Arginine-engrafted biodegradable polymer for the systemic delivery of therapeutic siRNA

Biomaterials

Volumn 33, Issue 5, 2012, Pages 1640-1650

  Beloor, Jagadish ^a   Choi, Chang Seon ^a   Nam, Hye Yeong ^b   Park, Minsun ^a   Kim, Sung Hwa ^a   Jackson, Andrew ^c   Lee, Kuen Yong ^a   Kim, Sung Wan ^b   Kumar, Priti ^c   Lee, Sang Kyung ^a  

^a Hanyang University   (South Korea)

^b UNIVERSITY OF UTAH   (United States)

^c YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

Arginine engrafted polymer; Cancer therapeutics; Cationic carriers; Combinatorial RNAi; SiRNA

Indexed keywords

ARGININE RESIDUE; BINDING PROPERTIES; BODY WEIGHT; CANCER THERAPEUTICS; COMBINATORIAL RNAI; DEOXYRIBONUCLEIC ACIDS; DISULPHIDE BONDS; GENE DELIVERY AGENTS; INTRACELLULAR COMPARTMENTS; PLASMID DNA; POLYCATIONS; RAPID RELEASE; SIRNA; SIRNA DELIVERY; SMALL INTERFERING RNA (SIRNA); SOLID CANCER; SUPERCOILED; SYSTEMIC ADMINISTRATION; TUMOR TISSUES;

ARGININE; BINDING ENERGY; BIODEGRADABLE POLYMERS; DISEASES; DNA; GENE THERAPY; GENE TRANSFER; ORGANIC COMPOUNDS; RNA; TUMORS;

FUNCTIONAL POLYMERS;

ARGININE; BIOPOLYMER; CELL MEMBRANE MARKER; DISULFIDE; DRUG CARRIER; MYC PROTEIN; NANOPARTICLE; POLY(DISULFIDE AMINE); POLYAMINE DERIVATIVE; POLYCATION; PROTEIN BCL 2; SMALL INTERFERING RNA; UNCLASSIFIED DRUG; VASCULOTROPIN;

ADVANCED CANCER; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTINEOPLASTIC ACTIVITY; ARTICLE; CANCER GENE THERAPY; CANCER TISSUE; CELL PROLIFERATION; COMBINATORIAL CHEMISTRY; COMPLEX FORMATION; CONTROLLED STUDY; CYTOTOXICITY; DISULFIDE BOND; DRUG ACCUMULATION; DRUG DOSE REGIMEN; DRUG EFFICACY; DRUG FORMULATION; DRUG PENETRATION; DRUG RELEASE; DRUG TARGETING; GENE DELIVERY SYSTEM; MALE; MELANOMA; MOLECULAR STABILITY; MOUSE; NONHUMAN; PARTICLE SIZE; PRIORITY JOURNAL; RNA BINDING; RNA INTERFERENCE; SOLID TUMOR; ZETA POTENTIAL;

ANIMALS; ARGININE; BIOCOMPATIBLE MATERIALS; CELL LINE, TUMOR; CELL PROLIFERATION; DISEASE MODELS, ANIMAL; GENE KNOCKDOWN TECHNIQUES; GENE SILENCING; GENE TRANSFER TECHNIQUES; MELANOMA; MICE; PHYSICOCHEMICAL PHENOMENA; POLYMERS; RNA, SMALL INTERFERING;

EID: 83355177991     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2011.11.008     Document Type: Article

References (55)

