Ribozyme: A clinical tool

Clinica Chimica Acta

Volumn 367, Issue 1-2, 2006, Pages 20-27

  Khan, Asad U ^a  

^a ALIGARH MUSLIM UNIVERSITY   (India)

Author keywords

Clinical applications; Hammerhead; Ribozyme; RNAse P

Indexed keywords

HAMMERHEAD RIBOZYME; RIBONUCLEASE P; RIBOZYME; SMALL INTERFERING RNA; ZINC FINGER PROTEIN;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ANTIVIRAL ACTIVITY; CANCER; CANCER THERAPY; CATALYSIS; DRUG MECHANISM; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; HUMAN; PRIORITY JOURNAL; PROTEIN FUNCTION; REVIEW; RNA CLEAVAGE; RNA STRUCTURE; VIRUS INFECTION;

ANIMALS; ANTIVIRAL AGENTS; HUMANS; NEOPLASMS; NUCLEIC ACID CONFORMATION; RNA; RNA, CATALYTIC;

RNA VIRUSES;

EID: 33645851037     PISSN: 00098981     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cca.2005.11.023     Document Type: Review

References (92)

1. Alifano P., Rivellini F., Piscitelli C., Arraiano C.M., Bruni C.B., and Carlomagno M.S. Ribonuclease E provides substrates for ribonuclease P-dependent processing of a polycistronic mRNA. Genes Dev 8 (1994) 3021-3031
2. Saville B. A site-specific self-cleavage reaction performed by a novel RNA in Neurospora mitochondria. Cell 61 (1990) 685-696
3. Sharmeen L., Kuo M.Y.P., Dinter-Gottlieb G., and Taylor J. Antigenomic RNA of human hepatitis delta virus can undergo self-cleavage. J Virol 62 (1988) 2674-2679
4. Kuo M.Y., Sharmeen L., Dinter-Gottlieb G., and Taylor J. Characterization of self-cleaving RNA sequences on the genome and antigenome of human hepatitis delta virus. J Virol 62 (1988) 4439-4444
5. Prody G.A., Bakos J.T., Buzayan J.M., Schneider I.R., and Bruening G. Science 231 (1986) 1577-1580
6. Forster A.C., and Symons R.H. Self-cleavage of plus and minus RNAs of a virusoid and a structural model for the active sites. Cell 49 (1987) 211-220
7. Hutchins C.J., Rathjen P.D., Forster A.C., and Symons R.H. Self-cleavage of plus and minus RNA transcripts of avocado sunblotch viroid. Nucleic Acids Res 14 (1986) 3627-3640
8. Buzayan J.M., Gerlach W.L., and Bruening G. Nature 323 (1986) 349-352
9. Khan A.U., and Lal S.K. Ribozymes: a modern tool in medicine. J Biomed Sci 10 (2003) 457-467
10. Branch A.D., and Robertson H.D. Science 223 (1984) 450-455
11. Kruger K., Grabowski P.J., Zaug A.J., Sands J., Gottschling D.E., and Cech T.R. Self-splicing RNA: autoexcision and autocyclization of the ribosomal RNA intervening sequence of Tetrahymena. Cell 31 (1982) 147-157
12. Guerrier-Takada C., Gardiner K., Marsh T., Pace N., and Altman S. The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme. Cell 35 (1983) 849-857
13. Peebles C., Perlman P., Mecklenburg K., et al. A self-splicing RNA excises an intron lariat. Cell 44 (1986) 213-223
14. Frank D.N., and Pace N.R. Ribonuclease P: unity and diversity in a tRNA processing ribozyme. Annu Rev Biochem 67 (1998) 153-180
15. Khan A.U., Lal S.K., and Ahmad M. Isolation and characterization of EMS induced splicing defective point mutations within the intron of the nrdB gene of bacteriophage T4. Biochem Biophys Res Commun 242 (1998) 10-15
16. Khan A.U., Ahmad M., and Lal S.K. Restoration of mRNA splicing by a second-site intragenic suppressor in the T4 ribonucleotide reductase (small subunit) self-splicing intron. Biochem Biophys Res Commun 268 (2000) 359-364
17. Cech T.R. Self-splicing of group I introns. Annu Rev Biochem 59 (1990) 543-568
18. Michel F., and Ferat J.-L. Structure and activities of group II introns. Annu Rev Biochem 64 (1995) 435-461
19. Jacquier A. Group II introns: elaborate ribozymes. Biochimie 78 (1996) 474-487
20. Khan A.U., and Lal S.K. Ribozyme: a structure and potential applications in medicine. Med Sci Res 27 (1999) 507-512
21. Khan A.U., and Lal S.K. The white halo plaque phenotype of bacteriophage T4: its uses and applications in screening and mapping of splicing-defective mutants. J Biochem Mol Biol Biophys 5 (2001) 237-242
