Antisense oligonucleotides: Basic concepts and mechanisms

Molecular Cancer Therapeutics

Volumn 1, Issue 5, 2002, Pages 347-355

  Dias, Nathalie ^a   Stein, C A ^a  

^a Och Spine at New York Presbyterian Hospitals   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DNA; MESSENGER RNA;

ANIMAL; CHEMICAL MODEL; CHEMISTRY; GENETICS; HUMAN; METABOLISM; NUCLEIC ACID HYBRIDIZATION; REVIEW;

ANIMALS; DNA; HUMANS; MODELS, CHEMICAL; NUCLEIC ACID HYBRIDIZATION; OLIGONUCLEOTIDES, ANTISENSE; RNA, MESSENGER;

EID: 0000015743     PISSN: 15357163     EISSN: 15388514     Source Type: Journal    
DOI: None     Document Type: Short Survey

References (116)

1. Orr, R. M. Technology evaluation: fomivirsen. Isis Pharmaceuticals Inc/CIBA vision. Curr. Opin. Mol. Ther., 3: 288-294, 2001. (Pubitemid 32631420)
2. Roehr, B. Fomivirsen approved for CMV retinitis. J. Int. Assoc. Physicians AIDS Care, 4: 14-16, 1998.
3. Agrawal, S., and Kandimalla, E. R. Antisense therapeutics: is it as simple as complementary base recognition? Mol. Med. Today, 6: 72-81, 2000. (Pubitemid 30070669)
4. Wickstrom, E. Oligodeoxynucleotide stability in subcellular extracts and culture media. J. Biochem. Biophys. Methods, 13: 97-102, 1986.
5. Akhtar, S., Kole, R., and Juliano, R. L. Stability of antisense DNA oligodeoxynucleotide analogs in cellular extracts and sera. Life Sci., 49: 1793-1801, 1991.
6. Eder, P. S., DeVine, R. J., Dagle, J. M., and Walder, J. A. Substrate specificity and kinetics of degradation of antisense oligonucleotides by a 3′ exonuclease in plasma. Antisense Res. Dev., 1: 141-151, 1991.
7. Vaerman, J. L., Moureau, P., Deldime, F., Lewalle, P., Lammineur, C., Morschhauser, F., and Martiat, P. Antisense oligodeoxyribonucleotides suppress hematologic cell growth through stepwise release of deoxyribonucleotides. Blood, 90: 331-339, 1997. (Pubitemid 27276578)
8. Koziolkiewicz, M., Gendaszewska, E., Maszewska, M., Stein, C. A., and Stec, W. J. The mononucleotide-dependent, nonantisense mechanism of action of phosphodiester and phosphorothioate oligonucleotides depends upon the activity of an ecto-5′-nucleotidase. Blood, 98: 995-1002, 2001.
9. Kara, J., and Duschinsky, R. Inhibition of thymidylate kinase and DNA synthesis in HeLa cells by 5′-deoxythymidine. Biochim. Biophys. Acta, 186: 223-225, 1969.
10. Miller, P. S., Yano, J., Yano, E., Carroll, C., Jayaraman, K., and Ts'o, P. O. Nonionic nucleic acid analogues. Synthesis and characterization of dideoxyribonucleoside methylphosphonates. Biochemistry, 18: 5134-5143, 1979.
11. Miller, P. S., McParland, K. B., Jayaraman, K., and Ts'o, P. O. Biochemical and biological effects of nonionic nucleic acid methylphosphonates. Biochemistry, 20: 1874-1880, 1981.
12. Blake, K. R., Murakami, A., Spitz, S. A., Glave, S. A., Reddy, M. P., Ts'o, P. O., and Miller, P. S. Hybridization arrest of globin synthesis in rabbit reticulocyte lysates and cells by oligodeoxyribonucleoside methylphosphonates. Biochemistry, 24: 6139-6145, 1985. (Pubitemid 16197366)
13. Tonkinson, J. L., and Stein, C. A. Patterns of intracellular compartmentalization, trafficking and acidification of 5′-fluorescein labeled phosphodiester and phosphorothioate oligodeoxynucleotides in HL60 cells. Nucleic Acids Res., 22: 4268-4275, 1994.
