Modified ribozymls

Organic compounds -- part of the class 532-570 series – Organic compounds – Carbohydrates or derivatives

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536 231, 536 245, 435 911, C07H 2104, C07H 2100, C12P 1934

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active

056986874

ABSTRACT:
The present invention refers to an RNA molecule with catalytic activity comprising at least one modified nucleoside, wherein the hydroxy group at the 2'-position of the fibose sugar is replaced by a modifier group, selected from halo, sulfhydryl, azido, amino, monosubstituted amino, and disubstituted andno groups, a process for the preparation of modified KNA molecules and the use of modified KNA molecules as therapeutic agents and biocatalysts.

REFERENCES:
patent: 5149796 (1992-09-01), Rossi et al.
patent: 5298612 (1994-03-01), Jennings et al.
patent: 5334711 (1994-08-01), Sproat et al.
Chowrira et al., Extensive phosphorothioate substitution yields highly ace and nuclease resistant hairpin ribozymes, Nucleic Acids Research, vol. 20(11) 2835-2840 Apr. 1992.
Pieken et al., Kinetic characterization of ribonuclease resistant 2'-modified hammerhead ribozymes, Science, vol. (253) pp. 314-317 Jul. 1991.
Barinaga, Ribozymes: Killing the messenger, Science, 1512-1514 Dec. 1993.
Aurup et al., 2'-fluoro- and 2'-amino-deoxynucleoside 5'-triphosphates as substrates for T7 RNA polymerase, Biochemistry, vol. (31) pp. 9636-9641 Oct. 1992.
Milligan et al., Oligoribnonucleotide synthesis using T7 RNA polymerase and synthetic DNA templates, Nucleic Acids Research, vol. 15 (21) pp. 8783-8798 Oct. 1987.
Baer et al., Nucleic Acids and Molecular Biology, vol. 3, pp. 231-250, Eckstein and Lilley eds, Springer Verlag, Berlin/Heidelberg (1988).
Bass and Cech, "Ribozyme Inhibitors: Deoxyguanosine and Dideoxyguanosine are Competitive Inhibitors of Self-Splicing of the Tetrahymena Ribosomal Ribonucleic Acid Precursor," Biochemistry 25:4473-4477 (1986).
Been and Cech, "One Binding Site Determines Sequence Specificity of Tetrahymena Pre-rRNA Self-Splicing, Trans-Splicing and RNA Enzyme Activity," Cell 47:207-216 (1986).
Black et al., Virology 48:537-545 (1972).
Cameron and Jennings, "Specific Gene Expression by Engineered Ribozymes in Monkey Cells," Proc. Natl. Acad. Sci. USA 86:9139-9143 (1989).
Cech, "A Model for the RNA-Catalyzed Replication of RNA," Proc. Natl. Acad. Sci. USA, 83: 4360-4363 (1986).
Cech, "RNA as an Enzyme," Scientific American 255:76-84 (1986).
Cech, "The Chemistry of Self-Splicing RNA and RNA Enzymes," Science 236:1532-1539 (1987).
Cech, "Self-Splicing of Group I Introns," Ann. Rev. Biochem. 59-543-568 (1990).
Cedergren et al., "Catalytic RNA as an Anti-HIV Agent: Design and Delivery to Cells," Abstract NIH Conference Oct. 21-24 1990, San Diego, California.
Cedergren et al., Abstract presentation at Cold Spring Harbor meeting: RNA Processing, May 15-19, 1991.
Cedergren et al., Abstract presentation at Cold Spring Harbor meeting: RNA Processing, May 16-20, 1990.
Chowrira et al., "Four Ribose 2'-Hydroxyl Groups Essential for Catalytic Function of the Hairpin Ribozyme," J. Biol. Chem. 268:19458-19462 (1993).
Chowrira and Burke, "Binding and Cleavage of Nucleic Acids by the Hairpin Ribozyme," Biochemistry 30:8518 (1991).
Codington et al., J. Org. Chem. 29:558-567 (1964).
Cotten, "The in vivo application of ribozymes," TIBS 8:174-178 (1990).
Cotten et al., "Ribozyme, Antisense RNA, and Antisense DNA Inhibition of U7 Small Nuclear Ribonucleoprotein-Mediated Histone Pre-mRNA Processing In Vitro," Mol Cell. Biol. 9:4479-4487 (1989).
Doerr & Fox, J. Org. Chem. 32:1462 (1976).
Doudna et al., "A Multisubunit that is a Catalyst of and Template for Complementary Strand RNA Synthesis," Science 251:1605 (1991).
Ellington and Szostak, Nature 346:818-822 (1990).
Engli et al., "Crystal Structure of an Okazaki Fragment of 2-A Resolution," Proc. Natl. Acad. Sci. USA 89:534-538 (1992).
Fedor and Uhlenbeck, "Substrate sequence effects on hammerhead RNA catalytic efficiency," Proc. Natl. Acad. Sci. USA 87:1668-1672 (1990).
Greider and Blackburn, Nature 337:331-337 (1989).
Hampel et al., "`Hairpin` Catalytic RNA Model: Evidence for Helices and Sequence Requirement for Substrate RNA," Nucleic Acids Research 18:299-304 (1990).
