Click conjugated polymeric immuno-nanoparticles for targeted siRNA and antisense oligonucleotide delivery

Biomaterials

Volumn 34, Issue 33, 2013, Pages 8408-8415

  Chan, Dianna P Y ^a,b   Deleavey, Glen F ^d   Owen, Shawn C ^a,b   Damha, Masad J ^d   Shoichet, Molly S ^a,b,c  

^a Department of Chemical Engineering and Applied Chemistry ^*  (Canada)

^b UNIVERSITY OF TORONTO   (Canada)

^c UNIVERSITY OF TORONTO   (Canada)

^d MCGILL UNIVERSITY   (Canada)

Author keywords

Antisense; Copolymer; Gene therapy; Micelle; Self assembly; Tumour targeting

Indexed keywords

ANTISENSE; ANTISENSE OLIGONUCLEOTIDES; CELLULAR DELIVERY; CONJUGATED LIGANDS; POLYMERIC DELIVERY; POLYMERIC NANOPARTICLES; SMALL INTERFERING RNA; TUMOUR-TARGETING;

COPOLYMERS; FLUORINE; GENE THERAPY; GENETIC ENGINEERING; LIPOSOMES; MICELLES; NANOPARTICLES; OLIGONUCLEOTIDES; RNA; SELF ASSEMBLY;

CHEMICAL MODIFICATION;

ANTIBODY; ANTISENSE OLIGONUCLEOTIDE; HER2 ANTIBODY; LIPOFECTAMINE; NANOPARTICLE; NUCLEIC ACID; PHOSPHOROTHIOIC ACID; POLYLACTIDE; POLYMERIC IMMUNO NANOPARTICLE; RNA; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ARTICLE; CHEMICAL MODIFICATION; CONTROLLED STUDY; GENE DELIVERY SYSTEM; GENE SILENCING; GENE THERAPY; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; PRIORITY JOURNAL;

ANTISENSE; COPOLYMER; GENE THERAPY; MICELLE; SELF-ASSEMBLY; TUMOUR-TARGETING;

CELL LINE, TUMOR; HUMANS; MICELLES; NANOPARTICLES; OLIGONUCLEOTIDES, ANTISENSE; POLYMERS; RNA, SMALL INTERFERING;

EID: 84881670224     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2013.07.019     Document Type: Article

References (61)