1. Pecot C.V., Calin G.A., Coleman R.L., Lopez-Berestein G., Sood A.K. RNA interference in the clinic: challenges and future directions. Nat Rev Cancer 2011, 11(1):59-67.
2. Chen S.-H., Zhaori G. Potential clinical applications of siRNA technique: benefits and limitations. Eur J Clin Invest 2011, 41(2):221-232.
3. Burckbuchler V., Wintgens V., Lecomte S., Percot A., Leborgne C., Danos O., et al. DNA compaction into new DNA vectors based on cyclodextrin polymer: surface enhanced Raman spectroscopy characterization. Biopolymers 2006, 81(5):360-370.
4. 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 2007, 121(1-2):64-73.
5. Spagnou S., Miller A.D., Keller M. Lipidic carriers of siRNA: differences in the formulation, cellular uptake, and delivery with plasmid DNA. Biochemistry 2004, 43(42):13348-13356.
6. Davis M. Nanoparticles for systemic medicines and imaging agents. Nanotechnology, Law Business 2006, 3(3):255.
7. Matsumura Y., Maeda H. A new concept for macromolecular therapeutics in cancer chemotherapy: mechanism of tumoritropic accumulation of proteins and the antitumor agent smancs. Cancer Res 1986, 46(12 Part 1):6387-6392.
8. Bundgaard M. Transport pathways in capillaries-in search of pores. Annu Rev Physiol 1980, 42(1):325-336.
9. Devi G.R. siRNA-based approaches in cancer therapy. Cancer Gene Ther 2006, 13(9):819-829.
10. Pirollo K.F., Rait A., Zhou Q., Hwang S.H., Dagata J.A., Zon G., et al. Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system. Cancer Res 2007, 67(7):2938-2943.
11. Murata N., Takashima Y., Toyoshima K., Yamamoto M., Okada H. Anti-tumor effects of anti-VEGF siRNA encapsulated with PLGA microspheres in mice. J Control Release 2008, 126(3):246-254.
12. Al-Abd A.M., Lee S.H., Kim S.H., Cha J.-H., Park T.G., Lee S.J., et al. Penetration and efficacy of VEGF siRNA using polyelectrolyte complex micelles in a human solid tumor model in-vitro. J Control Release 2009, 137(2):130-135.
13. Park K., Lee M.-Y., Kim K.S., Hahn S.K. Target specific tumor treatment by VEGF siRNA complexed with reducible polyethyleneimine-hyaluronic acid conjugate. Biomaterials 2010, 31(19):5258-5265.
14. Ofek P., Fischer W., Calderón M., Haag R., Satchi-Fainaro R. In vivo delivery of small interfering RNA to tumors and their vasculature by novel dendritic nanocarriers. FASEB J 2010, 24(9):3122-3134.
15. Howard K.A., Rahbek U.L., Liu X., Damgaard C.K., Glud S.Z., Andersen M.O., et al. RNA interference in vitro and in vivo using a chitosan/siRNA nanoparticle system. Mol Ther 2006, 14(4):476-484.
16. Minakuchi Y., Takeshita F., Kosaka N., Sasaki H., Yamamoto Y., Kouno M., et al. Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res 2004, 32(13):e109.
17. Davis M.E., Zuckerman J.E., Choi C.H.J., Seligson D., Tolcher A., Alabi C.A., et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 2010, 464(7291):1067-1070.
18. Kumar P., Wu H., McBride J.L., Jung K.-E., Hee Kim M., Davidson B.L., et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 2007, 448(7149):39-43.
19. Kumar P., Ban H.-S., Kim S.-S., Wu H., Pearson T., Greiner D.L., et al. T Cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 2008, 134(4):577-586.
20. Kenneth Alan H. Delivery of RNA interference therapeutics using polycation-based nanoparticles. Adv Drug Deliv Rev 2009, 61(9):710-720.
21. Kang H.C., Lee M., Bae Y.H. Polymeric gene carriers. Crit Rev Eukaryot Gene Expr 2005, 15(4):317-342.
