Properties of Therapeutic Aptamers

The Aptamer Handbook: Functional Oligonucleotides and Their Applications

Volumn , Issue , 2006, Pages 363-416

  Cload, Sharon T ^a   Mccauley, Thomas G ^a   Keefe, Anthony D ^a   Healy, Judith M ^a   Wilson, Charles ^a  

^a Archemix Corporation   (United States)

Author keywords

Alternative aptamer formulations; Applications; Aptamer binding characteristics; Aptamer biodistribution; Aptamer handbook; Aptamer manufacture; Aptamer pharmacokinetics; Aptamer targets; Chemical modification of aptamers; Immunogenicity of aptamers; Properties of therapeutic aptamers; Routes of administration of aptamers; Toxicity profile of aptamers

Indexed keywords

EID: 84889280097     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/3527608192.ch17     Document Type: Chapter

References (218)

1. Adler, B. K. (2004). Unfractionated heparin and other antithrombin mediated anticoagulants. Clin Lab Sci 17, 113-117.
2. Agrawal, S., Iyer, R. P. (1995). Modified oligonucleotides as therapeutic and diagnostic agents. Curr Opin Biotechnol 6, 12-19.
3. Agrawal, S., Zhang, R. (1997). Pharmacokinetics of oligonucleotides. Ciba Found Symp 209, 60-75; discussion 75-68.
4. Agrawal, S., Goodchild, J., Civeira, M. P., Thornton, A. H., Sarin, P. S., Zamecnik, P. C. (1988). Oligodeoxynucleoside phosphoramidates and phosphorothioates as inhibitors of human immunodeficiency virus. Proc Natl Acad Sci USA 85, 7079-7083.
5. Agrawal, S., Jiang, Z., Zhao, Q., Shaw, D., Cai, Q., Roskey, A., Channavajjala, L., Saxinger, C., Zhang, R. (1997). Mixed-backbone oligonucleotides as second generation antisense oligonucleotides: in vitro and in vivo studies. Proc Natl Acad Sci USA 94, 2620-2625.
6. Akhtar, S., Agrawal, S. (1997). In vivo studies with antisense oligonucleotides. Trends Pharmacol Sci 18, 12-18.
7. Akhtar, S., Basu, S., Wickstrom, E., Juliano, R. L. (1991). Interactions of antisense DNA oligonucleotide analogs with phospholipid membranes (liposomes). Nucleic Acids Res 19, 5551-5559.
8. Ali, S., Leonard, S. A., Kukoly, C. A., Metzger, W. J., Wooles, W. R., McGinty, J. F., Tanaka, M., Sandrasagra, A., Nyce, J. W. (2001). Absorption, distribution, metabolism, and excretion of a respirable antisense oligonucleotide for asthma. Am J Respir Crit Care Med 163, 989-993.
9. Allen, P., Worland, S., Gold, L. (1995). Isolation of high-affinity RNA ligands to HIV-1 integrase from a random pool. Virology 209, 327-336.
10. Andrews, C. L., Vouros, P., Harsch, A. (1999). Analysis of DNA adducts using high-performance separation techniques coupled to electrospray ionization mass spectrometry. J Chromatogr A 856, 515-526.
11. Antopolsky, M., Azhayeva, E., Tengvall, U., Auriola, S., Jaaskelainen, I., Ronkko, S., Honkakoski, P., Urtti, A., Lonnberg, H., Azhayev, A. (1999). Peptide-oligonucleotide phosphorothioate conjugates with membrane translocation and nuclear localization properties. Bioconjug Chem 10, 598-606.
12. Anwar, A., Ali, N., Tanveer, R., Siddiqui, A. (2000). Demonstration of functional requirement of polypyrimidine tract-binding protein by SELEX RNA during hepatitis C virus internal ribosome entry site-mediated translation initiation. J Biol Chem 275, 34231-34235.
13. Astriab-Fisher, A., Sergueev, D., Fisher, M., Shaw, B. R., Juliano, R. L. (2002). Conjugates of antisense oligonucleotides with the Tat and antennapedia cell-penetrating peptides: effects on cellular uptake, binding to target sequences, and biologic actions. Pharm Res 19, 744-754.
14. Bergers, G., Song, S., Meyer-Morse, N., Bergsland, E., Hanahan, D. (2003). Benefits of targeting both pericytes and endothelial cells in the tumor vasculature with kinase inhibitors. J Clin Invest 111, 1287-1295.
15. Biesecker, G., Gold, L. (2002). High affinity nucleic acid ligands to complement system proteins. (USA) Patent Number 6, 713, 616.
16. Biesecker, G., Dihel, L., Enney, K., Bendele, R. A. (1999). Derivation of RNA aptamer inhibitors of human complement C5. Immunopharmacology 42, 219-230.
17. Bijsterbosch, M. K., Rump, E. T., De Vrueh, R. L., Dorland, R., van Veghel, R., Tivel, K. L., Biessen, E. A., van Berkel, T. J., Manoharan, M. (2000). Modulation of plasma protein binding and in vivo liver cell uptake of phosphorothioate oligodeoxynucleotides by cholesterol conjugation. Nucleic Acids Res 28, 2717-2725.
18. Bijsterbosch, M. K., Manoharan, M., Dorland, R., Waarlo, I. H., Biessen, E. A., van Berkel, T. J. (2001). Delivery of cholesteryl-conjugated phosphorothioate oligodeoxynucleotides to Kupffer cells by lactosylated low-density lipoprotein. Biochem Pharmacol 62, 627-633.
19. Bijsterbosch, M. K., Manoharan, M., Dorland, R., Van Veghel, R., Biessen, E. A., Van Berkel, T. J. (2002). bis-Cholesteryl-conjugated phosphorothioate oligodeoxynucleotides are highly selectively taken up by the liver. J Pharmacol Exp Ther 302, 619-626.
20. Binkley, J., Allen, P., Brown, D. M., Green, L., Tuerk, C., Gold, L. (1995). RNA ligands to human nerve growth factor. Nucleic Acids Res 23, 3198-3205.
21. Biroccio, A., Hamm, J., Incitti, I., De Francesco, R., Tomei, L. (2002). Selection of RNA aptamers that are specific and high-affinity ligands of the hepatitis C virus RNA-dependent RNA polymerase. J Virol 76, 3688-3696.
22. Blank, M., Weinschenk, T., Priemer, M., 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.
23. Blind, M., Kolanus, W., Famulok, M. (1999). Cytoplasmic RNA modulators of an inside-out signal-transduction cascade. Proc Natl Acad Sci USA 96, 3606-3610.
24. Blount, K. F., Tor, Y. (2003). Using pyrene-labeled HIV-1 TAR to measure RNA-small molecule binding. Nucleic Acids Res 31, 5490-5500.
25. Bock, L. C., Griffin, L. C., Latham, J. A., Vermaas, E. H., Toole, J. J. (1992). Selection of single-stranded DNA molecules that bind and inhibit human thrombin. Nature 355, 564-566.