14. Shoji, Y., Akhtar, S., Periasamy, A., Herman, B., and Juliano, R. L. Mechanism of cellular uptake of modified oligodeoxynucleotides containing methylphosphonate linkages. Nucleic Acids Res., 19: 5543-5550, 1991. (Pubitemid 21912953)
15. Stein, C. A., and Cheng, Y. C. Antisense oligonucleotides as therapeutic agents - is the bullet really magical? Science, 261: 1004-1012, 1993. (Pubitemid 23300859)
16. Jansen, B., Wacheck, V., Heere-Ress, E., Schlagbauer-Wadl, H., Hoeller, C., Lucas, T., Hoermann, M., Hollenstein, U., Wolff, K., and Pehamberger, H. Chemosensitization of malignant melanoma by BCL2 antisense therapy. Lancet, 356: 1728-1733, 2000.
17. Geiger, T., Muller, M., Dean, N. M., and Fabbro, D. Antitumor activity of a PKC-α antisense oligonucleotide in combination with standard chemotherapeutic agents against various human tumors transplanted into nude mice. Anticancer Drug Des., 13: 35-45, 1998. (Pubitemid 28045537)
18. Benimetskaya, L., Loike, J. D., Khaled, Z., Loike, G., Silverstein, S. C., Cao, L., el Khoury, J., Cai, T. Q., and Stein, C. A. Mac-1 (CD11b/CD18) is an oligodeoxynucleotide-binding protein. Nat. Med., 3: 414-420, 1997. (Pubitemid 27145683)
19. Stein, C. A. Controversies in the cellular pharmacology of oligodeoxynucleotides. Ciba Found Symp., 209: 79-89, 1997.
20. Guvakova, M. A., Yakubov, L. A., Vlodavsky, I., Tonkinson, J. L., and Stein, C. A. 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: 2620-2627, 1995.
21. Fennewald, S. M., and Rando, R. F. Inhibition of high affinity basic fibroblast growth factor binding by oligonucleotides. J. Biol. Chem., 270: 21718-21721, 1995.
22. Monia, B. P., Lesnik, E. A., Gonzalez, C., Lima, W. F., McGee, D., Guinosso, C. J., Kawasaki, A. M., Cook, P. D., and Freier, S. M. Evaluation of 2′-modified oligonucleotides containing 2′-deoxy gaps as antisense inhibitors of gene expression. J. Biol. Chem., 268: 14514-14522, 1993. (Pubitemid 23195583)
23. Baker, B. F., Lot, S. S., Condon, T. P., Cheng-Flournoy, S., Lesnik, E. A., Sasmor, H. M., and Bennett, C. F. 2′-O-(2-Methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (ICAM-1) oligonucleotides selectively increase the ICAM-1 mRNA level and inhibit formation of the ICAM-1 translation initiation complex in human umbilical vein endothelial cells. J. Biol. Chem., 272: 11994-12000, 1997. (Pubitemid 27202775)
24. Egholm, M., Buchardt, O., Christensen, L., Behrens, C., Freier, S. M., Driver, D. A., Berg, R. H., Kim, S. K., Norden, B., and Nielsen, P. E. PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen-bonding rules. Nature, 365: 566-568, 1993. (Pubitemid 23317782)
25. Nielsen, P. E., Egholm, M., Berg, R. H., and Buchardt, O. Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Science, 254: 1497-1500, 1991. (Pubitemid 21917487)
26. Jensen, K. K., Orum, H., Nielsen, P. E., and Norden, B. Kinetics for hybridization of peptide nucleic acids (PNA) with DNA and RNA studied with the BIAcore technique. Biochemistry, 36: 5072-5077, 1997. (Pubitemid 27180989)
27. Cutrona, G., Carpaneto, E. M., Ulivi, M., Roncella, S., Landt, O., Ferrarini, M., and Boffa, L. C. Effects in live cells of a c-myc anti-gene PNA linked to a nuclear localization signal. Nat. Biotechnol., 18: 300-303, 2000. (Pubitemid 30159481)
28. Boffa, L. C., Morris, P. L., Carpaneto, E. M., Louissaint, M., and Allfrey, V. G. Invasion of the CAG triplet repeats by a complementary peptide nucleic acid inhibits transcription of the androgen receptor and TATA-binding protein genes and correlates with refolding of an active nucleosome containing a unique AR gene sequence. J. Biol. Chem., 271: 13228-13233, 1996.