22. Kim T.-i, Ou M., Lee M., Kim S.W. Arginine-grafted bioreducible poly(disulfide amine) for gene delivery systems. Biomaterials 2009, 30(4):658-664.
23. Willett C.G., Boucher Y., Duda D.G., di Tomaso E., Munn L.L., Tong R.T., et al. Surrogate markers for antiangiogenic therapy and dose-limiting toxicities for bevacizumab with radiation and chemotherapy: continued experience of a phase I trial in rectal cancer patients. J Clin Oncol 2005, 23(31):8136-8139.
24. Willett C.G., Boucher Y., di Tomaso E., Duda D.G., Munn L.L., Tong R.T., et al. Direct evidence that the VEGF-specific antibody bevacizumab has antivascular effects in human rectal cancer. Nat Med 2004, 10(2):145-147.
25. Del Poeta G., Venditti A., Del Principe M.I., Maurillo L., Buccisano F., Tamburini A., et al. Amount of spontaneous apoptosis detected by Bax/Bcl-2 ratio predicts outcome in acute myeloid leukemia (AML). Blood 2003, 101(6):2125-2131.
26. Minn A., Rudin C., Boise L., Thompson C. Expression of bcl-xL can confer a multidrug resistance phenotype. Blood 1995, 86(5):1903-1910.
27. Yoshino T., Shiina H., Urakami S., Kikuno N., Yoneda T., Shigeno K., et al. Bcl-2 expression as a predictive marker of hormone-refractory prostate cancer treated with taxane-based chemotherapy. Clin Cancer Res 2006, 12(20):6116-6124.
28. Chenevix-Trench G., Martin N.G., Ellem K.A. Gene expression in melanoma cell lines and cultured melanocytes: correlation between levels of c-src-1, c-myc and p53. Oncogene 1990, 5(8):1187-1193.
29. Grover R., Ross D.A., Richman P.I., Robinson B., Wilson G.D. C-myc oncogene expression in human melanoma and its relationship with tumour antigenicity. Eur J Surg Oncol 1996, 22(4):342-346.
30. Li S.-D., Chono S., Huang L. Efficient oncogene silencing and metastasis inhibition via systemic delivery of siRNA. Mol Ther 2008, 16(5):942-946.
31. Sonoke S., Ueda T., Fujiwara K., Sato Y., Takagaki K., Hirabayashi K., et al. Tumor regression in mice by delivery of Bcl-2 small interfering RNA with pegylated cationic liposomes. Cancer Res 2008, 68(21):8843-8851.
32. Pastorino F., Brignole C., Marimpietri D., Pagnan G., Morando A., Ribatti D., et al. Targeted liposomal c-myc antisense oligodeoxynucleotides induce apoptosis and inhibit tumor growth and metastases in human melanoma models. Clin Cancer Res 2003, 9(12):4595-4605.
33. Poeck H., Besch R., Maihoefer C., Renn M., Tormo D., Morskaya S.S., et al. 5'-triphosphate-siRNA: turning gene silencing and Rig-I activation against melanoma. Nat Med 2008, 14(11):1256-1263.
34. Kawasaki H., Mocarski E.S., Kosugi I., Tsutsui Y. Cyclosporine Inhibits Mouse Cytomegalovirus infection via a cyclophilin-dependent pathway specifically in neural stem/progenitor cells. J Virol 2007, 81(17):9013-9023.
35. Kim T.-I., Baek J.-U., Zhe Bai C., Park J.-S. Arginine-conjugated polypropylenimine dendrimer as a non-toxic and efficient gene delivery carrier. Biomaterials 2007, 28(11):2061-2067.
36. Bachelder R.E., Wendt M.A., Mercurio A.M. Vascular endothelial growth factor promotes breast carcinoma invasion in an autocrine manner by regulating the chemokine receptor CXCR4. Cancer Res 2002, 62(24):7203-7206.
37. Kranz A., Mattfeldt T., Waltenberger J. Molecular mediators of tumor angiogenesis: enhanced expression and activation of vascular endothelial growth factor receptor KDR in primary breast cancer. Int J Cancer Suppl 1999, 84(3):293-298.
38. Won Y.-W., Yoon S.-M., Lee K.-M., Kim Y.-H. Poly(oligo-D-arginine) with internal disulfide linkages as a cytoplasm-sensitive carrier for siRNA delivery. Mol Ther 2011, 19(2):372-380.