26. Bongartz, J. P., Aubertin, A. M., Milhaud, P. G., Lebleu, B. (1994). Improved biological activity of antisense oligonucleotides conjugated to a fusogenic peptide. Nucleic Acids Res 22, 4681-4688.
27. Boomer, R. M., Kurz, M., Healy, J. M., Wilson, C., McCauley, T. G. (2005). Conjugation to polyethylene glycol polymer promotes aptamer biodistribution to healthy and inflamed tissues. Oligonucleotides 15, 183-195.
28. Brenner, T., Hamra-Amitay, Y., Evron, T., Boneva, N., Seidman, S., Soreq, H. (2003). The role of readthrough acetylcholinesterase in the pathophysiology of myasthenia gravis. FASEB J 17, 214-222.
29. Burke, D. H., Nickens, D. G. (2002). Expressing RNA aptamers inside cells to reveal proteome and ribonome function. Brief Funct Genomic Proteomic 1, 169-188.
30. 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.
31. Butterworth, J., Lin, Y. A., Prielipp, R. C., Bennett, J., Hammon, J. W., James, R. L. (2002). Rapid disappearance of protamine in adults undergoing cardiac operation with cardiopulmonary bypass. Ann Thorac Surg 74, 1589-1595.
32. Caliceti, P., Veronese, F. M. (2003). Pharmacokinetic and biodistribution properties of poly(ethylene glycol)-protein conjugates. Adv Drug Deliv Rev 55, 1261-1277.
33. Carrasquillo, K. G., Ricker, J. A., Rigas, I. K., Miller, J. W., Gragoudas, E. S., Adamis, A. P. (2003). Controlled delivery of the anti-VEGF aptamer EYE001 with poly(lactic-coglycolic)acid microspheres. Invest Ophthalmol Vis Sci 44, 290-299.
34. Chen, C. H., Chernis, G. A., Hoang, V. Q., 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.
35. Chen, H. X., Marshall, J. L., Ness, E., Martin, R. R., Dvorchik, B., Rizvi, N., Marquis, J., McKinlay, M., Dahut, W., Hawkins, M. J. (2000). A safety and pharmacokinetic study of a mixed-backbone oligonucleotide (GEM231) targeting the type I protein kinase A by two-hour infusions in patients with refractory solid tumors. Clin Cancer Res 6, 1259-1266.
36. Crooke, S. T. (1997). Advances in understanding the pharmacological properties of antisense oligonucleotides. Adv Pharmacol 40, 1-49.
37. Danahay, H., Giddings, J., Christian, R. A., Moser, H. E., Phillips, J. A. (1999). Distribution of a 20-mer phosphorothioate oligonucleotide, CGP69846A (ISIS 5132), into airway leukocytes and epithelial cells following intratracheal delivery to brown-norway rats. Pharm Res 16, 1542-1549.
38. Daniels, D. A., Sohal, A. K., Rees, S., Grisshammer, R. (2002). Generation of RNA aptamers to the G-protein-coupled receptor for neurotensin, NTS-1. Anal Biochem 305, 214-226.
39. Daniels, D. A., Chen, H., Hicke, B. J., Swiderek, K. M., Gold, L. (2003). A tenascin-C aptamer identified by tumor cell SELEX: systematic evolution of ligands by exponential enrichment. Proc Natl Acad Sci USA 100, 15416-15421.
40. Darfeuille, F., Hansen, J. B., Orum, H., Di Primo, C., Toulme, J. J. (2004). LNA/DNA chimeric oligomers mimic RNA aptamers targeted to the TAR RNA element of HIV-1. Nucleic Acids Res 32, 3101-3107.
41. Davis, J. H., Szostak, J. W. (2002). Isolation of high-affinity GTP aptamers from partially structured RNA libraries. Proc Natl Acad Sci USA 99, 11616-11621.
42. de Smidt, P. C., Le Doan, T., de Falco, S., van Berkel, T. J. (1991). Association of antisense nucleotides with lipoproteins prolongs the plasma half-life and modifies tissue distribution. Nucleic Acids Res 19, 4695-4700.
43. DeAnda, A., Jr., Coutre, S. E., Moon, M. R., Vial, C. M., Griffin, L. C., Law, V. S., Komeda, M., Leung, L. L., Miller, D. C. (1994). Pilot study of the efficacy of a thrombin inhibitor for use during cardiopulmonary bypass. Ann Thorac Surg 58, 344-350.
44. Doudna, J. A., Cech, T. R., Sullenger, B. A. (1995). Selection of an RNA molecule that mimics a major autoantigenic epitope of human insulin receptor. Proc Natl Acad Sci USA 92, 2355-2359.
45. Dougan, H., Lyster, D. M., Vo, C. V., Stafford, A., Weitz, J. I., Hobbs, J. B. (2000). Extending the lifetime of anticoagulant oligodeoxynucleotide aptamers in blood. Nucl Med Biol 27, 289-297.
46. Drachman, D. B. (1994). Myasthenia gravis. N Engl J Med 330, 1797-1810.
47. Drolet, D. W., Nelson, J., Tucker, C. E., Zack, P. M., Nixon, K., Bolin, R., Judkins, M. B., Farmer, J. A., Wolf, J. L., Gill, S. C., Bendele, R. A. (2000). Pharmacokinetics and safety of an anti-vascular endothelial growth factor aptamer (NX1838) following injection into the vitreous humor of rhesus monkeys. Pharm Res 17, 1503-1510.
48. Eaton, B. E. (1997). The joys of in vitro selection: chemically dressing oligonucleotides to satiate protein targets. Curr Opin Chem Biol 1, 10-16.
49. Eaton, B. E., Gold, L., Zichi, D. A. (1995). Let's get specific: the relationship between specificity and affinity. Chem Biol 2, 633-638.
50. Eaton, B. E., Gold, L., Hicke, B. J., Janjic, N., Jucker, F. M., Sebesta, D. P., Tarasow, T. M., Willis, M. C., Zichi, D. A. (1997). Post-SELEX combinatorial optimization of aptamers. Bioorg Med Chem 5, 1087-1096.
51. Ehsan, A., Mann, M. J., Dell'Acqua, G., 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.
52. Ellington, A. D., Szostak, J. W. (1990). In vitro selection of RNA molecules that bind specific ligands. Nature 346, 818-822.
53. Eyetech Study Group (2002). Preclinical and phase 1A clinical evaluation of an anti-VEGF pegylated aptamer (EYE001) for the treatment of exudative age-related macular degeneration. Retina 22, 143-152.
54. Eyetech Study Group (2003). Anti-vascular endothelial growth factor therapy for sub-foveal choroidal neovascularization secondary to age-related macular degeneration: phase II study results. Ophthalmology 110, 979-986.
55. Famulok, M., Blind, M., Mayer, G. (2001). Intramers as promising new tools in functional proteomics. Chem Biol 8, 931-939.
