Enhanced intracellular delivery and multi-target gene silencing triggered by tripodal RNA structures

Journal of Gene Medicine

Volumn 14, Issue 2, 2012, Pages 138-146

  Chang, Chan Il ^a,d   Lee, Tae Yeon ^a   Kim, Sera ^a   Sun, Xiangao ^b   Hong, Sun Woo ^c   Yoo, Jae Wook ^a   Dua, Pooja ^a   Kang, Hye Suk ^a   Kim, Soyoun ^c   Li, Chiang J ^b,d   Lee, Dong Ki ^a  

^a Sungkyunkwan University   (South Korea)

^b DAINIPPON SUMITOMO PHARMA CO LTD   (Japan)

^c Dongguk University   (South Korea)

^d HARVARD MEDICAL SCHOOL   (United States)

Author keywords

Gene delivery; Gene silencing; HeLa; Nanomedicine; RNA interference

Indexed keywords

DOUBLE STRANDED DNA; RNA INDUCED SILENCING COMPLEX; SMALL INTERFERING RNA;

ANTINEOPLASTIC ACTIVITY; ARTICLE; CELL DEATH; GENE SILENCING; HUMAN; HUMAN TISSUE; IMMUNE RESPONSE; INTRACELLULAR TRANSPORT; PRIORITY JOURNAL; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA STRUCTURE; STRUCTURE ANALYSIS;

FLOW CYTOMETRY; GENE SILENCING; GENE TARGETING; GENE TRANSFER TECHNIQUES; HELA CELLS; HUMANS; LUCIFERASES; NANOMEDICINE; POLYETHYLENEIMINE; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA; RNA INTERFERENCE;

EID: 84863116793     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1653     Document Type: Article

References (36)

1. Fire A, Xu S, Montgomery MK, et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391: 806-811.
2. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411: 494-498.
3. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference-based therapeutics. Nature 2009; 457: 426-433.
4. Mercatelli N, Coppola V, Bonci D, et al. The inhibition of the highly expressed miR-221 and miR-222 impairs the growth of prostate carcinoma xenografts in mice. PLoS One 2008; 3: e4029.
5. Shan Y, Zheng J, Lambrecht RW, et al. Reciprocal effects of micro-RNA-122 on expression of heme oxygenase-1 and hepatitis C virus genes in human hepatocytes. Gastroenterology 2007; 133: 1166-1174.
6. Care A, Catalucci D, Felicetti F, et al. MicroRNA-133 controls cardiac hypertrophy. Nat Med 2007; 13: 613-618.
7. Chen JF, Callis TE, Wang DZ. MicroRNAs and muscle disorders. J Cell Sci 2009; 122: 13-20.
8. Boden D, Pusch O, Lee F, et al. Human immunodeficiency virus type 1 escape from RNA interference. J Virol 2003; 77: 11531-11535.
9. Das AT, Brummelkamp TR, Westerhout EM, et al. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J Virol 2004; 78: 2601-2605.
10. Westerhout EM, Ooms M, Vink M, et al. HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome. Nucleic Acids Res 2005; 33: 796-804.
11. Menendez JA, Vellon L, Mehmi I, et al. Inhibition of fatty acid synthase (FAS) suppresses HER2/neu (erbB-2) oncogene overexpression in cancer cells. Proc Natl Acad Sci USA 2004; 101: 10715-10720.
12. Grimm D, Kay MA. Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther 2007; 15: 878-888.
13. Goga A, Benz C. Anti-oncomir suppression of tumor phenotypes. Mol Interv 2007; 7: 199-202, 180.
14. Seeman NC. DNA in a material world. Nature 2003; 421: 427-431.
15. Guo P. The emerging field of RNA nanotechnology. Nat Nanotechnol 2010; 5: 833-842.
16. Guo P, Coban O, Snead NM, et al. Engineering RNA for targeted siRNA delivery and medical application. Adv Drug Deliv Rev 2010; 62: 650-666.
17. McNamara JO II, Andrechek ER, Wang Y, et al. Cell type-specific delivery of siRNAs with aptamer-siRNA chimeras. Nat Biotechnol 2006; 24: 1005-1015.
18. Zhou J, Li H, Li S, et al. Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy. Mol Ther 2008; 16: 1481-1489.
19. Ehsani A, Saetrom P, Zhang J, et al. Rational design of micro-RNA-like bifunctional siRNAs targeting HIV and the HIV coreceptor CCR5. Mol Ther 2010; 18: 796-802.
20. Lu Y, Xiao J, Lin H, et al. A single anti-microRNA antisense oligodeoxyribonucleotide (AMO) targeting multiple microRNAs offers an improved approach for microRNA interference. Nucleic Acids Res 2009; 37: e24.
21. Kim DH, Behlke MA, Rose SD, et al. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat Biotechnol 2005; 23: 222-226.
22. Chang CI, Yoo JW, Hong SW, et al. Asymmetric shorter-duplex siRNA structures trigger efficient gene silencing with reduced nonspecific effects. Mol Ther 2009; 17: 725-732.
23. Shchepinov MS, Udalova IA, Bridgman AJ, et al. Oligonucleotide dendrimers: synthesis and use as polylabelled DNA probes. Nucleic Acids Res 1997; 25: 4447-4454.
24. Chang CI, Hong SW, Kim S, et al. A structure-activity relationship study of siRNAs with structural variations. Biochem Biophys Res Commun 2007; 359: 997-1003.
25. Chang CI, Kang HS, Ban C, et al. Dual-target gene silencing by using long, synthetic siRNA duplexes without triggering antiviral responses. Mol Cells 2009; 27: 689-695.
26. Zhang K, Wang Q, Xie Y, et al. Receptor-mediated delivery of siRNAs by tethered nucleic acid base-paired interactions. RNA 2008; 14: 577-583.
27. Aigner A. Gene silencing through RNA interference (RNAi) in vivo: strategies based on the direct application of siRNAs. J Biotechnol 2006; 124: 12-25.
28. Urban-Klein B, Werth S, Abuharbeid S, et al. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther 2005; 12: 461-466.
29. Bolcato-Bellemin AL, Bonnet ME, Creusat G, et al. Sticky overhangs enhance siRNA-mediated gene silencing. Proc Natl Acad Sci USA 2007; 104: 16050-16055.
30. Mok H, Lee SH, Park JW, et al. Multimeric small interfering ribonucleic acid for highly efficient sequence-specific gene silencing. Nat Mater 2010; 9: 272-278.
31. Lee SY, Huh MS, Lee S, et al. Stability and cellular uptake of polymerized siRNA (poly siRNA)/polyethylenimine (PEI) complexes for efficient gene silencing. J Control Release 2010; 141: 339-346.
32. Altieri DC. Survivin, cancer networks and pathway-directed drug discovery. Nat Rev Cancer 2008; 8: 61-70.
33. Yu H, Pardoll D, Jove R. STATs in cancer inflammation and immunity: a leading role for STAT3. Nat Rev Cancer 2009; 9: 798-809.
34. Marques JT, Devosse T, Wang D, et al. A structural basis for discriminating between self and nonself double-stranded RNAs in mammalian cells. Nat Biotechnol 2006; 24: 559-565.
35. Tan GS, Garchow BG, Liu X, et al. Expanded RNA-binding activities of mammalian Argonaute 2. Nucleic Acids Res 2009; 37: 7533-7545.
36. Salomon W, Bulock K, Lapierre J, et al. Modified dsRNAs that are not processed by Dicer maintain potency and are incorporated into the RISC. Nucleic Acids Res 2010; 38: 3771-3779.