Gene silencing efficiency of siRNA-PEG conjugates: Effect of PEGylation site and PEG molecular weight

Journal of Controlled Release

Volumn 144, Issue 3, 2010, Pages 306-313

  Jung, Sooyeon ^a   Lee, Soo Hyeon ^a   Mok, Hyejung ^a   Chung, Hyun Jung ^a   Park, Tae Gwan ^a  

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

Author keywords

Cleavable linkage; Conjugate site; Gene inhibition; PEGylation; SiRNA

Indexed keywords

ANTISENSE; CONJUGATE SITE; CRITICAL FACTORS; GENE SILENCING; LIPOFECTAMINE; PEGYLATION; SMALL INTERFERING RNA (SIRNA); TARGET SEQUENCES; THIOETHER LINKAGES;

ETHYLENE; ETHYLENE GLYCOL; GENETIC ENGINEERING; MOLECULAR WEIGHT; POLYETHYLENE GLYCOLS; POLYETHYLENE OXIDES;

RNA;

DICER; LIPOFECTAMINE; LIPOFECTAMINE 2000; MACROGOL; MESSENGER RNA; SMALL INTERFERING RNA; SULFIDE; UNCLASSIFIED DRUG; ALPHA INTERFERON; DRUG CARRIER; GREEN FLUORESCENT PROTEIN; MACROGOL DERIVATIVE; MONOMETHOXYPOLYETHYLENE GLYCOL; RIBONUCLEASE III; VASCULOTROPIN A; VEGFA PROTEIN, HUMAN;

ARTICLE; COMPLEX FORMATION; CONJUGATE; CONTROLLED STUDY; DISULFIDE BOND; GENE SILENCING; HUMAN; HUMAN CELL; MOLECULAR WEIGHT; PRIORITY JOURNAL; RNA DEGRADATION; RNA INTERFERENCE; CHEMISTRY; DRUG EFFECTS; ENZYME LINKED IMMUNOSORBENT ASSAY; GENETIC TRANSFECTION; GENETICS; METABOLISM; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; TUMOR CELL LINE;

CELL LINE, TUMOR; DRUG CARRIERS; ENZYME-LINKED IMMUNOSORBENT ASSAY; GENE SILENCING; GREEN FLUORESCENT PROTEINS; HUMANS; INTERFERON-ALPHA; MOLECULAR WEIGHT; POLYETHYLENE GLYCOLS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RIBONUCLEASE III; RNA, SMALL INTERFERING; TRANSFECTION; VASCULAR ENDOTHELIAL GROWTH FACTOR A;

EID: 77953287100     PISSN: 01683659     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jconrel.2010.03.002     Document Type: Article

References (57)

