Drug delivery of antisense molecules to the brain for treatment of alzheimer's disease and cerebral aids

Journal of Pharmaceutical Sciences

Volumn 87, Issue 11, 1998, Pages 1308-1315

  Boado, Ruben J ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMYLOID BETA PROTEIN; ANTISENSE OLIGONUCLEOTIDE; ENDONUCLEASE; NUCLEIC ACID; RIBONUCLEASE H; STREPTAVIDIN; TRANSFERRIN RECEPTOR;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ALZHEIMER DISEASE; CONFERENCE PAPER; DRUG BINDING; DRUG BRAIN LEVEL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG TRANSPORT; ENZYME DEGRADATION; EXPRESSION VECTOR; HUMAN; HUMAN CELL; HUMAN TISSUE; MEMBRANE PERMEABILITY; MOLECULAR RECOGNITION;

EID: 0031763412     PISSN: 00223549     EISSN: None     Source Type: Journal    
DOI: 10.1021/js9800836     Document Type: Article

References (69)

1. Mercola, D.; Cohen J. S. Antisense approaches to cancer gene therapy. Cancer Gene Ther. 1995, 2, 47-59.
2. Wagner, R. W.; Flanagan, W. M. Antisense technology and prospects for therapy of viral infections and cancer. Mol. Med. Today 1997, 3, 31-38.
3. Curcio, L. D.; Bouffard, D. Y.; Scanlon, K. J. Oligonucleotides as modulators of cancer gene expression. Pharmacol. Ther. 1997, 74, 317-332.
4. Boado, R. J. Antisense drug delivery through the blood-brain barrier. Adv. Drug Del. Rev. 1995, 15, 73-107.
5. Pilowsky, P. M.; Suzuki, S.; Minson, J. B. Antisense oligonucleotides: a new tool in neuroscience. Clin. Exp. Pharmacol. Physiol. 1994, 21, 935-944.
6. Ogawa, S.; Pfaff, D. W. Application of antisense DNA method for the study of molecular bases of brain function and behavior. Behav. Genet. 1996, 26, 279-292.
7. Weiss, B.; Davidkova, G.; Zhang, S.-P. Antisense strategies in neurobiology. Neurochem. Int. 1997, 31, 321-348.
8. Le Corre, S. M.; Burnet, P. W. J.; Meiler, R.; Sharp, T.; Harrison, P. J. Critical issues in the antisense inhibiton of brain gene expression in vivo: experiences targeting the 5-HTiA receptor. Neurochem. Int. 1997, 31, 349-362.
9. Pardridge, W. M. Transport of small molecules through the blood-brain barrier: biology and methodology. Adv. Drug Del. Rev. 1995, 15, 5-36.
10. Dorri, F.; Hampson, D. R.; Baskys, A.; Wojtowicz, J. M. Down-regulation of rnGluRS by antisense deoxynucleotides alters pharmacological responses to applications of ACPD in the rat hippocampus. Exp. Neural. 1997, 147, 48-54.
11. 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. Sei. U.S.A. 1993, 90, 4665-4669.
12. Agrawal, S. Antisense oligonucleotides: a possible approach for chemotherapy of AIDS. In: Prospects for Antisense Nucleic Acid Therapy of Cancer and AIDS; Wickstrom, E., Ed.; Wiley-Liss: New York, 1991; pp 143-158.
13. Wojcik, W.,!.; Swoveland, P.; Zhang, X.; Vanguri, P. Chronic intrathecal infusion of phosphorothioate or phosphodiester antisense oligonucelotides against cytokine responsive gene2/IP-10 in experimental allergic encephalomyelitis of Lewis rat. J. Pharmacol. Exp. Ther. 1996, 278, 404-410.
14. Boado, R. J.; Pardridge, W. M. Complete inactivation of target mRNAby biotinylated antisense oligodeoxynucleotide-avidin conjugates. Bioconj. Chem. 1994, 5, 406-410.
15. Kang, Y.-S.; Boado, R. J.; Pardridge, W. M. Pharmacokinetics and organ clearance of a 3'-biotinylated, internally [^Pilabeled phosphodiester oligodeoxynucleotide coupled to a neutral avidin/monoclonal antibody conjugate. Drug Metab. Dispos. 1995, 23, 55-59.
16. Whiteside, C.; Silverman, M. Determination of postglomerular perme-selectivity to neutral dextrans in the dog. Am. J. Physiol. 1983, 245, 496-505.
17. Me, S.; Kishimoto, T.; Tavassoli, M. Desialation of transferrin by rat liver endothelium. J. Clin. Invest. 1988, 82, 508-513.