22. Famulok M., and Mayer G. Aptamers as tools in molecular biology and immunology. Curr Top Microbiol Immunol 243 (1999) 123-136
23. Famulok M., Mayer G., and Blind M. Nucleic acid aptamers-from selection in vitro to applications in vivo. Acc Chem Res 33 (2000) 591-599
24. Kurz M., and Breaker R.R. In vitro selection of nucleic acid enzymes. Curr Top Microbiol Immunol 243 (1999) 137-158
25. Koizumi M., Soukup G.A., Kerr J.N., and Breaker R.R. Allosteric selection of ribozymes that respond to the second messengers cGMP and cAMP. Nat Struct Biol 6 (1999) 1062-1071
26. Piganeau N., Jenne A., Thuillier V., and Famulok M. An Allosteric Ribozyme Regulated by Doxycyline This work was supported by Aventis Gencell and by a grant from the Volkswagen Foundation (Priority program "conformational control") to M.F. We thank M. Blind, G. Mayer, D. Proske, and G. Sengle (Universitat Bonn) for helpful discussions as well as J. Crouzet, J. F. Mayaux, and M. Finer (Aventis Gencell) for support. Angew Chem Int Ed Engl 40 (2001) 3503
27. Tassakka A.C., Savan R., Watanuki H., and Sakai M. The in vitro effects of CpG oligodeoxynucleotides on the expression of cytokine genes in the common carp (Cyprinus carpio L.) head kidney cells. Vet Immunol Immunopathol (2005 (Oct 13)) [Electonic publication ahead of print]
28. Bramlage B., Luzi E., and Eckstein F. HIV-1 LTR as a target for synthetic ribozyme-mediated inhibition of gene expression: site selection and inhibition in cell culture. Nucleic Acids Res 28 (2000) 4059-4067
29. Scherer L.J., and Rossi J.J. Approaches for the sequence-specific knockdown of mRNA. Nat Biotechnol 21 (2003) 1457-1465
30. Zamecnik P.C., and Stephenson M.L. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc Natl Acad Sci U S A 75 (1978) 280-284
31. Stein C.A., and Cheng Y.C. Antisense oligonucleotides as therapeutic agents-is the bullet really magical?. Science 261 (1993) 1004-1012
32. Sarver N., Cantin E.M., Chang P.S., et al. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247 (1990) 1222-1225
33. Yu M., Ojwang J., Yamada O., et al. A hairpin ribozyme inhibits expression of diverse strains of human immunodeficiency virus type 1. Proc Natl Acad Sci U S A 90 (1993) 6340-6344
34. Castanotto D., Rossi J.J., and Sarver N. Antisense catalytic RNAs as therapeutic agents. Adv Pharmacol 25 (1994) 289-317
35. Poeschla E., and Wong-Staal F. Antiviral and anticancer ribozymes. Curr Opin Oncol 6 (1994) 601-606
36. Rossi J.J. Ribozymes, genomics and therapeutics. Chem Biol 6 (1999) R33-R37
37. Frank D.N., and Pace N.R. Ribonuclease P: unity RNase P ribozymes as gene targeting agents 507 and diversity in a tRNA processing ribozyme. Annu Rev Biochem 67 (1998) 153-180
38. Altman S., and Kirsebom L. In: Gesteland R.F., Cech T.R., and Atkins J.F. (Eds). In the RNA World. 2nd edn. (1999), Cold Spring Harbor Laboratory Press, Cold Spring Harbor, NY 351-380
39. Guerrier-Takada C., Gardiner K., Marsh T., Pace N., and Altman S. The RNA moiety of ribonuclease P is the catalytic subunit of the enzyme. Cell 35 (1983) 849-857
40. Niranjanakumari S., Stams T., Crary S.M., Christianson D.W., and Fierke C.A. Protein component of the ribozyme ribonuclease P alters substrate recognition by directly contacting precursor tRNA. Proc Natl Acad Sci U S A 95 (1998) 15212-15217
41. Reich C., Olsen G.J., Pace B., and Pace N.R. Role of the protein moiety of ribonuclease P, a ribonucleoprotein enzyme. Science 239 (1988) 178-181
42. Forster A.C., and Altman S. External guide sequences for an RNA enzyme. Science 249 (1990) 783-786
43. Yuan Y., Hwang E.S., and Altman S. Targeted cleavage of mRNA by human RNase P. Proc Natl Acad Sci U S A 89 (1992) 8006-8010
44. Bothwell A.L., Garber R.L., and Altman S. Nucleotide sequence and in vitro processing of a precursor molecule to Escherichia coli 4.5S RNA. J Biol Chem 251 (1976) 7709-7716
