Therapeutic applications of DNA and RNA aptamers

Oligonucleotides

Volumn 19, Issue 3, 2009, Pages 209-222

  Thiel, Kristina W ^a   Giangrande, Paloma H ^a,b,c  

^a UNIVERSITY OF IOWA   (United States)

^b UNIVERSITY OF IOWA HOSPITALS AND CLINICS   (United States)

^c NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

APTAMER; ARC 1779; ARC 183; AS 1411; DNA; LOCKED NUCLEIC ACID; MACROGOL; NU 172; PEGAPTANIB; REG 1; RNA; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

CAROTID ENDARTERECTOMY; CLINICAL TRIAL; COMBINATORIAL CHEMISTRY; CORONARY ARTERY BYPASS GRAFT; DIABETIC RETINOPATHY; DRUG ACTIVITY; DRUG EFFICACY; HUMAN; IN VITRO STUDY; KIDNEY CARCINOMA; NONHUMAN; PERCUTANEOUS CORONARY INTERVENTION; PRIORITY JOURNAL; RETINA MACULA AGE RELATED DEGENERATION; RETINA MACULA DEGENERATION; REVIEW; THROMBOCYTOPENIC PURPURA; THROMBOTIC THROMBOCYTOPENIC PURPURA;

ANIMALS; APTAMERS, NUCLEOTIDE; DRUG DISCOVERY; HUMANS; INFECTION; NEOPLASMS; PROTEINS; SELEX APTAMER TECHNIQUE;

EID: 70349272617     PISSN: 15454576     EISSN: None     Source Type: Journal    
DOI: 10.1089/oli.2009.0199     Document Type: Review

References (115)

1. ADLER, A., FORSTER, N., HOMANN, M., and GORINGER, H.U. (2008). Post-SELEX chemical optimization of a trypanosome-specif c RNA aptamer. Comb. Chem. High Throughput Screen. 11, 16-23.
2. AKIYAMA, H., KACHI, S., SILVA, R.L., UMEDA, N., HACKETT, S.F., MCCAULEY, D., MCCAULEY, T., ZOLTOSKI, A., EPSTEIN, D.M., and CAMPOCHIARO, P.A. (2006). Intraocular injection of an aptamer that binds PDGFB: a potential treatment for prolifer-ative retinopathies. J. Cell Physiol. 207, 407-412.
3. ALEXANDER, J.H., FERGUSON, J.T.B., JOSEPH, D.M., MACK, M.J., WOLF, R.K., GIBSON, C.M., GENNEVOIS, D., LORENZ, T.J., HARRINGTON, R.A., PETERSON, E.D., LEE, K.L., CALIFF, R.M., and KOUCHOUKOS, N.T. (2005). The PRoject of Ex-vivo Vein graft ENgineering via Transfection IV (PREVENT IV) trial: Study rationale, design, and baseline patient characteristics. Am. Heart J. 150, 643-649.
4. BAGALKOT, V., FAROKHZAD, O.C., LANGER, R., and JON, S. (2006). An aptamer-doxorubicin physical conjugate as a novel targeted drug-delivery platform. Angew. Chem. Int. Ed. Engl. 45, 8149-8152.
5. BASSETT S.E., FENNEWALD S.M., KING D.J., LI X., HERZOG N.K., SHOPE R., ARONSON J.F., LUXON B.A., and GORENSTEIN D.G. (2004). Combinatorial selection and edited combinatorial selection of phosphorothioate aptamers targeting human nuclear factor-kappa B RelA/p50 and RelA/RelA. Biochemistry 43, 9105-9115.
6. BATES, P.J., KAHLON, J.B., THOMAS, S.D., TRENT, J.O., and MILLER, D.M. (1999). Antiproliferative activity of G-rich oligo-nucleotides correlates with protein binding. J. Biol. Chem. 274, 26369-26377.
7. BEHLKE, M.A. (2008). Chemical modif cation of siRNAs for in vivo use. Oligonucleotides. 18, 305-320.
8. BEREZOVSKI, M., DRABOVICH, A., KRYLOVA, S.M., MUSHEEV, M., OKHONIN, V., PETROV, A., and KRYLOV, S.N. (2005). Nonequilibrium capillary electrophoresis of equilibrium mixtures: a universal tool for development of aptamers. J. Am. Chem. Soc. 127, 3165-3171. (Pubitemid 40349896)
9. BLANK, M., WEINSCHENK, T., PRIEMER, M., and SCHLUESENER, H. (2001). Systematic evolution of a DNA aptamer binding to rat brain tumor microvessels: selective targeting of endothelial regulatory protein pigpen. J. Biol. Chem. 276, 16464-16468.