18. Zhao, Q.; Matson, S.; Herrera, C. J.; Fisher, E.; Yu, H.; Krieg, A. M. Comparison of cellular binding and uptake of antisense phosphodiester, phosphorothioate, and mixed phosphorothioate and methylphosphonate oligonucleotides. Antisense Res. Devel. 1993, 3, 53-66.
19. Giles, R. V.; Spiller, D. G.; Tidd, D. M. Chimeric oligodeoxynucleotide analogues: enhanced cell uptake of structures which direct ribonuclease H with high specificity. AntiCancer Drug Design 1993, 8, 33-51.
20. 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. 1994, 269, 2680126805.
21. Ehrlich, G.; Patinkin, D.; Ginzberg, D.; Zakut, H.; Eckstein, F.; Soreq, H. Use of partially phosphorothioated "antisense" oligodeoxynucleotides for sequence-dependent modulation of hematopoiesis in culture. Antisense Res. Devel. 1994,4,173183.
22. Agrawal, S.; Jiang, Z.; Zhao, Q.; Shaw, D.; Cai, Q.; Roskey, A.; Channavajjala, L.; Saxinger, C.; Zhang, R. Mixedbackbone oligonucleotides as second generation antisense oligonucelotides: in vitro and in vivo studies. Proc. Natl. Acad. Sei. U.S.A. 1997, 94, 2620-2625.
23. Boado, R. J.; Kang, Y.-S.; Wu, D.; Pardridge, W. M. Rapid plasma clearance and metabolism in vivo of a phosphorothioate oligodeoxynucleotide with a single, internal phosphodiester bond. Drug Metab. Dispos. 1995, 23, 1297-1300.
24. Boado, R. J.; Pardridge, W. M. Complete protection of antisense oligonucleotides against serum nuclease degradation by an avidin-biotin system. Bioconj. Chem. 1992, 3, 519-523.
25. Crooke, R. M. In vitro toxicology and pharmacokinetics of antisense oligonucleotides. Anti-Cancer Drug Design 1991, 6, 609-646.
26. Ratajczak, M. Z.; Kant, J. A.; Luger, S. M.; Hijiya, N.; Zhang, J.; Zon, G.; Gewirtz, A. M. In vivo treatment of human leukemia in a seid mouse model with c-myb antisense oligodeoxynucleotides. Proc. Natl. Acad. Sei. U.S.A. 1992,89, 11823-11827.
27. Srinivasan, S. K.; Iversen, P. Review of in vivo pharmacokinetics and toxicology of phosphorothioate oligonucleotides. J. Clin. Lab. Anal. 1995, 9, 129-137.
28. Wu, D.; Boado, R. J.; Pardridge, W. M. Pharmacokinetics and blood-brain barrier transport of [3H]-biotinylated phosphorothioate oliogodeoxynucleotide conjugated to a vector-mediated drug delivery system. J. Pharmacol. Exp. Ther. 1996, 276, 206-211.
29. Matsukura, M.; Mitsuya, H.; Broder, S. A new concept in AIDS treatment: an antisense approach and its current status towards clinical application. In Prospects for Antisense Nucleic Acid Therapy of Cancer and AIDS; Wickstrom, E., Ed.; Wiley-Liss: New York, 1991; pp 159-178.
30. Demidov, V. V.; Potaman, V. N.; Frank-Kamenetskii, M. D.; Egholm, M.; Buchard, O.; Sonnichsen, S. H.; Nielsen, P. E. Stability of peptide nucleic acids in human serum and cellular extracts. Biochem. Pharmacol. 1994,48,1310-1313.
31. Pardridge, W. M.; Eisenberg, J.; Yang, J. Human blood-brain barrier transferrin receptor. Metabolism 1987,36, 892-895.
32. Jefferies, A. W.; Brandon, M. R.; Hunt, S. V.; Williams, A. F.; Gatter, K. C.; Mason, D. Y. Transferrin receptor on endothelium of brain capillaries. Nature 1984,312,162-163.
33. Pardridge, W. M.; Boado, R. J.; Kang, Y.-S. Vector-mediated delivery of a polyamide ("peptide") nucleic acid analogue through the blood-brain barrier in vivo. Proc. Natl. Acad. Sei. U.S.A. 1995, 92, 5592-5596.
34. Wu, D.; Kang, Y.-S.; Bickel, U.; and Pardridge, W. M. Bloodbrain barrier permeability to morphine-6-glucuronide is markedly reduced compared to morphine. Drug Metab. Dispos. 1997, 25, 768-771.