29. Hanvey, J. C., Peffer, N. J., Bisi, J. E., Thomson, S. A., Cadilla, R., Josey, J. A., Ricca, D. J., Hassman, C. F., Bonham, M. A., Au, K. G. et al. Antisense and antigene properties of peptide nucleic acids. Science, 258: 1481-1485, 1992. (Pubitemid 23011003)
30. Vickers, T. A., Griffith, M. C., Ramasamy, K., Risen, L. M., and Freier, S. M. Inhibition of NF-κB specific transcriptional activation by PNA strand invasion. Nucleic Acids Res., 23: 3003-3008, 1995.
31. Karras, J. G., Maier, M. A., Lu, T., Watt, A., and Manoharan, M. Peptide nucleic acids are potent modulators of endogenous pre-mRNA splicing of the murine interleukin-5 receptor-α chain. Biochemistry, 40: 7853-7859, 2001. (Pubitemid 32622976)
32. Mologni, L., Marchesi, E., Nielsen, P. E., and Gambacorti-Passerini, C. Inhibition of promyelocytic leukemia (PML)/retinoic acid receptor-α and PML expression in acute promyelocytic leukemia cells by anti-PML peptide nucleic acid. Cancer Res., 61: 5468-5473, 2001. (Pubitemid 32694928)
33. Mologni, L., Nielsen, P. E., and Gambacorti-Passerini, C. In vitro transcriptional and translational block of the bcl-2 gene operated by peptide nucleic acid. Biochem. Biophys. Res. Commun., 264: 537-543, 1999. (Pubitemid 29516121)
34. Dias, N., Dheur, S., Nielsen, P. E., Gryaznov, S., Van Aerschot, A., Herdewijn, P., Helene, C., and Saison-Behmoaras, T. E. Antisense PNA tridecamers targeted to the coding region of Ha-ras mRNA arrest polypeptide chain elongation. J. Mol. Biol., 294: 403-416, 1999.
35. Good, L., and Nielsen, P. E. Antisense inhibition of gene expression in bacteria by PNA targeted to mRNA. Nat. Biotechnol., 16: 355-358, 1998. (Pubitemid 28164655)
36. Gambacorti-Passerini, C., Mologni, L., Bertazzoli, C., le Coutre, P., Marchesi, E., Grignani, F., and Nielsen, P. E. In vitro transcription and translation inhibition by anti-promyelocytic leukemia (PML)/retinoic acid receptor α and anti-PML peptide nucleic acid. Blood, 88: 1411-1417, 1996. (Pubitemid 26276818)
37. Summerton, J., and Weller, D. Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug Dev., 7: 187-195, 1997. (Pubitemid 27323077)
38. Hudziak, R. M., Barofsky, E., Barofsky, D. F., Weller, D. L., Huang, S. B., and Weller, D. D. Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation. Antisense Nucleic Acid Drug Dev., 6: 267-272, 1996. (Pubitemid 27045903)
39. Taylor, M. F., Weller, D. D., and Kobzik, L. Effect of TNF-α antisense oligomers on cytokine production by primary murine alveolar macrophages. Antisense Nucleic Acid Drug Dev., 8: 199-205, 1998. (Pubitemid 28333906)
40. Summerton, J., Stein, D., Huang, S. B., Matthews, P., Weller, D., and Partridge, M. Morpholino and phosphorothioate antisense oligomers compared in cell-free and in-cell systems. Antisense Nucleic Acid Drug Dev., 7: 63-70, 1997. (Pubitemid 27231917)
41. Partridge, M., Vincent, A., Matthews, P., Puma, J., Stein, D., and Summerton, J. A simple method for delivering morpholino antisense oligos into the cytoplasm of cells. Antisense Nucleic Acid Drug Dev., 6: 169-175, 1996. (Pubitemid 26394876)
42. Giles, R. V., Spiller, D. G., Clark, R. E., and Tidd, D. M. Antisense morpholino oligonucleotide analog induces missplicing of C-myc mRNA. Antisense Nucleic Acid Drug Dev., 9: 213-220, 1999. (Pubitemid 29248741)
43. Gryaznov, S. M., Lloyd, D. H., Chen, J. K., Schultz, R. G., DeDionisio, L. A., Ratmeyer, L., and Wilson, W. D. Oligonucleotide N3′->P5′ phosphoramidates. Proc. Natl. Acad. Sci. USA, 92: 5798-5802, 1995.