10. BOCK, C., COLEMAN, M., COLLINS, B., DAVIS, J., FOULDS, G., GOLD, L., GREEF, C., HEIL, J., HEILIG, J.S., HICKE, B., HURST, M.N., HUSAR, G.M., MILLER, D., OSTROFF, R., PETACH, H., SCHNEIDER, D., VANT-HULL, B., WAUGH, S., WEISS, A., WILCOX, S.K., and ZICHI, D. (2004). Photoaptamer arrays applied to multiplexed proteomic analysis. Proteomics. 4, 609-618.
11. BOCK, L.C., GRIFFIN, L.C., LATHAM, J.A., VERMAAS, E.H., and TOOLE, J.J. (1992). Selection of single-stranded DNA molecules that bind and inhibit human thrombin. Nature. 355, 564-566.
12. BOOMER, R.M., LEWIS, S.D., HEALY, J.M., KURZ, M., WILSON, C., and MCCAULEY, T.G. (2005). Conjugation to polyethylene gly-col polymer promotes aptamer biodistribution to healthy and inf amed tissues. Oligonucleotides. 15, 183-195.
13. BOUCARD, D., TOULME, J.J., and DI PRIMO, C. (2006). Bimodal loop-loop interactions increase the aff nity of RNA aptamers for HIV-1 RNA structures. Biochemistry. 45, 1518-1524.
14. BROWNING, C.M., CAGNON, L., GOOD, P.D., ROSSI, J., ENGELKE, D.R., and MARKOVITZ, D.M. (1999). Potent inhibition of human immunodef ciency virus type 1 (HIV-1) gene expression and virus production by an HIV-2 tat activation-response RNA decoy. J. Virol. 73, 5191-5195.
15. BUNKA, D.H.J., MANTLE, B.J., MORTEN, I.J., TENNENT, G.A., RADFORD, S.E., and STOCKLEY, P.G. (2007). Production and characterization of RNA aptamers specif c for amyloid f bril epitopes. J. Biol. Chem. 282, 34500-34509.
16. BURKE, D.H., SCATES, L., ANDREWS, K., and GOLD, L. (1996). Bent pseudoknots and novel RNA inhibitors of type 1 human immunodef ciency virus (HIV-1) reverse transcriptase. J. Mol. Biol. 264, 650-666.
17. BURMEISTER, P.E., LEWIS, S.D., SILVA, R.F., PREISS, J.R., HORWITZ, L.R., PENDERGRAST, P.S., MCCAULEY, T.G., KURZ, J.C., EPSTEIN, D.M., WILSON, C., and KEEFE, A.D. (2005). Direct in vitro selection of a 2′-O-methyl aptamer to VEGF. Chem. Biol. 12, 25-33.
18. CASSIDAY L.A., and MAHER L.J. (2003). Yeast genetic selections to optimize RNA decoys for transcription factor NF-kappaB. Proc. Natl. Acad. Sci. USA 100, 3930-3935.
19. CERCHIA, L., DUCONGE, F., PESTOURIE, C., BOULAY, J., AISSOUNI, Y., GOMBERT, K., TAVITIAN, B., DE FRANCISCIS, V., and LIBRI, D. (2005). Neutralizing aptamers from whole-cell SELEX inhibit the RET receptor tyrosine kinase. PLoS Biol. 3, e123.
20. CHAN, M.Y., COHEN, M.G., DYKE, C.K., MYLES, S.K., ABERLE, L.G., LIN, M., WALDER, J., STEINHUBL, S.R., GILCHRIST, I.C., KLEIMAN, N.S., VORCHHEIMER, D.A., CHRONOS, N., MELLONI, C., ALEXANDER, J.H., HARRINGTON, R.A., TONKENS, R.M., BECKER, R.C., and RUSCONI, C.P. (2008a). Phase 1b randomized study of antidote-controlled modulation of factor IXa activity in patients with stable coronary artery disease. Circulation. 117, 2865-2874.
21. CHAN, M.Y., RUSCONI, C.P., ALEXANDER, J.H., TONKENS, R.M., HARRINGTON, R.A., and BECKER, R.C. (2008b). A randomized, repeat-dose, pharmacodynamic and safety study of an antidote-controlled factor IXa inhibitor. J. Thromb. Haemost. 6, 789-796.
22. CHELLISERRYKATTIL, J., and ELLINGTON, A.D. (2004). Evolution of a T7 RNA polymerase variant that transcribes 2′-O-methyl RNA. Nat. Biotechnol. 22, 1155-1160.