45. Alifano P., Rivellini F., Piscitelli C., Arraiano C.M., Bruni C.B., and Carlomagno M.S. Ribonuclease E provides substrates for ribonuclease P dependent processing of a polycistronic mRNA. Genes Dev 8 (1994) 3021-3031
46. Komine Y., Kitabatake M., Yokogawa T., Nishikawa K., and Inokuchi H. A tRNA-like structure is present in 10Sa RNA, a small stable RNA from Escherichia coli. Proc Natl Acad Sci U S A 91 (1994) 9223-9227
47. McClain W.H., Guerrier-Takada C., and Altman S. Model substrates for an RNA enzyme. Science 238 (1987) 527-530
48. Guerrier-Takada C., Li Y., and Altman S. Artificial regulation of gene expression in Escherichia coli by Rnase P. Proc Natl Acad Sci U S A 92 (1995) 11115-11119
49. Guerrier-Takada C., Salavati R., and Altman S. Phenotypic conversion of drug-resistant bacteria to drug sensitivity. Proc Natl Acad Sci U S A 94 (1997) 8468-8472
50. McKinney J., Guerrier-Takada C., Wesolowski D., and Altman S. Inhibition of Escherichia coli viability by external guide sequences complementary to two essential genes. Proc Natl Acad Sci U S A 98 (2001) 6605-6610
51. Liu F., and Altman S. Inhibition of viral gene expression by the catalytic RNA subunit of RNase P from Escherichia coli. Genes Dev 9 (1995) 471-480
52. Forster A.C., and Symons R.H. Self-cleavage of plus and minus RNAs of a virusoid and a structural model for the active sites. Cell 49 (1987) 211-220
53. Branch A.D., and Robertson H.D. A replication cycle for viroids and other small infectious RNAs. Science 223 (1984) 450-455
54. Symons R.H. Self-cleavage of RNA in the replication of small pathogens of plants and animals. Trends Biochem Sci 14 (1989) 445-450
55. Uhlenbeck O.C. A small catalytic oligoribonucleotide. Nature 328 (1987) 596-600
56. Haseloff J., and Gerlach W.L. Simple RNA enzymes with new and highly specific endoribonuclease activities. Nature 334 (1988) 585-591
57. Hammann C., and Lilley D.M. Folding and activity of the hammerhead ribozyme. Chembiochem 2002; 3: divalent metal ion-independent channels in reactions catalyzed by a hammerhead ribozyme. Nucleic Acids Res 30 (2002) 2374-2382
58. Persson T., Hartmann R.K., and Eckstein F. Selection of hammerhead ribozyme variants with low Mg2{thorn} requirement: importance of stem-loop II. Chembiochem 3 (2002) 1066-1071
59. Conaty J., Hendry P., and Lockett T. Selected classes of minimised hammerhead ribozyme have very high cleavage rates at low Mg2{thorn} concentration. Nucleic Acids Res 27 (1999) 2400-2407
60. Pley H.W., Flaherty K.M., and McKay D.B. Three dimensional structure of a hammerhead ribozyme. Nature 372 (1994) 68-74
61. Scott W.G., Finch J.T., and Klug A. The crystal structure of an all-RNA hammerhead ribozyme: a proposed mechanism for RNA catalytic cleavage. Cell 81 (1995) 991-1002
62. Scott W.G., Murray J.B., Arnold J.R.P., Stoddard B.L., and Klug A. Capturing the structure of a catalytic RNA intermediate: the hammerhead ribozyme. Science 274 (1996) 2065-2069
63. McKay D. Structure and function of the hammerhead ribozyme: an unfinished story. RNA 2 (1996) 395-403
64. Lilley D.M. Structure, folding and mechanisms of ribozymes. Curr Opin Struct Biol 15 (2005) 313-323
65. McKnight K.L., and Heinz B.A. RNA as a target for developing antivirals. Antivir Chem Chemother 14 (2003) 61-73
66. Wong-Staal F., Poeschla E.M., and Looney D.J. A controlled, Phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA. Hum Gene Ther 9 (1998) 2407-2425
67. Amado R.G., Mitsuyasu R.T., Symonds G., et al. A phase I trial of autologous CD34{thorn} hematopoietic progenitor cells transduced with an anti-HIV ribozyme. Hum Gene Ther 10 (1999) 2255-2270
68. Sarver N., Cantin E.M., Chang P.S., Zaia J.A., Stephens D.A., and Rossi J.J. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 247 (1990) 1222-1225