44. Chen, J. K., Schultz, R. G., Lloyd, D. H., and Gryaznov, S. M. Synthesis of oligodeoxyribonucleotide N3′->P5′ phosphoramidates. Nucleic Acids Res., 23: 2661-2668, 1995.
45. Gryaznov, S., Skorski, T., Cucco, C., Nieborowska-Skorska, M., Chiu, C. Y., Lloyd, D., Chen, J. K., Koziolkiewicz, M., and Calabretta, B. Oligonucleotide N3′->P5′ phosphoramidates as antisense agents. Nucleic Acids Res., 24: 1508-1514, 1996.
46. Skorski, T., Perrotti, D., Nieborowska-Skorska, M., Gryaznov, S., and Calabretta, B. Antileukemia effect of c-myc N3′->P5′ phosphoramidate antisense oligonucleotides in vivo. Proc. Natl. Acad. Sci. USA, 94: 3966-3971, 1997.
47. McKay, R. A., Miraglia, L. J., Cummins, L. L., Owens, S. R., Sasmor, H., and Dean, N. M. Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-α expression. J. Biol. Chem., 274: 1715-1722, 1999.
48. Giles, R. V., Spiller, D. G., Grzybowski, J., Clark, R. E., Nicklin, P., and Tidd, D. M. Selecting optimal oligonucleotide composition for maximal antisense effect following streptolysin O-mediated delivery into human leukaemia cells. Nucleic Acids Res., 26: 1567-1575, 1998. (Pubitemid 28291789)
49. Agrawal, S., Jiang, Z., Zhao, Q., Shaw, D., Cai, Q., Roskey, A., Channavajjala, L., Saxinger, C., and Zhang, R. Mixed-backbone oligonucleotides as second generation antisense oligonucleotides: in vitro and in vivo studies. Proc. Natl. Acad. Sci. USA, 94: 2620-2625, 1997. (Pubitemid 27136925)
50. Shen, L. X., Kandimalla, E. R., and Agrawal, S. Impact of mixed-backbone oligonucleotides on target binding affinity and target cleaving specificity and selectivity by Escherichia coli RNase H. Bioorg. Med. Chem., 6: 1695-1705, 1998. (Pubitemid 28524225)
51. Dean, N. M., McKay, R., Condon, T. P., and Bennett, C. F. 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: 16416-16424, 1994. (Pubitemid 24209342)
52. Larrouy, B., Blonski, C., Boiziau, C., Stuer, M., Moreau, S., Shire, D., and Toulme, J. J. RNase H-mediated inhibition of translation by antisense oligodeoxyribonucleotides: use of backbone modification to improve specificity. Gene, 121: 189-194, 1992.
53. Cazenave, C., Frank, P., and Busen, W. Characterization of ribonuclease H activities present in two cell-free protein synthesizing systems, the wheat germ extract and the rabbit reticulocyte lysate. Biochimie, 75: 113-122, 1993. (Pubitemid 23140160)
54. Minshull, J., and Hunt, T. The use of single-stranded DNA and RNase H to promote quantitative 'hybrid arrest of translation' of mRNA/DNA hybrids in reticulocyte lysate cell-free translations. Nucleic Acids Res., 14: 6433-6451, 1986.
55. Shuttleworth, J., and Colman, A. Antisense oligonucleotide-directed cleavage of mRNA in Xenopus oocytes and eggs. EMBO J., 7: 427-434, 1988.
