Nanomedicines based on recombinant fusion proteins for targeting therapeutic siRNA oligonucleotides

Therapeutic Delivery

Volumn 2, Issue 7, 2011, Pages 891-905

  Winkler, Johannes ^a  

^a UNIVERSITY OF VIENNA   (Austria)

Author keywords

[No Author keywords available]

Indexed keywords

ALN RSV 01; ALN TTR01; ALN VSP; ANKYRIN; ANTISENSE OLIGONUCLEOTIDE; ATU 072; BEVASIRANIB; CALAA 01; DENDRIMER; DNA VACCINE; HYBRID PROTEIN; PF 655; PHOSPHOROTHIOIC ACID; POLYAMIDOAMINE; POLYMER; QPI 1002; QPI 1007; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; SIRNA 027; SMALL INTERFERING RNA; TD 101; TKM APOLIPOPROTEIN B; TKM POLO LIKE KINASE 1; UNCLASSIFIED DRUG;

AMYLOIDOSIS; CONGENITAL PACHYONYCHIA; DRUG CLEARANCE; DRUG ELIMINATION; DRUG EXTRAVASATION; DRUG INDUSTRY; DRUG STABILITY; ENDOCYTOSIS; GENE DELIVERY SYSTEM; GENE SILENCING; GENE TARGETING; GENE THERAPY; HUMAN; HYPERCHOLESTEROLEMIA; IN VITRO STUDY; IN VIVO STUDY; KIDNEY INJURY; NANOMEDICINE; NONHUMAN; OPTIC NERVE DISEASE; PRIORITY JOURNAL; RESPIRATORY SYNCYTIAL VIRUS INFECTION; RETINA MACULA AGE RELATED DEGENERATION; REVIEW; RNA INTERFERENCE; SOLID TUMOR; TOXICITY;

EID: 79961203099     PISSN: 20415990     EISSN: 20416008     Source Type: Journal    
DOI: 10.4155/tde.11.56     Document Type: Review

References (93)