1. Kim W.J., Kim S.W. Efficient siRNA delivery with non-viral polymeric vehicles. Pharm Res 2009, 26(3):657-666.
2. Rayburn E.R., Zhang R.W. Antisense, RNAi, and gene silencing strategies for therapy: mission possible or impossible?. Drug Discov Today 2008, 13(11-12):513-521.
3. Deleavey G.F., Damha M.J. Designing chemically modified oligonucleotides for targeted gene silencing. Chem Biol 2012, 19(8):937-954.
4. Jackson A.L., Burchard J., Leake D., Reynolds A., Schelter J., Guo J., et al. Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing. RNA 2006, 12(7):1197-1205.
5. Whitehead K.A., Dahlman J.E., Langer R.S., Anderson D.G. Silencing or stimulation? siRNA delivery and the immune system. Annu Rev Chem Biomol Eng 2011, 2:77-96.
6. Watts J.K., Deleavey G.F., Damha M.J. Chemically modified siRNA: tools and applications. Drug Discov Today 2008, 13(19-20):842-855.
7. Kalota A., Karabon L., Swider C.R., Viazovkina E., Elzagheid M., Damha M.J., et al. 2'-Deoxy-2'-fluoro-beta-d-arabinonucleic acid (2' F-ANA) modified oligonucleotides (ON) effect highly efficient, and persistent, gene silencing. Nucleic Acids Res 2006, 34(2):451-461.
8. Yokoyama M. Polymeric micelles as a new drug carrier system and their required considerations for clinical trials. Expert Opin Drug Deliv 2010, 7(2):145-158.
9. Saif M.W., Podoltsev N.A., Rubin M.S., Figueroa J.A., Lee M.Y., Kwon J., et al. Phase II clinical trial of paclitaxel loaded polymeric micelle in patients with advanced pancreatic cancer. Cancer Invest 2010, 28(2):186-194.
10. Lee S.Y., Kim S.Y., Lee K.Y., Jeon Y., Lee K., Kim Y., et al. Arandomized clinical trial of paclitaxel loaded polymeric micelle and cisplatin versus paclitaxel and cisplatin in advanced non-small cell lung cancer (NSCLC). Ann Oncol 2010, 21:136-137.
11. Polymer micelles for drug delivery 2010, CRC Press-Taylor & Francis Group. S. Kim, K. Park (Eds.).
12. Chen Z. Small-molecule delivery by nanoparticles for anticancer therapy. Trends Mol Med 2010, 16(12):594-602.
13. Belting M. Macromolecular drug delivery: methods and protocols 2009, 1-10. Humana Press, New York, NY.
14. Zhang L., Gu F.X., Chan J.M., Wang A.Z., Langer R.S., Farokhzad O.C. Nanoparticles in medicine: therapeutic applications and developments. Clin Pharmacol Ther 2007, 83(5):761-769.
15. Chan D.P.Y., Owen S.C., Shoichet M.S. Double click: dual functionalized polymeric micelles with antibodies and peptides. Bioconjug Chem 2013, 24(1):105-113.
16. Juliano R.L., Ming X., Nakagawa O. The chemistry and biology of oligonucleotide conjugates. Acc Chem Res 2012, 45(7):1067-1076.
17. Rettig G.R., Behlke M.A. Progress toward invivo use of siRNAs-II. Mol Ther 2012, 20(3):483-512.
18. Juliano R., Alam M.R., Dixit V., Kang H. Mechanisms and strategies for effective delivery of antisense and siRNA oligonucleotides. Nucleic Acids Res 2008, 36(12):4158-4171.
19. Yuan X.D., Naguib S., Wu Z.Q. Recent advances of siRNA delivery by nanoparticles. Expert Opin Drug Deliv 2011, 8(4):521-536.
20. Zhang C.L., Tang N., Liu X.J., Liang W., Xu W., Torchilin V.P. siRNA-containing liposomes modified with polyarginine effectively silence the targeted gene. JControl Release 2006, 112(2):229-239.
21. Dalby B., Cates S., Harris A., Ohki E.C., Tilkins M.L., Price P.J., et al. Advanced transfection with Lipofectamine 2000 reagent: primary neurons, siRNA, and high-throughput applications. Methods 2004, 33(2):95-103.
22. Kim H.K., Davaa E., Myung C.S., Park J.S. Enhanced siRNA delivery using cationic liposomes with new polyarginine-conjugated PEG-lipid. Int J Pharm 2010, 392(1-2):141-147.