35. Katzman, R. Alzheimer's disease. N. Engl. J. Med. 1986,314, 964-973.
36. Selkoe, D. J. The molecular pathology of Alzheimer's disease. Neuron 1991, 6, 487-498.
37. Brightman, M. W. Morphology of blood-brain interfaces. Exp. Eye Res. 1977, 25, 1-25.
38. Pardridge, W. M. Brain drug delivery and blood-brain barrier transport. Drug Deliv. 1993, 1, 83-101.
39. Bahmanyar, S.; Higgins, G. A.; Goldgaber, D.; Lewis, D. A.; Morrison, J. H.; Wilson, M. C.; Shankar, S. K.; Gajdusek, D. C. Localization of amyloid beta protein messenger RNA in brains from patients with Alzheimer's disease. Science 1987, 237, 77-80.
40. Goedert, M. Neuronal localization of amyloid beta protein precursor mRNA in normal human brain and in Alzheimer's disease. EMBO J. 1987, 6, 3627-3632.
41. Goldgaber, D.; Lerman, J. I.; McBride, O. W.; Saffiotti, U.; Gajdusek, D. C. Characterization and chromosomal localization of a cDNA encoding brain amyloid of Alzhaimer's disease. Science 1987, 235, 877-880.
42. Kang, J.; Lemaire, H. G.; Unterbeck, A.; Salbaum, J.; Masters, L.; Grzeschik, K. H.; Multhaup, G.; Beyreuther, K.; Muller-Hill, B. The precursor of Alzheimer's disease amyloid A4 protein resembles a cell-surface receptor. Nature 1987, 325, 733-736.
43. Robakis, N. K.; Ramakrishna, N.; Wolfe, G.; Wisniewski, H. M. Molecular cloning and characterization of a cDNA encoding the cerebrovascular and the neuritic plaque amyloid peptides. Proc. Natl. Acad. Sei. U.S.A. 1987,84,4190-4194.
44. Tanzi, R. E,; Gusella, J. F.; Watkins, P. C.; Bruns, G. A.; St. George-Hyslop; P.; VanKeuren, M. L.,; Patterson, S. P.; Kurnit, D. H.; Neve, R. L. Amyloid beta protein gene: cDNA, mRNA distribution, and genetic likage near the Alzheimer locus. Science 1987, 235, 880-884.
45. Goldgaber, D.; Schmechel, D. E. Expression of the amyloid beta-protein precursor gene. Adv. Neural. 1990, 51, 163169.
46. Higgins, G. A.; Lewis, D. A.; Bahmanyar, S.,; Goldgaber, D.; Gajdusek, D. C.; Young, W. G.; Morrison, J. H.; Wilson, M. C. Differential regulation of amyloid-beta-protein mRNA expression within hippocampal neuronal subpopulations in Alzheimer disease. Proc. Natl. Acad. Sei. U.S.A. 1987, 85, 1297-1301.
47. Lewis, D. A.; Higgins, G. A.; Young, W. G.; Goldgaber, D.; Gajdusek, D. C.,; Wilson, M. C.; Morrison, J. H. Distribution of precursor amyloid-beta-protein messenger RNA in human cerebral cortex: relationship to neurofibrillary tangles and neuritic plaques. Proc. Natl. Acad. Sei. U.S.A. 1987, 85, 1691-1695.
48. Neve, R. L.; Finch, E. A.; Dawes, L. R. Expression of the Alzheimer amyloid precursor gene transcripts in the human brain. Neuron 1988, 1, 669-677.
49. Tanzi, R. E.; Hyman, B. T. Studies of amyloid beta-protein precursor expression in Alzheimer's disease. Ann. N.Y. Acad. Sei. 1991, 640, 149-154.
50. Palmert, M. R.; Golde, T. E.; Cohen, M. L.; Kovacs, D. M.; Tanzi, R. E.; Gusella, J. F.; Usiak, M. F.; Younkin, L. H.; Younkin, S. C. Amyloid protein precursor messenger RNAs: differential expression in Alzheimer's disease. Science 1988, 242, 1080-1084.
51. Jacobsen, S. J.; Blume, A. J.; Vitek, M. P. Quantitative measurement of alternatively spliced amyloid precursor protein mRNA expression in Alzheimer's disease and normal brain by SI nuclease protection analysis. Neurobiol. Aging 1991, 12, 585-592.
52. Takashima, A.; Noguchi, K.; Sato, K.; Hoshino, T.; Imahori, K. Tau protein kinase I is essential for amyloid beta-proteininduced neurotoxicity. Proc. Natl. Acad. Sei. U.S.A. 1993, 90, 7789-7793.
53. Donnelly, R. J.; Friedhoff, A. J.; Beer, B.; Blume, A. J.; Vitek, M. J. Interleukin-1 stimulates the beta-amyloid precursor protein promoter. Cell. Mol. Neurobiol. 1990, 10, 485-495.
