Maxizymes and Small Hairpin-Type RNAs that Are Driven by a tRNA Promoter Specifically Cleave a Chimeric Gene Associated with Leukemia in Vitro and in Vivo

Cancer Research

Volumn 63, Issue 20, 2003, Pages 6809-6814

  Oshima, Keisuke ^a   Kawasaki, Hiroaki ^a,b   Soda, Yasushi ^a   Tani, Kenzaburo ^c   Asano, Shigetaka ^a   Taira, Kazunari ^a,b  

^a UNIVERSITY OF TOKYO   (Japan)

^b NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGY AIST   (Japan)

^c KYUSHU UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

BCR ABL PROTEIN; MAXIZYME; ONCOPROTEIN; RETINOIC ACID RECEPTOR ALPHA; RIBOZYME; RNA; SHORT HAIRPIN RNA; TRANSFER RNA; UNCLASSIFIED DRUG;

ALLOSTERISM; ARTICLE; CHIMERA; CHROMOSOME TRANSLOCATION; CONTROLLED STUDY; DNA VECTOR; GENE EXPRESSION REGULATION; GENE SILENCING; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; ONCOGENE; PRIORITY JOURNAL; PROMOTER REGION; PROMYELOCYTIC LEUKEMIA; RNA SEQUENCE;

BASE SEQUENCE; GENE EXPRESSION REGULATION, LEUKEMIC; GENE SILENCING; HELA CELLS; HUMANS; NEOPLASM PROTEINS; NUCLEIC ACID CONFORMATION; ONCOGENE PROTEINS, FUSION; PLASMIDS; PROMOTER REGIONS (GENETICS); RECOMBINANT FUSION PROTEINS; RNA, CATALYTIC; RNA, MESSENGER; RNA, SMALL INTERFERING; RNA, TRANSFER, VAL; TRANSFECTION;

EID: 0142157679     PISSN: 00085472     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (45)