23. CHEN, C.-H.B., CHERNIS, G.A., HOANG, V.Q., and LANDGRAF, R. (2003). Inhibition of heregulin signaling by an aptamer that preferentially binds to the oligomeric form of human epidermal growth factor receptor-3. Proc. Natl. Acad. Sci. USA. 100, 9226-9231.
24. CHEN, C.-H.B., DELLAMAGGIORE, K.R., OUELLETTE, C.P., SEDANO, C.D., LIZADJOHRY, M., CHERNIS, G.A., GONZALES, M., BALTASAR, F.E., FAN, A.L., MYEROWITZ, R., and NEUFELD, E.F. (2008). Aptamer-based endocytosis of a lysosomal enzyme. Proc. Natl. Acad. Sci. USA. 105, 15908-15913.
25. CHEN, F., ZHOU, J., LUO, F., MOHAMMED, A.-B., and ZHANG, X.-L. (2007). Aptamer from whole-bacterium SELEX as new therapeutic reagent against virulent Mycobacterium tuberculosis. Biochem. Biophys. Res. Commun. 357, 743-748
26. CHENG, C., DONG, J., YAO, L., CHEN, A., JIA, R., HUAN, L., GUO, J., SHU, Y., and ZHANG, Z. (2008). Potent inhibition of human inf uenza H5N1 virus by oligonucleotides derived by SELEX. Biochem. Biophys. Res. Commun. 366, 670-674.
27. CHENG, J., TEPLY, B.A., SHERIFI, I., SUNG, J., LUTHER, G., GU, F.X., LEVY-NISSENBAUM, E., RADOVIC-MORENO, A.F., LANGER, R., and FAROKHZAD, O.C. (2007). Formulation of functional-ized PLGA-PEG nanoparticles for in vivo targeted drug delivery. Biomaterials. 28, 869-876. (Pubitemid 44667020)
28. CHU, T.C., MARKS, J.W., III, LAVERY, L.A., FAULKNER, S., ROSENBLUM, M.G., ELLINGTON, A.D., and LEVY, M. (2006a). Aptamer:toxin conjugates that specif cally target prostate tumor cells. Cancer Res. 66, 5989-5992.
29. CHU, T.C., TWU, K.Y., ELLINGTON, A.D., and LEVY, M. (2006b). Aptamer mediated siRNA delivery. Nucleic Acids Res. 34, e73.
30. COLLETT, J.R., CHO, E.J., and ELLINGTON, A.D. (2005). Production and processing of aptamer microarrays. Methods. 37, 4-15.
31. CONTE, M.S., BANDYK, D.F., CLOWES, A.W., MONETA, G.L., SEELY, L., LORENZ, T.J., NAMINI, H., HAMDAN, A.D., RODDY, S.P., BELKIN, M., BERCELI, S.A., DEMASI, R.J., SAMSON, R.H., and BERMAN, S.S. (2006). Results of PREVENT III: a multicen-ter, randomized trial of edifoligide for the prevention of vein graft failure in lower extremity bypass surgery. J. Vasc. Surg. 43, 742-751; discussion 751.
32. COX, J.C., RUDOLPH, P., and ELLINGTON, A.D. (1998). Automated RNA selection. Biotechnol. Prog. 14, 845-850.
33. CUNNINGHAM, E.T., Jr., ADAMIS, A.P., ALTAWEEL, M., AIELLO, L.P., BRESSLER, N.M., D'AMICO, D.J., GOLDBAUM, M., GUYER, D.R., KATZ, B., PATEL, M., and SCHWARTZ, S.D. (2005). A phase II randomized double-masked trial of pegaptanib, an anti-vascular endothelial growth factor aptamer, for diabetic macular edema. Ophthalmology. 112, 1747-1757.
34. DANIELS, D.A., CHEN, H., HICKE, B.J., SWIDEREK, K.M., and GOLD, L. (2003). A tenascin-C aptamer identif ed by tumor cell SELEX: systematic evolution of ligands by exponential enrichment. Proc. Natl. Acad. Sci. USA. 100, 15416-15421.
35. DAPIC, V., BATES, P.J., TRENT, J.O., RODGER, A., THOMAS, S.D., and MILLER, D.M. (2002). Antiproliferative activity of G-quartet-forming oligonucleotides with backbone and sugar modif cations. Biochemistry. 41, 3676-3685.
36. DEY, A.K., KHATI, M., TANG, M., WYATT, R., LEA, S.M., and JAMES, W. (2005). An aptamer that neutralizes R5 strains of human immunodef ciency virus type 1 blocks gp120-CCR5 interaction. J. Virol. 79, 13806-13810.