1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in caenorhabditis elegans. Nature 391(6669), 806-811 (1998). (Pubitemid 28099681)
2. Whitehead KA, Langer R, Anderson DG. Knocking down barriers: Advances in siRNA delivery. Nat. Rev. Drug Discov. 8(2), 129-138 (2009).
3. Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 457(7228), 426-433 (2009).
4. Lares MR, Rossi JJ, Ouellet DL. RNAi and small interfering RNAs in human disease therapeutic applications. Trends Biotechnol. 28(11), 570-579 (2010).
5. Wang J, Lu Z, Wientjes M, Au J. Delivery of siRNA therapeutics: barriers and carriers. AAPS J. 12(4), 492-503 (2010).
6. Corey DR. RNA learns from antisense. Nature Chem. Biol. 3(1), 8-11 (2007). (Pubitemid 46005687)
7. Shukla S, Sumaria CS, Pradeepkumar PI. Exploring chemical modifications for siRNA therapeutics: A structural and functional outlook. ChemMedChem 5(3), 328-349 (2010).
8. Bhattacharjee H, Balabathula P, Wood GC. Targeted nanoparticulate drug-delivery systems for treatment of solid tumors: a review. Therapeutic Delivery 1(5), 713-734 (2010).
9. Wang M, Thanou M. Targeting nanoparticles to cancer. Pharmacol. Res. 62(2), 90-99 (2010).
10. Davis ME. The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: From concept to clinic. Mol. Pharm. 6(3), 659-668 (2009).
11. Davis ME, Zuckerman JE, Choi CHJ et al. Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles. Nature 464(7291), 1067-1070 (2010).
12. Chiu S-J, Ueno NT, Lee RJ. Tumor-targeted gene delivery via anti-her2 antibody (trastuzumab, herceptin®) conjugated polyethylenimine. J. Control. Release 97(2), 357-369 (2004). (Pubitemid 38759492)
13. Kunath K, Merdan T, Hegener O, Haberlein H, Kissel T. Integrin targeting using RGD-PEI conjugates for in vitro gene transfer. J. Gene Med. 5(7), 588-599 (2003). (Pubitemid 40310402)
14. Watkins J, Marsh A, Taylor PC, Singer DR. Personalized medicine: The impact on chemistry. Therapeutic Delivery 1(5), 651-665 (2010).
15. Caravella J, Lugovskoy A. Design of next-generation protein therapeutics. Curr. Opin. Chem. Biol. 14(4), 520-528 (2010).
16. Chen SY, Zani C, Khouri Y, Marasco WA. Design of a genetic immunotoxin to eliminate toxin immunogenicity. Gene Ther. 2(2), 116-123 (1995).
17. Li X, Stuckert P, Bosch I, Marks JD, Marasco WA. Single-chain antibody-mediated gene delivery into erbb2-positive human breast cancer cells. Cancer Gene Ther. 8(8), 555-565 (2001). (Pubitemid 32786558)
18. Song E, Zhu P, Lee SK et al. Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors. Nat. Biotechnol. 23(6), 709-717. (2005). (Pubitemid 41716361)
19. Peer D, Zhu P, Carman CV, Lieberman J, Shimaoka M. Selective gene silencing in activated leukocytes by targeting siRNAs to the integrin lymphocyte function-associated antigen-1. Proc. Natl Acad. Sci. USA 104(10), 4095-4100 (2007). (Pubitemid 47181584)
20. Wen WH, Liu JY, Qin WJ et al. Targeted inhibition of hbv gene expression by single-chain antibody mediated small interfering RNA delivery. Hepatol. 46(1), 84-94 (2007).
21. Kumar P, Ban HS, Kim SS et al. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 134(4), 577-586 (2008).
22. Lee Y, Kim K, Kim J et al. Leukemia-specific siRNA delivery by immunonanoplexes consisting of anti-JL1 minibody conjugated to oligo-9 Arg-peptides. Mol. Cells 29(5), 457-462 (2010).
23. Zhang T, Wang C-Y, Zhang W et al. Generation and characterization of a fusion protein of single-chain fragment variable antibody against hemagglutinin antigen of avian influenza virus and truncated protamine. Vaccine 28(23), 3949-3955 (2010).
24. Li S, Tan Y, Viroonchatapan E, Pitt BR, Huang L. Targeted gene delivery to pulmonary endothelium by anti-PECAM antibody. Am. J. Physiol.-Lung C. 278(3), L504-L511 (2000). (Pubitemid 30152507)
25. Stumpp MT, Binz HK, Amstutz P. DARPins: a new generation of protein therapeutics. Drug Discov. Today 13(15-16), 695-701 (2008).
26. Zahnd C, Kawe M, Stumpp MT et al. Efficient tumor targeting with high-affinity designed ankyrin repeat proteins: Effects of affinity and molecular size. Cancer Res. 70(4), 1595-1605 (2010).
