Progress in antisense oligonucleotide therapeutics

Annual Review of Pharmacology and Toxicology

Volumn 36, Issue , 1996, Pages 107-129

  Crooke, Stanley T ^a   Bennett, C Frank ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

DNA; medicinal chemistry; nucleosides; pharmacokinetics; RNA

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; OLIGODEOXYNUCLEOTIDE PHOSPHOROTHIOATE;

CANCER; CARDIOVASCULAR DISEASE; DRUG DEVELOPMENT; HUMAN; INFLAMMATION; INTRACEREBRAL DRUG ADMINISTRATION; INTRADERMAL DRUG ADMINISTRATION; INTRATUMORAL DRUG ADMINISTRATION; INTRAVENOUS DRUG ADMINISTRATION; NEUROLOGIC DISEASE; NONHUMAN; PHARMACODYNAMICS; PRIORITY JOURNAL; REVIEW; SUBCUTANEOUS DRUG ADMINISTRATION; TOPICAL DRUG ADMINISTRATION;

PRIMATES; RODENTIA;

EID: 0030000245     PISSN: 00664251     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev.pa.36.040196.000543     Document Type: Review

References (185)

1. Nielsen PE, Egholm M, Berg RH, Buchardt O. 1991. Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Science 254:1497-500
2. Mollegaard NE, Buchardt O, Egholm M, Nielsen PE. 1994. PNA-DNA strand displacement loops as artificial transcription promoters. Proc. Natl. Acad. Sci. USA 91:3892-95
3. Sun J-S, Helene C. 1993. Oligonucleotide-directed triple-helix formation. Curr. Opin. Struct. Biol. 3:345-56
4. Bielinska A, Shivdasani RA, Zhang L, Nabel GJ. 1990. Regulation of gene expression with double-stranded phosphorothioate oligonucleotides. Science 250:997-1000
5. Stein CA, Cheng YC. 1993. Antisense oligonucleotides as therapeutic agents - Is the bullet really magical? Science 261:1004-12
6. Wagner RW. 1994. Gene inhibition us-ing antisense oligodeoxynucleotides. Nature 372:333-35
7. Crooke ST. 1995. Therapeutic Applications of Oligonueleotides. Austin, TX: Landes
8. Breslauer KJ, Frank R, Blocker H, Marky LA. 1986. Predicting DNA duplex stability from base sequence. Proc. Natl. Acad. Sci. USA 83:3746-50
9. Freier SM, Kierzek R, Jaeger JA, Sugimoto N, Caruthers MH, et al. 1986. Improved free-energy parameters for predictions of RNA duplex stability. Proc. Natl. Acad Sci. USA 83:9373-77
10. Freier SM, Lima WF, Sanghvi YS, Vickers T, Zounes M, et al. 1992. Thermodynamics of antisense oligonucleotide hybridization. See Ref. 184, pp. 95-107
11. Thein SL, Wallace RB. 1986. The use of synthetic oligonucleotides as specific hybridization probes in the diagnosis of genetic disorders. In Human Genetic Diseases: A Practical Approach, ed. KE Davies, pp. 33-50. Oxford: IRL Press
12. Helene C, Tpulme J. 1989. Control of gene expression by oligonucleotides covalently linked to intercalating agents and nucleic acid-cleaving reagents. In Oligonucleotides: Antisense Inhibitors of Gene Expression, ed. JS Cohen, pp. 137-72. Boca Raton, FL: CRC Press
13. Helene C, Toulme J-J. 1990. Specific regulation of gene expression by antisense, sense and antigene nucleic acids. Biochim. Biophys. Acta 1049:99-125
14. Monia BP, Johnston JF, Ecker DJ, Zounes M, Lima WF, Freier SM. 1992. Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides. J. Biol. Chem. 267:19954-62
15. Crooke ST. 1993. Progress toward oligonucleotide therapeutics: pharmacodynamic properties. FASEB J. 7:533-39
16. Bennett CF, Crooke ST. 1994. Regulation of endothelial cell adhesion molecule expression with antisense oligonucleotides. Adv. Pharmacol. 28:1-43
17. Chiang M-Y, Chan H, Zounes MA, Freier SM, Lima WF, Bennett CF. 1991. Antisense oligonucleotides inhibit intercellular adhesion molecule 1 expression by two distinct mechanisms. J. Biol. Chem. 266:18162-71
18. Cowsert LM, Fox MC, Zoo G, Mirabelli CK. 1993. In vitro evaluation of phosphorothioate oligonucleotides targeted to the E2 mRNA of papillomavirus: potential treatment for genital warts. Antimicrob. Agents Chemother. 37:171-77
19. Dean NM, McKay R, Condon TP, Bennett CF. 1994. Inhibition of protein kinase C-α expression in human A549 cells by antisense oligonucleotides inhibits induction of intercellular adhesion molecule 1 (ICAM-1) mRNA by phorbol esters. J. Biol. Chem. 269:16416-24
20. Azad RF, Tanaka K, Anderson KP. 1993. Antiviral activity of a phosphorothioate oligonucleotide complementary to RNA of the human cytomegalovirus major immediate early region. Antimicrob. Agents Chemother. 37: 1945-54