39. Furuhata M., Kawakami H., Toma K., Hattori Y., Maitani Y. Design, synthesis and gene delivery efficiency of novel oligo-arginine-linked PEG-lipids: effect of oligo-arginine length. Int J Pharm 2006, 316(1-2):109-116.
40. Hoon Jeong J., Christensen L.V., Yockman J.W., Zhong Z., Engbersen J.F.J., Jong Kim W., et al. Reducible poly(amido ethylenimine) directed to enhance RNA interference. Biomaterials 2007, 28(10):1912-1917.
41. McKenzie D.L., Kwok K.Y., Rice K.G. A potent new class of reductively activated peptide gene delivery agents. J Biol Chem 2000, 275(14):9970-9977.
42. Oupický D., Parker A.L., Seymour L.W. Laterally stabilized complexes of DNA with linear reducible polycations: strategy for triggered intracellular activation of DNA delivery dectors. J Am Chem Soc 2001, 124(1):8-9.
43. Manickam D.S., Oupický D. Multiblock reducible copolypeptides containing histidine-rich and nuclear localization sequences for gene delivery. Bioconjug Chem 2006, 17(6):1395-1403.
44. Lin C., Zhong Z., Lok M.C., Jiang X., Hennink W.E., Feijen J., et al. Novel Bioreducible poly(amido amine)s for highly efficient gene delivery. Bioconjug Chem 2006, 18(1):138-145.
45. Schiffelers R.M., Storm G. ICS-283: a system for targeted intravenous delivery of siRNA. Expert Opin Drug Deliv 2006, 3(3):445-454.
46. Strumberg D. First-in-human phase I study of Atu027, a liposomal small interfering RNA formulation, targeting protein kinase N3 (PKN3) in patients with advanced solid tumors. ASCO Annual Meeting 2011, ASCO, Chicago. U. Traugott, C. Vank, A. Santel, O. Keil, K. Giese, J. Kaufmann, J. Drevs (Eds.).
47. Gollob J. Interim safety and pharmacodynamic results for ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement. ASCO Annual Meeting 2010, ASCO, Chicago. G. Shapiro, P. LoRusso, B.J. Dezube, J. Heymach, J. Cehelsky, R. Falzone, A. Vaishnaw, H.A. Burris (Eds.).
48. Jackson A.L., Linsley P.S. Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat Rev Drug Discov 2010, 9(1):57-67.
49. Love K.T., Mahon K.P., Levins C.G., Whitehead K.A., Querbes W., Dorkin J.R., et al. Lipid-like materials for low-dose, in vivo gene silencing. Proc Natl Acad Sci U S A 2010, 107(5):1864-1869.
50. Wu S.Y., Singhania A., Burgess M., Putral L.N., Kirkpatrick C., Davies N.M., et al. Systemic delivery of E6/7 siRNA using novel lipidic particles and its application with cisplatin in cervical cancer mouse models. Gene Ther 2011, 18(1):14-22.
51. Alshamsan A., Hamdy S., Samuel J., El-Kadi A.O., Lavasanifar A., Uludaĝ H. The induction of tumor apoptosis in B16 melanoma following STAT3 siRNA delivery with a lipid-substituted polyethylenimine. Biomaterials 2010, 31(6):1420-1428.
52. Kim W.J., Christensen L.V., Jo S., Yockman J.W., Jeong J.H., Kim Y.H., et al. Cholesteryl oligoarginine delivering vascular endothelial growth factor siRNA effetively inhibits tumor growth in colon adenocarcinoma. Mol Ther 2006, 14(3):343-350.
53. Lee S.-K., Kumar P. Conditional RNAi: towards a silent gene therapy. Adv Drug Deliv Rev 2009, 61(7-8):650-664.
54. Lv H., Zhang S., Wang B., Cui S., Yan J. Toxicity of cationic lipids and cationic polymers in gene delivery. J Control Release 2006, 114(1):100-109.
55. Nam H.Y., Kim J., Kim S., Yockman J.W., Kim S.W., Bull D.A. Cell penetrating peptide conjugated bioreducible polymer for siRNA delivery. Biomaterials 2011, 32(22):5213-5222.