Modification of phosphorothioate oligonucleotides yields potent analogs with minimal toxicity for antisense experiments in the CNS

Molecular Brain Research

Volumn 62, Issue 1, 1998, Pages 1-11

  Peng Ho, Siew ^a   Livanov, Valentin ^a   Zhang, Wei ^a   Li, Jing Hua ^a   Lesher, Treena ^a  

^a DUPONT COMPANY   (United States)

Author keywords

Antisense oligonucleotides; Corticotropin releasing factor type 2 receptor; Phosphorothioates; Ribonuclease H

Indexed keywords

ANTISENSE OLIGODEOXYNUCLEOTIDE; CORTICOTROPIN RELEASING FACTOR; IODINE 125; MESSENGER RNA; OLIGODEOXYNUCLEOTIDE PHOSPHOROTHIOATE; RIBONUCLEASE H; SAUVAGINE;

ANIMAL EXPERIMENT; ARTICLE; CONFORMATIONAL TRANSITION; DRUG POTENCY; GENE TECHNOLOGY; MALE; NEUROTOXICITY; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN RNA BINDING; PROTEIN TARGETING; RAT; STRUCTURE ACTIVITY RELATION;

ANIMALS; BRAIN; CENTRAL NERVOUS SYSTEM; DRUG TOXICITY; FEVER; IN SITU HYBRIDIZATION; INJECTIONS, INTRAVENTRICULAR; IODINE RADIOISOTOPES; MALE; MOLECULAR STRUCTURE; OLIGONUCLEOTIDES, ANTISENSE; PEPTIDES; RATS; RATS, SPRAGUE-DAWLEY; RECEPTORS, CORTICOTROPIN-RELEASING HORMONE; RIBONUCLEASE H, CALF THYMUS; RNA, MESSENGER; THIONUCLEOTIDES; WEIGHT LOSS;

ANIMALIA;

EID: 0345628579     PISSN: 0169328X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0169-328X(98)00185-5     Document Type: Article

References (59)