56. Ferrara, N. (2004). Vascular endothelial growth factor: basic science and clinical progress. Endocr Rev 25, 581-611.
57. Floege, J., Ostendorf, T., Janssen, U., Burg, M., Radeke, H. H., Vargeese, C., Gill, S. C., Green, L. S., Janjic, N. (1999). Novel approach to specific growth factor inhibition in vivo: antagonism of platelet-derived growth factor in glomerulonephritis by aptamers. Am J Pathol 154, 169-179.
58. Gal, S. W., Amontov, S., Urvil, P. T., Vishnuvardhan, D., Nishikawa, F., Kumar, P. K., Nishikawa, S. (1998). Selection of a RNA aptamer that binds to human activated protein C and inhibits its protease function. Eur J Biochem 252, 553-562.
59. Geary, R. S., Watanabe, T. A., Truong, L., Freier, S., Lesnik, E. A., Sioufi, N. B., Sasmor, H., Manoharan, M., Levin, A. A. (2001). Pharmacokinetic properties of 2l-O-(2methoxyethyl)-modified oligonucleotide analogs in rats. J Pharmacol Exp Ther 296, 890-897.
60. Geary, R. S., Henry, S. P., Grillone, L. R. (2002). Fomivirsen: clinical pharmacology and potential drug interactions. Clin Pharmacokinet 41, 255-260.
61. Gonzalez Ferreiro, M., Tillman, L. G., Hardee, G., Bodmeier, R. (2002). Alginate/poly-L-lysine microparticles for the intestinal delivery of antisense oligonucleotides. Pharm Res 19, 755-764.
62. Good, P. D., Krikos, A. J., Li, S. X., Bertrand, E., Lee, N. S., Giver, L., Ellington, A., Zaia, J. A., Rossi, J. J., Engelke, D. R. (1997). Expression of small, therapeutic RNAs in human cell nuclei. Gene Ther 4, 45-54.
63. Goodchild, J., Agrawal, S., Civeira, M. P., Sarin, P. S., Sun, D., Zamecnik, P. C. (1988). Inhibition of human immunodeficiency virus replication by antisense oligodeoxynucleotides. Proc Natl Acad Sci USA 85, 5507-5511.
64. Green, L. S., Jellinek, D., Bell, C., Beebe, L. A., Feistner, B. D., Gill, S. C., Jucker, F. M., Janjic, N. (1995). Nuclease-resistant nucleic acid ligands to vascular permeability factor/vascular endothelial growth factor. Chem Biol 2, 683-695.
65. Green, L. S., Jellinek, D., Jenison, R., Ostman, A., Heldin, C. H., Janjic, N. (1996). Inhibitory DNA ligands to platelet-derived growth factor B-chain. Biochemistry 35, 14413-14424.
66. Griffin, L. C., Tidmarsh, G. F., Bock, L. C., Toole, J. J., Leung, L. L. (1993). In vivo anticoagulant properties of a novel nucleotide-based thrombin inhibitor and demonstration of regional anticoagulation in extracorporeal circuits. Blood 81, 3271-3276.
67. Grindel, J. M., Musick, T. J., Jiang, Z., Roskey, A., Agrawal, S. (1998). Pharmacokinetics and metabolism of an oligodeoxynucleotide phosphorothioate (GEM91) in cynomolgus monkeys following intravenous infusion. Antisense Nucleic Acid Drug Dev 8, 43-52.
68. Harris, J. M., Chess, R. B. (2003). Effect of pegylation on pharmaceuticals. Nat Rev Drug Discov 2, 214-221.
69. Healy, J. M., Lewis, S. D., Kurz, M., Boomer, R. M., Thompson, K. T., Wilson, C., McCauley, T. G. (2004). Pharmacokinetics and biodistribution of novel aptamer compositions. Pharm Res 21, 2234-2246.
70. Held, H. A., Benner, S. A. (2002). Challenging artificial genetic systems: thymidine analogs with 5-position sulfur functionality. Nucleic Acids Res 30, 3857-3869.
71. Heldin, C. H., Westermark, B. (1999). Mechanism of action and in vivo role of platelet-derived growth factor. Physiol Rev 79, 1283-1316.
72. Hellstrom, M., Gerhardt, H., Kalen, M., Li, X., Eriksson, U., Wolburg, H., Betsholtz, C. (2001). Lack of pericytes leads to endothelial hyperplasia and abnormal vascular morphogenesis. J Cell Biol 153, 543-553.
73. Heuchel, R., Berg, A., Tallquist, M., Ahlen, K., Reed, R. K., Rubin, K., Claesson-Welsh, L., Heldin, C. H., Soriano, P. (1999). Platelet-derived growth factor beta receptor regulates interstitial fluid homeostasis through phosphatidylinositol-3l kinase signaling. Proc Natl Acad Sci USA 96, 11410-11415.
74. Hicke, B. J., Stephens, A. W. (2000). Escort aptamers: a delivery service for diagnosis and therapy. J Clin Invest 106, 923-928.
75. Hicke, B. J., Watson, S. R., Koenig, A., Lynott, C. K., Bargatze, R. F., Chang, Y. F., Ringquist, S., Moon-McDermott, L., Jennings, S., Fitzwater, T. et al. (1996). DNA aptamers block L-selectin function in vivo. Inhibition of human lymphocyte trafficking in SCID mice. J Clin Invest 98, 2688-2692.
76. Hicke, B. J., Marion, C., Chang, Y. F., Gould, T., Lynott, C. K., Parma, D., Schmidt, P. G., Warren, S. (2001). Tenascin-C aptamers are generated using tumor cells and purified protein. J Biol Chem 276, 48644-48654.
77. Homann, M., Goringer, H. U. (1999). Combinatorial selection of high affinity RNA ligands to live African trypanosomes. Nucleic Acids Res 27, 2006-2014.
78. Homann, M., Goringer, H. U. (2001). Uptake and intracellular transport of RNA aptamers in African trypanosomes suggest therapeutic "piggy-back" approach. Bioorg Med Chem 9, 2571-2580.
79. Huang, D. B., Vu, D., Cassiday, L. A., Zimmerman, J. M., Maher, L. J., III, Ghosh, G. (2003). Crystal structure of NF-kappaB (p50)2 complexed to a high-affinity RNA aptamer. Proc Natl Acad Sci USA 100, 9268-9273.
80. Huang, Y., Eckstein, F., Padilla, R., Sousa, R. (1997). Mechanism of ribose 2l-group discrimination by an RNA polymerase. Biochemistry 36, 8231-8242.
81. Hwang, B., Han, K., Lee, S. W. (2003). Prevention of passively transferred experimental autoimmune myasthenia gravis by an in vitro selected RNA aptamer. FEBS Lett 548, 85-89.
82. Jaaskelainen, I., Peltola, S., Honkakoski, P., Monkkonen, J., Urtti, A. (2000). A lipid carrier with a membrane active component and a small complex size are required for efficient cellular delivery of anti-sense phosphorothioate oligonucleotides. Eur J Pharm Sci 10, 187-193.