1. Zamore P.D., Tuschl T., Sharp P.A., Bartel D.P. RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell 2000, 101(1):25-33.
2. Dykxhoorn D.M., Novina C.D., Sharp P.A. Killing the messenger: short RNAs that silence gene expression. Nat. Rev. Mol. Cell Biol. 2003, 4(6):457-467.
3. McManus M.T., Sharp P.A. Gene silencing in mammals by small interfering RNAs. Nat. Rev. Genet. 2002, 3(10):737-747.
4. Song E., Lee S.K., Wang J., Ince N., Ouyang N., Min J., Chen J., Shankar P., Lieberman J. RNA interference targeting Fas protects mice from fulminant hepatitis. Nat. Med. 2003, 9(3):347-351.
5. Gartel A.L., Kandel E.S. RNA interference in cancer. Biomol. Eng. 2006, 23(1):17-34.
6. Takeshita F., Minakuchi Y., Nagahara S., Honma K., Sasaki H., Hirai K., Teratani T., Namatame N., Yamamoto Y., Hanai K., Kato T., Sano A., Ochiya T. Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc. Natl. Acad. Sci. U S A 2005, 102(34):12177-12182.
7. Dorsett Y., Tuschl T. siRNAs: applications in functional genomics and potential as therapeutics. Nat. Rev. Drug Discov. 2004, 3(4):318-329.
8. Ryther R.C., Flynt A.S., Phillips J.A., Patton J.G. siRNA therapeutics: big potential from small RNAs. Gene Ther. 2005, 12(1):5-11.
9. Braasch D.A., Paroo Z., Constantinescu A., Ren G., Oz O.K., Mason R.P., Corey D.R. Biodistribution of phosphodiester and phosphorothioate siRNA. Bioorg. Med. Chem. Lett. 2004, 14(5):1139-1143.
10. Schiffelers R.M., Ansari A., Xu J., Zhou Q., Tang Q., Storm G., Molema G., Lu P.Y., Scaria P.V., Woodle M.C. Cancer siRNA therapy by tumor selective delivery with ligand-targeted sterically stabilized nanoparticle. Nucleic Acids Res. 2004, 32(19):e149.
11. Jackson A.L., Bartz S.R., Schelter J., Kobayashi S.V., Burchard J., Mao M., Li B., Cavet G., Linsley P.S. Expression profiling reveals off-target gene regulation by RNAi. Nat. Biotechnol. 2003, 21(6):635-637.
12. Judge A.D., Sood V., Shaw J.R., Fang D., McClintock K., MacLachlan I. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat. Biotechnol. 2005, 23(4):457-462.
13. Hornung V., Guenthner-Biller M., Bourquin C., Ablasser A., Schlee M., Uematsu S., Noronha A., Manoharan M., Akira S., de Fougerolles A., Endres S., Hartmann G. Sequence-specific potent induction of IFN-alpha by short interfering RNA in plasmacytoid dendritic cells through TLR7. Nat. Med. 2005, 11(3):263-270.
14. Soutschek J., Akinc A., Bramlage B., Charisse K., Constien R., Donoghue M., Elbashir S., Geick A., Hadwiger P., Harborth J., John M., Kesavan V., Lavine G., Pandey R.K., Racie T., Rajeev K.G., Rohl I., Toudjarska I., Wang G., Wuschko S., Bumcrot D., Koteliansky V., Limmer S., Manoharan M., Vornlocher H.P. Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Nature 2004, 432(7014):173-178.
15. Wolfrum C., Shi S., Jayaprakash K.N., Jayaraman M., Wang G., Pandey R.K., Rajeev K.G., Nakayama T., Charrise K., Ndungo E.M., Zimmermann T., Koteliansky V., Manoharan M., Stoffel M. Mechanisms and optimization of in vivo delivery of lipophilic siRNAs. Nat. Biotechnol. 2007, 25(10):1149-1157.
16. Lorenz C., Hadwiger P., John M., Vornlocher H.P., Unverzagt C. Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Bioorg. Med. Chem. Lett. 2004, 14(19):4975-4977.
17. Chiu Y.L., Ali A., Chu C.Y., Cao H., Rana T.M. Visualizing a correlation between siRNA localization, cellular uptake, and RNAi in living cells. Chem. Biol. 2004, 11(8):1165-1175.
18. Xia C.F., Zhang Y., Boado R.J., Pardridge W.M. Intravenous siRNA of brain cancer with receptor targeting and avidin-biotin technology. Pharm. Res. 2007, 24(12):2309-2316.
19. Chu T.C., Twu K.Y., Ellington A.D., Levy M. Aptamer mediated siRNA delivery. Nucleic Acids Res. 2006, 34(10):e73.
20. Chiu Y.L., Rana T.M. RNAi in human cells: basic structural and functional features of small interfering RNA. Mol. Cell 2002, 10(3):549-561.
21. Czauderna F., Fechtner M., Dames S., Aygun H., Klippel A., Pronk G.J., Giese K., Kaufmann J. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 2003, 31(11):2705-2716.
22. Chiu Y.L., Rana T.M. siRNA function in RNAi: a chemical modification analysis. RNA 2003, 9(9):1034-1048.
23. Nguyen Q.N., Chavli R.V., Marques J.T., Conrad P.G., Wang D., He W., Belisle B.E., Zhang A., Pastor L.M., Witney F.R., Morris M., Heitz F., Divita G., Williams B.R., McMaster G.K. Light controllable siRNAs regulate gene suppression and phenotypes in cells. Biochim. Biophys. Acta 2006, 1758(3):394-403.
24. Nykanen A., Haley B., Zamore P.D. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell 2001, 107(3):309-321.
25. Shah S., Friedman S.H. Tolerance of RNA interference toward modifications of the 5' antisense phosphate of small interfering RNA. Oligonucleotides 2007, 17(1):35-43.
26. Kim S.H., Jeong J.H., Lee S.H., Kim S.W., Park T.G. PEG conjugated VEGF siRNA for anti-angiogenic gene therapy. J. Control Release 2006, 116(2):123-129.
27. Lee S.H., Kim S.H., Park T.G. 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):511-516.
28. 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 2008, 129(2):107-116.