21. Colige A, Sokolov BP, Nugent P, Baserga R, Prockop DJ. 1993. Use of antisense oligonucleotide to inhibit expression of a mutated human procollagen gene (COL1A1) in transfected mouse 3T3 cells. Biochemistry 32:7-11
22. Bennett CF, Condon T, Grimm S, Chan H, Chiang M-Y. 1994. Inhibition of endothelial cell-leukocyte adhesion molecule expression with antisense oligonucleotides. J. Immunol. 152:3530-40
23. Stepkowski SM, Tu Y, Condon TP, Bennett CF. 1994. Blocking of heart allograft rejection by intercellular adhesion molecule-1 antisense oligonucleotides alone or in combination with other immunosuppressive modalities. J. Immunol. 153:5336-46
24. Duff JL, Monia BP, Berk BC. 1995. Mitogen-activated protein (MAP) kinase is regulated by the MAP kinase phosphatase (MKP-1) in vascular smooth muscle cells. Effect of actinomycin D and antisense oligonucleotides. J. Biol. Chem. 270:7161-66
25. De Clercq E, Eckstein F, Sternbach H, Merigan TC. 1969. Interferon induction by and ribonuclease sensitivity of thio phosphate-substituted polyribonucleotides. Antimicrob. Agents Chemother. 9: 187-91
26. Campbell JM, Bacon TA, Wickstrom E. 1990. Oligodeoxynucleoside phosphorothioate stability in subcellular extracts, culture media, sera and cerebrospinal fluid. J. Biochem. Biophys. Methods 20:259-67
27. Akhtar S, Kole R, Juliano RL. 1991. Stability of antisense DNA oligodeoxynucleotide analogs in cellular extracts and sera. Life Sci. 49:1793-801
28. Shaw J-P, Kent K, Bird J, Fishback J, Froehler B. 1991. Modified deoxyoligonucleotides stable to exonuciease degradation in serum. Nucleic Acids Res. 19:747-50
29. Crooke ST. 1992. Therapeutic applications of oligonucleotides. Annu. Rev. Pharmacol. Toxicol. 32:329-76
30. Freier SM. 1993. Hybridization considerations affecting antisense drugs. See Ref. 185, pp. 67-82
31. Vickers T, Baker BF, Cook PD, Zounes M, Buckheit RW Jr, et al. 1991. Inhibition of HIV-LTR gene expression by oligonucleotides targeted to the TAR element. Nucleic Acids Res. 19:3359-68
32. Lima WF, Monia BP, Ecker DJ, Freier SM. 1992. Implication of RNA structure on antisense oligonucleotide hybridization kinetics. Biochemistry 31:12055-61
33. Wickstrom E. 1986. Oligodeoxynucleotide stability in subcellular extracts and culture media. J. Biochem. Biophys. Methods 13:97-102
34. Hoke GD, Draper K, Freier SM, Gonzalez C, Driver VB, et al. 1991. Effects of phosphorothioate capping on antisense oligonucleotide stability, hybridization and antiviral efficacy versus herpes simplex virus infection. Nucleic Acids Res. 19:5743-48
35. Crooke RM, Graham MJ, Cooke ME, Crooke ST. 1995. In vitro pharmacokinetics of pbosphorothioate antisense oligonucleotides. J. Pharmacol. Exp. Ther. In press
36. Gao W-Y, Han F-S, Storm C, Egan W, Cheng Y-C. 1992. Phosphorothioate oligonucleotides aie inhibitors of human DNA polymerases and RNase H: implications for antisense technology. Mol. Pharmacol. 41:223-29
37. Agrawal S, Temsamani J, Tang JY. 1991. Pharmacokinetics, biodistribution, and stability of oligodeoxynucleotide phosphorothioates in mice. Proc. Natl. Acad. Sci. USA 88:7595-99
38. 14C-labeled phosphorothioate oligonucleotide ISIS 2105 after intravenous administration to rats. J. Pharmacol. Exp. Ther. 261: 1181-40
39. 3H-labeled oligonucleotides. I. Comparison of a phosphodiester and a phosphorothioate. Mol. Pharmacol. 45:932-43
40. Loke SL, Stein CA, Zhang XH, Mori K, Nakanishi M, et al. 1989. Characterization of oligonucleotide transport into living cells. Proc. Natl. Acad. Sci. USA 86:3474-78
41. Neckers LM. 1993. Cellular internalization of oligodeoxynucleotides. See Ref. 185, pp. 451-60
42. Gao W-Y, Storm C, Egan W, Cheng Y-C. 1993. Cellular pharmacology of phosphorothioate homooligodeoxynucleotides in human cells. Mol. Pharmacol. 43:45-50
43. Iversen PL, Zhu S, Meyer A, Zon G. 1992. Cellular uptake and subcellular distribution of phosphorothioate oligonucleotides into cultured cells. Antisense Res. Dev. 2:211-22