37. DHAR, S., GU, F.X., LANGER, R., FAROKHZAD, O.C., and LIPPARD, S.J. (2008). Targeted delivery of cisplatin to prostate cancer cells by aptamer functionalized Pt(IV) prodrug-PLGA-PEG nanopar-ticles. Proc. Natl. Acad. Sci. USA. 105, 17356-17361.
38. DOLLINS, C.M., NAIR, S., BOCZKOWSKI, D., LEE, J., LAYZER, J.M., GILBOA, E., and SULLENGER, B.A. (2008). Assembling OX40 aptamers on a molecular scaffold to create a receptor-activating aptamer. Chem. Biol. 15, 675-682.
39. DYKE, C.K., STEINHUBL, S.R., KLEIMAN, N.S., CANNON, R.O., ABERLE, L.G., LIN, M., MYLES, S.K., MELLONI, C., HARRINGTON, R.A., ALEXANDER, J.H., BECKER, R.C., and RUSCONI, C.P. (2006). First-in-human experience of an antidote-controlled anticoagulant using RNA aptamer technology: A Phase 1a pharmacodynamic evaluation of a drug-antidote pair for the controlled regulation of factor IXa activity. Circulation. 114, 2490-2497.
40. EHSAN, A., MANN, M.J., DELL'ACQUA, G., and DZAU, V.J. (2001). Long-term stabilization of vein graft wall architecture and prolonged resistance to experimental atherosclerosis after E2F decoy oligonucleotide gene therapy. J. Thorac. Cardiovasc. Surg. 121, 714-722.
41. ELLINGTON, A.D., and SZOSTAK, J.W. (1990). In vitro selection of RNA molecules that bind specif c ligands. Nature. 346, 818-822.
42. EULBERG, D., BUCHNER, K., MAASCH, C., and KLUSSMANN, S. (2005). Development of an automated in vitro selection protocol to obtain RNA-based aptamers: identif cation of a biostable substance P antagonist. Nucleic Acids Res. 33, e45.
43. EULBERG, D., and KLUSSMANN, S. (2003). Spiegelmers: biostable aptamers. Chembiochem. 4, 979-983.
44. FAROKHZAD, O.C., CHENG, J., TEPLY, B.A., SHERIFI, I., JON, S., KANTOFF, P.W., RICHIE, J.P., and LANGER, R. (2006). Targeted nanoparticle-aptamer bioconjugates for cancer chemotherapy in vivo. Proc. Natl. Acad. Sci. USA. 103, 6315-6320.
45. FERREIRA, C.S.M., CHEUNG, M.C., MISSAILIDIS, S., BISLAND, S., and GARIEPY, J. (2009). Phototoxic aptamers selectively enter and kill epithelial cancer cells. Nucleic Acids Res. 37, 866-876.
46. GIANGRANDE, P.H., ZHANG, J., TANNER, A., ECKHART, A.D., REMPEL, R.E., ANDRECHEK, E.R., LAYZER, J.M., KEYS, J.R., HAGEN, P.O., NEVINS, J.R., KOCH, W.J., and SULLENGER, B.A. (2007). Distinct roles of E2F proteins in vascular smooth muscle cell proliferation and intimal hyperplasia. Proc. Natl. Acad. Sci. USA. 104, 12988-12993.
47. GILBERT, J.C., DEFEO-FRAULINI, T., HUTABARAT, R.M., HORVATH, C.J., MERLINO, P.G., MARSH, H.N., HEALY, J.M., BOUFAKHREDDINE, S., HOLOHAN, T.V., and SCHAUB, R.G. (2007). First-in-human evaluation of anti von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers. Circulation. 116, 2678-2686.
48. GIRVAN, A.C., TENG, Y., CASSON, L.K., THOMAS, S.D., JULIGER, S., BALL, M.W., KLEIN, J.B., PIERCE, W.M., Jr., BARVE, S.S., and BATES, P.J. (2006). AGRO100 inhibits activation of nuclear factor-kappaB (NF-kappaB) by forming a complex with NF-kappaB essential modulator (NEMO) and nucleolin. Mol. Cancer Ther. 5, 1790-1799.
49. GREEN, L.S., JELLINEK, D., JENISON, R., OSTMAN, A., HELDIN, C.-H., and JANJIC, N. (1996). Inhibitory DNA ligands to platelet-derived growth factor B-chain. Biochemistry. 35, 14413-14424.
50. HEALY, J.M., LEWIS, S.D., KURZ, M., BOOMER, R.M., THOMPSON, K.M., WILSON, C., and MCCAULEY, T.G. (2004). Pharmacokinetics and biodistribution of novel aptamer compositions. Pharm. Res. 21, 2234-2246.