1. S. Agrawal, Antisense oligonucleotides: a possible approach for chemotherapy of AIDS, in: E. Wickstrom (Ed.), Prospects for Antisense Nucleic Acid Therapy of Cancer and AIDS, Wiley-Liss, New York, 1991, pp. 143.
2. Agrawal S., Temsamani J., Tang J.Y. Pharmacokinetics, biodistribution and stability of olideoxynucleotide phosphorothioates in mice. Proc. Natl. Acad. Sci. USA. 88:1991;7595-7599.
3. Akabayashi A., Wahlestedt C., Alexander J.T., Leibowitz S.F. Specific inhibition of endogenous neuropeptide Y synthesis in arcuate nucleus by antisense oligonucleotides suppresses feeding behavior and insulin secretion. Mol. Brain Res. 21:1994;55-61.
4. Brown D.A., Kang S.H., Gryaznov S.M., DeDionisio L., Heidenreich O., Sullivan S., Xu X., Nerenberg M.I. Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding. J. Biol. Chem. 269:1994;26801-26805.
5. Campbell J.M., Bacon T.A., Wickstrom E. Oligodeoxynucleoside phosphorothioate stability in subcellular extracts, culture media, sera and cerebrospinal fluid. J. Biochem. Biophys. Mtds. 20:1990;259-267.
6. Chiang M.Y., Chan H., Zounes M.A., Freier S.M., Lima W.F., Bennett C.F. Antisense oligonucleotides inhibit intercellular adhesion molecule 1 expression by two distinct mechanisms. J. Biol. Chem. 266:1991;18162-18171.
7. Chiasson B.J., Hooper M.L., Murphy P.R., Robertson H.A. Antisense oligonucleotide eliminates in vivo expression of c-fos in mammalian brain. Eur. J. Pharmacol. 227:1992;451-453.
8. S.T. Crooke, B. Lebleu (Eds.), Antisense Research and Applications, CRC Press, Boca Raton, 1993, 579 pp.
9. R.M. Crooke, In vitro and in vivo toxicology of first generation analogs, in: S.T. Crooke, B. Lebleu (Eds.), Antisense Research and Applications, CRC Press, Boca Raton, 1993, pp. 471-492.
10. Crooke S.T., Lemonidis K.M., Neilson L., Griffey R., Lesnik E.A., Monia B.P. Kinetic characteristics of Escherichia coli RNase H1: cleavage of various antisense oligonucleotide-RNA duplexes. Biochem. J. 312:1995;599-608.
11. Crooke S.T., Graham M.J., Zukerman J.E., Brooks D., Conklin B.S., Cummins L.L., Greig M.J., Gionosso C.J., Kornburst D., Manoharan M., Sasmor H.M., Schleich T., Tivel K.L., Giffery R.H. J. Pharmacol. Exp. Ther. 227:1996;923-937.
12. Giles R.V., Spiller D.G., Tidd D.M. Detection of ribonuclease G-generated mRNA fragments in human leukemia cells following reversible membrane permeabilization in the presence of antisense oligodeoxynucleotides. Antisense Res. Develop. 5:1995;23-31.
13. Gryaznov S., Chen J.K. Oligodeoxyribonucleotide N3′-P5′ phosphoramidates: synthesis and hybridization properties. J. Am. Chem. Soc. 116:1994;3143-3144.
14. Gryaznov S.M. Oligodeoxyribonucleotide N3′-P5′ phosphoramidates. Proc. Natl. Acad. Sci. USA. 92:1995;5798-5802.
15. R. Grzanna, J.R. Dubin, G.W. Dent, Z. Ji, W. Zhang, S.P. Ho, P.R. Hartig, Intrastriatal and intraventricular injections of oligodeoxynucleotides in the rat brain: tissue penetration, intracellular distribution and c-fos antisense effects, submitted to Mol. Brain Res.
16. Guvakova M.A., Yakubov L.A., Vlodavsky I., Tonkinson J.L., 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. 270:1995;2620-2627.
17. Hebb M.O., Robertson H.A. End-capped antisense oligodeoxynucleotides effectively inhibit gene expression in vivo and offer a low-toxicity alternative to fully modified phosphorothioate oligodeoxynucleotides. Mol. Brain Res. 47:1997;223-228.
18. Heinrichs S.C., Lapsansky J., Lovenberg T.W., Souza E.B.D., Chalmers D.T. Corticotropin-releasing factor CRF1, but not CRF2, receptors mediate anxiogenic-like behavior. Reg. Peptides. 71:1997;15-21.
19. Henry S.P., Grillone L.R., Orr J.L., Bruner R.H., Kornbrust D.J. Comparison of the toxicity profiles of ISIS 1082 and ISIS 2105, phosphorothioate oligonucleotides, following subacute intradermal administration in Sprague Dawley rats. Toxicology. 116:1997;77-88.
