Expression of ribozymes in gene transfer systems to modulate target RNA levels

Current Opinion in Biotechnology

Volumn 9, Issue 5, 1998, Pages 486-496

  Welch, Peter J ^a   Barber, Jack R ^a   Wong Staal, Flossie ^b  

^a Immusol Inc   (United States)

^b UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

RIBOSOME RNA; RNA; VIRUS RNA;

CANCER; CELL CULTURE; GENE EXPRESSION; GENE FUNCTION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC DISORDER; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INFECTION; MAMMAL CELL; NONHUMAN; ONCOGENE; PHASE 1 CLINICAL TRIAL; PRIORITY JOURNAL; REVIEW; RIBOSOME; RNA CLEAVAGE; SOMATIC MUTATION; TRANSGENIC ANIMAL; VIRUS INFECTION;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS; MAMMALIA;

EID: 0032192170     PISSN: 09581669     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0958-1669(98)80034-7     Document Type: Article

References (107)

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81. Vaish NK, Heaton PA, Fedorova O, Eckstein F. In vitro selection of a purine nucleotide-specific hammerheadlike ribozyme. Proc Natl Acad Sci USA. 95:1998;2158-2162.
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84. + peripheral blood lymphocytes by targeted retroviral vectors pseudotyped with the HIV envelope. Transduced cells resisted HIV infection for up to 20 days.
85. Lee CG, Jeang KT, Martin MA, Pastan I, Gottesman MM. Efficient long-term coexpression of a hammerhead ribozyme targeted to the U5 region of HIV-1 LTR by linkage to the multidrug-resistance gene. of special interest Antisense Nucleic Acid Drug Dev. 7:1997;511-522 Microinjection of ribozymes into human cells indicated that both the length of antisense target-binding arms and the subcellular localization play a role in ribozyme efficacy, as measured by anti-HIV activity.
86. Michienzi A, Conti L, Varano B, Prislei S, Gessani S, Bozzoni I. Inhibition of human immunodeficiency virus type 1 replication by nuclear chimeric anti-HIV ribozymes in a human T lymphoblastoid cell line. of special interest Hum Gene Ther. 9:1998;621-628 The authors demonstrate the efficacy of anti-HIV ribozymes localized to the nucleus by linking the ribozyme to the Rev pre-mRNA splice site, thus colocalizing it with the Rev pre-mRNA.
87. Paik SY, Banerjea A, Chen CJ, Ye Z, Harmison GG, Schubert M. Defective HIV-1 provirus encoding a multitarget-ribozyme inhibits accumulation of spliced and unspliced HIV-1 mRNAs, reduces infectivity of viral progeny, and protects the cells from pathogenesis. of special interest Hum Gene Ther. 8:1997;1115-1124 Using a multi-ribozyme approach, the authors demonstrate the specificity of ribozymes in cells. Ribozymes against HIV-1 inhibit replication of HIV-1 but not HIV-2.
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91. Alvarez-Salas LM, Cullinan AE, Siwkowski A, Hampel A, DiPaolo JA. Inhibition of HPV-16 E6/E7 immortalization of normal keratinocytes by hairpin ribozymes. of special interest Proc Natl Acad Sci USA. 95:1998;1189-1194 Ribozymes directed against the E6/E7 genes of HPV reduced cell growth of normal human keratinocytes expressing E6/E7 and prevented immortalization. Inactive ribozymes against the same site also reduced immortalization indicating an antisense effect from the ribozyme.
92. Gebhard JR, Perry CM, Mahadeviah S, Whitton JL. Use of a nonviral vector to express a chimeric tRNA-ribozyme against lymphocytic choriomeningitis virus: cytoplasmic accumulation of a catalytically competent transcript but minimal antiviral effect. Antisense Nucleic Acid Drug Dev. 7:1997;3-11.
93. Heusch M, Kraus G, Johnson P, Wong-Staal F. Intracellular immunization against SIVmac utilizing a hairpin ribozyme. Virology. 216:1996;241-244.
94. Yokoyama Y, Morishita S, Takahashi Y, Hashimoto M, Tamaya T. Modulation of c-fms proto-oncogene in an ovarian carcinoma cell line by a hammerhead ribozyme. of special interest Br J Cancer. 76:1997;977-982 Expression of ribozyme against c-fms reduced target RNA and cell growth potential, and appeared to block proposed autocrine loop between c-fms and macrophage colony-stimulating factor in ovarian epithelial carcinoma.
95. Ahmad M, Theofanidis P, Medford RM. Role of activating protein-1 in the regulation of the vascular cell adhesion molecule-1 gene expression by tumor necrosis factor-alpha. J Biol Chem. 273:1998;4616-4621.
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