Antisense approaches to the gene therapy of cancer - 'Recnac'

Cancer and Metastasis Reviews

Volumn 15, Issue 3, 1996, Pages 287-299

  Gibson, Ian ^a  

^a UNIVERSITY OF EAST ANGLIA   (United Kingdom)

Author keywords

antisense; oncogenes; ribozymes

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; COMPLEMENTARY DNA; COMPLEMENTARY RNA; RIBOZYME;

CANCER; GENE THERAPY; HUMAN; PRIORITY JOURNAL; REVIEW;

EID: 0029805590     PISSN: 01677659     EISSN: None     Source Type: Journal    
DOI: 10.1007/BF00046343     Document Type: Review

References (73)

1. Bos JL: Ras gene mutations and human cancer. In: Crossman J (ed) Molecular Biology of Human Cancer, Elsevier. Amsterdam, pp 273-287, 1990
2. Nauber U, Schuh R, Jäckle H: Applications of antisense nucleic acids in developmental biology: Drosophila. In: Mol JNM, van der Krol AR (eds) Antisense Nucleic Acids and Proteins, Fundamentals and Applications. Marcel Dekker, Paris, pp 133-124, 1991
3. Hawkins JW: Oligonucleotide Therapeutics: Coming 'Round the Clubhouse Turn'. Antisense Res & Dev 5: 1, 1995
4. Neckers L, Whitesell L, Rosolen A, Geselowitz DA: Antisense inhibition of oncogene expression. Crit Rev Oncogenes 3: 175-231, 1992
5. Egholm M, Nielsen PE, Buchardt O, Berg RH: Recognition of guanine and adenine in DNA by cytosine and thymine containing peptide nucleic acids (PNA). J Am Chem Soc 114: 9677-9678, 1992
6. Zamecnik PC, Stephenson ML: Inhibition of Rous sarcoma virus replication and transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci USA 75: 285-288, 1978
7. Matsukura M, Zon G, Shinozuka K, Stein CA, Mitsuya H, Cohen JS, Broder S: Synthesis of phosphorothioate analogues of oligodeoxyribonucleotides and their antiviral activity against human immunodeficiency virus (HIV). Gene 72: 343-347, 1988
8. Ratajayak MZ, Hijyva N, Catani L, DeRiel K, Luger SM, McGlave P, Gewirtz AM: Acute- and chronic-phase chronic myelogenous leukemia colony-forming units are highly sensitive to the growth inhibitory effects of c-myb antisense oligodeoxynucleotides. Blood 79: 1956-1961 (1992a)
9. Skorski T, Nieborowska-Skorska M, Nicolaides NC, Szczylyk C, Iversen P, Iozzo RV, Zon G, Calabretta B: Suppression of Philadelphia-leukemia cell growth in mice by bcr-abl antisense oligodeoxynucleotides. Proc Natl Acad Sci USA 91: 4504-4508, 1994
10. Bayever E, Iversen PL, Bishop MR, Sharp JG, Tewary HK, Arneson MA, Pirrucello SJ, Ruddon RW, Kessinger A, Zon G: Systemic administration of a phosphorothioate oligonucleotide with a sequence complementary to p53 for acute myelogenous leukemia and myelodysplastic syndrome: Initial results of a phase I trial. Antisense Res & Dev 3: 383-390, 1993
11. Jansen B, Wadl H, Inoue S, Trülzsch B, Selzer E, Duchêne M, Eichler H-G, Wolff K, Pehamberger H: Phosphorothioate oligonucleotides reduce melanoma growth in a SCID-hu mouse model by a nonantisense mechanism. Antisense Res & Dev 5: 271-277, 1995
12. Kobayashi M, Yamamaguchi Y, Yamaguchi K, Tanaka A: Transient expression assay for antisense RNAs using episomal replication of plasmids: Effective reduction of retinoblastoma gene (Rb-1) product by its antisense RNA complementary to 3′-untranslated region. Antisense Res & Dev 5: 141-148, 1995
13. Spiller DG, Tidd DM: The uptake kinetics of chimeric oligodeoxynucleotide analogues in human leukaemia MOLT-4 cells. Anti-Cancer Drug Res 7: 115-119, 1992
14. Giles RV, Spiller DG, Green JA, Clark RE, Tidd DM: Optimization of antisense oligodeoxynucleotide structure for targeting bcr/abl mRNA. Blood 86: 744-754, 1995
15. Giles RV, Spiller DG, Tidd DM: Chimeric oligodeoxynucleotide analogues: Enhanced cell uptake of structures which direct ribonuclease H with high specificity. Anti-Cancer Drug Des 8: 33-51, 1993
