The expression cassette determines the functional activity of ribozymes in mammalian cells by controlling their intracellular localization

RNA

Volumn 3, Issue 1, 1997, Pages 75-88

  Bertrand, Edouard ^a,b   Castanotto, Daniela ^a   Zhou, Chen ^c   Carbonnelle, Cecile ^a   Lee, Nan Sook ^a   Good, Paul ^d   Chatterjee, Saswati ^a   Grange, Thierry ^b   Pictet, Raymond ^b   Kohn, Donald ^c   Engelke, David ^d   Rossi, John J ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

^b UNIVERSITÉ PARIS DESCARTES   (France)

^c UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

^d UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

HIV; regulatory RNA; RNA localization; SIV

Indexed keywords

RIBOZYME; TRANSFER RNA;

ARTICLE; CELLULAR DISTRIBUTION; DNA FLANKING REGION; ENZYME ACTIVITY; ENZYME LOCALIZATION; EXPRESSION VECTOR; GENE CASSETTE; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; REPORTER GENE; RNA TRANSCRIPTION; SIMIAN IMMUNODEFICIENCY VIRUS; T LYMPHOCYTE;

ANIMALS; AVIAN SARCOMA VIRUSES; CELLS, CULTURED; CLONING, MOLECULAR; COS CELLS; DEPENDOVIRUS; GENE EXPRESSION REGULATION; GENES, REPORTER; GENES, TAT; GENETIC VECTORS; HIV-1; HUMANS; IN SITU HYBRIDIZATION; KIDNEY; PROMOTER REGIONS (GENETICS); REPETITIVE SEQUENCES, NUCLEIC ACID; RNA; RNA, CATALYTIC; RNA, MESSENGER; RNA, SMALL NUCLEAR; RNA, TRANSFER, MET; SIMIAN IMMUNODEFICIENCY VIRUS; SUBCELLULAR FRACTIONS; T-LYMPHOCYTES;

EID: 12644252947     PISSN: 13558382     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (49)

