Multimeric small interfering ribonucleic acid for highly efficient sequence-specific gene silencing

Nature Materials

Volumn 9, Issue 3, 2010, Pages 272-278

  Mok, Hyejung ^a   Lee, Soo Hyeon ^a   Park, Ji Won ^a   Park, Tae Gwan ^a  

^a Korea Advanced Institute of Science and Technology (KAIST)   (South Korea)

Author keywords

[No Author keywords available]

Indexed keywords

CROSSLINKING; GENE THERAPY; GENES; GENETIC ENGINEERING; NUCLEIC ACIDS; POLYELECTROLYTES; TRANSCRIPTION;

CLINICAL APPLICATION; CONDENSING AGENTS; NEW THERAPEUTIC AGENT; POLYELECTROLYTE COMPLEXES; SELF-CROSSLINKED; SMALL INTERFERING RNA (SIRNA); TARGET MESSENGERS; TRANSCRIPTION LEVEL;

RNA;

GREEN FLUORESCENT PROTEIN; SMALL INTERFERING RNA; VASCULOTROPIN A;

ARTICLE; CHEMISTRY; CONFORMATION; GENE SILENCING; GENETICS; POLYACRYLAMIDE GEL ELECTROPHORESIS; RNA INTERFERENCE; RNA PROCESSING;

ELECTROPHORESIS, POLYACRYLAMIDE GEL; GENE SILENCING; GREEN FLUORESCENT PROTEINS; NUCLEIC ACID CONFORMATION; RNA INTERFERENCE; RNA PROCESSING, POST-TRANSCRIPTIONAL; RNA, SMALL INTERFERING; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 77249154018     PISSN: 14761122     EISSN: 14764660     Source Type: Journal    
DOI: 10.1038/nmat2626     Document Type: Article

References (30)

1. Jeong, J. H., Kim, S. W. & Park, T. G. Molecular design of functional polymers for gene therapy. Prog. Polym. Sci. 32, 1239-1274 (2007).
2. Jeong, J. H., Mok, H., Oh, Y. & Park, T. G. siRNA conjugate delivery systems. Bioconjug. Chem. 20, 5-14 (2009).
3. Elbashir, S. M., Lendeckel, W. & Tuschl, T. RNA interference is mediated by 21-and 22-nucleotide RNAs. Genes Dev. 15, 188-200 (2001). (Pubitemid 32107664)
4. Kim, D. H. et al. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nature Biotech. 23, 222-226 (2005).
5. Bolcato-Bellemin, A. L., Bonnet, M. E., Creusat, G., Erbacher, P. & Behr, J. P. Sticky overhangs enhance siRNA-mediated gene silencing. Proc. Natl Acad. Sci. USA 104, 16050-16055 (2007).
6. Mok, H. & Park, T. G. Self-crosslinked and reducible fusogenic peptides for intracellular delivery of siRNA. Biopolymers 89, 881-888 (2008).
7. Wang, W. & Ballatori, N. Endogenous glutathione conjugates: Occurrence and biological functions. Pharmacol. Rev. 50, 335-356 (1998).
8. Mok, H., Park, J. W. & Park, T. G. Antisense oligodeoxynucleotide- conjugated hyaluronic acid/protamine nanocomplexes for intracellular gene inhibition. Bioconjug. Chem. 18, 1483-1489 (2007).
9. Gary, D. J., Puri, N. & Won, Y. Y. Polymer-based siRNA delivery: Perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery. J. Control. Release 121, 64-73 (2007).
10. Shah, S. A. & Brunger, A. T. The 1.8 A crystal structure of a statically disordered 17 base-pair RNA duplex: Principles of RNA crystal packing and its effect on nucleic acid structure. J. Mol. Biol. 285, 1577-1588 (1999).
11. Kebbekus, P., Draper, D. E. & Hagerman, P. Persistence length of RNA. Biochemistry 34, 4354-4357 (1995).
12. Neu, M., Sitterberg, J., Bakowsky, U. & Kissel, T. Stabilized nanocarriers for plasmids based on cross-linked poly(ethylene imine). Biomacromolecules 7, 3428-3438 (2006).
13. Lee, J. H. et al. All-in-one target-cell-specific magnetic nanoparticles for simultaneous molecular imaging and siRNA delivery. Angew. Chem. Int. Ed. 48, 4174-4179 (2009).
14. Wack, S. et al. Feasibility, sensitivity, and reliability of laser-induced fluorescence imaging of green fluorescent protein-expressing tumours in vivo. Mol. Ther. 7, 765-773 (2003).
15. Banerjee, P., Weissleder, R. & Bogdanov, A. Jr Linear polyethyleneimine grafted to a hyperbranched poly(ethylene glycol)-like core: A copolymer for gene delivery. Bioconjug. Chem. 17, 125-131 (2006).
16. Mehrotra, S., Lee, I. & Chan, C. Multilayer mediated forward and patterned siRNA transfection using linear-PEI at extended N/P ratios. Acta Biomater. 5, 1474-1488 (2009).
17. Rose, S. D. et al. Functional polarity is introduced by Dicer processing of short substrate RNAs. Nucleic Acids Res. 33, 4140-4156 (2005).
18. Zhang, H., Kolb, F. A., Brondani, V., Billy, E. & Filipowicz, W. Human Dicer preferentially cleaves dsRNAs at their termini without a requirement for ATP. EMBO J. 21, 5875-5885 (2002).
19. Castanotto, D. & Rossi, J. J. The promises and pitfalls of RNA-interference-based therapeutics. Nature 457, 426-433 (2009).
20. Wassenegger, M. The role of the RNAi machinery in heterochromatin formation. Cell 122, 13-16 (2005).
21. Matzke, M. A. & Birchler, J. A. RNAi-mediated pathways in the nucleus. Nature Rev. Genet. 6, 24-35 (2005).
22. Robbins, M. et al. Misinterpreting the therapeutic effects of siRNA caused by immune stimulation. Hum. Gene Ther. 19, 991-999 (2008).
23. Zamanian-Daryoush, M. et al. Determinants of cytokine induction by small interfering RNA in human peripheral blood mononuclear cells. J. Interferon Cytokine Res. 28, 221-233 (2008).
24. Judge, A. & MacLachlan, I. Overcoming the innate immune response to small interfering RNA. Hum. Gene Ther. 19, 111-124 (2008).
25. Sioud, M. RNA interference and innate immunity. Adv. Drug Deliv. Rev. 59, 153-163 (2007).
26. Aliyari, R. & Ding, S. W. RNA-based viral immunity initiated by the Dicer family of host immune receptors. Immunol. Rev. 227, 176-188 (2009).
27. Castanotto, D. et al. Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC. Nucleic Acids Res. 35, 5154-5164 (2007).
28. Wightman, L. et al. Different behaviour of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J. Gene Med. 3, 362-372 (2001).
29. Judge, A. D. et al. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nature Biotech. 23, 457-462 (2005).
30. Kim, S. H., Jeong, J. H., Lee, S. H., Kim, S. W. & Park, T. G. Local and systemic delivery of VEGF siRNA using polyelectrolyte complex micelles for effective treatment of cancer. J. Control. Release 129, 107-116 (2008).