83. Jaeger, J., Restle, T., Steitz, T. A. (1998). The structure of HIV-1 reverse transcriptase complexed with an RNA pseudoknot inhibitor. EMBO J 17, 4535-4542.
84. Janjic, N., Gold, L. (2004). High affinity vascular endothelial growth factor (VEGF) receptor nucleic acid ligands and inhibitors. US Patent 6, 762, 290.
85. Jellinek, D., Green, L. S., Bell, C., Janjic, N. (1994). Inhibition of receptor binding by high-affinity RNA ligands to vascular endothelial growth factor. Biochemistry 33, 10450-10456.
86. Jellinek, D., Green, L. S., Bell, C., Lynott, C. K., Gill, N., Vargeese, C., Kirschenheuter, G., McGee, D. P., Abesinghe, P., Pieken, W. A. et al. (1995). Potent 2l-amino-2l-deoxypyrimidine RNA inhibitors of basic fibroblast growth factor. Biochemistry 34, 11363-11372.
87. Jenison, R. D., Jennings, S. D., Walker, D. W., Bargatze, R. F., Parma, D. (1998). Oligonucleotide inhibitors of P-selectin-dependent neutrophil-platelet adhesion. Antisense Nucleic Acid Drug Devel 8, 265-279.
88. Jeong, J. H., Kim, S. W., Park, T. G. (2003). Novel intracellular delivery system of antisense oligonucleotide by self-assembled hybrid micelles composed of DNA/PEG conjugate and cationic fusogenic peptide. Bioconjug Chem 14, 473-479.
89. Jeter, M. L., Ly, L. V., Fortenberry, Y. M., Whinna, H. C., White, R. R., Rusconi, C. P., Sullenger, B. A., Church, F. C. (2004). RNA aptamer to thrombin binds anion-binding exosite-2 and alters protease inhibition by heparin-binding serpins. FEBS Lett 568, 10-14.
90. Jones, D. S., Hachmann, J. P., Osgood, S. A., Hayag, M. S., Barstad, P. A., Iverson, G. M., Coutts, S. M. (1994). Conjugates of double-stranded oligonucleotides with poly(ethylene glycol) and keyhole limpet hemocyanin: a model for treating systemic lupus erythematosus. Bioconjug Chem 5, 390-399.
91. Karande, P., Jain, A., Mitragotri, S. (2004). Discovery of transdermal penetration enhancers by high-throughput screening. Nat Biotechnol 22, 192-197.
92. Kawaguchi, T., Asakawa, H., Tashiro, Y., Juni, K., Sueishi, T. (1995). Stability, specific binding activity, and plasma concentration in mice of an oligodeoxynucleotide modified at 5l-terminal with poly(ethylene glycol). Biol Pharm Bull 18, 474-476.
93. Kensch, O., Connolly, B. A., Steinhoff, H. J., McGregor, A., Goody, R. S., Restle, T. (2000). HIV-1 reverse transcriptase-pseudoknot RNA aptamer interaction has a binding affinity in the low picomolar range coupled with high specificity. J Biol Chem 275, 18271-18278.
94. Khati, M., Schuman, M., Ibrahim, J., Sattentau, Q., Gordon, S., James, W. (2003). Neutralization of infectivity of diverse R5 clinical isolates of human immunodeficiency virus type 1 by gp120-binding 2lF-RNA aptamers. J Virol 77, 12692-12698.
95. Kim, Y. M., Choi, K. H., Jang, Y. J., Yu, J., Jeong, S. (2003). Specific modulation of the anti-DNA autoantibody-nucleic acids interaction by the high affinity RNA aptamer. Biochem Biophys Res Commun 300, 516-523.
96. Kimoto, M., Shirouzu, M., Mizutani, S., Koide, H., Kaziro, Y., Hirao, I., Yokoyama, S. (2002). Anti-(Raf-1) RNA aptamers that inhibit Ras-induced Raf-1 activation. Eur J Biochem 269, 697-704.
97. King, D. J., Bassett, S. E., Li, X., Fennewald, S. A., Herzog, N. K., Luxon, B. A., Shope, R., Gorenstein, D. G. (2002). Combinatorial selection and binding of phosphorothioate aptamers targeting human NF-kappa B RelA(p65) and p50. Biochemistry 41, 9696-9706.
98. Klickstein, L., Moore, D., Atkinson, J. (2001). Therapeutic Immunology. Oxford: Blackwell Science, pp. 287-301.
99. Kraus, E., James, W., Barclay, A. N. (1998). Cutting edge: novel RNA ligands able to bind CD4 antigen and inhibit CD4+ T lymphocyte function. J Immunol 160, 5209-5212.
100. Kubik, M. F., Bell, C., Fitzwater, T., Watson, S. R., Tasset, D. M. (1997). Isolation and characterization of 2l-fluoro-, 2l-amino-, and 2l-fluoro-/amino-modified RNA ligands to human IFN-gamma that inhibit receptor binding. J Immunol 159, 259-267.
101. Kumar, P. K., Machida, K., Urvil, P. T., Kakiuchi, N., Vishnuvardhan, D., Shimotohno, K., Taira, K., Nishikawa, S. (1997). Isolation of RNA aptamers specific to the NS3 protein of hepatitis C virus from a pool of completely random RNA. Virology 237, 270-282.
102. Kumar, R., Singh, S. K., Koshkin, A. A., Rajwanshi, V. K., Meldgaard, M., Wengel, J. (1998). The first analogues of LNA (locked nucleic acids): phosphorothioate-LNA and 2l-thio-LNA. Bioorg Med Chem Lett 8, 2219-2222.
103. Kwon, Y. (2001). Handbook of Essential Pharmacokinetics, Pharmacodynamics, and Drug Metabolism for the Industrial Scientist. New York: Kluwer/Plenum, p. 89.
104. Latham, J. A., Johnson, R., Toole, J. J. (1994). The application of a modified nucleotide in aptamer selection: novel thrombin aptamers containing 5-(1-pentynyl)-2'-deoxyuridine. Nucleic Acids Res 22, 2817-2822.
105. Lato, S. M., Ozerova, N. D., He, K., Sergueeva, Z., Shaw, B. R., Burke, D. H. (2002). Boron-containing aptamers to ATP. Nucleic Acids Res 30, 1401-1407.
106. Lebedeva, I., Benimetskaya, L., Stein, C. A., Vilenchik, M. (2000). Cellular delivery of antisense oligonucleotides. Eur J Pharm Biopharm 50, 101-119.
107. Lebruska, L. L., Maher, L. J., III (1999). Selection and characterization of an RNA decoy for transcription factor NF-kappa B. Biochemistry 38, 3168-3174.
108. Lee, S. W., Sullenger, B. A. (1996). Isolation of a nuclease-resistant decoy RNA that selectively blocks autoantibody binding to insulin receptors on human lymphocytes. J Exp Med 184, 315-324.