29. Prakash T.P., Allerson C.R., Dande P., Vickers T.A., Sioufi N., Jarres R., Baker B.F., Swayze E.E., Griffey R.H., Bhat B. Positional effect of chemical modifications on short interference RNA activity in mammalian cells. J. Med. Chem. 2005, 48(13):4247-4253.
30. Oishi M., Nagasaki Y., Itaka K., Nishiyama N., Kataoka K. Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile β-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells. J. Am. Chem. Soc. 2005, 127(6):1624-1625.
31. Tanaka S., Ataka M., Onuma K., Kubota T. Rationalization of membrane protein crystallization with polyethylene glycol using a simple depletion model. Biophys. J. 2003, 84(5):3299-3306.
32. Schwarz D.S., Hutvagner G., Du T., Xu Z., Aronin N., Zamore P.D. Asymmetry in the assembly of the RNAi enzyme complex. Cell 2003, 115(2):199-208.
33. Parker J.S., Roe S.M., Barford D. Crystal structure of a PIWI protein suggests mechanisms for siRNA recognition and slicer activity. EMBO J. 2004, 23(24):4727-4737.
34. Nicholson A.W. Function, mechanism and regulation of bacterial ribonucleases. FEMS Microbiol. Rev. 1999, 23(3):371-390.
35. Filippov V., Solovyev V., Filippova M., Gill S.S. A novel type of RNase III family proteins in eukaryotes. Gene 2000, 245(1):213-221.
36. Cook A., Conti E. Dicer measures up. Nat. Struct. Mol. Biol. 2006, 13(3):190-192.
37. Rose S.D., Kim D.H., Amarzguioui M., Heidel J.D., Collingwood M.A., Davis M.E., Rossi J.J., Behlke M.A. Functional polarity is introduced by Dicer processing of short substrate RNAs. Nucleic Acids Res. 2005, 33(13):4140-4156.
38. Chang C.I., Hong S.W., Kim S., Lee D.K. A structure-activity relationship study of siRNAs with structural variations. Biochem. Biophys. Res. Commun. 2007, 359(4):997-1003.
39. Nishina K., Unno T., Uno Y., Kubodera T., Kanouchi T., Mizusawa H., Yokota T. Efficient in vivo delivery of siRNA to the liver by conjugation of α-tocopherol. Mol. Ther. 2008, 16(4):734-740.
40. Elbashir S.M., Lendeckel W., Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev. 2001, 15(2):188-200.
41. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411(6836):494-498.
42. Lima W.F., Murray H., Nichols J.G., Wu H., Sun H., Prakash T.P., Berdeja A.R., Gaus H.J., Crooke S.T. 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(4):2535-2548.
43. 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. 2002, 21(21):5875-5885.
44. Vermeulen A., Behlen L., Reynolds A., Wolfson A., Marshall W.S., Karpilow J., Khvorova A. The contributions of dsRNA structure to Dicer specificity and efficiency. RNA 2005, 11(5):674-682.
45. Kim D.H., Behlke M.A., Rose S.D., Chang M.S., Choi S., Rossi J.J. Synthetic dsRNA Dicer substrates enhance RNAi potency and efficacy. Nat. Biotechnol. 2005, 23(2):222-226.
46. Elbashir S.M., Martinez J., Patkaniowska A., Lendeckel W., Tuschl T. Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate. EMBO J. 2001, 20(23):6877-6888.
47. Ma J.B., Ye K., Patel D.J. Structural basis for overhang-specific small interfering RNA recognition by the PAZ domain. Nature 2004, 429(6989):318-322.
48. Takeda K., Akira S. Toll-like receptors in innate immunity. Int. Immunol. 2005, 17(1):1-14.
49. Kleinman M.E., Yamada K., Takeda A., Chandrasekaran V., Nozaki M., Baffi J.Z., Albuquerque R.J., Yamasaki S., Itaya M., Pan Y., Appukuttan B., Gibbs D., Yang Z., Kariko K., Ambati B.K., Wilgus T.A., DiPietro L.A., Sakurai E., Zhang K., Smith J.R., Taylor E.W., Ambati J. Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Nature 2008, 452(7187):591-597.
50. Sioud M. RNA interference and innate immunity. Adv. Drug Deliv. Rev. 2007, 59(2-3):153-163.
51. Robbins M., Judge A., Ambegia E., Choi C., Yaworski E., Palmer L., McClintock K., MacLachlan I. Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation. Hum. Gene Ther. 2008, 19(10):991-999.
52. Zamanian-Daryoush M., Marques J.T., Gantier M.P., Behlke M.A., John M., Rayman P., Finke J., Williams B.R. Determinants of cytokine induction by small interfering RNA in human peripheral blood mononuclear cells. J. Interferon. Cytokine Res. 2008, 28(4):221-233.
53. Hornung V., Guenthner-Biller M., Bourquin C., Ablasser A., Schlee M., Uematsu S., Noronha A., Manoharan M., Akira S., de Fougerolles A., Endres S., Hartmann G. Sequence-specific potent induction of IFN-α by short interfering RNA in plasmacytoid dendritic cells through TLR7. Nat. Med. 2005, 11(3):263-270.
54. Hansmann L., Groeger S., von Wulffen W., Bein G., Hackstein H. Human monocytes represent a competitive source of interferon-α in peripheral blood. Clin. Immunol. 2008, 127(2):252-264.
55. Mok H., Palmer D.J., Ng P., Barry M.A. Evaluation of polyethylene glycol modification of first-generation and helper-dependent adenoviral vectors to reduce innate immune responses. Mol. Ther. 2005, 11(1):66-79.
56. Croyle M.A., Chirmule N., Zhang Y., Wilson J.M. "Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung. J. Virol 2001, 75(10):4792-4801.
57. Malek A., Czubayko F., Aigner A. PEG grafting of polyethylenimine (PEI) exerts different effects on DNA transfection and siRNA-induced gene targeting efficacy. J. Drug Target. 2008, 16(2):124-139.