51. HICKE, B.J., MARION, C., CHANG, Y.-F., GOULD, T., LYNOTT, C.K., PARMA, D., SCHMIDT, P.G., and WARREN, S. (2001). Tenascin-C aptamers are generated using tumor cells and purif ed protein. J. Biol. Chem. 276, 48644-48654.
52. HICKE, B.J., STEPHENS, A.W., GOULD, T., CHANG, Y.-F., LYNOTT, C.K., HEIL, J., BORKOWSKI, S., HILGER, C.-S., COOK, G., WARREN, S., and SCHMIDT, P.G. (2006). Tumor targeting by an aptamer. J. Nucl. Med. 47, 668-678.
53. HOMANN, M., and GORINGER, H. (1999). Combinatorial selection of high aff nity RNA ligands to live African trypanosomes. Nucleic Acids Res. 27, 2006-2014.
54. HUANG, Y., ECKSTEIN, F., PADILLA, R., and SOUSA, R. (1997). Mechanism of ribose 2′-group discrimination by an RNA poly-merase. Biochemistry. 36, 8231-8242.
55. HUANG, Y.F., SHANGGUAN, D., LIU, H., PHILLIPS, J.A., ZHANG, X., CHEN, Y., and TAN, W. (2009). Molecular assembly of an aptamer-drug conjugate for targeted drug delivery to tumor cells. Chembiochem. 10, 862-868.
56. IRESON, C.R., and KELLAND, L.R. (2006). Discovery and development of anticancer aptamers. Mol. Cancer Ther. 5, 2957-2962.
57. ISHIZAKI, J., NEVINS, J.R., and SULLENGER, B.A. (1996). Inhibition of cell proliferation by an RNA ligand that selectively blocks E2F function. Nat. Med. 2, 1386-1389.
58. JASON, T.L., KOROPATNICK, J., and BERG, R.W. (2004). Toxicology of antisense therapeutics. Toxicol. Appl. Pharmacol. 201, 66-83.
59. KEEFE, A.D., and CLOAD, S.T. (2008). SELEX with modif ed nucle-otides. Curr. Opin. Chem. Biol. 12, 448-456.
60. KHATI, M., SCHUMAN, M., IBRAHIM, J., SATTENTAU, Q., GORDON, S., and JAMES, W. (2003). Neutralization of infectivity of diverse R5 clinical isolates of human immunodef ciency virus type 1 by gp120-binding 2′F-RNA aptamers. J. Virol. 77, 12692-12698.
61. KIM, Y., CAO, Z., and TAN, W. (2008). Molecular assembly for high-performance bivalent nucleic acid inhibitor. Proc. Natl. Acad. Sci. USA. 105, 5664-5669.
62. KIRBY, R., CHO, E.J., GEHRKE, B., BAYER, T., PARK, Y.S., NEIKIRK, D.P., MCDEVITT, J.T., and ELLINGTON, A.D. (2004). Aptamer-based sensor arrays for the detection and quantitation of proteins. Anal. Chem. 76, 4066-4075.
63. KOHN, D.B., BAUER, G., RICE, C.R., ROTHSCHILD, J.C., CARBONARO, D.A., VALDEZ, P., HAO, Q., ZHOU, C., BAHNER, I., KEARNS, K., BRODY, K., FOX, S., HADEN, E., WILSON, K., SALATA, C., DOLAN, C., WETTER, C., AGUILAR-CORDOVA, E., and CHURCH, J. (1999). A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodef ciency virus-1-infected children. Blood. 94, 368-371.
64. LEBRUSKA L.L., and MAHER L.J. (1999). Selection and characterization of an RNA decoy for transcription factor NF-kappaB. Biochemistry 38, 3168-3174.
65. LEE, T.C., SULLENGER, B.A., GALLARDO, H.F., UNGERS, G.E., and GILBOA, E. (1992). Overexpression of RRE-derived sequences inhibits HIV-1 replication in CEM cells. New Biol. 4, 66-74.
66. LI, N., EBRIGHT, J., STOVALL, G., CHEN, X., NGUYEN, H., SINGH, A., SYRETT, H., and ELLINGTON, A. (2009). Technical and biological issues relevant to cell typing by aptamers. J. Proteome Res. 8:2438-2448.
67. LI, N., WANG, Y., POTHUKUCHY, A., SYRETT, A., HUSAIN, N., GOPALAKRISHA, S., KOSARAJU, P., and ELLINGTON, A.D. (2008). Aptamers that recognize drug-resistant HIV-1 reverse transcriptase. Nucleic Acids Res. 36, 6739-6751.