56. Giles, R. V., Spiller, D. G., Green, J. A., Clark, R. E., and Tidd, D. M. Optimization of antisense oligodeoxynucleotide structure for targeting bcr-abl mRNA. Blood, 86: 744-754, 1995.
57. Damha, M. J., Noronha, A. M., Wilds, C. J., Trempe, J. F., Denisov, A., Pon, R. T., and Gehring, K. Properties of arabinonucleic acids (ANA & 20′F-ANA): implications for the design of antisense therapeutics that invoke RNase H cleavage of RNA. Nucleosides Nucleotides Nucleic Acids, 20: 429-440, 2001. (Pubitemid 32821808)
58. Wilds, C. J., and Damha, M. J. 2′-Deoxy-2′-fluoro-β-D- arabinonucleosides and oligonucleotides (2′F- ANA): synthesis and physicochemical studies. Nucleic Acids Res., 28: 3625-3635, 2000.
59. Benimetskaya, L., Takle, G. B., Vilenchik, M., Lebedeva, I., Miller, P., and Stein, C. A. Cationic porphyrins: novel delivery vehicles for antisense oligodeoxynucleotides. Nucleic Acids Res., 26: 5310-5317, 1998. (Pubitemid 28542729)
60. Giles, R. V., Spiller, D. G., and Tidd, D. M. Detection of ribonuclease H-generated mRNA fragments in human leukemia cells following reversible membrane permeabilization in the presence of antisense oligodeoxynucleotides. Antisense Res. Dev., 5: 23-31, 1995.
61. Cazenave, C., Chevrier, M., Nguyen, T. T., and Helene, C. Rate of degradation of [α]- and [β]-oligodeoxynucleotides in Xenopus oocytes. Implications for anti-messenger strategies. Nucleic Acids Res., 15: 10507-10521, 1987.
62. Kole, R., and Sazani, P. Antisense effects in the cell nucleus: modification of splicing. Curr. Opin. Mol. Ther., 3: 229-234, 2001. (Pubitemid 32624570)
63. Hudziak, R. M., Summerton, J., Weller, D. D., and Iversen, P. L. Antiproliferative effects of steric blocking phosphorodiamidate morpholino antisense agents directed against c-myc. Antisense Nucleic Acid Drug Dev., 10: 163-176, 2000. (Pubitemid 30487617)
64. van Deutekom, J. C., Bremmer-Bout, M., Janson, A. A., Ginjaar, I. B., Baas, F., den Dunnen, J. T., and van Ommen, G. J. Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum. Mol. Genet., 10: 1547-1554, 2001. (Pubitemid 32734306)
65. Sierakowska, H., Sambade, M. J., Agrawal, S., and Kole, R. Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides. Proc. Natl. Acad. Sci. USA, 93: 12840-12844, 1996. (Pubitemid 26382730)
66. Mann, C. J., Honeyman, K., Cheng, A. J., Ly, T., Lloyd, F., Fletcher, S., Morgan, J. E., Partridge, T. A., and Wilton, S. D. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc. Natl. Acad. Sci. USA, 98: 42-47, 2001. (Pubitemid 32095860)
67. Tu, G. C., Cao, Q. N., Zhou, F., and Israel, Y. Tetranucleotide GGGA motif in primary RNA transcripts. Novel target site for antisense design. J. Biol. Chem., 273: 25125-25131, 1998. (Pubitemid 28443271)
68. Mologni, L., leCoutre, P., Nielsen, P. E., and Gambacorti-Passerini, C. Additive antisense effects of different PNAs on the in vitro translation of the PML/RARα gene. Nucleic Acids Res., 26: 1934-1938, 1998. (Pubitemid 28291725)
69. Tyler, B. M., Jansen, K., McCormick, D. J., Douglas, C. L., Boules, M., Stewart, J. A., Zhao, L., Lacy, B., Cusack, B., Fauq, A., and Richelson, E. Peptide nucleic acids targeted to the neurotensin receptor and administered i.p. cross the blood-brain barrier and specifically reduce gene expression. Proc. Natl. Acad. Sci. USA, 96: 7053-7058, 1999. (Pubitemid 29275008)
70. Pooga, M., Soomets, U., Hallbrink, M., Valkna, A., Saar, K., Rezaei, K., Kahl, U., Hao, J. X., Xu, X. J., Wiesenfeld-Hallin, Z., Hokfelt, T., Bartfai, T., and Langel, U. Cell penetrating PNA constructs regulate galanin receptor levels and modify pain transmission in vivo. Nat. Biotechnol., 16: 857-861, 1998. (Pubitemid 28405857)