109. Lee, S. W., Sullenger, B. A. (1997). Isolation of a nuclease-resistant decoy RNA that can protect human acetylcholine receptors from myasthenic antibodies. Nat Biotechnol 15, 41-45.
110. Lee, W. A., Fishback, J. A., Shaw, J. P., Bock, L. C., Griffin, L. C., Cundy, K. C. (1995). A novel oligodeoxynucleotide inhibitor of thrombin. II. Pharmacokinetics in the cynomolgus monkey. Pharm Res 12, 1943-1947.
111. Leppanen, O., Janjic, N., Carlsson, M. A., Pietras, K., Levin, M., Vargeese, C., Green, L. S., Bergqvist, D., Ostman, A., Heldin, C. H. (2000). Intimal hyperplasia recurs after removal of PDGF-AB and-BB inhibition in the rat carotid artery injury model. Arterioscler Thromb Vasc Biol 20, E89-95.
112. Leveen, P., Pekny, M., Gebre-Medhin, S., Swolin, B., Larsson, E., Betsholtz, C. (1994). Mice deficient for PDGF B show renal, cardiovascular, and hematological abnormalities. Genes Dev 8, 1875-1887.
113. Lin, Y., Padmapriya, A., Morden, K. M., Jayasena, S. D. (1995). Peptide conjugation to an in vitro-selected DNA ligand improves enzyme inhibition. Proc Natl Acad Sci USA 92, 11044-11048.
114. Lin, Y., Nieuwlandt, D., Magallanez, A., Feistner, B., Jayasena, S. D. (1996). High-affinity and specific recognition of human thyroid stimulating hormone (hTSH) by in vitro-selected 2l-amino-modified RNA. Nucleic Acids Res 24, 3407-3414.
115. Lobov, I. B., Brooks, P. C., Lang, R. A. (2002). Angiopoietin-2 displays VEGF-dependent modulation of capillary structure and endothelial cell survival in vivo. Proc Natl Acad Sci USA 99, 11205-11210.
116. Lopes, D., Mayer, L. D. (2002). Pharmacokinetics of Bcl-2 antisense oligonucleotide (G3139) combined with doxorubicin in SCID mice bearing human breast cancer solid tumor xenografts. Cancer Chemother Pharmacol 49, 57-68.
117. Lorger, M., Engstler, M., Homann, M., Goringer, H. U. (2003). Targeting the variable surface of African trypanosomes with variant surface glycoprotein-specific, serum-stable RNA aptamers. Eukaryot Cell 2, 84-94.
118. Lupold, S. E., Hicke, B. J., Lin, Y., Coffey, D. S. (2002). Identification and characterization of nuclease-stabilized RNA molecules that bind human prostate cancer cells via the prostate-specific membrane antigen. Cancer Res 62, 4029-4033.
119. Makrides, S. C. (1998). Therapeutic inhibition of the complement system. Pharmacol Rev 50, 59-87.
120. 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.
121. Manoharan, M., Tivel, K. L., Ross, B., Cook, P.D. (1994). A 2l-O-thiol tether in the ribose moiety of nucleic acids for conjugation chemistry. Gene 149, 147-156.
122. Masud, M. M., Kuwahara, M., Ozaki, H., Sawai, H. (2004). Sialyllactose-binding modified DNA aptamer bearing additional functionality by SELEX. Bioorg Med Chem 12, 1111-1120.
123. Mayer, G., Blind, M., Nagel, W., Bohm, T., Knorr, T., Jackson, C. L., Kolanus, W., Famulok, M. (2001). Controlling small guanine-nucleotide-exchange factor function through cytoplasmic RNA intramers. Proc Natl Acad Sci USA 98, 4961-4965.
124. Mehta, R. C., Stecker, K. K., Cooper, S. R., Templin, M. V., Tsai, Y. J., Condon, T. P., Bennett, C. F., Hardee, G. E. (2000). Intercellular adhesion molecule-1 suppression in skin by topical delivery of anti-sense oligonucleotides. J Invest Dermatol 115, 805-812.
125. Modi, N. (2002). In: Pharmacokinetics in Drug Discovery and Development, Schoenwald, R. D., ed. New Yory: CRC Press, p. 66.
126. Monteith, D. K., Levin, A. A. (1999). Synthetic oligonucleotides: the development of antisense therapeutics. Toxicol Pathol 27, 8-13.
127. Mordenti, J. (1985) Forecasting cephalosporin and monobactam antibiotic half-lives in humans from data collected in laboratory animals. Antimicrob. Agents Chemother. 27, 887-891.
128. Moulton, H. M., Hase, M. C., Smith, K. M., Iversen, P. L. (2003). HIV Tat peptide enhances cellular delivery of antisense morpholino oligomers. Antisense Nucleic Acid Drug Dev 13, 31-43.
129. Nicklin, P. L., Bayley, D., Giddings, J., Craig, S. J., Cummins, L. L., Hastewell, J. G., Phillips, J. A. (1998). Pulmonary bioavailability of a phosphorothioate oligonucleotide (CGP 64128A): comparison with other delivery routes. Pharm Res 15, 583-591.
130. Nieuwlandt, D., Wecker, M., Gold, L. (1995). In vitro selection of RNA ligands to substance P. Biochemistry 34, 5651-5659.
131. Nobile, V., Russo, N., Hu, G., Riordan, J. F. (1998). Inhibition of human angiogenin by DNA aptamers: nuclear colocalization of an angiogenin-inhibitor complex. Biochemistry 37, 6857-6863.
132. Nyce, J. W., Metzger, W. J. (1997). DNA antisense therapy for asthma in an animal model. Nature 385, 721-725.
133. O'Connell, D., Koenig, A., Jennings, S., Hicke, B., Han, H. L., Fitzwater, T., Chang, Y. F., Varki, N., Parma, D., Varki, A. (1996). Calcium-dependent oligonucleotide antagonists specific for L-selectin. Proc Natl Acad Sci USA 93, 5883-5887.
134. Ono, T., Scalf, M., Smith, L. M. (1997). 2lFluoro modified nucleic acids: polymerase-directed synthesis, properties and stability to analysis by matrix-assisted laser desorption/ionization mass spectrometry. Nucleic Acids Res 25, 4581-4588.
135. Ostendorf, T., Kunter, U., Grone, H. J., Bahlmann, F., Kawachi, H., Shimizu, F., Koch, K. M., Janjic, N., Floege, J. (2001). Specific antagonism of PDGF prevents renal scarring in experimental glomerulonephritis. J Am Soc Nephrol 12, 909-918.
136. Padilla, R. and Sousa, R. (1999). Efficient synthesis of nucleic acids heavily modified with non-canonical ribose 2'-groups using a mutant T7 RNA polymerase (RNAP). Nucleic Acids Res. 27, 1561-1563.
137. Pagratis, N. C., Bell, C., Chang, Y. F., Jennings, S., Fitzwater, T., Jellinek, D., Dang, C. (1997). Potent 2l-amino-, and 2l-fluoro2l-deoxyribonucleotide RNA inhibitors of keratinocyte growth factor. Nat Biotechnol 15, 68-73.