20. S.P. Ho, Y. Bao, T. Lesher, D. Conklin, Antisense inhibition of the angiotensin type-1 receptor occurs through an RNase H-type mechanism, submitted.
21. Ho S.P., Bao Y., Lesher T., Malhotra R., Ma L.Y., Fluharty S.J., Sakai R.R. Mapping of RNA accessible sites for antisense experiments with oligonucleotide libraries. Nature Biotechnology. 16:1998;59-63.
22. Inoue H., Hayase Y., Imura A., Iwai S., Miura K., Ohtsuka E. Synthesis and hybridization studies on two complementary nona (2′-O-methyl) ribonucleotides. Nucl. Acids Res. 15:1987;6131-6148.
23. Inoue H., Hayase Y., Iwai S., Ohtsuka E. Sequence dependent hydrolysis of RNA using modified oligonucleotide splints and RNase H. FEBS Lett. 215:1987;327-330.
24. Iverson P. In vivo studies with phosphorothioate oligonucleotides: pharmacokinetic prologue. Anti-Cancer Drug Des. 6:1992;531.
25. Keck B.J., Lakoski J.M. Region-specific serotonin 1A receptor turnover following irreversible blockade with EEDQ. NeuroReport. 7:1996;2717-2721.
26. M.J. Kuhar, J.R. Unnerstall, Receptor autoradiography, in: H.I. Yamamura (Ed.), Methods in Neurotransmitter Receptor Analysis, Raven Press, New York, 1990, pp. 177-218.
27. R. Leslie, J. Hunter, H. Robertson (Eds.), Antisense Oligonucleotide Gene Knockdown in the CNS, Oxford University Press, Oxford, 1998, in press.
28. C.K. Mirabelli, S.T. Crooke, Antisense oligonucleotides in the context of modern molecular drug discovery and development, in: S.T. Crooke, B. Lebleu (Eds.), Antisense Research and Applications, CRC Press, Boca Raton, 1993, pp. 7-36.
29. Monia B.P., Lesnik E.A., Gonzalez C., Lima W.F., McGee D., Guinosso C.J., Kawasaki A.M., Cook P.D., Freier S.M. Evaluation of 2′-modified inhibitors of gene expression. J. Biol. Chem. 268:1993;14514-14522.
30. Ogawa S., Brown H.E., Okano H.J., Pfaff D.W. Cellular uptake of intracerebrally administered oligodeoxynucleotides in mouse brain. Reg. Peptides. 59:1995;143-149.
31. Ortigao J.F.R., Rosch H., Selter H., Frohlich A., Lorenz A., Montenarh M., Seliger H. Antisense effect of oligodeoxynycleotides with inverted terminal internucleolytic linkages: a minimal modification protecting against nucleolytic degradation. Antisense Res. Develop. 2:1992;129-146.
32. Pasternak G.W., Childers S.R., Snyder S.H. opiate analgesia: evidence for mediation by a subpopulation of opiate receptors. Science. 208:1980;514-516.
33. Pasternak G.W., Childers S.R., Snyder S.H. Naloxazone, a long-acting opiate antagonist: effects on analgesia in intact animals and on opiate binding in vitro. J. Pharmacol. Exp. Ther. 214:1980;455-462.
34. Pezeshki G., Schobitz B., Pohl T., Reul J.M. Intracerebroventricular administration of missence oligodeoxynucleotide induces interleukin-6 mRNA expression in brain and spleen of rats. Neurosci. Lett. 217:1996;97-100.
35. Qin Z.H., Chen J.F., Weiss B. Lesions of mouse striatum induced by 6-hydroxydopamine differentially alter the density, rate of synthesis, and level of gene expression of D1 and D2 dopamine receptors. J. Neurochem. 62:1994;411-420.
36. R.B. Raffa, F. Porreca (Eds.), Antisense Strategies for the Study of Receptor Mechanisms, R.G. Landes, Austin, 1996, 224 pp.
37. D.H. Rominger, C.M. Rominger, L.W. Fitzgerald, R. Grzanna, B.L. Largent, R.J. Zaczek, Pharmacol. Exp. Ther., in press.
38. Sakai R.R., He P.F., Yang X.D., Ma L.Y., Guo Y.F., Reilly J.J., Moga C.N., Fluharty S.J. Intracerebroventricular administration of AT1 receptor antisense oligonucleotides inhibits the behavioral actions of angiotensin II. J. Neurochem. 62:1994;2053-2056.
39. Sands H., Gorey-Feret L.J., Cocuzza A.J., Hobbs F.W., Chidester D., Trainor G.L. Biodistribution and metabolism of internally 3H-labeled oligonucleotides: I. Comparison of a phosphodiester and a phosphorothioate. Mol. Pharmacol. 45:1994;932-943.