71. Sharp, P. A. RNA interference-2001. Genes Dev., 15: 485-490, 2001.
72. Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E., and Mello, C. C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature, 391: 806-811, 1998. (Pubitemid 28099681)
73. Bernstein, E., Caudy, A. A., Hammond, S. M., and Hannon, G. J. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature, 409: 363-366, 2001. (Pubitemid 32151243)
74. Lipardi, C., Wei, Q., and Paterson, B. M. RNAi as random degradative PCR: siRNA primers convert mRNA into dsRNAs that are degraded to generate new siRNAs. Cell, 107: 297-307, 2001. (Pubitemid 33049974)
75. Zamore, P. D., Tuschl, T., Sharp, P. A., and Bartel, D. P. RNAi. double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell, 101: 25-33, 2000.
76. Loke, S. L., Stein, C. A., Zhang, X. H., Mori, K., Nakanishi, M., Subasinghe, C., Cohen, J. S., and Neckers, L. M. Characterization of oligonucleotide transport into living cells. Proc. Natl. Acad. Sci. USA, 86: 3474-3478, 1989. (Pubitemid 19141813)
77. Yakubov, L. A., Deeva, E. A., Zarytova, V. F., Ivanova, E. M., Ryte, A. S., Yurchenko, L. V., and Vlassov, V. V. Mechanism of oligonucleotide uptake by cells: involvement of specific receptors? Proc. Natl. Acad. Sci. USA, 86: 6454-6458, 1989. (Pubitemid 19236298)
78. Vlassov, V. V., Balakireva, L. A., and Yakubov, L. A. Transport of oligonucleotides across natural and model membranes. Biochim. Biophys. Acta, 1197: 95-108, 1994.
79. de Diesbach, P., Berens, C., N'Kuli, F., Monsigny, M., Sonveaux, E., Wattiez, R., and Courtoy, P. J. Identification, purification and partial characterization of an oligonucleotide receptor in membranes of HepG2 cells. Nucleic Acids Res., 28: 868-874, 2000. (Pubitemid 30085311)
80. Gray, G. D., Basu, S., and Wickstrom, E. Transformed and immortalized cellular uptake of oligodeoxynucleoside phosphorothioates, 3′-alkylamino oligodeoxynucleotides, 2′-O-methyl oligoribonucleotides, oligodeoxynucleoside methylphosphonates, and peptide nucleic acids. Biochem. Pharmacol., 53: 1465-1476, 1997. (Pubitemid 27356119)
81. Stein, C. A., Tonkinson, J. L., Zhang, L. M., Yakubov, L., Gervasoni, J., Taub, R., and Rotenberg, S. A. Dynamics of the internalization of phosphodiester oligodeoxynucleotides in HL60 cells. Biochemistry, 32: 4855-4861, 1993. (Pubitemid 23162043)
82. Bennett, C. F., Chiang, M. Y., Chan, H., Shoemaker, J. E., and Mirabelli, C. K. Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides. Mol. Pharmacol., 41: 1023-1033, 1992. (Pubitemid 23026100)
83. Ma, D. D., and Wei, A. Q. Enhanced delivery of synthetic oligonucleotides to human leukaemic cells by liposomes and immunoliposomes. Leuk. Res., 20: 925-930, 1996. (Pubitemid 27042326)