1. Smith, M. A., Parkinson, D. R., Cheson, B. D., and Friedman, M. A. Retinoids in cancer therapy. J. Clin. Oncol., 10: 839-864, 1992.
2. de The, H., Lavau, C., Marchio, A., Chomienne, C., Degos, L., and Dejean, A. The PML-RAR a fusion mRNA generated by the t(15;17) translocation in acute promyelocytic leukemia encodes a functionally altered RAR. Cell, 66: 675-684, 1991.
3. Kakizuka, A., Miller, W. H., Jr., Umesono, K., Warrell, R. P., Jr., Frankel, S. R., Murty, V. V., Dmitrovsky, E., and Evans, R. M. Chromosomal translocation t(15;17) in human acute promyelocytic leukemia fuses RAR α with a novel putative transcription factor, PML. Cell, 66: 663-674, 1991.
4. Pandolfi, P. P., Grignani, F., Alcalay, M., Mencarelli, A., Biondi, A., LoCoco, F., Grignani, F., and Pelicci, P. G. Structure and origin of the acute promyelocytic leukemia myl/RAR α cDNA and characterization of its retinoid-binding and transactivation properties, Oncogene, 6: 1285-1292, 1991.
5. Rousselot, P., Hardas, B., Patel, A., Guidez, F., Gaken, J., Castaigne, S., Dejean, A., de The, H., Degos, L., Farzaneh, F., et al. The PML-RAR α gene product of the t(15;17) translocation inhibits retinoic acid-induced granulocytic differentiation and mediated transactivation in human myeloid cells. Oncogene, 9: 545-551, 1994.
6. Kastner, P., Perez, A., Lutz, Y., Rochette-Egly, C., Gaub, M. P., Durand, B., Lanotte, M., Berger, R., and Chambon, P. Structure, localization and transcriptional properties of two classes of retinoic acid receptor α fusion proteins in acute promyelocytic leukemia (APL): structural similarities with a new family of oncoproteins. EMBO J., 11: 629-642, 1992.
7. Grignani, F., Ferrucci, P. F., Testa, U., Talamo, G., Fagioli, M., Alcalay, M., Mencarelli, A., Grignani, F., Peschle, C., Nicoletti, I., et al. The acute promyelocytic leukemia-specific PML-RAR a fusion protein inhibits differentiation and promotes survival of myeloid precursor cells. Cell, 74: 423-431, 1993.
8. Nason-Burchenal, K., Takle, G., Pace, U., Flynn, S., Allopenna, J., Martin, P., George, S. T., Goldberg, A. R., and Dmitrovsky, E. Targeting the PML/RAR α translocation product triggers apoptosis in promyelocytic leukemia cells. Oncogene, 17:1759-1768, 1998.
9. Nason-Burchenal, K., Allopenna, J., Begue, A., Stehelin, D., Dmitrovsky, E., and Martin, P. Targeting of PML/RAR α is lethal to retinoic acid-resistant promyelocytic leukemia cells. Blood, 92: 1758-1767, 1998.
10. Pace, U., Bockman, J. M., MacKay, B. J., Miller, W. H., Jr., Dmitrovsky, E., and Goldberg, A. R. A ribozyme which discriminates in vitro between PML/RAR α the t(15;17)-associated fusion RNA of αcute promyelocytic leukemia, and PML and RAR α, the transcripts from the nonrearranged alleles. Cancer Res., 54: 6365-6369, 1994.
11. Shimayama, T., Nishikawa, S., and Taira, K. Generality of the NUX rule: kinetic analysis of the results of systematic mutations in the trinucleotide at the cleavage site of hammerhead ribozymes. Biochemistry, 34: 3649-3654, 1995.
12. Kuwabara, T., Warashina, M., Tanabe, T., Tani, K., Asano, S., and Taira, K. A novel allosterically trans-activated ribozyme, the maxizyme, with exceptional specificity in vitro and in vivo. Mol. Cell, 2: 617-627, 1998.
13. 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 (Lond.), 391: 806-811, 1998.
14. Robertson, H. D., and Mathews, M. B. The regulation of the protein kinase PKR by RNA. Biochimie (Paris), 78: 909-914, 1996.
15. Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., and Tuschl, T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature (Lond.), 411: 494-498, 2001.
16. Paddison, P. J., Caudy, A. A., Bernstein, E., Hannon, G. J., and Conklin, D. S. Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev., 16: 948-958, 2002.
17. Yu, J. Y., DeRuiter, S. L., and Turner, D. L. RNA interference by expression of short-interfering RNAs and hairpin RNAs in mammalian cells. Proc. Natl. Acad. Sci. USA, 99: 6047-6052, 2002.
18. Sui, G., Soohoo, C., el Affar, B., Gay, F., Shi, Y., Forrester, W. C., and Shi, Y. A DNA vector-based RNAi technology to suppress gene expression in mammalian cells. Proc. Natl. Acad. Sci. USA, 99: 5515-5520, 2002.
19. Val promoter significantly induce RNAi-mediated gene silencing in the cytoplasm of human cells. Nucleic Acids Res., 31: 700-707, 2003.
20. Kitamura, T., Onishi, M.. Kinoshita, S., Shibuya, A., Miyajima, A., and Nolan, G. P. Efficient screening of retroviral cDNA expression libraries. Proc. Natl. Acad. Sci. USA, 92: 9146-9150, 1995.
21. Dubrez, L., Eymin, B., Sordel, O., Droin, N., Turban, A. G., and Solary, E. BCR-ABL delays apoptosis upstream of procaspase-3 activation. Blood, 91: 2415-2422, 1998.
22. Yu, M., Leavitt, M. C., Maruyama, M., Yamada, O., Young, D., Ho, A. D., and Wong-Staal, F. Intracellular immunization of human fetal cord blood stem/progenitor cells with a ribozyme against human immunodeficiency virus type 1. Proc. Natl. Acad. Sci. USA, 92: 699-703, 1995.