40. Sands H., Gorey-Feret L.J., Ho S.P., Bao Y., Cocuzza A.J., Chidester D., Hobbs F.W. Biodistribution and metabolism of internally 3H-labeled oligonucleotides: II. 3′. 5′-blocked oligonucleotides. Mol. Pharmacol. 47:1995;636-646.
41. Schobitz B., Pezeshki G., Probst J.C., Reul J.M., Skutella T., Stohr T., Holsboer F., Spanagel R. Centrally administered oligodeoxynucleotides in rats: occurence of non-specific effects. Eur. J. Pharmacol. 331:1997;97-107.
42. 4. Neuropsychopharmacology. 7:1992;261-284.
43. Seeman P. Therapeutic receptor-blocking concentrations of neuroleptics. Int. Clin. Psychopharm. 10:1995;5-13. Suppl. 3.
44. Shaw J.P., Kent K., Bird J., Fishback J., Froehler B. Nucl. Acids Res. 19:1991;747-750.
45. Skutella T., Stohr T., Probst J.C., Ramalho-Ortigao F.J., Holsboer F., Jirikowski G.F. Antisense oligodeoxynucleotides for in vivo targeting of corticotropin-releasing hormone mRNA: comparison of phosphorothioate and 3′-inverted probe performance. Horm. Metab. Res. 26:1994;460-464.
46. Skutella T., Montkowski A., Stohr T., Probst J.C., Landgraf R., Holsboer F., Jirikowski G.F. Corticotropin releasing hormone antisense oligodeoxynucleotide treatment attenuates social defeat-induced anxiety in rats. Cell. Mol. Neurobiol. 14:1994;579-588.
47. Standifer K.M., Chien C.C., Wahlestedt C., Brown G.P., Pasternak G.W. Selective loss of δ opioid analgesia and binding by antisense oligodeoxynucleotides to a δ opioid receptor. Neuron. 12:1994;805-810.
48. Szklarczyk A., Kaczmarek L. Antisense oligodeoxyribonucleotides: stability and distribution after intracerebral injection into rat brain. J. Neurosci. Mtds. 60:1995;181-187.
49. Tonkinson J.L., Guvakova M., Khaled Z., Lee J., Yakubov L., Marshall W.S., Caruthers M.H., Stein C.A. Cellular Pharmacology and protein binding of phosphoromonothioate and phosphorodithioate oligodeoxynucleotides: a comparative study. Antisense Res. Develop. 4:1994;269-278.
50. Wagner R.W., Matteucci M.D., Lewis J.G., Gutierrez A.J., Moulds C., Froehler B.C. Antisense gene inhibition by oligonucleotides containing C-5 propyne pyrimidines. Science. 260:1993;1510-1513.
51. Wagner R.W., Matteucci M.D., Grant D., Huang T., Froehler B.C. Potent and selective inhibition of gene expression by an antisense heptanucleotide. Nature Biotechnol. 14:1996;840-844.
52. Wahlestedt C., Pich E.M., Koob G.F., Yee F., Heilig M. Modulation of anxiety and neuropeptide Y-Y1 receptors by antisense oligodeoxynucleotides. Science. 259:1993;528-531.
53. Wahlestedt C., Golanov E., Yamamoto S., Yee F., Ericson H., Yoo H., Inturrisi C.E., Reis D.J. Antisense oligodeoxynucleotides to the NMDAR1 receptor channel protect corticol neurones from excitotoxicity and reduce focal ischemic infarctions in rat. Nature. 363:1993;260-262.
54. Wahlestedt C. Antisense oligonucleotide strategies in neuropharmacology. Trends Pharmacol. Sci. 15:1994;42-46.
55. Ward S.J., Portoghese P.S., Takemori A.E. Pharmacological characterization in vivo of the novel opiate, β-funaltrexamine. J. Pharmacol. Exp. Ther. 220:1982;494-498.
56. Weiss B., Zhou L.W., Zhang S.P., Qin Z.H. Neuroscience. 55:1993;607-612.
57. Whitesell L., Geselowitz D., Chavany C., Fahmy B., Walbridge S., Alger J.R., Neckers L.M. Stability, clearance and disposition of intraventricularly administered oligodeoxynucleotides: implications for therapeutic application within the central nervous system. Proc. Natl. Acad. Sci. USA. 90:1993;4665-4669.
58. Yaida Y., Nowak J., Nowak T.S. Distribution of phosphodiester and phosphorothioate oligonucleotides in rat brain after intraventricular and intrahippocampal administration determined by in situ hybridization. Reg. Peptides. 59:1995;193-199.
59. Zhang M., Creese I. Antisense oligodeoxynucleotide reduces brain dopamine D2 receptors: behavioral correlates. Neurosci. Lett. 161:1993;223-226.