44. Nestle FO, Mitra RS, Bennett CF, Chan H, Nickoloff BJ. 1994. Cationic lipid is not required for uptake and selective inhibitory activity of ICAM-1 phosphorothioate antisense oligonucleotides in keratinocytes. J. Invest. Dermatol. 103:569-75
45. Chrissey LA, Walz SE, Pazirandeh M. 1993. Internalization of oligodeoxyribonucleotides by vibrio parahemolyticus. Antisense Res. Dev. 3:367
46. Krieg AM, Gmelig-Meyling F, Gourley MF, Kisch WJ, Chrisey LA, Steinberg AD. 1991. Uptake of oligodeoxyribonucleotides by lymphoid cells is heterogenerous and inducible. Antisense Res. Dev. 1:161-71
47. Iversen PL, Crouse D, Zon G, Perry G. 1992. Binding of antisense phosphorothioate oligonucleotides to murine lymphocytes is lineage specific and inducible. Antisense Res. Dev. 2:223-33
48. Beltinger C, Saragovi HU, Smith RM, LeSauteur L, Shah N, et al. 1995. Binding, uptake, and intracellular trafficking of phosphorothioate-modified oligodeoxynucleotides. J. Clin. Invest. 95:1814-23
49. Noonberg SB, Garovoy MR, Hunt CA. 1993. Characteristics of oligonucleotide uptake in human keratinocyte cultures. J. Invest. Dermatol. 101:727-31
50. Bennett CF, Chiang M-Y, Chan H, Shoemaker JEE, Mirabelli CK. 1992. Cationic Eipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides. Mol. Pharmacol. 41:1023-33
51. Bennett CF, Chiang MY, Chan H, Grimm S. 1993. Use of cationic lipids to enhance the biological activity of antisense oligonucleotides. J. Liposome Res. 3:85-102
52. Greig MJ, Gaus H, Cummins LL, Sasmor H, Griffey RH. 1995. Measurement of macromolecular binding using electrospray mass spectrometry. Determination of dissociation constants for oligonucleotide: serum albumin complexes. J. Am. Chem. Soc. In press
53. Joos RW, Hall WH. 1969. Determination of binding constants of serum albumin for penicillin. J. Pharmacol. Exp. Ther. 166:113
54. Srinivasan SK, Iversen P. 1995. Review of in vivo pharmacokinetics and toxicology of phosphorothioate oligonucleotides. J. Clin. Lab. Anal. 9:129-37
55. 14C-labeled phosphorothioate oligonucleotide, ISIS 2105, after intradermal administration to rats. J. Pharmacol. Exp. Ther. 269: 89-94
56. 14C-labeled afovirsen sodium in patients with genital warts. Clin. Pharmacol. Ther. 56:641-46
57. 3H- labeled oligonucleotides. II. 3′,5- blocked oligonucleotides. Mol. Pharmacol. 47:636-46
58. Stein CA, Krieg AM. 1994. Problems in interpretation of data derived from in vitro and in vivo use of antisense oligodeoxynucleotides. Antisense Res. Dev. 4:67-69
59. Crooke ST, Lemonidis KM, Nielson L, Griffey R, Monia BP. 1995. Kinetic characteristics of E. coli RNase H1: cleavage of various antisense oligonucleotides-RNA duplexes. Biochem. J. In press
60. Majumdar C, Stein CA, Cohen JS, Broder S, Wilson SH. 1989. Stepwise mechanism of HIV reverse transcriptase: primer function of phosphorothioate oligodeoxynucleotide. Biochemistry 28:1340-46
61. Perez JR, Li Y, Stein CA, Majumder S, Van Oorschot A, Narayanan R. 1994. Sequence-independent induction of Sp1 transcription factor activity by phosphorothioate oligodeoxynucleotides. Proc. Natl. Acad. Sci. USA 91:5957-61
62. Brown DA, Kang SH, Gryaznov SM, DeDionisio L, Heidenreich O, et al. 1994. Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding. J. Biol. Chem. 269:26801-5
63. Yakubov L, Khaled Z, Zhang L-M, Truneh A, Vlassov V, Stein CA. 1993. Oligodeoxynucleotides interact with recombinant CD4 at multiple sites. J. Biol. Chem. 268:18818-23
64. Stein CA, Neckers M, Nair BC, Mumbauer S, Hoke G, Pal R. 1991. Phosphorothioate oligodeoxycytidine interferes with binding of HIV-1 gp120 to CD4. J. Acquired Immune Defic. Syndr. 4:686-93
65. Stein CA, Cleary AM, Yakubov L, Lederman S. 1993. Phosphorothioate oligodeoxynucleotides bind to the third variable loop domain (v3) of human immunodeficiency virus type 1 gp120. Antisense Res. Dev. 3:19-31
66. Guvakova MA, Yakubov LA, Vlodavsky I, Tonkinson JL, Stein CA. 1995. Phosphorothioate oligodeoxynucleotides bind to basic fibroblast growth factor, inhibit its binding to cell surface receptors, and remove it from low affinity binding sites on extracellular matrix. J. Biol. Chem. 270:2620-27
67. Stein CA, Tonkinson JL, Zhang L-M, Yakubov L, Gervasoni J, et al. 1993. Dynamics of the internalization of phosphodiester oligodeoxynucleotides in HL60 cells. Biochemistry 32:4855-61