68. LOU, X., QIAN, J., XIAO, Y., VIEL, L., GERDON, A.E., LAGALLY, E.T., ATZBERGER, P., TARASOW, T.M., HEEGER, A.J., and SOH, H.T. (2009). Micromagnetic selection of aptamers in microf uidic channels. Proc. Natl. Acad. Sci. USA. 106, 2989-2994.
69. LUPOLD, S.E., HICKE, B.J., LIN, Y., and COFFEY, D.S. (2002). Identif cation and characterization of nuclease-stabilized RNA molecules that bind human prostate cancer cells via the prostate-specif c membrane antigen. Cancer Res. 62, 4029-4033.
70. MANOHARAN, M. (2002). Oligonucleotide conjugates as potential antisense drugs with improved uptake, biodistribution, targeted delivery, and mechanism of action. Antisense Nucleic Acid Drug Dev. 12, 103-128.
71. MATTEUCCI, M. (1997). Oligonucleotide analogues: an overview. Ciba Found. Symp. 209, 5-14; discussion 14-18.
72. MAYER, G., MULLER, J., MACK, T., FREITAG, D.F., HOVER, T., POTZSCH, B., and HECKEL, A. (2009). Differential regulation of protein subdomain activity with caged bivalent ligands. Chembiochem. 10, 654-657.
73. MCNAMARA, J.O., II, ANDRECHEK, E.R., WANG, Y., VILES, K.D., REMPEL, R.E., GILBOA, E., SULLENGER, B.A., and GIANGRANDE, P.H. (2006). Cell type-specif c delivery of siRNAs with aptamer-siRNA chimeras. Nat. Biotechnol. 24, 1005-1015.
74. MCNAMARA, J.O., KOLONIAS, D., PASTOR, F., MITTLER, R.S., CHEN, L., GIANGRANDE, P.H., SULLENGER, B., and GILBOA, E. (2008). Multivalent 4-1BB binding aptamers costimulate CD8+ T cells and inhibit tumor growth in mice. J. Clin. Invest. 118, 376-386.
75. MENDONSA, S.D., and BOWSER, M.T. (2004). In vitro evolution of functional DNA using capillary electrophoresis. J. Am. Chem. Soc. 126, 20-21.
76. MICHIENZI, A., LI, S., ZAIA, J.A., and ROSSI, J.J. (2002). A nucleolar TAR decoy inhibitor of HIV-1 replication. Proc. Natl. Acad. Sci. USA. 99, 14 0 4 7-14 0 52.
77. MULLER, J., WULFFEN, B., POTZSCH, B., and MAYER, G. (2007). Multidomain targeting generates a high-aff nity thrombin-inhibiting bivalent aptamer. Chembiochem. 8, 2223-2226.
78. NG, E.W.M., SHIMA, D.T., CALIAS, P., CUNNINGHAM, E.T., GUYER, D.R., and ADAMIS, A.P. (2006). Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease. Nat. Rev. Drug Discov. 5, 123-132.
79. OHUCHI, S.P., OHTSU, T., and NAKAMURA, Y. (2006). Selection of RNA aptamers against recombinant transforming growth factor-beta type III receptor displayed on cell surface. Biochimie. 88, 897-904.
80. ONEY, S., NIMJEE, S.M., LAYZER, J., QUE-GEWIRTH, N., GINSBURG, D., BECKER, R.C., AREPALLY, G., and SULLENGER, B.A. (2007). Antidote-controlled platelet inhibition targeting von Willebrand factor with aptamers. Oligonucleotides. 17, 265-274.
81. PESTOURIE, C., CERCHIA, L., GOMBERT, K., AISSOUNI, Y., BOULAY, J., DE FRANCISCIS, V., LIBRI, D., TAVITIAN, B., and DUCONGE, F. (2006). Comparison of different strategies to select aptamers against a transmembrane protein target. Oligonucleotides. 16, 323-335.
82. PIEKEN, W.A., OLSEN, D.B., BENSELER, F., AURUP, H., and ECKSTEIN, F. (1991). Kinetic characterization of ribonuclease-resistant 2′-modif ed hammerhead ribozymes. Science. 253, 314-317.
83. PREVENT IV INVESTIGATORS. (2005). Eff cacy and safety of Edifoligide, an E2F transcription factor decoy, for prevention of vein graft failure following coronary artery bypass graft surgery: PREVENT IV: A randomized controlled trial. JAMA. 294, 2446-2454.