16. Grigoriev M, Praseuth D, Robin P, Hemar A, Saison-Behmoaras T, Dautry-Varsat A, Thuong NT, Helen C, Harel-Bellan A: A triple helix-forming oligonucleotide-intercalator conjugate acts as a transcriptional represser via inhibition of NF KB binding to IL-2-receptor α regulatory sequence. J Biol Chem 267: 3389-3395, 1992
17. Grigoriev M, Praseuth D, Guieysse RL, Robin P, Thuong NT, Helene C, Harel-Bellan A: Inhibition of gene expression by triple helix-directed site-specific crosslinking. Proc Natl Acad Sci USA 90: 35-1-3505, 1993
18. Giovannangeli C, Thuong NT, Helen C: Oligonucleotide clamps arrest DNA synthesis on a single-stranded DNA target. Proc Natl Acad Sci USA 90: 10013-10017, 1993
19. Tidd D, Hawley T, Gibson I: Synthesis and evaluation of N-ras antisense and nonsense methylphosphonate oligonucleotide analogues. Brit J Cancer 58: 225, 1988
20. O'Brien SG, Kirkland MA, Davidson RJ, McDonald C, Goldman JM: Antisense oligodeoxynucleotides for in vitro purging of autologous hone marrow in chronic phase chronic myeloid leukaemia. Blood 82: Suppl 1, 119a, 1993
21. Kirkand MA, Rao H, O'Brien SG, McDonald C, Davidson RJ, Goldman JM: Non-sequence specific effects of bcr-abl antisense oligomers on CML cell lines. Blood 82, Suppl. 1, 40a, 1993
22. Smetsers TFCM, Skorski T, van de Locht LTF, Wessels HMC, Pennings AHM, de Witte T, Calabreta B, Mensink EJBM: Antisense BCR-ABL oligonucleotides induce apoptosis in the Philadelphia chromosome-positive cell line BV173. Leukemia 129: 140, 1994
23. Bennett CF, Chiang M-Y, Chang H, Shoemaker JEE, Mirabelli CK: Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides. Mol Pharmacol 41: 1023-1033, 1992
24. Zelphati O, Zon G, Leserman L: Inhibition of HIV-1 replication in cultured cells with antisense oligonucleotides encapsulated in immunoliposomes. Antisense Res & Dev 3: 323-338, 1993
25. Thierry AR, Dritschilo S: Intercellular availability of unmodified, phosphorothioated and liposomally encapsulated oligodeoxynucleotides for antisense activity. Nucleic Acid Res 20: 5961-5968, 1992
26. Lemaitre M, Bayard B, Lebleu B: Specific antiviral activity of a poly(L-lysine)-conjugated oligodeoxyribonucleotide sequence complementary to vesicular stomatitis virus N protein mRNA initiation site. Proc Nail Acad Sci USA 84: 648-652, 1987
27. Saison-Behmoaras T, Tocque B, Rev I, Chassignol M, Thuong NT, Helene C: Short modified antisense oligonucleotides directed against Ha-ras point mutation induce selective cleavage of the mRNA and inhibit T24 cells proliferation. EMBO J 10: 1111-1118, 1991
28. Krieg AM, Tonkinson J, Matson S, Zhao Q, Saxon M, Zhang L-M, Bhanja U, Yakubov L, Stein CA: Modification of antisense phosphodiester oligodeoxynucleotides by a 5′ cholesteryl moiety increases cellular association and improves efficacy. Proc Natl Acad Sci USA 90: 1048-1052, 1993
29. Boutorine AS, Kostina EV: Reversible covalent attachment of cholesterol to oligodeoxyribonucleotides for studies of the mechanisms of their penetration into eucaryotic cells. Biochimie 75: 35-41, 1993
30. Loke SL, Stein CA, Zhang XH, Mori K, Nakanishi M, Subasinghe C, Cohen JS, Neckers LM: Characterization of oligonucleotide transport into living cells. Proc Natl Acad Sci USA 86: 3474-3478, 1989
31. Shoyi Y, Akhtar S, Periasamy A, Herman B, Juliano RL: Mechanism of cellular uptake of modified oligodeoxynucleotides containing methylphosphonate linkages. Nucleic Acid Res 19: 5543-5550, 1991
32. Bergan R, Connell Y, Fahmy B, Neckers L: Electroporation enhances c-myc antisense oligodeoxynucleotide efficacy. Nucleic Acids Res 21: 3567-3573, 1993
33. Noonberg SB, Garovoy MR, Hunt CA: Characteristics of oligonucleotide uptake in human keratinocyte cultures. J Invest Dermatol 101: 727-731, 1993
34. Leonetti J-P, Mechti N, Degols G, Gagnor C, Lehleu B: Intracellular distribution of microinjected antisense oligonucleotides. Proc Natl Acad Sci USA 88: 2702-2706, 1991