54. Goldgaber, D.; Harris, H. W.; Hla, T.; Maciag, T.; Donnelly, R. J.; Jacobsen, J. S.; Vitek, M. P.; Gajdusek, D. C. Interleukin l regulates synthesis of amyloid beta-protein precursor mRNA in human endothelial cells. Proc. Natl. Acad. Sei. U.S.A. 1989, 86, 7606-7610.
55. Mobley, W. C.; Neve, R. L.; Prusiner, S. B.; McKinley, M. P. Nerve growth factor increases mRNA levels for the prion protein and the beta-amyloid protein precursor in developing hamster brain. Proc. Natl. Acad. Sei. U.S.A. 1988,85, 98119815.
56. Games, D.; Adams, D.; Alessandrini, R.; Barbour, R.; Berthelette, P.; Blackwell, C.; Carr, T.; Clemens, J.; Donaldson T.; Gillespie, F.; Guido, T.; Hagoplan, S.; Johnson-Wood, K. Khan, K.; Lee, M.; Leibowitz, P.; Lieberburg, L; Little, S. Masilah, E.; McConlogue, L.; Montoya-Zavala; M., Mucke L.; Paganini, L.; Penniman, E.; Power, M.; Schenk, D. Seubert, P.; Snyder, B.; Soriano, F.; Tan, H.; Vitale, J. Wadsworth, S.; Wolozin, B.; Zhao, J. Alzheimer-type neuropathology in transgenic mice overexpressing V717F betaamyloid precursor protein. Nature 1995, 373, 523-527.
57. Coulson, E. J.; Barrett, G. L.; Storey, E.; Bartlett, P. F.; Beyreuther, K.; Masters, C. L. Down-regulation of the amyloid protein precursor of Alzheimer's disease by antisense oligonucleotides reduces neuronal adhesion to specific substrata. Brain Res. 1997, 770, 72-80.
58. Navia, B. A.; Jordan, B. D.; Price, R. W. The AIDS dementia complex: I. Clinical features. Ann. Neural. 1986, 19, 517524.
59. Navia, B. A.; Cho, E.-S.; Petito, C. K.; Price, R. W. The AIDS dementia complex: II. Neuropathology. Ann. Neural. 1986, 19, 525-535.
60. Resnick, L.; Berger, J. R.; Shapshak, P.; Tourtellotte, W. W. Early penetration of the blood-brain barrier by HIV. Neurology 1988, 38, 9-14.
61. Atwood, W. J.; Berger, J. R.; Kaderman, R.; Tornatore, C. S.; Major, E. O. Human immunodeficiency virus type 1 infection of the brain. Clin. Microbiol. Rev. 1993, 6, 339366.
62. Perry, V. H.; Gordon, S. Modulation of CD4 antigen on macrophages and microglia in rat brain. J. Exp. Med. 1987, 166, 1138-1143.
63. Erickson, J. W.; Burt, S. K. Structural mechanisms of HIV drug resistance. Annu. Rev. Pharmacol. Toxicol. 1996, 36, 545-571.
64. Noble, S.; Faulds, D. Saquinavir. A review of its pharmacology and clinical potential in the management of HIV infection. Drugs 1996, 52, 93-112.
65. Lewis, J. S. 2nd; Terriff, C. M.; Coulston, D. R.; Garrison, M. W. Protease inhibitors: a therapeutic breakthrough for the treatment of patients with human immunodeficiency virus. Clin. Ther. 1997, 19, 187-214.
66. Matsukura, M.; Mitsuya, H.; Broder, S. A new concept in AIDS treatment: an antisense approach and its current status towards clinical application. In Prospects for Antisense Nucleic Acid Therapy of Cancer and AIDS; Wickstrom, E., Ed.; Wiley-Liss: New York, 1991; pp 159-178.
67. Agrawal, S. Antisense oligonucleotides: a possible approach for chemotherapy of AIDS. In: Prospects for Antisense Nucleic Acid Therapy of Cancer and AIDS; Wickstrom, E., Ed.; Wiley-Liss: New York, 1991; pp 143-158.
68. Lisziewicz, J.; Sun, D.; Weichold, F. F.; Thierry, A. R.; Lusso, P.; Tang, J.; Gallo, R. C.; Agrawal, S. Antisense oligodeoxynucleotide phosphorothioate complementary to Gag mRNA blocks replication of human immunodeficiency virus type 1 in human peripheral blood cells. Proc. Natl. Acad. Sei. U.S.A. 1994, 91, 7942-7946.
69. Terasaki, T.; Pardridge, W. M. Restricted transport of 3'azido-3'-deoxythymidine and dideoxynucleosides through the blood-brain barrier. J. Infect. Dis. 1988, 158, 630-632.