84. Farhood, H., Serbina, N., and Huang, L. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta, 1235: 289-295, 1995.
85. Felgner, J. H., Kumar, R., Sridhar, C. N., Wheeler, C. J., Tsai, Y. J., Border, R., Ramsey, P., Martin, M., and Felgner, P. L. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem., 269: 2550-2561, 1994. (Pubitemid 24235749)
86. Fraley, R., Straubinger, R. M., Rule, G., Springer, E. L., and Papahadjopoulos, D. Liposome-mediated delivery of deoxyribonucleic acid to cells: enhanced efficiency of delivery related to lipid composition and incubation conditions. Biochemistry, 20: 6978-6987, 1981. (Pubitemid 12185950)
87. Stewart, A. J., Pichon, C., Meunier, L., Midoux, P., Monsigny, M., and Roche, A. C. Enhanced biological activity of antisense oligonucleotides complexed with glycosylated poly-L-lysine. Mol. Pharmacol., 50: 1487-1494, 1996. (Pubitemid 26425103)
88. Clarenc, J. P., Degols, G., Leonetti, J. P., Milhaud, P., and Lebleu, B. Delivery of antisense oligonucleotides by poly(L-lysine) conjugation and liposome encapsulation. Anticancer Drug Des., 8: 81-94, 1993. (Pubitemid 23119934)
89. Bielinska, A., Kukowska-Latallo, J. F., Johnson, J., Tomalia, D. A., and Baker, J. R., Jr. Regulation of in vitro gene expression using antisense oligonucleotides or antisense expression plasmids transfected using starburst PAMAM dendrimers. Nucleic Acids Res., 24: 2176-2182, 1996. (Pubitemid 26174562)
90. Zobel, H. P., Kreuter, J., Werner, D., Noe, C. R., Kumel, G., and Zimmer, A. Cationic polyhexylcyanoacrylate nanoparticles as carriers for antisense oligonucleotides. Antisense Nucleic Acid Drug Dev., 7: 483-493, 1997. (Pubitemid 27491371)
91. Chavany, C., Le Doan, T., Couvreur, P., Puisieux, F., and Helene, C. Polyalkylcyanoacrylate nanoparticles as polymeric carriers for antisense oligonucleotides. Pharm. Res., 9: 441-449, 1992.
92. Boussif, O., Lezoualc'h, F., Zanta, M. A., Mergny, M. D., Scherman, D., Demeneix, B., and Behr, J. P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. USA, 92: 7297-7301, 1995.
93. Dheur, S., Dias, N., van Aerschot, A., Herdewijn, P., Bettinger, T., Remy, J. S., Helene, C., and Saison-Behmoaras, E. T. Polyethylenimine but not cationic lipid improves antisense activity of 3′-capped phosphodiester oligonucleotides. Antisense Nucleic Acid Drug Dev., 9: 515-525, 1999. (Pubitemid 30040674)
94. Hughes, J. A., Aronsohn, A. I., Avrutskaya, A. V., and Juliano, R. L. Evaluation of adjuvants that enhance the effectiveness of antisense oligodeoxynucleotides. Pharm. Res., 13: 404-410, 1996. (Pubitemid 26112266)
95. Schwab, G., Chavany, C., Duroux, I., Goubin, G., Lebeau, J., Helene, C., and Saison-Behmoaras, T. 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: 10460-10464, 1994. (Pubitemid 24329017)
96. Derossi, D., Chassaing, G., and Prochiantz, A. Trojan peptides: the penetratin system for intracellular delivery. Trends Cell Biol., 8: 84-87, 1998. (Pubitemid 28056816)
97. Vives, E., Brodin, P., and Lebleu, B. 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, 1997. (Pubitemid 27265584)
98. Pichon, C., Freulon, I., Midoux, P., Mayer, R., Monsigny, M., and Roche, A. C. Cytosolic and nuclear delivery of oligonucleotides mediated by an amphiphilic anionic peptide. Antisense Nucleic Acid Drug Dev., 7: 335-343, 1997. (Pubitemid 27450404)
99. Spiller, D. G., Giles, R. V., Grzybowski, J., Tidd, D. M., and Clark, R. E. Improving the intracellular delivery and molecular efficacy of antisense oligonucleotides in chronic myeloid leukemia cells: a comparison of streptolysin-O permeabilization, electroporation, and lipophilic conjugation. Blood, 91: 4738-4746, 1998. (Pubitemid 28270324)
100. Spiller, D. G., and Tidd, D. M. Nuclear delivery of antisense oligodeoxynucleotides through reversible permeabilization of human leukemia cells with streptolysin O. Antisense Res. Dev., 5: 13-21, 1995.