35. Fisher TL, Terhorst T, Cao X, Wagner RW: Intracellular disposition and metabolism of fluorescently-labelled unmodified and modified oligonucleotides microinjected into mammalian cells. Nucleic Acid Res 21: 3857-3865, 1993
36. Yakubov L, Deeva EA, Zarytova F, Ivanova M, Ryte AS, Yrchenko LV, Vlassov VV: Mechanism of oligonucleotide uptake by cells: Involvement of specific receptors? Proc Natl Acad Sci USA 86: 6454-6458, 1989
37. Zhao O, Matson S, Herrera CJ, Fisher E, Yu H, Krieg AM: Comparison of cellular binding and uptake of antisense phosphodiester, phosphorothioate, and mixed phosphorothioate and methylphosphonate oligonucleotides. Antisense Res & Dev 3: 53-66, 1993
38. Temsamani J, Tang J-Y, Padmapriya A, Kubert M, Agrawal S: Pharmacokinetics, biodistribution, and stability of capped oligodeoxynucleotide phosphorothioates in mice. Antisense Res & Dev 3: 277-284, 1993
39. Barry ELR, Gesek FA, Friedman PA: Introduction of antisense oligonucleotides into cells by permeabilization with streptolysin O. Bio Techniques 15: 1016-1020, 1993
40. Thieringer HA, Takayama KM, Kang C, Inouye M: Antisense RNA-mediated inhibition of mouse hepatitis virus replication in L2 cells. Antisense Res & Dev 5: 289-294, 1995
41. Woolf TM: To Cleave or Not to Cleave: Ribozymes and Antisense Res & Dev 5: 227-232, 1995
42. Stein CA, Cheng Y-C: Antisense oligonucleotides as therapeutic agents - is the bullet really magical? Science 261: 1004-1012, 1993
43. Stein CA, Krieg AM: Problems in interpretation of data derived from in vitro and in vivo use of antisense oligodeoxynucleotides. Antisense Res & Dev 4: 69-69, 1994
44. Milligan JF, Matteucci MD, Martin JC: Current concepts in antisense drug design. J Med Chem 36: 1923-1937, 1993
45. Yaswen P, Stampfer MR, Ghosh K, Cohen JS: Effects of sequence of thioated oligonucleotides on cultured human mammary epithelial cells. Antisense Res & Dev 3: 67-77, 1993
46. Burgess TL, Fisher EF, Ross SL, Bready JV, Qian Y-X, Baywitch LA, Cohen AM, Herrera CH, Hu SS-F, Kramer TB, Lott FD, Martin FH, Pierce GF, Simonet L, Farrell CL: The antiproliferative activity of c-myb and c-myc antisense oligonucleotide in smooth muscle cells is caused by a nonantisense mechanism. Proc Natl Acad Sci USA 92: 4051-4055, 1995
47. Agrawal S, Temsamani J, Tang JY: Pharmacokinetics, biodistribution, and stability ot oligodeoxynucleotide phosphorothioates in mice. Proc Natl Acad Sci USA 88: 7595-7599, 1991
48. Miyao T, Takakura Y, Akiyama T, Yoneda F, Sezaki H, Hashida M: Stability and pharmacokinetic characteristics of oligonucleotides modified at terminal linkages in mice. Antisense Res & Dev 5: 115-121, 1995
49. Zendegni JG, Vasquez KM, Tinsley JH, Kessler DJ, Hogan ME: In vivo stability and kinetics of absorption and disposition of 3′ phosphopropyl amine oligonucleotides. Nucleic Acid Res 20: 307-314, 1992
50. Sawai K, Miyao T, Takakura Y, Hashida M: Renal disposition characteristics of oligonucleotides modified at terminal linkages in the perfused rat kidney. Antisense Res & Dev 5: 279-287, 1995
51. Ohta Y, Kijima H, Ohkawa T, Kashani-Sabet K, Scanlon KJ: Tissue-specific expression of an anti-ras ribozyme inhibits proliferation of human malignant melanoma cells. Nucleic-Acid Res 24, No. 5, 1996
52. Haseloff J, Gerlach WL: Simple RNA enzymes with new and highly specific endonuclease activities. Nature (Lond) 334: 585-591
53. Tabler M: Antisense RNA in plants: a tool for analysis and suppression of gene function. In: Roubelakis-Angelakis KA, Tran Thanh Van K (eds) Morphogenesis in Plants: Molecular Approaches, NATO ASI Series, A243, Plenum, New York, pp 237-258, 1993