Haseloff and Gerlach, "Simple RNA Enzymes with New and Highly Specific Endoribonuclease Activities," Nature 334:585-591 (1988).
Herschlag and Cech, Nature 344:405-409 (1990).
Hobbs et al., Biochemistry 11:43367-4334 (1972).
Hobbs et al., Biochemistry 12:5138-5145 (1973).
Hutchins et al., "Self-cleavage of Plus and Minus RNA Transcripts of Avocado Sunblotch Viroid," Nucleic Acids Research 14:3627-3640 (1986).
Ikehara and Miki, Chem. Pharm. Bull. 26:2449-2453 (1978).
Imazawa and Eckstein, J. Org. Chem. 44:2039-2041 (1979).
Ludwig, "A New Route to Nucleoside 5'-Triphosphates," Acta Biochim. Biophys. Acad. Sci. Hung. 16:131-133 (1981).
Ludwig and Eckstein, "Rapid and Efficient Synthesis of Nucleoside 5'-O-(1-Thiotriphosphates), 5'-Triphosphates and 2',3'-Cyclophosphorothioates Using 2-Chloro-4H-1,3,2-Benzodioxaphosphorin-4-one," J. Org. Chem. 54:631-635 (1989).
Mengel and Guschlbauer, Ang. Chem. 90:557-558 (1979).
Mungall et al., J. Org. Chem. 40:1659 (1975).
Nielsen et al., "Sequence-Selective Recognition of DNA by Strand Displacement with a Thymine-Substituted Polyamide," Science 254:1497 (1991).
Paolella et al., "Nuclease Resistant Ribozymes with High Catalytic Activity", EBMO J. 11:1913-1919 (1992).
Perreault et al., "Mixed Deoxyribo- and Ribo-Oligonucleotides with Catalytic Activity," Nature 344:565-567 (1990).
Perreault et al., "Relationship between 2'-Hydroxyls and Magensium Binding in the Hammerhead RNA Domain: A Model for Ribozyme Catalysis," Biochemistry 30:4020-4025 (1991).
Pieken et al., "Kinetic Characterization of Ribonuclease-Resistant 2'-Modified Hammerhead Ribozymes," Science 253:314-317 (1991).
Pieken et al., "Influence of 2'-Amino and 2'-Fluoro Modifications on the Catalytic Properties of Hammerhead Ribozymes," Abstract of the 14th International tRNA workshop May 4-9, 1991, Ridzyna, Poland.
Price and Cech, "Coupling of Tetrahymena Ribosomal RNA Splicing to .beta.-Galactosidase Expression in Escherichia coli," Science 228:719 (1985).
Rossi and Sarver, "RNA enzymes, ribozymes as antiviral therapeutic agents," TIBTECH 8:179-183 (1990).
Ruffner et al., "Thiophosphate interference experiments locate phosphates important for the hammerhead RNA self-cleavage reaction," Gene 82:34-41 (1989).
Sarver et al., "Ribozymes as Potential Anti-HIV-1 Therapeutic Agents" Science 247:1222-1225 (1990).
Scaringe et al., "Chemical synthesis of biologically active oligoribonucleotides using .beta.-cyanoethyl protected ribonucleoside phosphoramidites," Nucl Acids Res. 18:5433-5441 (1990).
Sheldon et al., Nucleic Acids and Molecular Biology, vol. 4., pp. 227-242, eds Eckstein and Lilley, Springer Verlag Berlin Heidelberg (1990).
Sinha et al., Nucleic Acids Research 12:4539-4577 (1984).
Strobel and Cech, "Tertiary Interactions with the Internal Guide Sequence Mediate Docking of the P1 Helix into the Catalytic Core of the T. Ribozyme," Biochemistry 32:13593 (1993).
Sung, J. Org. Chem. 47:3623-3628 (1982).
Symons, TIBS 14:445-450 (1989).
Tuerk and Gold, "Systematic Evolution of Ligands by Exponential Enrichment: RNA Ligands to Bacteriophage T4 DNA Polymerase," Science 249:505-510 (1990).
Uchida and Egami, The Enzymes vol. IV, 3rd ed., pp. 205-250, P.D. Boyer editor, Academic Press (1971).
Uhlenbeck, "A Small Catalytic Oligoribonucleotide," Nature 328:596-600 (1987).
Uhlenbeck, Proc. Natl. Acad. Sci. USA 87:1668-1672 (1990).
Uhlmann and Peyman, "Antisense Oligonucleotides: A New Therapeutic Principle," Chemical Reviews 90:544 (1990).
Verheyden et al., J. Org. Chem. 36:250 (1971).
Williams et al., "Properties of 2'-Fluorothymidine-Containing Oloigonucleotides: Interaction with Restriction Endonuclease EcoRV," Biochemistry 30:4001-4009 (1991).
Wu et al., "Convenient Procedure for the Preparation of Specific Mixed DNA-RNA Polymers," J. Am. Chem. Soc. 111:8531-8533 (1991).
Yang et al., "Minimum Ribonucleotide Requirement for Catalysis by the RNA Hammerhead Domain," Biochemistry 31:5005 (1992).
Yang et al., "Mixed DNA/RNA Polymers Are Cleaved by the Hammerhead Ribozyme," Biochemistry 29:1156 (1990).
Zang et al., Biochemistry 27:8924-8931 (1988).
Zaug and Cech, "The Intervening Sequence RNA of Tetrahymena

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