101. Fisher, T. L., Terhorst, T., Cao, X., and Wagner, R. W. Intracellular disposition and metabolism of fluorescently-labeled unmodified and modified oligonucleotides microinjected into mammalian cells. Nucleic Acids Res., 21: 3857-3865, 1993. (Pubitemid 23254510)
102. Liu, Y., and Bergan, R. Improved intracellular delivery of oligonucleotides by square wave electroporation. Antisense Nucleic Acid Drug Dev., 11: 7-14, 2001. (Pubitemid 32199391)
103. Bergan, R., Connell, Y., Fahmy, B., and Neckers, L. Electroporation enhances c-myc antisense oligodeoxynucleotide efficacy. Nucleic Acids Res., 21: 3567-3573, 1993. (Pubitemid 23241494)
104. Juliano, R. L., Alahari, S., Yoo, H., Kole, R., and Cho, M. Antisense pharmacodynamics: critical issues in the transport and delivery of antisense oligonucleotides. Pharm. Res., 16: 494-502, 1999. (Pubitemid 29191292)
105. Ding, Y., and Lawrence, C. E. Statistical prediction of single-stranded regions in RNA secondary structure and application to predicting effective antisense target sites and beyond. Nucleic Acids Res., 29: 1034-1046, 2001. (Pubitemid 32186188)
106. Sczakiel, G. Theoretical and experimental approaches to design effective antisense oligonucleotides. Front. Biosci., 5: D194-D201, 2000.
107. Scherr, M., Rossi, J. J., Sczakiel, G., and Patzel, V. RNA accessibility prediction: a theoretical approach is consistent with experimental studies in cell extracts. Nucleic Acids Res., 28: 2455-2461, 2000. (Pubitemid 30420841)
108. Patzel, V., Steidl, U., Kronenwett, R., Haas, R., and Sczakiel, G. A theoretical approach to select effective antisense oligodeoxyribonucleotides at high statistical probability. Nucleic Acids Res., 27: 4328-4334, 1999. (Pubitemid 29533171)
109. Chiang, M. Y., Chan, H., Zounes, M. A., Freier, S. M., Lima, W. F., and Bennett, C. F. Antisense oligonucleotides inhibit intercellular adhesion molecule 1 expression by two distinct mechanisms. J. Biol. Chem., 266: 18162-18171, 1991. (Pubitemid 21908061)
110. Stull, R. A., Taylor, L. A., and Szoka, F. C., Jr. Predicting antisense oligonucleotide inhibitory efficacy: a computational approach using histograms and thermodynamic indices. Nucleic Acids Res., 20: 3501-3508, 1992.
111. Ding, Y., and Lawrence, C. E. A bayesian statistical algorithm for RNA secondary structure prediction. Comput. Chem., 23: 387-400, 1999. (Pubitemid 129752345)
112. Lloyd, B. H., Giles, R. V., Spiller, D. G., Grzybowski, J., Tidd, D. M., and Sibson, D. R. Determination of optimal sites of antisense oligonucleotide cleavage within TNFα mRNA. Nucleic Acids Res., 29: 3664-3673, 2001. (Pubitemid 32898782)
113. Mir, K. U., and Southern, E. M. Determining the influence of structure on hybridization using oligonucleotide arrays. Nat. Biotechnol., 17: 788-792, 1999. (Pubitemid 29365108)
114. Sohail, M., Hochegger, H., Klotzbucher, A., Guellec, R. L., Hunt, T., and Southern, E. M. Antisense oligonucleotides selected by hybridization to scanning arrays are effective reagents in vivo. Nucleic Acids Res., 29: 2041-2051, 2001. (Pubitemid 32472074)
115. Altman, R. K., Schwope, I., Sarracino, D. A., Tetzlaff, C. N., Bleczinski, C. F., and Richert, C. Selection of modified oligonucleotides with increased target affinity via MALDI-monitored nuclease survival assays. J. Comb. Chem., 1: 493-508, 1999.
116. Stein, C. A. The experimental use of antisense oligonucleotides: a guide for the perplexed. J. Clin. Investig., 108: 641-644, 2001. (Pubitemid 32906188)