68. Hodges D, Crooke ST. 1995. Inhibition of splicing of wild-type and mutated luciferase-adenovirus pre-mRNA by antisense oligonucleotides. Mol. Pharmacol. In press
69. Wyatt JR, Vickers TA, Roberson JL, Buckheit RW Jr, Klimkait T, et al. 1994. Combinatorially selected guanosine- quartet structure is a potent inhibitor of human immunodeficiency virus envelope-mediated cell fusion. Proc. Natl. Acad. Sci. USA 91:1356-60
70. 2 enzyme activity by phosphorothioate oligonucleotides. Nucleic Acids Res. 22:3202-9
71. Ramanathan M, Lantz M, MacGregor RD, Garovoy MR, Hunt CA. 1994. Characterization of the oligodeoxynucleotide-mediated inhibition of interferon-Y-induced major histocompatibility complex class I and intercellular adhesion molecule-1. J, Biol. Chem. 269:24564-74
72. Watson PH, Pon RT, Shiu RPC. 1992. Inhibition of cell adhesion to plastic substratum by phosphorothioate oligonucleotide. Exp. Cell Res. 202:391-97
73. KB phosphorothioates determines specificity. Nucleic Acids Res. 23:1146-51
74. Yaswen P, Stampfer MR, Ghosh K, Cohen JS. 1993. Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells. Antisense Res. Dev. 3:67-77
75. Burgess TL, Fisher EF, Ross SL, Bready JV, Qian Y-X, et al. 1995. The antiproliferative activity of c-myb and c-myc antisense oligonucleotides in smooth muscle cells is caused by a nonantisense mechanism. Proc. Natl. Acad. Sci. USA 92:4051-55
76. Ecker DJ, Vickers TA, Hanecak R, Driver V, Anderson K. 1993. Rational screening of oligonucleotide combinatorial libraries for drug discovery. Nucleic Acids Res. 21:1853-56
77. Monia BP, Lesnik EA, Gonzalez C, Lima WF, McGee D, et al. 1993. Evaluation of 2′ modified oligonucleotides containing deoxy gaps as antisense inhibitors of gene expression. J. Biol. Chem. 268:14514-22
78. 1 cells. J. Biol. Chem. 268:21875-82
79. Dean NM, McKay R. 1994. Inhibition of protein kinase C-alpha expression in mice after systemic administration of phosphorothioate antisense oligodeoxynucleotides. Proc. Natl. Acad. Sci. USA 91:11762-66
80. Fenster SD, Wagner RW, Froehler BC, Chin DJ. 1994. Inhibition of human immunodeficiency virus Type-1 env expression by C-5 propyne oligonucleotides specific for rev-response element stem-loop V. Biochemistry 33: 8391-98
81. Sawa H, Sobel BE, Fujii S. 1994. Inhibition of type-1 plasminogen activator inhibitor production by antisense oligonucleotides in human vascular endothelial and smooth muscle cells. J. Biol. Chem. 269:14149-52
82. Anazodo MI, Wainberg MA, Friesen AD, Wright JA. 1995. Sequence-specific inhibition of gene expression by a novel antisense oligodeoxynucleotide phosphorothioale directed against a non- regulatory region of the human immunodeficiency virus type 1 genome. J. Virol. 69:1794-801
83. Ulloa L, Diaz-Nido J, Avila J. 1993. Depletion of casein kinase II by antisense oligonucleotide prevents neuritogenesis in neuroblastoma cells. EMBO J. 12:1633-40
84. Agrawal S, Sarin PS, Zamecnik M, Zamecnik PC. 1992. Cellular uptake and anti-HIV activity of oligonucleotides and their analogs. See Ref. 184, pp. 273-83
85. Lisziewicz J, Sun D, Klotman M, Agrawal S, Zamecnik P, Gallo R. 1992. Specific inhibition of human immunodeficiency virus type 1 replication by antisense oligonucleotides: an in vitro model for treatment. Proc. Natl. Acad. Sci. USA 89:11209-13
86. Lisziewicz J, Sun D, Symthe J, Lusso P, Lori F, et al. 1993. Inhibition of human immunodeficiency virus type 1 replication by regulated expression of a polymeric Tat activation response RNA decoy as a strategy for gene therapy in AIDS. Proc. Natl. Acad. Sci. USA 90: 8000-4
87. Lisziewicz J, Sun D, Weichold FF, Thierry AR, Lusso P, et al. 1994. Antisense oligodeoxynucleotide phosphorothioate complementary to Gag mRNA blocks replication of human immunodeficiency virus type 1 in human peripheral blood cells. Proc. Natl. Acad. Sci. USA 91:7942-46
88. Azad RF, Driver VB, Tanaka K, Crooke RM, Anderson KP. 1993. Antiviral activity of a phosphorothioate oligonucleotide complementary to RNA of the human cytomegalovirus major immediate-early region. Antimicrob. Agents Chemother. 37:1945-54