23. Kuwabara, T., Warashina, M., Koseki, S., Sano, M., Ohkawa, J., Nakayama, K., and Taira, K. Significantly higher activity of a cytoplasmic hammerhead ribozyme than a corresponding nuclear counterpart: engineered tRNAs with an extended 3′ end can be exported efficiently and specifically to the cytoplasm in mammalian cells. Nucleic Acids Res., 29: 2780-2788, 2001.
24. Bertrand, E., Castanotto, D., Zhou, C., Carbonnelle, C., Lee, G. P., Chatterjee, S., Grange, T., Pictet, R., Kohn, D., Engelke, D., and Rossi, J. J. The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization. RNA, 3: 75-88, 1997.
25. Zeng, Y., and Cullen, B. R. RNA interference in human cells is restricted to the cytoplasm. RNA, 8: 855-860, 2002.
26. Pandolfï, P. P., Alcalay, M., Fagioli, M., Zangrilli, D., Mencarelli, A., Diverio, D., Biondi, A., LoCoco, F., Rambaldi, A., Grignani, F., et al. Genomic variability and alternative splicing generate multiple PML/RAR α transcripts that encode aberrant PML proteins and PML/RAR α isoforms in acute promyelocytic leukaemia. EMBO J., 11: 1397-1407, 1992.
27. Val-heterodimeric maxizymes with high potential as gene inactivating agents: simultaneous cleavage at two sites in HIV-1 Tat tuRNA in cultured cells. Proc. Natl. Acad. Sci. USA, 96: 1886-1891, 1999.
28. Val-driven short ribozymes. Nat. Biotechnol., 16: 961-965, 1998.
29. Stark, G. R., Kerr, I. M., Williams, B. R., Silverman, R. H., and Schreiber, R. D. How cells respond to interferons. Annu. Rev. Biochem., 67: 227-264, 1998.
30. Hamada, M., Kuwabara, T., Warashina, M., Nakayama, A., and Taira, K. Specificity of novel allosterically trans- and cis-activated connected maxizymes that are designed to suppress BCR-ABL expression. FEBS Lett., 461: 77-85, 1999.
31. Tanabe, T., Takata, I., Kuwabara, T., Warashina, M., Kawasaki, H., Tani, K., Ohta, S., Asano, S., and Taira, K. Maxizymes, novel allosterically controllable ribozymes, can be designed to cleave various substrates. Biomacromolecules, 1: 108-117, 2000.
32. Tanabe, T., Kuwabara, T., Warashina, M., Tani, K., Taira, K., and Asano, S. Oncogene inactivation in a mouse model. Nature (Lond.), 406: 473-474, 2000.
33. Zhou, J. M., Nakamatsu, Y., Kuwabara, T., Warashina, M., Tanaka, Y., Yoshinari, K., and Taira, K. Chemical and enzymatic probing of effector-mediated changes in the conformation of a maxizyme. J. Inorg. Biochem., 78: 261-268, 2000.
34. Tuschl, T. Expanding small RNA interference. Nat. Biotechnol., 20: 446-448, 2002.
35. Brummelkamp, T. R., Bemards, R., and Agami, R. A system for stable expression of short interfering RNAs in mammalian cells. Science (Wash. DC), 296: 550-553, 2002.
36. Paul, C. P., Good, P. D., Winer, I., and Engelke, D. R. Effective expression of small interfering RNA in human cells. Nat. Biotechnol., 20: 505-508, 2002.
37. Lee, N. S., Dohjima, T., Bauer, G., Li, H., Li, M. J., Ehsani, A., Salvaterra, P., and Rossi, J. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat. Biotechnol., 20: 500-505, 2002.
38. Miyagishi, M., and Taira, K. U6 promoter-driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells. Nat. Biotechnol., 20: 497-500, 2002.
39. Kawasaki, H., Eckner, R., Yao, T-P., Taira, K., Chiu, R., Livingston, D. M., and Yokoyama, K. K. Distinct roles of the co-activators p300 and CBP in retinoic-acid-induced F9-cell differentiation. Nature (Lond.), 393: 284-289, 1998.
40. Kuwabara, T., Warashina, M., Sano, M., Tang, H., Wong-Staal, F., Munekata, E., and Taira, K. Recognition of engineered iRNAs with an extended 3′ end by Exportin-t (Xpo-t) and transport of tRNA-attached ribozymes to the cytoplasm in somatic cells. Biomacromolecules, 2: 1229-1242, 2001.
41. Qin, X. F., An, D. S., Chen, I. S., and Baltimore, D. Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc. Natl. Acad. Sci. USA, 100: 183-188. 2003.
42. Rubinson, D. A., Dillon, C. P., Kwiatkowski, A. V., Sievers, C., Yang, L., Kopinja, J., Zhang, M., McManus, M. T., Gertler, F. B., Scott, M. L., et al. A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells and transgenic mice by RNA interference. Nat. Genet., 33: 401-406, 2003.
43. Stewart, S. A., Dykxhoorn, D. M., Palliser, D., Mizuno, H., Yu, E. Y., An, D. S., Sabatini, D. M., Chen, I. S., Hahn, W. C., Sharp, P. A., et al. Lentivirus-delivered stable gene silencing by RNAi in primary cells. RNA, 9: 493-501, 2003.
44. Bai, Y., Soda, Y., Izawa, K., Tanabe, T., Kang, X., Tojo, A., Hoshino, H., Miyoshi, H., Asano, S., and Tani, K. Effective transduction and stable transgene expression in human blood cells by a third-generation lentiviral vector. Gene Ther., 10: 1446-1457. 2003.
45. Wilda, M., Fuchs, U., Wossmann, W., and Borkhardt, A. Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi). Oncogene, 21: 5716-5724, 2002.