A potent and specific morpholino antisense inhibitor of hepatitis C translation in mice

Hepatology

Volumn 38, Issue 2, 2003, Pages 503-508

  McCaffrey, Anton P ^a   Meuse, Leonard ^a   Karimi, Mobin ^a   Contag, Christopher H ^a   Kay, Mark A ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

5 METHYLCYTIDINE ANTISENSE OLIGONUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; LUCIFERASE; MESSENGER RNA; MORPHOLINE DERIVATIVE; PHOSPHOROTHIOIC ACID; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DOSE RESPONSE; ENCEPHALOMYOCARDITIS VIRUS; EUROPE; FEMALE; HEPATITIS C; HEPATITIS C VIRUS; LIVER FAILURE; LIVER TRANSPLANTATION; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA TRANSLATION; RNA VIRUS; TRANSLATION REGULATION; UNITED STATES;

EID: 0042265573     PISSN: 02709139     EISSN: None     Source Type: Journal    
DOI: 10.1053/jhep.2003.50330     Document Type: Article

References (28)

1. Alt M, Renz R, Hofschneider PH, Paumgartner G, Caselmann WH. Specific inhibition of hepatitis C viral gene expression by antisense phosphorothioate oligodeoxynucleotides. HEPATOLOGY 1995;22:707-717.
2. Lima WF, Brown-Driver V, Fox M, Hanecak R, Bruice TW. Combinatorial screening and rational optimization for hybridization to folded hepatitis C virus RNA of oligonucleotides with biological antisense activity. J Biol Chem 1997;272:626-638.
3. Wu CH, Wu GY. Targeted inhibition of hepatitis C virus-directed gene expression in human hepatoma cell lines. Gastroenterology 1998;114:1304-1312.
4. Zhang H, Hanecak R, Brown-Driver V, Azad R, Conklin B, Fox MC, Anderson KP. Antisense oligonucleotide inhibition of hepatitis C virus (HCV) gene expression in livers of mice infected with an HCV-vaccinia virus recombinant. Antimicrob Agents Chemother 1999;43:347-353.
5. Brown-Driver V, Eto T, Lesnik E, Anderson KP, Hanecak RC. Inhibition of translation of hepatitis C virus RNA by 2-modified antisense oligonucleotides. Antisense Nucleic Acid Drug Dev 1999;9:145-154.
6. Alt M, Eisenhardt S, Serwe M, Renz R, Engels JW, Caselmann WH. Comparative inhibitory potential of differently modified antisense oligodeoxynucleotides on hepatitis C virus translation. Eur J Clin Invest 1999;29:868-876.
7. Jubin R, Vantuno NE, Kieft JS, Murray MG, Doudna JA, Lau JY, Baroudy BM. Hepatitis C virus internal ribosome entry site (IRES) stem loop IIId contains a phylogenetically conserved GGG triplet essential for translation and IRES folding. J Virol 2000;74:10430-10437.
8. Welch PJ, Tritz R, Yei S, Leavitt M, Yu M, Barber J. A potential therapeutic application of hairpin ribozymes: in vitro and in vivo studies of gene therapy for hepatitis C virus infection. Gene Ther 1996;3:994-1001.
9. Macejak DG, Jensen KL, Jamison SF, Domenico K, Roberts EC, Chaudhary N, von Carlowitz I, et al. Inhibition of hepatitis C virus (HCV)-RNA-dependent translation and replication of a chimeric HCV poliovirus using synthetic stabilized ribozymes. HEPATOLOGY 2000;31:769-776.
10. Macejak DG, Jensen KL, Pavco PA, Phipps KM, Heinz BA, Colacino JM, Blatt LM. Enhanced antiviral effect in cell culture of type 1 interferon and ribozymes targeting HCV RNA. J Viral Hepat 2001;8:400-405.
11. Oketani M, Asahina Y, Wu CH, Wu GY. Inhibition of hepatitis C virus-directed gene expression by a DNA ribonuclease. J Hepatol 1999;31:628-634.
12. McCaffrey AP, Ohashi K, Meuse L, Shen S, Lancaster AM, Lukavsky PJ, Sarnow P, et al. Determinants of hepatitis C translational initiation in vitro, in cultured cells and mice. Mol Ther 2002;5:676-684.
13. Borman AM, Le Mercier P, Girard M, Kean KM. Comparison of picornaviral IRES-driven internal initiation of translation in cultured cells of different origins. Nucleic Acids Res 1997;25:925-932.
14. Kamoshita N, Tsukiyama-Kohara K, Kohara M, Nomoto A. Genetic analysis of internal ribosomal entry site on hepatitis C virus RNA: implication for involvement of the highly ordered structure and cell type-specific transacting factors. Virology 1997;233:9-18.
15. Honda M, Kaneko S, Matsushita E, Kobayashi K, Abell GA, Lemon SM. Cell cycle regulation of hepatitis C virus internal ribosomal entry site-directed translation. Gastroenterology 2000;118:152-162.
16. Mercer DF, Schiller DE, Elliott JF, Douglas DN, Hao C, Rinfret A, Addison WR, et al. Hepatitis C virus replication in mice with chimeric human livers. Nat Med 2001;7:927-933.
17. Summerton J, Weller D. Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucleic Acid Drug Dev 1997;7:187-195.
18. Arora V, Knapp DC, Reddy MT, Weller DD, Iversen PL. Bioavailability and efficacy of antisense morpholino oligomers targeted to c-myc and cytochrome P-450 3A2 following oral administration in rats. J Pharm Sci 2002;91:1009-1018.
19. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999;6:1258-1266.
20. Contag CH, Spilman SD, Contag PR, Oshiro M, Eames B, Dennery P, Stevenson DK, et al. Visualizing gene expression in living mammals using a bioluminescent reporter. Photochem Photobiol 1997;66:523-531.
21. Zhang G, Budker V, Wolff JA. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 1999;10:1735-1737.
22. McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. Gene expression: RNA interference in adult mice. Nature 2002;418:38-39.
23. Lukavsky PJ, Otto GA, Lancaster AM, Sarnow P, Puglisi JD. Structures of two RNA domains essential for hepatitis C virus internal ribosome entry site function. Nat Struct Biol 2000;7:1105-1110.
24. Contag CH, Bachmann MH. Advances in in vivo bioluminescence imaging of gene expression. Annu Rev Biomed Eng 2002;4:235-260.
25. Hanecak R, Brown-Driver V, Fox MC, Azad RF, Furusako S, Nozaki C, Ford C, et al. Antisense oligonucleotide inhibition of hepatitis C virus gene expression in transformed hepatocytes. J Virol 1996;70:5203-5212.
26. Johannes G, Carter MS, Eisen MB, Brown PO, Sarnow P. Identification of eukaryotic mRNAs that are translated at reduced cap binding complex eIF4F concentrations using a cDNA microarray. Proc Natl Acad Sci U S A 1999;96:13118-13123.
27. Hudziak RM, Barofsky E, Barofsky DF, Weller DL, Huang SB, Weller DD. Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation. Antisense Nucleic Acid Drug Dev 1996;6:267-272.
28. Zhang G, Budker V, Williams P, Subbotin V, Wolff JA. Efficient expression of naked DNA delivered intraarterially to limb muscles of nonhuman primates. Hum Gene Ther 2001;12:427-438.