89. Barton CM, Lemoine NR. 1995. Antisense oligonucleotides directed against p53 have antiproliferative effects unrelated to effects on p53 expression. Br. J. Cancer 71:429-37
90. Burch RM, Mahan LC. 1991. Oligonucleotides antisense to the interleukin 1 receptor mRNA block the effects of interleukin 1 in cultured marine and human fibroblasts and in mice. J. Clin. Invest. 88:1190-96
91. Kitajima I, Shinohara T, Bilakovics J, Brown DA, Xu X, Nerenberg M. 1992. Ablation of transplanted HTLV-1 tax- transformed tumors in mice by antisense inhibition of NF-kB. Science 258:1792-95
92. Chiasson BJ, Hooper ML, Murphy PR, Robertson HA. 1992. Antisense oligonucleotide eliminates in vivo expression of c-fos in mammalian brain. Eur. J. Pharmacol. 227:451-53
93. Sommer W, Bjelke B, Ganten D, Fuxe K. 1993. Antisense oligonucleotide to c-fos induces ipsilateral rotational behaviour to d-amphetamine. NeuroReport 5:277-80
94. Nesterova M, Cho-Chung YS. 1995. A single-injection protein kinase A-directed antisense treaiment to inhibit tumor growth. Nat. Med. 1:528-33
95. 2 dopamine receptors in mouse striatum. J. Pharmacol. Exp. Ther. 268:1015-23
96. Skorski T, Nieborowska-Skorska M, Nicolaides NC, Szczylik C, Iversea P, et al. 1994. Suppression of Philadelphia leukemia cell growth in mice by BCR- ABL antisense oligodeoxynucleotide. Proc. Natl. Acad. Sci. USA 91:4504-8
97. Hijiya N, Zhang J, Ratajczak MZ, Kant JA, DeRiel K, et al. 1994. Biologic and therapeutic significance of MYB expression in human melanoma. Proc. Natl. Acad. Sci. USA 91:4499-503
98. 2 messenger RNA. Neuroscience 55:607-12
99. Mani SK, Blaustein JD, Allen JMC, Law SW, O'Malley BW, Clark JH. 1994. Inhibition of rat sexual behavior by antisense oligonucleotides to the progesterone receptor. Endocrinology 135: 1409-14
100. Hunter JC, Woodburn VL, Durieux C, Pettersson EKE, Poat JA, Hughes J. 1995. C-fos antisense oligodeoxynucleotide increases formalin-induced nociception and regulates preprodynorphin expression. Neuroscience 65:485-92
101. Cornish KG, Iversen P, Smith L, Arneson M, Bayever E. 1993. Cardiovascular effects of a phosphorothioate oligonucleotide to p53 in the conscious rhesus monkey. Pharmacol. Commun. 3:239-47
102. Galbraith WM, Hobson WC, Giclas PC, Schechter PJ, Agrawal S. 1994. Complement activation and hemodynamic changes following intravenous administration of phosphorothioate oligonucleotides in the monkey. Antisense Res. Dev. 4:201-6
103. Lisziewicz J, Sun D, Metelev V, Zamecnik P, Gallo RC, Agrawal S. 1993. Long-term treatment of human immunodeficiency virus-infected cells with antisense oligonucleotide phosphorothioates. Proc. Natl. Acad. Sci. USA 90:3860-64
104. Paroli M, Carloni G, Franco A, De Petrillo G, Alfani E, et al. 1994. Human hepatoma cells expressing MHC antigens display accessory cell function: dependence on LFA-1/ICAM-1 interaction. Immunology 82:215-21
105. Calabretta B, Sims RB, Valtieri M, Caracciolo D, Szczylik C, et al. 1991. Normal and leukemic hematopoietic cells manifest differential sensitivity to inhibitory effects of c-myb anti sense oligodeoxynucleotides: an in vitro study relevant to bone marrow purging. Proc. Natl. Acad. Sci. USA 88:2351-55
106. Ratajczak MZ, Kant JA, Luger SM, Hijiya N, Zhang J, et al. 1992. In vivo treatment of human leukemia in a scid mouse model with c-myb antisense oligodeoxynucleotides. Proc. Natl. Acad. Sci. USA 89:11823-27
107. Milligan JF, Matteucci MD, Martin JC. 1993. Current concepts in antisense drug design. J. Med. Chem. 36:1923-37
108. Sanghvi YS, Cook PD. 1993. Towards second-generation synthetic backbones for antisense oligonucleosides. In Nucleosides, Nucleotides as Antiviral and Antitumor Agents, ed. CK Chu, DC Baker, pp. 309-22. New York: Plenum
109. Beaucage SL, Iyer RP. 1993. The synthesis of modified oligonucleotides by the phosphoramidite approach and their applications. Tetrahedron 49: 6123-94
110. Sanghvi YS, Cook PD. 1994. Carbohydrates: synthetic methods and applications in antisense therapeutics: an overview. In Carbohydrate Modifications in Antisense Research, ACS Symposium Series No. 580, ed. YS Sanghvi, PD Cook, pp. 1-22. Washington, DC: Am. Chem. Soc.