138. Pagratis, N., Lochrie, M., Gold, L. (2002). High affinity TGFb nucleic acid ligands and inhibitors. US Patent Number 63, 466, 111.
139. Peng, B., Andrews, J., Nestorov, I., Brennan, B., Nicklin, P., Rowland, M. (2001). Tissue distribution and physiologically based pharmacokinetics of antisense phosphorothioate oligonucleotide ISIS 1082 in rat. Antisense Nucleic Acid Drug Dev 11, 15-27.
140. Pichon, C., Roufai, M. B., Monsigny, M., Midoux, P. (2000). Histidylated oligolysines increase the transmembrane passage and the biological activity of antisense oligonucleotides. Nucleic Acids Res 28, 504-512.
141. Pietersz, G. A., Li, W., Apostolopoulos, V. (2001). A 16-mer peptide (RQIKIWFQNRRMKWKK) from antennapedia preferentially targets the Class I pathway. Vaccine 19, 1397-1405.
142. Pietras, K., Ostman, A., Sjoquist, M., Buchdunger, E., Reed, R. K., Heldin, C. H., Rubin, K. (2001). Inhibition of platelet-derived growth factor receptors reduces interstitial hypertension and increases transcapillary transport in tumors. Cancer Res 61, 2929-2934.
143. Pietras, K., Rubin, K., Sjoblom, T., Buchdunger, E., Sjoquist, M., Heldin, C. H., Ostman, A. (2002). Inhibition of PDGF receptor signaling in tumor stroma enhances antitumor effect of chemotherapy. Cancer Res 62, 5476-5484.
144. Pietras, K., Sjoblom, T., Rubin, K., Heldin, C. H., Ostman, A. (2003). PDGF receptors as cancer drug targets. Cancer Cell 3, 439-443.
145. Plater-Zyberk, C., Buckton, J., Thompson, S., Spaull, J., Zanders, E., Papworth, J., Life, P.F. (2001). Amelioration of arthritis in two murine models using antibodies to oncostatin M. Arthritis Rheum 44, 2697-2702.
146. Proske, D., Hofliger, M., Soll, R. M., Beck-Sickinger, A. G., Famulok, M. (2002). A Y2 receptor mimetic aptamer directed against neuropeptide Y. J Biol Chem 277, 11416-11422.
147. Putney, S. D., Brown, J., Cucco, C., Lee, R., Skorski, T., Leonetti, C., Geiser, T., Calabretta, B., Zupi, G., Zon, G. (1999). Enhanced anti-tumor effects with microencapsulated c-myc antisense oligonucleotide. Antisense Nucleic Acid Drug Dev 9, 451-458.
148. Raoof, A. A., Ramtoola, Z., McKenna, B., Yu, R. Z., Hardee, G., Geary, R. S. (2002). Effect of sodium caprate on the intestinal absorption of two modified antisense oligonucleotides in pigs. Eur J Pharm Sci 17, 131-138.
149. Reyderman, L., Stavchansky, S. (1997). Quantitative determination of short single-stranded oligonucleotides from blood plasma using capillary electrophoresis with laser-induced fluorescence. Anal Chem 69, 3218-3222.
150. Rhie, A., Kirby, L., Sayer, N., Wellesley, R., Disterer, P., Sylvester, I., Gill, A., Hope, J., James, W., Tahiri-Alaoui, A. (2003). Characterization of 2l-fluoro-RNA aptamers that bind preferentially to disease-associated conformations of prion protein and inhibit conversion. J Biol Chem 278, 39697-39705.
151. Rhodes, A., Deakin, A., Spaull, J., Coomber, B., Aitken, A., Life, P., Rees, S. (2000). The generation and characterization of antagonist RNA aptamers to human oncostatin M. J Biol Chem 275, 28555-28561.
152. Rhodes, A., Smithers, N., Chapman, T., Parsons, S., Rees, S. (2001). The generation and characterisation of antagonist RNA aptamers to MCP-1. FEBS Lett 506, 85-90.
153. Richardson, F. C., Tennant, B. C., Meyer, D. J., Richardson, K. A., Mann, P. C., McGinty, G. R., Wolf, J. L., Zack, P. M., Bendele, R. A. (1999). An evaluation of the toxicities of 2l-fluorouridine and 2l-fluorocytidine-HCl in F344 rats and woodchucks (Marmota monax). Toxicol Pathol 27, 607-617.
154. Richardson, F. C., Kuchta, R. D., Mazurkiewicz, A., Richardson, K. A. (2000). Polymerization of 2l-fluoro-and 2l-O-methyldNTPs by human DNA polymerase alpha, polymerase gamma, and primase. Biochem Pharmacol 59, 1045-1052.
155. Richardson, F. C., Zhang, C., Lehrman, S. R., Koc, H., Swenberg, J. A., Richardson, K. A., Bendele, R. A. (2002). Quantification of 2l-fluoro-2l-deoxyuridine and 2l-fluoro-2ldeoxycytidine in DNA and RNA isolated from rats and woodchucks using LC/MS/MS. Chem Res Toxicol 15, 922-926.
156. Ringquist, S., Parma, D. (1998). Anti-L-selectin oligonucleotide ligands recognize CD62L-positive leukocytes: binding affinity and specificity of univalent and bivalent ligands. Cytometry 33, 394-405.
157. Roth, R. A., Maddux, B. A., Cassell, D. J., Goldfine, I. D. (1983). Regulation of the insulin receptor by a monoclonal anti-receptor antibody. Evidence that receptor down regulation can be independent of insulin action. J Biol Chem 258, 12094-12097.
158. Rothbard, J. B., Kreider, E., VanDeusen, C. L., Wright, L., Wylie, B. L., Wender, P. A. (2002). Arginine-rich molecular transporters for drug delivery: role of backbone spacing in cellular uptake. J Med Chem 45, 3612-3618.
159. Ruckman, J., Green, L. S., Beeson, J., Waugh, S., Gillette, W. L., Henninger, D. D., Claesson-Welsh, L., Janjic, N. (1998). 2l-Fluoropyrimidine RNA-based aptamers to the 165amino 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.
160. Rusconi, C. P., Yeh, A., Lyerly, H. K., Lawson, J. H., Sullenger, B. A. (2000). Blocking the initiation of coagulation by RNA aptamers to factor VIIa. Thromb Haemost 84, 841-848.
161. Rusconi, C. P., Scardino, E., Layzer, J., Pitoc, G. A., Ortel, T. L., Monroe, D., Sullenger, B.A. (2002). RNA aptamers as reversible antagonists of coagulation factor IXa. Nature 419, 90-94.
162. Rusconi, C. P., Roberts, J. D., Pitoc, G. A., Nimjee, S. M., White, R. R., Quick, G., Jr., Scardino, E., Fay, W. P., Sullenger, B. A. (2004). Antidote-mediated control of an anticoagulant aptamer in vivo. Nat Biotechnol 22, 1423-1428.