54. Flory CM, Pavco PA, Jarvis TC, Lesch ME, Wincott FE, Beigelman L, Hunt SW, Schrier DJ: Nuclease-resistant ribozymes decrease stromelysin mRNA levels in rabbit synovium following exogenous delivery to the knee joint. Proc Natl Acad Sci USA 93: 754-758, 1996
55. Zhao JJ, Pick L: Generating loss-of-function phenotypes of the fushi tarazu gene with a targeted ribozyme in Drosophila. Nature 365: 448-451, 1993
56. Heinrich J-C, Tabler M, Louis C: Attenuation of white gene-expression in transgenic Drosophila melanogaster. Possible role of a catalytic antisense RNA. Dev Genet 14: 258-265, 1993
57. Heinrich J-C, Tabler M, Louis C: Influence of chromosomal position and copy number of a white-directed ribozyme gene on the suppression of eye pigmentation in Drosophila melanogaster. Antisense Res & Dev 5: 155-160, 1995
58. Bass B: The double-stranded RNA unwinding-modifying activity. In: Murray JAH (ed) Antisense RNA and DNA. Wiley, New York pp 159-175, 1992
59. Daley GQ, Van Etten RA, Baltimore D: Induction of chronic myelogenous leukemia in mice by the p210 bcr/abl gene of the Philadelphia chromosome. Science 247: 824-830, 1990
60. Bernards A, Rubin CM, Westbrook CA, Paskind M, Baltimore D: The first intron in the human c-abl gene is at least 200 kilobases long and is a target for translocations in chronic myelogenous leukemia. Mol Cell Biol 7: 3231-3236, 1987
61. Leopold LH, Shore SK, Newkirk TA, Reddy RMV, Reddy EP: Multi-unit ribozyme-mediated cleavage of bcr-abl mRNA in myeloid leukemias. Blood 85: 2162-2170, 1995
62. Pachuk CJ, Yoon K, Moelling K, Coney LR: Selective cleavage of bcr-abl chimeric RNAs by a ribozyme targeted to non-contiguous sequences. Nucleic Acid Res 22: 301-307, 1994
63. Wright L, Wilson SB, Milliken S, Biggs J, Kearney P: Ribozyme-mediated cleavage of the bcr-abl transcript expressed in chronic myeloid leukemia. Expt Hematology 21: 1714-1718, 1993
64. Szczylik C, Skorski T, Nicolaides NC, Manzella L, Malaguarnera L, Venturelli D, Gewirtz AM, Calabretta B: Selective inhibition of leukemia cell proliferation by bcr-abl antisense oligonucleotides. Science 253: 562-565, 1991
65. Bergen R, Connell Y, Fahmy B, Kyle E, Neckers L: Aptameric inhibition of p210 bcrabl tyrosine kinase autophosphorylation by oligodeoxynucleotides of defined sequence and backbone structure. Nucleic Acid Res 22: 2150-2154, 1994
66. Bergen RC, Kyle E, Connell Y, Neckers L: Inhibition of protein-kinase activity in intact cells by the aptameric action of oligodeoxynucleotides. Antisense Res & Dev 5: 33-38, 1995
67. O'Brien SG, Kirkland MA, Melo JV, Rao MH, Davidson RJ, McDonald C, Goldman JM: Antisense bcr-abl oligomers cause non-specific inhibition of chronic myeloid leukemia cell lines. Leukemia 8: 2156-2162, 1994
68. Thomas M, Kosciolek B, Wang N, Rowley P: Capping of bcr-abl antisense oligonucleotides enhances anti-proliferative activity against chronic myeloid leukemia cell lines. Leuk Res 18: 401-408, 1994
69. Tari AM, Tucker SD, Deisseroth A, Lopez-Berestin G: Liposomal delivery of methylphosphonate antisense oligodeoxynucleotides in chronic myelogous leukemia. Blood 84: 601-607, 1994
70. Lange W, Cantin EM, Finke J, Dolken G: In vitro and in vivo effects of synthetic ribozymes targeted against bcr/abl mRNA. Leukemia 7: 1786-1794, 1993
71. Shore SK, Nabissa DM, Reddy EP: Ribozyme-mediated cleavage of the bcr/abl oncogene transcript: in vitro cleavage of RNA and in vivo loss of p210 protein-kinase activity. Oncogene 8: 3183-3188, 1993
72. Snyder DS, Wu Y, Wang JL, Rossi JJ, Swiderski P, Kaplan BE, Forman SJ: Ribozyme-mediated inhibition of bcr-abl gene expression in a Philadelphia chromosome-positive cell line. Blood 82: 600-605, 1993
73. Wright L, Wilson SB, Milliken S, Biggs J, Kearney P: Ribozyme-mediated cleavage of the bcr/abl transcript expressed in chronic myeloid leukemia. Expt Hematology 21: 1714-1718, 1993