111. Sagi J, Szemzo A, Ebinger K, Szabolocs A, Sagi G, et al. 1993. Base-modified oligodeoxynucleotides. I. Effect of 5-alkyl, 5-(1-alkenyl) and 5-(1-alkynyl) substitution of the pyrimidines on duplex stability and hydrophobicity. Tetrahedron Lett. 34:2191-94
112. Wagner RW, Matteucci MD, Lewis JG, Gutierrez AJ, Moulds C, Froehler BC. 1993. Antisense gene inhibition by oligonucleotides containing C-5 propyne pyrimidines. Science 260:1510-13
113. Iribarren AM, Sproat BS, Neuner P, Sulston I, Ryder U, Lamond AI. 1990. 2′-O-alkyl oligoribonucleotides as antisense probes. Proc. Natl. Acad. Sci. USA 87:7747-51
114. Kawasaki AM, Casper MD, Freier SM, Lesnik EA, Zounes MC, et al. 1993. Uniformly modified 2′-deoxy-2′-fluoro pbosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets. J. Med. Chem. 36:831-41
115. Lesnik EA, Guinosso CI, Kawasaki AM, Sasmor H, Zounes M, et al. 1993. Oligodeoxynucleotides containing 2′-O- modified adenosine: synthesis and effects on stability of DNA:RNA duplexes. Biochemistry 32:7832-38
116. Martin P. 1995. Ein neuer zugang zu 2′-O-alkylribonucleosiden und eigenschaften deren oligonucleotide. Helv. Chim. Acta 78:486-89
117. Cummins LL, Owens SR, Risen LM, Lesnik EA, Freier SM, et al. 1995. Effects of uniform 2′ modifications on oligonucleotide hybridization and nuclease sensitivity. Nucleic Acids Res. 23: 2019-24
118. Agrawal S, Mayrand SH, Zamecnik PC, Pederson T. 1990. Site-specific excision from RNA by RNase H and mixed- phosphate-backbone oligodeoxynucleotides. Proc. Natl. Acad. Sci. USA 87: 1401-5
119. Lamond AI, Barabino S, Blencowe BJ, Sproat B, Ryder U. 1990. Studying pre- mRNA splicing using antisense 2-OMe RNA oligonucleotictes. Mol. Biol. Rep. 14:201
120. Miller PS, McParland KB, Jayaraman K, Ts'o POP. 1981. Biochemical and biological effects of nonionic nucleic acid methylphosphonates. Biochemistry 20:1874-80
121. Miller PS, Ts'o POP. 1987. A new approach to chemotherapy based on molecular biology and nucleic acid chemistry: matagen (masking tape for gene expression). Anti-Cancer Drug Des. 2: 117-28
122. Miller PS. 1991. Oligonucleoside methylphosphonates as antisense reagents. BioTechnology 9:358-62
123. Arnold LJ. 1995. The development of oligonucleotides containing chirally pure methylphosphonates as therapeutic agents. Antisense Res. Dev. 5:108
124. Vasseur J-J, Debart F, Sanghvi YS, Cook PD. 1992. Oligonucleosides: synthesis of a novel methylhydroxylamine- linked nucleoside dimer and its incorporation into antisense sequences. J. Am. Chem. Soc. 114:4006-7
125. De Mesmaeker A, Lebreton J, Waldner A, Fritsch V, Wolf RM, Freier SM. 1993. Amides as substitute for the phosphodiester linkage in antisense oligonucleotides. Synlett 10:733-36
126. Jones RJ, Lin K-Y, Milligan JF, Wadwani S, Matteucci MD, 1993. Synthesis and binding properties of pyrimidine oligodeoxynucleoside analogs containing neutral phosphodiester replacements. The formacetal and 3′- thioformacetal internucleoside linkages. J. Org. Chem. 58:2983-91
127. Buchardt O, Egholm M, Berg RH, Nielsen PE. 1993. Peptide nucleic acids (PNA) and their potential applications in medicine and biotechnology. Trends BioTechnol. 11:384-86
128. Egholm M, Buchardt O, Christensen L, Behrens C, Freier SM, et al. 1993. PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen-bonding rules. Nature 365: 566-68
129. Stirchak EP, Summerton JE, Weller DD. 1989. Uncharged stereoregular nucleic acid analogs: 2. Morpholino nucleoside oligomers with carbamate internucleotide linkages. Nucleic Acids Res. 17:6129-41
130. Letsinger RL, Zhang GR, Sun DK, Ikeuchi T, Sarin PS. 1989. Cholesteryl- conjugated oligonucleotides: synthesis, properties, and activity as inhibitors of replication of human immunodeficiency virus in cell culture. Proc. Natl. Acad. Sci. USA 86:6553-56
131. Torrence PF, Maitra RK, Leisak K, Khamnei S, Zhou A, Silverman RH. 1993 Targeting RNA for degradation with a (2′-5′)oligoadenylate-antisense chimera. Proc. Natl. Acad. Sci. USA 90:1300-4
132. Manoharan M, Johnson LK, Bennett CF, Vickers TA, Ecker DJ, et al. 1994. Cholic acid-oligonucleotide conjugates for antisense applications. Bioorg. Med. Chem. Lett 4:1053-60
133. Manoharan M, Tivel KL, Andrade LK, Mohan V, Condon TP, et al. 1995. Oligonucleotide conjugates: alteration of the pharmacokinetic properties of antisense agents. Nucleos. Nucleot. 14: 969-73
134. Bock LC, Griffin LC, Latham JA, Vermaas EH, Toole JJ. 1992. Selection of single-stranded DNA molecules that bind and inhibit human thrombin. Nature 355:564-66
135. Gao W, Stein CA, Cohen JS, Dutschman GE, Cheng Y-C. 1989. Effect of phosphorothioate homo-oligodeoxynucleotides on herpes simplex virus type 2-induced DNA polymerase. J. Biol. Chem. 264:11521-26