27. Binz HK, Amstutz P, Kohl A et al. High-affinity binders selected from designed ankyrin repeat protein libraries. Nat. Biotechnol. 22(5), 575-582 (2004). (Pubitemid 38590636)
28. Kramer MA, Wetzel SK, Plückthun A, Mittl PRE, Grütter MG. Structural determinants for improved stability of designed ankyrin repeat proteins with a redesigned c-capping module. J. Mol. Biol. 404(3), 381-391 (2010).
29. Steiner D, Forrer P, Plückthun A. Efficient selection of Darpins with sub-nanomolar affinities using srp phage display. J. Mol. Biol. 382(5), 1211-1227 (2008).
30. Dreier B, Pluckthun A. Ribosome display: a technology for selecting and evolving proteins from large libraries. Methods Mol. Biol. 687, 283-306 (2011).
31. Winkler J, Martin-Killias P, Pluckthun A, Zangemeister-Wittke U. EpCAM-targeted delivery of nanocomplexed siRNA to tumor cells with designed ankyrin repeat proteins. Mol. Cancer Ther. 8(9), 2674-2683 (2009).
32. Wagner E, Zenke M, Cotten M, Beug H, Birnstiel ML. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl Acad. Sci. USA 87(9), 3410-3414 (1990).
33. Zenke M, Steinlein P, Wagner E, Cotten M, Beug H, Birnstiel ML. Receptor-mediated endocytosis of transferrin-polycation conjugates: An efficient way to introduce DNA into hematopoietic cells. Proc. Natl Acad. Sci. USA 87(10), 3655-3659 (1990).
34. Citro G, Perrotti D, Cucco C et al. Inhibition of leukemia cell proliferation by receptor-mediated uptake of c-myb antisense oligodeoxynucleotides. Proc. Natl Acad. Sci. USA 89(15), 7031-7035 (1992).
35. Henke S, Rohmann A, Bertling WM, Dingermann T, Zimmer A. Enhanced in vitro oligonucleotide and plasmid DNA transport by vp1 virus-like particles. Pharm. Res. 17(9), 1062-1070 (2000).
36. Ochiya T, Takahama Y, Nagahara S et al. New delivery system for plasmid DNA in vivo using atelocollagen as a carrier material: the minipellet. Nat. Med. 5(6), 707-710 (1999). (Pubitemid 29289437)
37. Minakuchi Y, Takeshita F, Kosaka N et al. Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res. 32(13), E109 (2004).
38. Tasaki M, Shimada K, Kimura H, Tsujikawa K, Konishi N. Alkbh3, a human alkb homologue, contributes to cell survival in human non-small-cell lung cancer. Br. J. Cancer 104(4), 700-706 (2011).
39. Subramanya S, Kim SS, Abraham S et al. Targeted delivery of small interfering RNA to human dendritic cells to suppress dengue virus infection and associated proinflammatory cytokine production. J. Virol. 84(5), 2490-2501 (2010).
40. Kumar P, Wu H, Mcbride JL et al. Transvascular delivery of small interfering RNA to the central nervous system. Nature 448(7149), 39-43 (2007). (Pubitemid 47037001)
41. Kim SS, Ye C, Kumar P et al. Targeted delivery of siRNA to macrophages for anti-inflammatory treatment. Mol. Ther. 18(5), 993-1001 (2010).
42. Shukla R, Thomas TP, Peters J, Kotlyar A, Myc A, Baker JJR. Tumor angiogenic vasculature targeting with pamam dendrimer-RGD conjugates. Chem. Commun. (46), 5739-5741 (2005). (Pubitemid 43028741)
43. Waite CL, Roth CM. Pamam-RGD conjugates enhance siRNA delivery through a multicellular spheroid model of malignant glioma. Bioconjug. Chem. 20(10), 1908-1916 (2009).
44. Dharap SS, Wang Y, Chandna P et al. Tumor-specific targeting of an anticancer drug delivery system by LHRH peptide. Proc. Natl Acad. Sci. USA 102(36), 12962-12967 (2005). (Pubitemid 41279472)
45. Lindberg S, Copolovici DM, Langel Ü. Therapeutic delivery opportunities, obstacles and applications for cell-penetrating peptides. Ther. Deliv. 2(1), 71-82 (2011).
46. Said Hassane F, Saleh AF, Abes R, Gait MJ, Lebleu B. Cell penetrating peptides: overview and applications to the delivery of oligonucleotides. Cell. Mol. Life Sci. 67(5), 715-726 (2010).
47. Fotin-Mleczek M, Fischer R, Brock R. Endocytosis and cationic cell-penetrating peptide - a merger of concepts and methods. Curr. Pharm. Des. 11(28), 3613-3628 (2005). (Pubitemid 41630988)
48. Gump JM, June RK, Dowdy SF. Revised role of glycosaminoglycans in TAT protein transduction domain-mediated cellular transduction. J. Biol. Chem. 285(2), 1500-1507 (2010).