23. Guo J.F., Fisher K.A., Darcy R., Cryan J.F., O'Driscoll C. Therapeutic targeting in thesilent era: advances in non-viral siRNA delivery. Mol Biosyst 2010, 6(7):1143-1161.
24. Ozpolat B., Sood A.K., Lopez-Berestein G. Nanomedicine based approaches for the delivery of siRNA in cancer. JIntern Med 2010, 267(1):44-53.
25. Howard K.A., Rahbek U.L., Liu X.D., Damgaard C.K., Glud S.Z., Andersen M.O., et al. RNA interference invitro and invivo using a chitosan/siRNA nanoparticle system. Mol Ther 2006, 14(4):476-484.
26. Miyata K., Nishiyama N., Kataoka K. Rational design of smart supramolecular assemblies for gene delivery: chemical challenges in the creation of artificial viruses. Chem Soc Rev 2012, 41(7):2562-2574.
27. Cutler J.I., Zhang K., Zheng D., Auyeung E., Prigodich A.E., Mirkin C.A. Polyvalent nucleic acid nanostructures. JAm Chem Soc 2011, 133(24):9254-9257.
28. Felber A.E., Castagner B., Elsabahy M., Deleavey G.F., Damha M.J., Leroux J.C. siRNA nanocarriers based on methacrylic acid copolymers. JControl Release 2011, 152(1):159-167.
29. Elsabahy M., Wazen N., Bayo-Puxan N., Deleavey G., Servant M., Damha M.J., et al. Delivery of nucleic acids through the controlled disassembly of multifunctional nanocomplexes. Adv Funct Mater 2009, 19(24):3862-3867.
30. Patel P.C., Hao L.L., Yeung W.S.A., Mirkin C.A. Duplex end breathing determines serum stability and intracellular potency of siRNA-Au NPs. Mol Pharm 2011, 8(4):1285-1291.
31. Lee H., Lytton-Jean A.K.R., Chen Y., Love K.T., Park A.I., Karagiannis E.D., et al. Molecularly self-assembled nucleic acid nanoparticles for targeted invivo siRNA delivery. Nat Nanotechnol 2012, 7(6):389-393.
32. Jin H.L., Lovell J.F., Chen J., Lin Q.Y., Ding L., Ng K.K., et al. Mechanistic insights intoLDL nanoparticle-mediated siRNA delivery. Bioconjug Chem 2012, 23(1):33-41.
33. Dowler T., Bergeron D., Tedeschi A.L., Paquet L., Ferrari N., Damha M.J. Improvements in siRNA properties mediated by 2'-deoxy-2'-fluoro-beta-d-arabinonucleic acid (FANA). Nucleic Acids Res 2006, 34(6):1669-1675.
34. Deleavey G.F., Watts J.K., Alain T., Robert F., Kalota A., Aishwarya V., et al. Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing. Nucleic Acids Res 2010, 38(13):4547-4557.
35. Min K.L., Viazovkina E., Galarneau A., Parniak M.A., Damha M.J. Oligonucleotides comprised of alternating 2'-deoxy-2'-fluoro-beta-d-arabinonucleosides and d-2'-deoxyribonucleosides (2' F-ANA/DNA 'altimers') induce efficient RNA cleavage mediated by RNase H. Bioorg Med Chem Lett 2002, 12(18):2651-2654.
36. Watts J.K., Damha M.J. 2' F-arabinonucleic acids (2' F-ANA) - history, properties, and new frontiers. Can J Chem revue Canadienne De Chim 2008, 86(7):641-656.
37. Eckstein F. Nucleoside phophorothioates. JAm Chem Soc 1966, 88(18):4292-4294.
38. Eckstein F.A. Dinucleoside phosphorothioate. Tetrahedron Lett 1967, (13):1157-1160.
39. Eckstein F. Phosphorothioate oligodeoxynucleotides: what is their origin and whatis unique about them?. Antisense Nucleic Acid Drug Dev 2000, 10(2):117-121.
40. Watts J.K., Katolik A., Viladoms J., Damha M.J. Studies on the hydrolytic stability of 2'-fluoroarabinonucleic acid (2' F-ANA). Org Biomol Chem 2009, 7(9):1904-1910.
41. Damha M.J., Wilds C.J., Noronha A., Brukner I., Borkow G., Arion D., et al. Hybrids of RNA and arabinonucleic acids (ANA and 2' F-ANA) are substrates of ribonuclease H. JAm Chem Soc 1998, 120(49):12976-12977.