136. Pisetsky DS, Reich C. 1993. Stimulation of in vitro proliferation of murine lymphocytes by synthetic oligodeoxynucleotides. Mol. Biol. Rep. 18: 217-21
137. Pisetsky DS, Reich CF. 1994. Stimulation of murine lymphocyte proliferation by a phosphorothioate oligonucleotide with antisense activity for herpes simplex virus. Life Sci. 54:101-7
138. Krieg AM, Yi A-K, Matson S, Waldschmidt TJ, Bishop GA, et al. 1995. CpG motifs in bacterial DNA trigger direct B-cell activation. Nature 374:546-49
139. Clark DL, Chrisey LA, Campbell JR, Davidson EA. 1994. Non-sequence-specific antimalarial activity of oligodeoxynucleotides. Mol. Biochem. Parasitol. 63:129-34
140. Gao W-Y, Jaroszewski JW, Cohen JS, Cheng Y-C. 1990. Mechanisms of inhibition of herpes simplex virus type 2 growth by 28-mer phosphorothioate oligodeoxycytidine. J. Biol. Chem. 265: 20172
141. Narayanan R, Higgins KA, Perez JR, Coleman TA, Rosen CA. 1993. Evidence for differential functions of the p50 and p65 subunits of NF-kB with a cell adhesion model. Mol. Cell Biol. 13:3802-10
142. Ghosh MK, Ghosh K, Cohen JS. 1992. Translation inhibition by phosphorothioate oligodeoxynucleotides in cell- free systems. Antisense Res. Dev. 2: 111-18
143. Ho PTC, Ishiguro K, Wickstrom E, Sartorelli AC. 1991. Non-sequence-specific inhibition of transferrin receptor expression in HL-60 leukemia cells by phosphorothioate oligodeoxynucleotides. Antisense Res. Dev. 1:329-42
144. Simons M, Edelman ER, DeKeyser J-L, Langer R, Rosenberg RD. 1992. Antisense c-myb oligonucleotides inhibit arterial smooth muscle cell accumulation in vivo. Nature 359:67-70
145. Bennett MR, Anglin S, McEwan JR, Jagoe R, Newby AC, Evan GI. 1994. Inhibition of vascular smooth muscle cell proliferation in vitro and in vivo by C-myc antisense oligodeoxynucleotides. J. Clin. Invest. 93:820-28
146. Morishita R, Gibbons GH, Ellison KE, Nakajima M, Zhang L, et al. 1993. Single intraluminal delivery of antisense cdc2 kinase and proliferating-cell nuclear antigen oligonucleotides results in chronic inhibition of neointimal hyperplasia. Proc. Natl. Acad. Sci. USA 90: 8474-78
147. Abe J, Zhou W, Taguchi J, Takuwa N, Miki K, et al. 1994. Suppression of neointimal smooth muscle cell accumulation in vivo by antisense CDC2 and CDK2 oligonucleotides in rat carotid artery. Biochem. Biophys. Res. Commun. 198:16-24
148. Simons M, Edelman ER, Rosenberg RD. 1994. Antisense proliferating cell nuclear antigen oligonucleotides inhibit in- timal hyperplasia in a rat carotid artery injury model. J. Clin. Invest. 93:2351-56
149. 1 and CDC 2 kinase) in the vessel wall in vivo: enhanced therapeutic utility for restenosis by HVJ- liposome delivery. Gene 149:13-19
150. 1 receptor mRNA and angiotensinogen mRNA in the brain of spontaneously hypertensive rats reduces hypertension of neurogenic origin. Regul. Pept. 49:167-74
151. Phillips MI, Wielbo D, Gyurko R. 1994. Antisense inhibition of hypertension: a new strategy for renin-angiotensin candidate genes. Kidney Int. 46:1554-56
152. Suzuki S, Pilowsky P, Minson J, Arnolda L, Llewellyn-Smith IJ, Chalmers J. 1994. c-fos antisense in rostral ventral medulla reduces arterial blood pressure. Am. J. Physiol. 266:R1418-22
153. Stepkowski SM, Tu Y, Condon TP, Bennett CF. 1995. Induction of transplantation tolerance by treatment with ICAM-1 antisense oligonucleotides and anti-LFA-1 monoclonal antibodies. Transplant. Proc. 27:113
154. Whitsell L, Rosolen A, Neckers LM. 1991. In vivo modulation of N-myc expression by continuous perfusion with an antisense oligonucleotide. Antisense Res. Dev. 1:343-50
155. Kitajima I, Shinohara T, Bilakovics J, Brown DA, Xiao X, Nerenberg M. 1992. Ablation of transplanted HTLV-1 tax-transformed tumors in mice by antisense inhibition of NF-KB. Science 258:1792-95
156. Higgins KA, Perez JR, Coleman TA, Dorshkind JC, McComas WA, et al. 1993. Antisense inhibition of the p65 subunit of NF-kappaB blocks tumorigenicity and causes tumor regression. Proc. Nati. Acad Sci. U.S.A. 90: 9901-5
157. Schwab G, Chavany C, Duroux I, Goubin G, Lebeau J, Hélène C, Saison- Behmoaras T. 1994. Antisense oligonucleotides adsorbed to polyalkylcyanoacrylate nanoparticles specifically inhibit mutated Ha-ras-mediated cell proliferation and tumorigenicity in nude mice. Proc. Natl. Acad. Sci. USA 91: 10460-64
158. Heilig M, Engel JA, Söderpalm B. 1993. C-fos antisense in the nucleus accumbens blocks the locomotor stimulant action of cocaine. Eur. J. Pharmacol. 236: 339-40
159. Hooper ML, Chiasson BJ, Robertson HA. 1994. Infusion into the brain of an antisense oligonucleotide to the immediate-early gene c-fos suppresses production of Fos and produces a behavioral effect. Neuroscience 63:917-24