84. PROSKE, D., GILCH, S., WOPFNER, F., SCHATZL, H.M., WINNACKER, E.L., and FAMULOK, M. (2002). Prion-protein-specif c aptamer reduces PrPSc formation. Chembiochem. 3, 717-725.
85. RENTMEISTER, A., BILL, A., WAHLE, T., WALTER, J., and FAMULOK, M. (2006). RNA aptamers selectively modulate protein recruitment to the cytoplasmic domain of beta-secretase BACE1 in vitro. RNA. 12, 1650-1660.
86. RHIE, A., KIRBY, L., SAYER, N., WELLESLEY, R., DISTERER, P., SYLVESTER, I., GILL, A., HOPE, J., JAMES, W., and TAHIRI-ALAOUI, A. (2003). Characterization of 2′-f uoro-RNA aptam-ers that bind preferentially to disease-associated conformations of prion protein and inhibit conversion. J. Biol. Chem. 278, 39697-39705.
87. RUCKMAN, J., GREEN, L.S., BEESON, J., WAUGH, S., GILLETTE, W.L., HENNINGER, D.D., CLAESSON-WELSH, L., and JANJIC, N. (1998). 2′-f uoropyrimidine RNA-based aptamers to the 165-amino acid form of vascular endothelial growth factor (VEGF165): inhibition of receptor binding and VEGF-induced vascular permeability through interactions requiring the exon 7-encoded domain. J. Biol. Chem. 273, 20556-20567.
88. RUSCONI, C.P., ROBERTS, J.D., PITOC, G.A., NIMJEE, S.M., WHITE, R.R., QUICK, G., Jr., SCARDINO, E., FAY, W.P., and SULLENGER, B.A. (2004). Antidote-mediated control of an anticoagulant aptamer in vivo. Nat. Biotechnol. 22, 1423-142.
89. RUSCONI, C.P., SCARDINO, E., LAYZER, J., PITOC, G.A., ORTEL, T.L., MONROE, D., and SULLENGER, B.A. (2002). RNA aptamers as reversible antagonists of coagulation factor IXa. Nature. 419, 90-94.
90. RUSCONI, C.P., YEH, A., LYERLY, H.K., LAWSON, J.H., and SULLENGER, B.A. (2000). Blocking the initiation of coagulation by RNA aptamers to factor VIIa. Thromb. Haemost. 84, 841-848.
91. SANTULLI-MAROTTO, S., NAIR, S.K., RUSCONI, C., SULLENGER, B., and GILBOA, E. (2003). Multivalent RNA aptamers that inhibit CTLA-4 and enhance tumor immunity. Cancer Res. 63, 7483-7489.
92. SAYER, N.M., CUBIN, M., RHIE, A., BULLOCK, M., TAHIRI-ALAOUI, A., and JAMES, W. (2004). Structural determinants of conformationally selective, prion-binding aptamers. J. Biol. Chem. 279, 13102-13109.
93. SCHMIDT, K.S., BORKOWSKI, S., KURRECK, J., STEPHENS, A.W., BALD, R., HECHT, M., FRIEBE, M., DINKELBORG, L., and ERDMANN, V.A. (2004). Application of locked nucleic acids to improve aptamer in vivo stability and targeting function. Nucleic Acids Res. 32, 5757-5765.
94. SCHNEIDER, D.J., FEIGON, J., HOSTOMSKY, Z., and GOLD, L. (1995). High-aff nity ssDNA inhibitors of the reverse transcrip-tase of type 1 human immunodef ciency virus. Biochemistry. 34, 9599-9610.
95. SENNINO, B., FALCON, B.L., MCCAULEY, D., LE, T., MCCAULEY, T., KURZ, J.C., HASKELL, A., EPSTEIN, D.M., and MCDONALD, D.M. (2007). Sequential loss of tumor vessel pericytes and endo-thelial cells after inhibition of platelet-derived growth factor B by selective aptamer AX102. Cancer Res. 67, 7358-7367.
96. SHAMAH, S.M., HEALY, J.M., and CLOAD, S.T. (2008). Complex target SELEX. Acc. Chem. Res. 41, 130-138.
97. SHANGGUAN, D., CAO, Z.C., LI, Y., and TAN, W. (2007). Aptamers evolved from cultured cancer cells reveal molecular differences of cancer cells in patient samples. Clin. Chem. 53, 1153-1155.
98. SHI, H., HOFFMAN, B.E., and LIS, J.T. (1999). RNA aptamers as effective protein antagonists in a multicellular organism. Proc. Natl. Acad. Sci. USA. 96, 10033-10038.