49. Arthanari Y, Pluen A, Rajendran R, Aojula H, Demonacos C. Delivery of therapeutic shRNA and siRNA by tat fusion peptide targeting BCR-ABL fusion gene in chronic myeloid leukemia cells. J. Control. Release 145(3), 272-280 (2010).
50. Saleh AF, Aojula H, Arthanari Y, Offerman S, Alkotaji M, Pluen A. Improved TAT-mediated plasmid DNA transfer by fusion to LK15 peptide. J. Control. Release 143(2), 233-242 (2010).
51. Leonetti JP, Degols G, Lebleu B. Biological activity of oligonucleotide-poly(l-lysine) conjugates: mechanism of cell uptake. Bioconjug. Chem. 1(2), 149-153 (1990).
52. Clarenc JP, Degols G, Leonetti JP, Milhaud P, Lebleu B. Delivery of antisense oligonucleotides by poly(l-lysine) conjugation and liposome encapsulation. Anticancer Drug Des. 8(1), 81-94 (1993). (Pubitemid 23119934)
53. Winkler J, Urban E, Noe CR. Oligonucleotides conjugated to short lysine chains. Bioconjug. Chem. 16(4), 1038-1044. (2005). (Pubitemid 41026191)
54. Winkler J, Gilbert M, Kocourková A, Stessl M, Noe CR. 2́-O-lysylaminohexyl oligonucleotides: Modifications for antisense and siRNA. ChemMedChem 3(1), 102-110 (2008).
55. Winkler J, Saadat K, Díaz Gavilán M, Urban E, Noe CR. Oligonucleotide polyamine conjugates: Influence of length and position of 2́-attached polyamines on duplex stability and antisense effect. Eur. J. Med. Chem. 44(2), 670-677 (2009).
56. Domenjoud L, Nussbaum G, Adham IM, Greeske G, Engel W. Genomic sequences of human protamines whose genes, PRM1 and PRM2, are clustered. Genomics 8(1), 127-133 (1990). (Pubitemid 20302285)
57. Chang LC, Lee HF, Yang Z, Yang VC. Low molecular weight protamine (LMWP) as nontoxic heparin/low molecular weight heparin antidote (i): preparation and characterization. AAPS PharmSci. 3(3), E17 (2001).
58. Junghans M, Kreuter J, Zimmer A. Phosphodiester and phosphorothioate oligonucleotide condensation and preparation of antisense nanoparticles. Biochim. Biophys. Acta 1544(1-2), 177-188 (2001). (Pubitemid 32121856)
59. Lochmann D, Weyermann J, Georgens C, Prassl R, Zimmer A. Albumin-protamine- oligonucleotide nanoparticles as a new antisense delivery system. Part 1: physicochemical characterization. Eur. J. Pharm. Biopharm. 59(3), 419-429 (2005). (Pubitemid 40342516)
60. Mayer G, Vogel V, Weyermann J et al. Oligonucleotide-protamine-albumin nanoparticles: Protamine sulfate causes drastic size reduction. J. Control. Release 106(1-2), 181-187 (2005). (Pubitemid 41140093)
61. Vogel V, Lochmann D, Weyermann J et al. Oligonucleotide-protamine-albumin nanoparticles: preparation, physical properties, and intracellular distribution. J. Control. Release 103(1), 99-111 (2005). (Pubitemid 40248565)
62. Jensen MG, Hansen M, Brock B, Rungby J. Differences between long-acting insulins for the treatment of Type 2 diabetes. Expert Opin. Pharmacother. 11(12), 2027-2035 (2010).
63. Chang LC, Liang JF, Lee HF, Lee LM, Yang VC. Low molecular weight protamine (LMWP) as nontoxic heparin/low molecular weight heparin antidote (ii): in vitro evaluation of efficacy and toxicity. AAPS PharmSci. 3(3), E18 (2001).
64. Lee LM, Chang LC, Wrobleski S, Wakefield TW, Yang VC. Low molecular weight protamine as nontoxic heparin/low molecular weight heparin antidote (iii): Preliminary in vivo evaluation of efficacy and toxicity using a canine model. AAPS PharmSci. 3(3), E19 (2001).
65. Choi Y-S, Lee JY, Suh JS et al. The systemic delivery of siRNAs by a cell-penetrating peptide, low molecular weight protamine. Biomaterials 31(6), 1429-1443 (2010).
66. Kim SW, Kim NY, Choi YB, Park SH, Yang JM, Shin S. RNA interference in vitro and in vivo using an arginine peptide/siRNA complex system. J. Control. Release 143(3), 335-343 (2010).
67. Eguchi A, Meade BR, Chang Y-C et al. Efficient siRNA delivery into primary cells by a peptide transduction domain-dsRNA binding domain fusion protein. Nat. Biotechnol. 27(6), 567-571 (2009).
68. Boussif O, Lezoualc'h F, Zanta MA et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl Acad. Sci. USA 92(16), 7297-7301 (1995).