69. Ngok F.K., Mitsuyasu R.T., Macpherson J.L., Boyd M.P., Symonds G.P., and Amado R.G. Clinical gene therapy research utilizing ribozymes: application to the treatment of HIV/AIDS. Methods Mol Biol 252 (2004) 581-598
70. Baringa M. Ribozyme linking messenger. Science 262 (1993) 1512
71. Koizumi M., Ozawa Y., Yagi R., Wishigaki T., Kamatsua Y., and Ohtsuaka E. Nucleic Acids Symp Ser 34 (1995) 125-126
72. Macejak D.G., Jensen K.L., Pavco P.A., et al. Enhanced antiviral effect in cell culture of type 1 interferon and ribozymes targeting HCV RNA. J Viral Hepat 8 (2001) 400-405
73. Macejak D.G., Jensen K.L., Jamison S.F., et al. Inhibition of hepatitis C virus (HCV)-RNA-dependent translation and replication of a chimeric HCV poliovirus using synthetic stabilized ribozymes. Hepatology 31 (2000) 769-776
74. Cornberg M., Wedemeyer H., and Manns M.P. Hepatitis C: therapeutic perspectives. Forum (Genova) 11 (2001) 154-162
75. Gomez J., Nadal A., Sabariegos R., Beguiristain N., Martell M., and Piron M. Three properties of the hepatitis C virus RNA genome related to antiviral strategies based on RNA-therapeutics: variability, structural conformation and tRNA mimicry. Curr Pharm Des 10 (2004) 3741-3756
76. Xu R., Cai K., Zheng D., Ma H., Xu S., and Fan S.T. Molecular therapeutics of HBV. Curr Gene Ther 3 (2003) 341-355
77. Hsieh S.Y., and Taylor J. Delta virus is a vector, for the delivery of biologically active RNA: possibly a ribozyme specific for chronic hepatitis B virus infection. Adv Exp Med Biol 312 (1992) 125-128
78. Netter H.J., Hsieh S.Y., Lazinski D., and Taylor H. Modified BDV as a vector for the delivery of biologically active RNAs. Prog Clin Biol Res 382 (1993) 373-376
79. Koizumi M., Ozawa Y., Yagi R., et al. Design and anti-HIV-I activity of hammerhead and hairpin ribozymes containing a stable loop. Nucl Nucl 17 (1998) 207-218
80. Wang L., Witherington C., King A., et al. Preclinical characterization of an anti-tat ribozyme for therapeutic application. Hum Gene Ther 9 (1998) 1283-1291
81. Muotri A.R., da Veiga Pereira L., dos Reis Vasques L., and Menck C.F. Ribozyme and anti-gene therapy: how a catalytic RNA can be used to inhibit gene function. Gene 37 (1999) 303-310
82. Rossi J.J. The application of ribozymes to HIV infection. Curr Opin Mol Ther 1 (1999) 316-322
83. Jacque J.M., Triques K., and Stevenson M. Modulation of HIV-1 replication by RNA interference. Modulation of HIV-1 replication by RNA interference. Nature 418 (2002) 435-438
84. Lee N.S., Dohjima T., Bauer G., et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 20 (2002) 500-505
85. Novina C.D., Murray M.F., Dykxhoorn D.M., et al. siRNA-directed inhibition of HIV-1 infection. Med 8 (2002) 681-686
86. Sanchez-Garcia I. Consequences of chromosomal translocations in tumor development. Annu Rev Genet 31 (1997) 429-453
87. Cobaleda C., Perez-Losada J., and Sanchez-Garcia I. Chromosomal abnormalities and tumor development: from gene to therapeutic mechanisms. Bioassay 20 (1998) 922
88. Choo Y., Sanchez-Garcia I., and Klug A. In vivo repression by a site-specific DNA-binding protein designed against an oncogenic sequence. Nature 372 (1994) 642-645
89. Takagi Y., Suyama E., Kawasaki H., Miyagishi M., and Taira K. Mechanism of action of hammerhead ribozymes and their applications in vivo: rapid identification of functional genes in the post-genome era by novel hybrid ribozyme libraries. Biochem Soc Trans 30 (2001) 1145-1149
90. Forster A.C., and Altman S. External guide sequence for and RNA enzyme. Science 249 (1990) 783-786
91. Cobeleda C., and Sanchez-Garcia I. In vivo inhibition by a site-specific catalytic RNA subunit of RNase P designed against the BCR-ABL oncogenic products: a novel approach for cancer treatment. Blood 95 (2000) 731-737
92. Trang P., Kim K., and Liu F. Developing RNase P ribozymes for gene-targeting and antiviral therapy. Cell Microbiol 6 (2004) 499-508