163. Saitoh, H., Nakamura, A., Kuwahara, M., Ozaki, H., Sawai, H. (2002). Modified DNA aptamers against sweet agent aspartame. Nucleic Acids Res Suppl, 215-216.
164. Sakamoto, T., Miyazaki, E., Aramaki, Y., Arima, H., Takahashi, M., Kato, Y., Koga, M., Tsuchiya, S. (2004). Improvement of dermatitis by iontophoretically delivered antisense oligonucleotides for interleukin10 in NC/Nga mice. Gene Ther 11, 317-324.
165. Sandrasagra, A., Leonard, S. A., Tang, L., Teng, K., Li, Y., Ball, H. A., Mannion, J. C., Nyce, J. W. (2002). Discovery and development of respirable antisense therapeutics for asthma. Antisense Nucleic Acid Drug Dev 12, 177-181.
166. Santulli-Marotto, S., Nair, S. K., Rusconi, C., Sullenger, B., Gilboa, E. (2003). Multivalent RNA aptamers that inhibit CTLA-4 and enhance tumor immunity. Cancer Res 63, 7483-7489.
167. Seiwert, S. D., Stines Nahreini, T., Aigner, S., Ahn, N. G., Uhlenbeck, O. C. (2000). RNA aptamers as pathway-specific MAP kinase inhibitors. Chem Biol 7, 833-843.
168. Shadidi, M., Sioud, M. (2003). Identification of novel carrier peptides for the specific delivery of therapeutics into cancer cells. FASEB J 17, 256-258.
169. Shargel, L., Yu, A. (1999). Applied Biopharmaceutics and Pharmacokinetics. New York: McGraw-Hill, pp. 108-154.
170. Shaw, J. P., Fishback, J. A., Cundy, K. C., Lee, W. A. (1995). A novel oligodeoxynucleotide inhibitor of thrombin. I. In vitro metabolic stability in plasma and serum. Pharm Res 12, 1937-1942.
171. Shea, R. G., Marsters, J. C., Bischofberger, N. (1990). Synthesis, hybridization properties and antiviral activity of lipid-oligodeoxynucleotide conjugates. Nucleic Acids Res 18, 3777-3783.
172. Shi, H., Hoffman, B. E., Lis, J. T. (1999). RNA aptamers as effective protein antagonists in a multicellular organism. Proc Natl Acad Sci USA 96, 10033-10038.
173. Schmidt, K. S., Borkowski, S., Kurreck, J., Stephens, A. W., Hecht, M., Friebe, M., Dinkelborg, L., Erdmann, V. A. (2004). Application of locked nucleic acids to improve aptamer in vivo stability and targeting function. Nucleic Acids Res 32, 5757-5765.
174. Smith, D., Kirschenheuter, G. P., Charlton, J., Guidot, D. M., Repine, J. E. (1995). In vitro selection of RNA-based irreversible inhibitors of human neutrophil elastase. Chem Biol 2, 741-750.
175. Smith, C.J. and Steitz, T.A. (1997). Sno storm in the nucleolus: new roles for myriad small RNPs. Cell 89, 669-672.
176. Solon, E. G., Kraus, L. (2001). Quantitative whole-body autoradiography in the pharmaceutical industry. Survey results on study design, methods, and regulatory compliance. J Pharmacol Toxicol Methods 46, 73-81.
177. Solon, E. G., Balani, S. K., Lee, F. W. (2002). Whole-body autoradiography in drug discovery. Curr Drug Metab 3, 451-462.
178. Srinivasan, S. K., Iversen, P. (1995). Review of in vivo pharmacokinetics and toxicology of phosphorothioate oligonucleotides. J Clin Lab Anal 9, 129-137.
179. Stein, C. A., Subasinghe, C., Shinozuka, K., Cohen, J. S. (1988). Physicochemical properties of phosphorothioate oligodeoxynucleotides. Nucleic Acids Res 16, 3209-3221.
180. Stevenson, M., Iversen, P. L. (1989). Inhibition of human immunodeficiency virus type 1-mediated cytopathic effects by poly(L-lysine)-conjugated synthetic antisense oligodeoxyribonucleotides. J Gen Virol 70 (Pt 10), 2673-2682.
181. Sullenger, B. A., Gilboa, E. (2002). Emerging clinical applications of RNA. Nature 418, 252-258.
182. Sussman, H. E. (2003). Success for oral antisense therapy. Drug Discov Today 8, 516-517.
183. Sutton, B. J., Gould, H. J. (1993). The human IgE network. Nature 366, 421-428.
184. Symensma, T. L., Giver, L., Zapp, M., Takle, G. B., Ellington, A. D. (1996). RNA aptamers selected to bind human immunodeficiency virus type 1 Rev in vitro are Rev responsive in vivo. J Virol 70, 179-187.
185. Tam, R. C., Wu-Pong, S., Pai, B., Lim, C., Chan, A., Thomas, D. F., Milovanovic, T., Bard, J., Middleton, P. J. (1999). Increased potency of an aptameric G-rich oligonucleotide is associated with novel functional properties of phosphorothioate linkages. Antisense Nucleic Acid Drug Dev 9, 289-300.
186. Tasset, D. M., Kubik, M. F., Steiner, W. (1997). Oligonucleotide inhibitors of human thrombin that bind distinct epitopes. J Mol Biol 272, 688-698.
187. Templin, M. V., Levin, A. A., Graham, M. J., Aberg, P. M., Axelsson, B. I., Butler, M., Geary, R. S., Bennett, C. F. (2000). Pharmacokinetic and toxicity profile of a phosphorothioate oligonucleotide following inhalation delivery to lung in mice. Antisense Nucleic Acid Drug Dev 10, 359-368.
188. Thomas, M., Chedin, S., Carles, C., Riva, M., Famulok, M., Sentenac, A. (1997). Selective targeting and inhibition of yeast RNA polymerase II by RNA aptamers. J Biol Chem 272, 27980-27986.
189. Tsai, D. E., Keene, J. D. (1993). In vitro selection of RNA epitopes using autoimmune patient serum. J Immunol 150, 1137-1145.
190. Tucker, C. E., Chen, L. S., Judkins, M. B., Farmer, J. A., Gill, S. C., Drolet, D. W. (1999). Detection and plasma pharmacokinetics of an anti-vascular endothelial growth factor oligonucleotide-aptamer (NX1838) in rhesus monkeys. J Chromatogr B Biomed Sci Appl 732, 203-212.
191. Tuerk, C., Gold, L. (1990). Systematic evolution of ligands by exponential enrichment: RNA ligands to bacteriophage T4 DNA polymerase. Science 249, 505-510.
192. Tuerk, C., MacDougal-Waugh, S. (1993). In vitro evolution of functional nucleic acids: high-affinity RNA ligands of HIV-1 proteins. Gene 137, 33-39.
193. Tuerk, C., MacDougal, S., Gold, L. (1992). RNA pseudoknots that inhibit human immunodeficiency virus type 1 reverse transcriptase. Proc Natl Acad Sci USA 89, 6988-6992.