42. Souleimanian N., Deleavey G.F., Soifer H., Wang S., Tiemann K., Damha M.J., et al. Antisense 2[prime]-deoxy, 2[prime]-fluoroarabino nucleic acid (2[prime]F-ANA) oligonucleotides:Invitro gymnotic silencers of gene expression whose potency is enhanced by fatty acids. Mol Ther Nucleic Acids 2012, 1:e43.
43. Kolb H.C., Finn M.G., Sharpless K.B. Click chemistry: diverse chemical function from a few good reactions. Angew Chem Int Edit 2001, 40(11):2004-2021.
44. Shi M., Wosnick J.H., Ho K., Keating A., Shoichet M.S. Immuno-polymeric nanoparticlesby Diels-Alder chemistry. Angew Chem Int Edit 2007, 46(32):6126-6131.
45. Lu J., Shoichet M.S. Self-assembled polymeric nanoparticles of organocatalytic copolymerizated d, l-lactide and 2-methyl 2-carboxytrimethylene carbonate. Macromolecules 2010, 43(11):4943-4953.
46. Lu J., Shi M., Shoichet M.S. Click chemistry functionalized polymeric nanoparticles target corneal epithelial cells through RGD-cell surface receptors. Bioconjug Chem 2009, 20(1):87-94.
47. Shi M., Shoichet M.S. Furan-functionalized co-polymers for targeted drug delivery: characterization, self-assembly and drug encapsulation. JBiomat Sci-polym E 2008, 19(9):1143-1157.
48. 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.
49. Meldal M., Tornoe C.W. Cu-catalyzed azide-alkyne cycloaddition. Chem Rev 2008, 108(8):2952-3015.
50. Foster D.J., Barros S., Duncan R., Shaikh S., Cantley W., Dell A., et al. Comprehensive evaluation of canonical versus Dicer-substrate siRNA invitro and invivo. RNA 2012, 18(3):557-568.
51. Ho K., Lapitsky Y., Shi M., Shoichet M.S. Tunable immunonanoparticle binding to cancer cells: thermodynamic analysis of targeted drug delivery vehicles. Soft Matter 2009, 5(5):1074-1080.
52. Shi M., Ho K., Keating A., Shoichet M.S. Doxorubicin-conjugated immuno-nanoparticles for intracellular anticancer drug delivery. Adv Funct Mater 2009, 19(11):1689-1696.
53. Yoo H.S., Park T.G. Folate-receptor-targeted delivery of doxorubicin nano-aggregates stabilized by doxorubicin-PEG-folate conjugate. JControl Release 2004, 100(2):247-256.
54. Park J.W., Hong K., Carter P., Asgari H., Guo L.Y., Keller G.A., et al. Development of anti-P185(Her2) immunoliposomes for cancer-therapy. PNatl Acad Sci USA 1995, 92(5):1327-1331.
55. Alam M.R., Dixit V., Kang H.M., Li Z.B., Chen X.Y., Trejo J., et al. Intracellular delivery of an anionic antisense oligonucleotide via receptor-mediated endocytosis. Nucleic Acids Res 2008, 36(8):2764-2776.
56. Panyam J., Zhou W.Z., Prabha S., Sahoo S.K., Labhasetwar V. Rapid endo-lysosomal escape of poly(dl-lactide-co-glycolide) nanoparticles: implications for drug and gene delivery. FASEB J 2002, 16(10).
57. Tang Y., Li Y.B., Wang B., Lin R.Y., van Dongen M., Zurcher D.M., et al. Efficient invitro siRNA delivery and intramuscular gene silencing using PEG-modified PAMAM dendrimers. Mol Pharmaceut 2012, 9(6):1812-1821.
58. Lu J., Owen S.C., Shoichet M.S. Stability of self-assembled polymeric micelles in serum. Macromolecules 2011, 44(15):6002-6008.
59. Kow S.C., McCarroll J., Valade D., Boyer C., Dwarte T., Davis T.P., et al. Dicer-labile PEG conjugates for siRNA delivery. Biomacromolecules 2011, 12(12):4301-4310.
60. Vermeulen A., Behlen L., Reynolds A., Wolfson A., Marshall W.S., Karpilow J., et al. The contributions of dsRNA structure to dicer specificity and efficiency. RNA 2005, 11(5):674-682.
61. Singh N., Agrawal A., Leung A.K.L., Sharp P.A., Bhatia S.N. Effect of nanoparticle conjugation on gene silencing by RNA interference. JAm Chem Soc 2010, 132(24):8241-8243.