160. Woodburn VL, Hunter JC, Durieux C, Poat JA, Hughes J. 1994. The effect of C-FOS antisense in the formalin-paw test. Regul. Pepr. 54:327-28
161. Gillardon F, Beck H, Uhlmann E, Herdegen T, Sandkühler J, et al. 1994. Inhibition of c-fos protein expression in rat spinal cord by antisense oligodeoxynucleotide superfusion. Eur. J. Neurosci. 6:880-84
162. Tischmeyer W, Grimm R, Schicknick H, Brysch W, Schlingensiepen K-H. 1994. Sequence-specific impairment of learning by c-jun antisense oligonucleotides. NeuroReport 5:1501-4
163. Konradi C, Cole RL, Heckers S, Hyman SE. 1994. Amphetamine regulates gene expression in rat striatum via transcription factor CREB. J. Neurosci. 14:5623-34
164. Osen-Sand A, Catsicas M, Staple JK, Jones KA, Ayala G, et al. 1993. Inhibition of axonal growth by SNAP-25 antisense oligonucleotides in vitro and in vivo. Nature 364:445-48
165. Amaratunga A, Morin PJ, Kosik KS, Fine RE. 1993. Inhibition of kinesin synthesis and rapid anterograde axonal transport in vivo by an antisense oligonucleotide. J. Biol. Chem. 268(23): 17427-30
166. Skutella T, Probst JC, Jirikowski GF, Holsboer F, Spanagel R. 1994. Ventral tegmental area (VTA) injections of tyrosine hydroxylase phosphorothioate antisense oligonucleotide suppress operant behavior in rats. Neurosci. Lett. 167:55-58
167. 67 mRNAs modulate reproductive behavior in the female rat. Brain Res. 636:209-20
168. Flanagan LM, McCarthy MM, Brooks PJ, Pfaff DW, McEwen BS. 1993. Arginine vasopressin levels after daily infusions of antisense oligonucleotides into the supraoptic nucleus. Ann. NY Acad. Sci. 689:520-21
169. Castro-Alamancos MA, Torres-Aleman I. 1994. Learning of the conditioned eye-blink response is impaired by an antisense insulin-like growth factor I oligonucleotide. Proc. Natl. Acad. Sci. USA 91:10203-7
170. Pollio G, Xue P, Zanisi M, Nicolin A, Maggi A. 1993. Antisense oligonucleotide blocks progesterone-induced lordosis behavior in ovariectomized rats. Mol. Brain Res. 19:135-39
171. McCarthy MM, Schlenker EH, Pfaff DW. 1993. Enduring consequences of neonatal treatment with antisense oligodeoxynucleotides to estrogen receptor messenger ribonucleic acid on sexual differentiation of rat brain. Endocrinology 133:433-39
172. Neumann I, Porter DWF, Landgraf R, Pittman QJ. 1994. Rapid effect on suckling of an oxytocin antisense oligonucleotide administered into rat supraoptic nucleus. Am. J. Physiol. Regul. Integr. Comp. Physiol. 267:R852-58
173. 2 receptors: behavioral correlates. Neurosci. Lett. 161:223-26
174. 1 dopamine receptor-mediated behaviors in normal mice and in mice lesioned with 6-hydroxydopamine. J. Pharmacol. Exp. Ther. 271:1462-70
175. Ogo H, Hirai Y, Miki S, Nishio H, Akiyama M, Nakata Y. 1994. Modulation of substance P/neurokinin-1 receptor in human astrocytoma cells by antisense oligodeoxynucleotides. Gen. Pharmacol. 25:1131-35
176. Akabayashi A, Wahlestedt C, Alexander JT, Leibowitz SF. 1994. Specific inhibition of endogenous neuropeptide Y synthesis in arcuate nucleus by antisense oligonucleotides suppresses feeding behavior and insulin secretion. Mol. Brain Res. 21:55-61
177. Wahlestedt C, Golanov E, Yamamoto S, Yee F, Ericson H, et al. 1993. Antisense oligodeoxynucleotides to NMDA-R1 receptor channel protect cortical neurons from excitotoxicity and reduce focal ischaemic infarctions. Nature 363:260-63
178. Adams JU, Chen XH, deRiel JK, Adler MW, Liu-Chen LY. 1994. In vivo treatment with antisense oligodeoxynucleotide to kappa-opioid receptors inhibited kappa-agonist-induced analgesia in rats. Regul. Pept. 54:1-2
179. 2-agonist antinociception. NeuroReport 5:1049-52
180. Kindy MS. 1994. NMDA receptor inhibition using antisense oligonucleotides prevents delayed neuronal death in gerbil hippocampus following cerebral ischemia. Neurosci. Res. Commun. 14:175-83
181. 4]deltorphin with d-opioid receptor subtypes in vivo. Eur. J. Pharmacol. 252:133-37
182. Offensperger W-B, Offensperger S, Walter E, Teubner K, et al. 1993. In vivo inhibition of duck hepatitis B virus replication and gene expression by phosphorothioate modified antisense oligodeoxynucleotides. EMBO J. 12: 1257-62
183. Kulka M, Smith CC, Aurelian L, Fishelevich R, Meade K, Miller P, Ts'o POP. 1989. Site specificity of the inhibitory effects of oligonucleoside methylphosphonates complementary to the acceptor splice junction of herpes simplex virus type 1 immediate early mRNA 4. Proc. Natl. Acad. Sci. USA 86:6868-72
184. Erickson RP, Izant RP, eds. 1992. Gene Regulation: Biology of Antisense RNA and DNA. New York: Raven
185. Crooke ST, Lebleu B, eds. 1993. Antisense Research and Applications. Boca Raton, FL: CRC Press