99. SOUNDARARAJAN, S., CHEN, W., SPICER, E.K., COURTENAY-LUCK, N., and FERNANDES, D.J. (2008). The nucleolin targeting aptamer AS1411 destabilizes Bcl-2 messenger RNA in human breast cancer cells. Cancer Res. 68, 2358-2365.
100. SULLENGER, B.A., GALLARDO, H.F., UNGERS, G.E., and GILBOA, E. (1990). Overexpression of TAR sequences renders cells resistant to human immunodef ciency virus replication. Cell. 63, 601-608.
101. SULLENGER, B.A., GALLARDO, H.F., UNGERS, G.E., and GILBOA, E. (1991). Analysis of trans-acting response decoy RNA-mediated inhibition of human immunodefciency virus type 1 transactivation. J. Virol. 65, 6811-6816.
102. TANG, Z., SHANGGUAN, D., WANG, K., SHI, H., SEFAH, K., MALLIKRATCHY, P., CHEN, H.W., LI, Y., and TAN, W. (2007). Selection of aptamers for molecular recognition and characterization of cancer cells. Anal. Chem. 79, 4900-4907.
103. TIAN, L., and HEYDUK, T. (2009). Bivalent ligands with long nanometer-scale f exible linkers. Biochemistry. 48, 264-275.
104. TUERK, C., and GOLD, L. (1990). Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science. 249, 505-510.
105. TUERK, C., MACDOUGAL, S., and GOLD, L. (1992). RNA pseudo-knots that inhibit human immunodef ciency virus type 1 reverse transcriptase. Proc. Natl. Acad. Sci. USA. 89, 6988-6992.
106. VENTO, M.T., IUORIO, M., NETTI, P.A., DUCONGE, F., TAVITIAN, B., FRANCISCIS, V., and CERCHIA, L. (2008). Distribution and bioactivity of the Ret-specif c D4 aptamer in three-dimensional collagen gel cultures. Mol. Cancer Ther. 7, 3381-3388.
107. WEISS, S., PROSKE, D., NEUMANN, M., GROSCHUP, M., KRETZSCHMAR, H., FAMULOK, M., and WINNACKER, E. (1997). RNA aptamers specif cally interact with the prion protein PrP. J. Virol. 71, 8790-8797.
108. WHITE, R., RUSCONI, C., SCARDINO, E., WOLBERG, A., LAWSON, J., HOFFMAN, M., and SULLENGER, B. (2001). Generation of species cross-reactive aptamers using "toggle" SELEX. Mol. Ther. 4, 567-573.
109. WILLIS, M.C., COLLINS, B., ZHANG, T., GREEN, L.S., SEBESTA, D.P., BELL, C., KELLOGG, E., GILL, S.C., MAGALLANEZ, A., KNAUER, S., BENDELE, R.A., GILL, P.S., and JANJIC, N. (1998). Liposome-anchored vascular endothelial growth factor aptam-ers. Bioconjug. Chem. 9, 573-582.
110. WULLNER, U., NEEF, I., ELLER, A., KLEINES, M., TUR, M.K., and BARTH, S. (2008). Cell-specif c induction of apoptosis by rationally designed bivalent aptamer-siRNA transcripts silencing eukary-otic elongation factor 2. Curr. Cancer Drug Targets 8, 554-565.
111. WURSTER S.E., and MAHER L.J. (2008). Selection and characterization of anti-NF-kappaB p65 RNA aptamers. RNA 14, 1037-1047.
112. YAMAMOTO, R., KATAHIRA, M., NISHIKAWA, S., BABA, T., TAIRA, K., and KUMAR, P.K. (2000). A novel RNA motif that binds eff ciently and specif cally to the Ttat protein of HIV and inhibits the trans-activation by Tat of transcription in vitro and in vivo. Genes Cells 5, 371-388.
113. YLERA, F., LURZ, R., ERDMANN, V.A., and FÜRSTE, J.P. (2002). Selection of RNA aptamers to the Alzheimer's disease amyloid peptide. Biochem. Biophys. Res. Commun. 290, 1583-1588.
114. ZHOU, J., LI, H., LI, S., ZAIA, J., and ROSSI, J.J. (2008). Novel dual inhibitory function aptamer-siRNA delivery system for HIV-1 therapy. Mol. Ther. 16, 1481-1489.
115. ZHOU, J., SWIDERSKI, P., LI, H., ZHANG, J., NEFF, C.P., AKKINA, R., and ROSSI, J.J. (2009). Selection, characterization and application of new RNA HIV gp 120 aptamers for facile delivery of Dicer substrate siRNAs into HIV infected cells. Nucleic Acids Res. 37, 3094-3109.