1. imet gene. Nucleic Acids Res 12:1101-1115.
2. Bertrand E, Fromont-Racine M, Pictet R, Grange T. 1993. Visualization of the in vivo interaction of a regulatory protein with RNA. Proc Natl Acad Sci USA 90:3496-3500.
3. Bertrand E, Pictet R, Grange G. 1994. Can hammerhead ribozymes be efficient tools for inactivating gene function? Nucleic Acids Res 22:293-300.
4. Bertrand E, Rossi J. 1996. Anti-HIV therapeutic hammerhead ribozymes: Targeting strategies and optimization of intracellular function. Nucleic Acids Mol Biol 10:301-313.
5. Boelens W, Palacios I, Mattaj I. 1995. Nuclear retention of RNA as a mechanism for localization. RNA 1:273-283.
6. Brawerman G. 1990. Mechanisms of mRNA decay. Trends Biotechnol 8:1990.
7. Chatterjee S, Johnson P, Wong K. 1992. Dual-target inhibition of HIV-1 in vitro by means of an Adeno-associated antisense vector. Science 258:1485.
8. TM B method. Cinna/Biotecx Bulletin 3.
9. Cotten M, Birnstiel ML. 1989. Ribozyme mediated destruction of RNA in vivo. EMBO J 8:3861-3866.
10. Crisell P, Thompson S, James W. 1993. Inhibition of HIV-1 replication by ribozymes that show poor activity in vitro. Nucleic Acids Res 21:5251-5255.
11. Dropulic B, Lin NH, Martin MA, Kuan-Teh J. 1992. Functional characterization of a U5 ribozyme: Intracellular suppression of human immunodeficiency virus type 1 expression. J Virol 66:1432-1441.
12. Emerman M, Temin H. 1984. Genes with promoters in retrovirus vectors can be independently suppressed by an epigenetic mechanism. Cell 39:459-467.
13. Emerman M, Temin H. 1986. Quantitative analysis of gene suppression in integrated retrovirus vectors. Mol Cell Biol 6:792-800.
14. Fedor M, Uhlenbeck O. 1990. Substrate sequence effects on hammerhead catalytic efficiency. Proc Natl Acad Sci USA 87:1668-1672.
15. Fedor M, Uhlenbeck O. 1992. Kinetics of intermolecular cleavage by hammerhead ribozymes. Biochemistry 31:12042-12054.
16. + human hematopoietic progenitor cells after transduction. Blood 88:492-504.
17. Good P, Krikos A, Li L, Bertrand E, Lee N, Giver L, Ellington A, Zaia J, Rossi J, Engelke D. 1996. Expression of small, therapeutic RNAs in human cell nuclei. Gene Therapy. Forthcoming.
18. Guerrier-Takada C, Gardiner K, Marsh T, Pace N, Altman S. 1983. The RNA moeity of ribonuclease P is the catalytic subunit of the enzyme. Cell 35:849-869.
19. Hernandez N. 1985. Formation of the 3′ end of U1 snRNA is directed by a conserved sequence located downstream of the coding region. EMBO J 4:1827-1837.
20. Hertel KJ, Herschlag D, Uhlenbeck OC. 1994. A kinetic and thermodynamic framework for the hammerhead ribozyme reaction. Biochemistry 33:3374-3385.
21. Hill M, Gunning P. 1993. Beta and gamma actin mRNAs are differentially located within myoblasts. J Cell Biol 122:825-832.
22. Hirsch V, Olmstead R, Murphey-Corb M, Purcell R, Johnson P. 1989. SIV from Sooty mangabeys: An African non-human primate lentivirus closely related to HIV-2. Nature 339:389-391.
23. Jennings PA, Molloy PL. 1987. Inhibition of SV40 replicon function by engineered antisense RNA transcribed by RNA polymerase III. EMBO J 6:3043-3047.
24. Kotin RM, Linden RM, Berns KI. 1992. Characterization of a preferred site on human chromosome 19q for integration of adenoassociated virus DNA by non-homologous recombination. EMBO J 11:5071-5078.
25. Kruger K, Grabowski PJ, Zaug A, Sands J, Gottschling DE, Cech TR. 1982. Self-splicing RNA: Autoexcision and autocyclization of the ribosomal RNA intervening sequences of Tetrahymena. Cell 31:147-157.
26. L'Huillier PJ, Davis SR, Bellamy R. 1992. Cytoplasmic delivery of ribozymes leads to efficient reduction in α-lactalbumin mRNA levels in C127I mouse cells. EMBO J 11:4411-4418.
27. Larsson S, Hotchkiss G, Andang M, Nyholm T, Inzunza J, Jansson I, Ahrlund-Richter L. 1994. Reduced Beta2-microglobulin mRNA levels in transgenic mice expressing a designed hammerhead ribozyme. Nucleic Acids Res 22:2242-2248.
28. Lo KM, Biasolo MA, Dehnhi G, Palu G, Haseltine WA. 1992. Inhibition of replication of HIV-1 by retroviral vectors expressing tat-antisense and anti-tat ribozyme RNA. Virology 190:176-183.
29. Lobo SM, Ifill S, Hernandez N. 1990. Cis-acting elements required for RNA polymerase II and III transcription: The human U2 and U6 snRNA promoters. Nucleic Acids Res 18:2891-9899.
30. Maniatis T, Fritsch EF, Sambrook J. 1982. Molecular cloning: A laboratory manual. Cold Spring Harbor, New York: Cold Spring Harbor Laboratory Press.
31. Neumann JR, Morency CA, Russian KO. 1987. A novel rapid assay for chloramphenicol acetyltransferase gene expression. BioTechniques 5:444-447.
32. Noonberg S, Scott G, Garovoy M, Benz C, Hunt C. 1994. In vivo generation of highly abundant sequence-specific oligonucleotides for antisense and triplex gene regulation. Nucleic Acids Res 22:2830-2836.
33. Parry HD, Scherly D, Mattaj IW. 1989. "Snurpogenesis:" the transcription and assembly of U snRNP components. Trends Biochem Sci 14:15-19.
34. Persson C, Gerhart E, Wagner H, Nordstrom K. 1990a. Control replication of plasmid R1: Formation of an initial transient complex is rate-limiting for antisens RNA-target RNA pairing. EMBO J 9:3777-3785.
35. Persson C, Gerhart E, Wagner H, Nordström K. 1990b. Control of replication of plasmid R1: Structures and sequences of the antisense RNA, CopA, required for its binding to the target RNA, CopT. EMBO J 9:3767-3775.
36. Rittner C, Burmester C, Sczakiel G. 1993. In vitro selection of fasthybridizing and effective antisense RNAs directed against the human immunodeficiency virus type 1. Nucleic Acids Res 21:1381-1387.
37. Sarver N, Cantin EM, Chang PS, Zaia JA, Ladne PA, Stephens DA, Rossi JJ. 1990. Ribozymes as potential anti-HIV-1 therapeutics agents. Science 247:1222-1225.
38. Saxena SK, Ackerman EJ. 1990. Ribozymes correctly cleave a model substrate and endogenous RNA in vivo. J Biol Chem 265:17106-17109.
39. Selden R, Byrke-howie K, Rowe M, Goodman H, Moore D. 1986. Human growth hormone as a reporter gene in regulation studies employing transient gene expression. Mol Cell Biol 6:3173-3179.
40. Singh R, Gupta S, Reddy R. 1990. Capping of mammalian U6 small nuclear RNA in vitro is directed by a conserved stem-loop and AUAUAC sequence: Conversion of a non-capped RNA into a capped RNA. Mol Cell Biol 10:939-946.
41. Sioud M, Drlica K. 1991. Prevention of human immunodeficiency virus type 1 integrase expression in Escherichia coli by a ribozyme. Proc Nail Acad Sci USA 88:7303-7307.
42. Steinecke P, Herget T, Schreier PH. 1992. Expression of a chimeric ribozyme gene results in endonucleolytic cleavage of target mRNA and a concomitant reduction of gene expression in vivo. EMBO J 11:1525-1530.
43. Sullenger B, Lee T, Smith C, Ungers G, Gilboa E. 1990. Expression of chimeric tRNA-driven antisense transcripts renders NIH 3T3 cells highly resistant to Moloney murine leukemia virus replication. Mol Cell Biol 10:6512-6523.
44. Sullenger BA, Cech TR. 1993. Tethering ribozymes to a retroviral packaging signal for destruction of viral RNA. Science 262:1566-1569.
45. Taneja K, Lifshitz L, Fay F, Singer R. 1992. Poly(A) RNA codistribution with microfilaments: Evaluation by in situ hybridization and quantitative digital imaging microscopy. J Cell Biol 119:1245-1260.
46. Terns M, Dahlberg J, Lund E. 1993. Multiple cis-acting signals for export of pre-U1 snRNA from the nucleus. Genes & Dev 7:1898-1908.
47. Thompson J, Ayers D, Malmstrom T, McKenzie T, Ganousis L, Chowrira B, Couture L, Stinchcomb D. 1995. Improved accumulation and activity of ribozymes expressed from a tRNA-based polymerase III promoter. Nucleic Acids Res 23:2259-2268.
48. Zhao JJ, Pick L. 1993. Generating loss-of-function phenotypes of the fushi tarazu gene with a targeted ribozyme in Drosophila. Nature 365:448-451.
49. Zhou C, Bahner I, Larson G, Zaia J, Rossi J, Kohn D. 1994. Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes. Gene 149:3-39.