction of folate-conjugated pRNA of bacteriophage phi29 DNA packaging motor for delivery of chimeric siRNA to nasopharyngeal carcinoma cells. Gene. Ther. 13 (2006) 814-820
56. Pirollo K.F., et al. Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system. Cancer Res. 67 (2007) 2938-2943
57. Park J.W., et al. Anti-HER2 immunoliposomes: enhanced efficacy attributable to targeted delivery. Clin. Cancer Res. 8 (2002) 1172-1181
58. Mamot C., et al. Epidermal growth factor receptor-targeted immunoliposomes significantly enhance the efficacy of multiple anticancer drugs in vivo. Cancer Res. 65 (2005) 11631-11638
59. Schiffelers R.M., et al. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic. Acids Res. 32 (2004) e149
60. Alshamsan A., et al. Formulation and delivery of siRNA by oleic acid and stearic acid modified polyethylenimine. Mol. Pharm. 6 (2009) 121-133
61. Zintchenko A., et al. Simple modifications of branched PEI lead to highly efficient siRNA carriers with low toxicity. Bioconjug. Chem. 19 (2008) 1448-1455
62. Howard K.A., et al. Chitosan/siRNA nanoparticle-mediated TNF-alpha knockdown in peritoneal macrophages for anti-inflammatory treatment in a murine arthritis model. Mol. Ther. 17 (2009) 162-168
63. Howard K.A., et al. RNA interference in vitro and in vivo using a novel chitosan/siRNA nanoparticle system. Mol. Ther. 14 (2006) 476-484
64. Zhang W., et al. Inhibition of respiratory syncytial virus infection with intranasal siRNA nanoparticles targeting the viral NS1 gene. Nat. Med. 11 (2005) 56-62
65. Hu-Lieskovan S., 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. 65 (2005) 8984-8992
66. Aouadi M., et al. Orally delivered siRNA targeting macrophage Map4k4 suppresses systemic inflammation. Nature 458 (2009) 1180-1184
67. Vangasseri D.P., et al. Lipid-protamine-DNA-mediated antigen delivery. Curr. Drug Deliv. 2 (2005) 401-406
68. Song E., et al. Antibody mediated in vivo delivery of small interfering RNAs via cell surface receptors. Nat. Biotechnol. 23 (2005) 709-717
69. Peer D., et al. 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 (2007) 4095-4100
70. Schwarze S.R., et al. In vivo protein transduction: delivery of a biologically active protein into the mouse. Science 285 (1999) 1569-1572
71. Torchilin V.P., et al. 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 (2001) 8786-8791
72. Kim W.J., et al. Cholesteryl oligoarginine delivering vascular endothelial growth factor siRNA effectively inhibits tumor growth in colon adenocarcinoma. Mol. Ther. 14 (2006) 343-350
73. McNamara II J.O., et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat. Biotechnol. 24 (2006) 1005-1015
74. Chu T.C., et al. Aptamer mediated siRNA delivery. Nucleic. Acids Res. 34 (2006) e73
75. Zhou J., et al. Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy. Mol. Ther. 16 (2008) 1481-1489
76. Zhou J., et al. 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 (2009) 3094-3109
77. Marshall E. Gene therapy. Second child in French trial is found to have leukemia. Science 299 (2003) 320
78. Hacein-Bey-Abina S., et al. Gene therapy of X-linked severe combined immunodeficiency. Int. J. Hematol. 76 (2002) 295-298
79. Song E., et al. Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages. J. Virol. 77 (2003) 7174-7181
80. Omi K., et al. Long-lasting RNAi activity in mammalian neurons. FEBS Lett. 558 (2004) 89-95
81. Cardoso A.L., et al. Tf-lipoplexes for neuronal siRNA delivery: a promising system to mediate gene silencing in the CNS. J. Control Release 132 (2008) 113-123