194. Ulrich, H., Magdesian, M. H., Alves, M. J., Colli, W. (2002). In vitro selection of RNA aptamers that bind to cell adhesion receptors of Trypanosoma cruzi and inhibit cell invasion. J Biol Chem 277, 20756-20762.
195. Vaish, N. K., Larralde, R., Fraley, A. W., Szostak, J. W., McLaughlin, L. W. (2003). A novel, modification-dependent ATP-binding aptamer selected from an RNA library incorporating a cationic functionality. Biochemistry 42, 8842-8851.
196. Vant-Hull, B., Payano-Baez, A., Davis, R. H., Gold, L. (1998). The mathematics of SELEX against complex targets. J Mol Biol 278, 579-597.
197. Vater, A., Klussmann, S. (2003). Toward third-generation aptamers: Spiegelmers and their therapeutic prospects. Curr Opin Drug Discov Devel 6, 253-261.
198. Vinogradov, S. V., Suzdaltseva, Y. G., Kabanov, A. V. (1996). Block polycationic oligonucleotide derivative: synthesis and inhibition of herpes virus reproduction. Bioconjug Chem 7, 3-6.
199. Vives, E., Brodin, P., Lebleu, B. (1997). A truncated HIV-1 Tat protein basic domain rapidly translocates through the plasma membrane and accumulates in the cell nucleus. J Biol Chem 272, 16010-16017.
200. Wang, H., Cai, Q., Zeng, X., Yu, D., Agrawal, S., Zhang, R. (1999). Antitumor activity and pharmacokinetics of a mixed-backbone antisense oligonucleotide targeted to the RIalpha subunit of protein kinase A after oral administration. Proc Natl Acad Sci USA 96, 13989-13994.
201. Wang, K. Y., McCurdy, S., Shea, R. G., Swaminathan, S., Bolton, P. H. (1993). A DNA aptamer which binds to and inhibits thrombin exhibits a new structural motif for DNA. Biochemistry 32, 1899-1904.
202. Warkentin, T. E., Greinacher, A. (2003). Heparin-induced thrombocytopenia and cardiac surgery. Ann Thorac Surg 76, 2121-2131.
203. Watson, S. R., Chang, Y. F., O'Connell, D., Weigand, L., Ringquist, S., Parma, D. H. (2000). Anti-L-selectin aptamers: binding characteristics, pharmacokinetic parameters, and activity against an intravascular target in vivo. Antisense Nucleic Acid Drug Dev 10, 63-75.
204. White, P. J., Gray, A. C., Fogarty, R. D., Sinclair, R. D., Thumiger, S. P., Werther, G. A., Wraight, C. J. (2002). C-5 propyne-modified oligonucleotides penetrate the epidermis in psoriatic and not normal human skin after topical application. J Invest Dermatol 118, 1003-1007.
205. White, R., Rusconi, C., Scardino, E., Wolberg, A., Lawson, J., Hoffman, M., Sullenger, B. (2001). Generation of species cross-reactive aptamers using "toggle" SELEX. Mol Ther 4, 567-573.
206. White, R. R., Shan, S., Rusconi, C. P., Shetty, G., Dewhirst, M. W., Kontos, C. D., Sullenger, B. A. (2003). Inhibition of rat corneal angiogenesis by a nuclease-resistant RNA aptamer specific for angiopoietin-2. Proc Natl Acad Sci USA 100, 5028-5033.
207. Wiegand, T. W., Williams, P. B., Dreskin, S. C., Jouvin, M. H., Kinet, J. P., Tasset, D. (1996). High-affinity oligonucleotide ligands to human IgE inhibit binding to Fc epsilon receptor I. J Immunol 157, 221-230.
208. Williams, K. P., Liu, X. H., Schumacher, T. N., Lin, H. Y., Ausiello, D. A., Kim, P. S., Bartel, D. P. (1997). Bioactive and nuclease-resistant L-DNA ligand of vasopressin. Proc Natl Acad Sci USA 94, 11285-11290.
209. Willis, M. C., Collins, B. D., Zhang, T., Green, L. S., Sebesta, D. P., Bell, C., Kellogg, E., Gill, S. C., Magallanez, A., Knauer, S. et al. (1998). Liposome-anchored vascular endothelial growth factor aptamers. Bioconjug Chem 9, 573-582.
210. Wlotzka, B., Leva, S., Eschgfaller, B., Burmeister, J., Kleinjung, F., Kaduk, C., Muhn, P., Hess-Stumpp, H., Klussmann, S. (2002). In vivo properties of an anti-GnRH Spiegelmer: an example of an oligonucleotide-based therapeutic substance class. Proc Natl Acad Sci USA 99, 8898-8902.
211. Yamaoka, T., Tabata, Y., Ikada, Y. (1994). Distribution and tissue uptake of poly(ethylene glycol) with different molecular weights after intravenous administration to mice. J Pharm Sci 83, 601-606.
212. Yamaoka, T., Tabata, Y., Ikada, Y. (1995). Fate of water-soluble polymers administered via different routes. J Pharm Sci 84, 349-354.
213. Yang, X., Bassett, S. E., Li, X., Luxon, B. A., Herzog, N. K., Shope, R. E., Aronson, J., Prow, T. W., Leary, J. F., Kirby, R. et al. (2002). Construction and selection of bead-bound combinatorial oligonucleoside phosphorothioate and phosphorodithioate aptamer libraries designed for rapid PCR-based sequencing. Nucleic Acids Res 30, 132.
214. Yang, X., Li, X., Prow, T. W., Reece, L. M., Bassett, S. E., Luxon, B. A., Herzog, N. K., Aronson, J., Shope, R. E., Leary, J. F., Gorenstein, D. G. (2003). Immunofluorescence assay and flow-cytometry selection of bead-bound aptamers. Nucleic Acids Res 31, 54.
215. Younes, C. K., Boisgard, R., Tavitian, B. (2002). Labelled oligonucleotides as radiopharmaceuticals: pitfalls, problems and perspectives. Curr Pharm Des 8, 1451-1466.
216. Yu, R. Z., Baker, B., Chappell, A., Geary, R. S., Cheung, E., Levin, A. A. (2002). Development of an ultrasensitive noncompetitive hybridization-ligation enzyme-linked immunosorbent assay for the determination of phosphorothioate oligodeoxynucleotide in plasma. Anal Biochem 304, 19-25.
217. Zhang, B., Roth, R. A. (1991). A region of the insulin receptor important for ligand binding (residues 450-601) is recognized by patients' autoimmune antibodies and inhibitory monoclonal antibodies. Proc Natl Acad Sci USA 88, 9858-9862.
218. Zubin, E. M., Romanova, E. A., Volkov, E. M., Tashlitsky, V. N., Korshunova, G. A., Shabarova, Z. A., Oretskaya, T. S. (1999). Oligonucleotide-peptide conjugates as potential antisense agents. FEBS Lett 456, 59-62.