69. Ravina M, Paolicelli P, Seijo B, Sanchez A. Knocking down gene expression with dendritic vectors. Mini Rev. Med. Chem. 10(1), 73-86 (2010).
70. Patil ML, Zhang M, Betigeri S, Taratula O, He H, Minko T. Surface-modified and internally cationic polyamidoamine dendrimers for efficient siRNA delivery. Bioconjug. Chem. 19(7), 1396-1403 (2008). (Pubitemid 352032339)
71. Yuan Q, Lee E, Yeudall WA, Yang H. Dendrimer-triglycine-EGF nanoparticles for tumor imaging and targeted nucleic acid and drug delivery. Oral Oncol. 46(9), 698-704 (2010).
72. Patil ML, Zhang M, Taratula O, Garbuzenko OB, He H, Minko T. Internally cationic polyamidoamine PAMAM-OH dendrimers for siRNA delivery: effect of the egree of quaternization and cancer targeting. Biomacromolecules 10(2), 258-266 (2009).
73. Kang H, Delong R, Fisher MH, Juliano RL. TAT-conjugated PAMAM dendrimers as delivery agents for antisense and siRNA oligonucleotides. Pharm. Res. 22(12), 2099-2106 (2005). (Pubitemid 41821589)
74. Dominska M, Dykxhoorn DM. Breaking down the barriers: siRNA delivery and endosome escape. J. Cell Sci. 123(8), 1183-1189 (2010).
75. Bramsen JB, Laursen MB, Nielsen AF et al. A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity. Nucleic Acids Res. 37(9), 2867-2881 (2009).
76. Cuesta ÁM, Sainz-Pastor N, Bonet J, Oliva B, Álvarez- Vallina L. Multivalent antibodies: when design surpasses evolution. Trends Biotechnol. 28(7), 355-362 (2010).
77. Danquah MK, Zhang XA, Mahato RI. Extravasation of polymeric nanomedicines across tumor vasculature. Adv. Drug Deliv. Rev. 63(8), 623-639 (2011) .
78. Wong C, Stylianopoulos T, Cui J et al. Multistage nanoparticle delivery system for deep penetration into tumor tissue. Proc. Natl. Acad. Sci. USA 108(6), 2426-2431 (2011).
79. Kumari S, Mg S, Mayor S. Endocytosis unplugged: multiple ways to enter the cell. Cell Res. 20(3), 256-275 (2010).
80. Hoekstra D, Rejman J, Wasungu L, Shi F, Zuhorn I. Gene delivery by cationic lipids: in and out of an endosome. Biochem. Soc. Trans. 35(1), 68-71 (2007).
81. Varkouhi AK, Scholte M, Storm G, Haisma HJ. Endosomal escape pathways for delivery of biologicals. J. Control. Release 151(3), 220-228 (2011).
82. Samuel-Abraham S, Leonard JN. Staying on message: design principles for controlling nonspecific responses to siRNA. FEBS J. 277(23), 4828-4836 (2010).
83. Walton SP, Wu M, Gredell JA, Chan C. Designing highly active siRNAs for therapeutic applications. FEBS J. 277(23), 4806-4813 (2010).
84. Sioud M. Recent advances in small interfering RNA sensing by the immune system. Nat. Biotechnol. 27(3), 236-242 (2010).
85. Poeck H, Besch R, Maihoefer C et al. 5'-triphosphate-siRNA: turning gene silencing and Rig-i activation against melanoma. Nat. Med. 14(11), 1256-1263 (2008).
86. Sioud M. RNA interference and innate immunity. Adv. Drug Deliv. Rev. 59(2-3), 153-163 (2007). (Pubitemid 46783844)
87. Stein CA, Colombini M. Specific VDAC inhibitors: phosphorothioate oligonucleotides. J. Bioenerg. Biomembr. 40(3), 157-162 (2008).
88. Stessl M, Marchetti-Deschmann M, Winkler J, Lachmann B, Allmaier G, Noe CR. A proteomic study reveals unspecific apoptosis induction and reduction of glycolytic enzymes by the phosphorothioate antisense oligonucleotide oblimersen in human melanoma cells. J. Proteomics 72(6), 1019-1030 (2009).
89. Winkler J, Stessl M, Amartey J, Noe CR. Off-target effects related to the phosphorothioate modification of nucleic acids. ChemMedChem 5(8), 1344-1352 (2010).
90. Akinc A, Zumbuehl A, Goldberg M et al. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol. 26(5), 561-569 (2008). (Pubitemid 351668042)
91. Semple SC, Akinc A, Chen J et al. Rational design of cationic lipids for siRNA delivery. Nat. Biotechnol. 28(2), 172-176 (2010).
92. Vester B, Wengel J. LNA (locked nucleic acid): high-affinity targeting of complementary RNA and DNA. Biochemistry 43(42), 13233-13241 (2004). (Pubitemid 39391124)
93. Leachman SA, Hickerson RP, Schwartz ME et al. First-in-human mutation-targeted siRNA Phase Ib trial of an inherited skin disorder. Mol. Ther. 18(2), 442-446 (2010).