Modified ribozymes

Details Modified ribozymes Modified ribozymes

2005-05-10

2005-05-10

Wang, Andrew (Department: 1635)

Drug, bio-affecting and body treating compositions

Designated organic active ingredient containing

Carbohydrate doai

C435S006120, C435S325000, C435S375000, C435S091100, C536S023100, C536S024500

Reexamination Certificate

active

06890908

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 ribose sugar is replaced by a modifier group, selected from halo, sulfhydryl, azido, amino, monosubstituted amino and disubstituted amino groups, a process for the preparation of modified RNA molecules and the use of modified RNA 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.
patent: 5817635 (1998-10-01), Eckstein et al.
patent: 0387775 (1990-09-01), None
patent: 0321201 (1994-12-01), None
patent: 8804300 (1988-06-01), None
patent: 9103162 (1991-03-01), None
patent: 9207065 (1992-04-01), None
Branch TIBS,23:45-50, Feb. 1998.*
Baer et al., “Structure and Function of Bacterial RNase P,”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,”Biochemistry25:4473-4477 (1986).
Been and Cech, “One Binding Site Determines Sequence Specificity of Tetrahymena Pre-rRNA Self-Splicing, Trans-Splicing and RNA Enzyme Activity,”Cell47:207-216 (1986).
Black et al., “The Antiviral Activity of Certain Thiophosphate and 2′-Chloro Substituted Polynucleotide Homopolymer Duplexes,”Virology48:537-545 (1972).
Cameron and Jennings, “Specific Gene Expression by Engineered Ribozymes in Monkey Cells,”Proc. Natl. Acad. Sci. USA86:9139-9143 (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 American255:76-84 (1986).
Cech, “The Chemistry of Self-Splicing RNA and RNA Enzymes,”Science236:1532-1539 (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 ConferenceOct. 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 Nuclei Acids by the “Hairpin” Ribozyme,”Biochemistry30:8518 (1991).
Codington et al., “Nucleosides. XVIII. Synthesis of 2′-Fluorothymidine, 2′-Fluorodeoxyuridine, and Other 2′-Halogeno-2′-Deoxy Nucleosides,”J. Org. Chem.29:558-567 (1964).
Cotten, “The in vivo application of ribozymes,”TIBS8:174-178 (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 and Fox, “Nucleosides. XXXIX. 2′-Deoxy-2′-fluorocytidine, 1-β-D-Arabinofuranosylk-2-amino-1,4(2H)-4-iminopyrimidine, and Related Derivatives,”J. Org. Chem.32:1462-1471 (1976).
Doudna et al., “A Multisubunit that is a Catalyst of and Template for Complementary Strand RNA Synthesis,”Science251:1605 (1991).
Ellington and Szostak, “In Vitro selection of RNA molecules that bind specific ligands,”Nature346:818-822 (1990).
Egli et al., “Crystal Structure of an Okazaki Fragment at 2-A Resolution,”Proc. Natl. Acad. Sci. USA89:534-538 (1992).
Fedor and Uhlenbeck, “Substrate sequence effect on “hammerhead” RNA catalytic efficiency,”Proc. Natl. Acad. Sci. USA87:1668-1672 (1990).
Greider and Blackburn, “A telomeric sequence in the RNA of Tetrahymena telomerase required for telomere repeat synthesis,”Nature337:331-337 (1989).
Hampel et al., “Hairpin' Catalytic RNA Model: Evidence for Helices and Sequence Requirement for Substrate RNA,”Nucleic Acids Research18:299-304 (1990).
Haseloff and Gerlach, “Simple RNA Enzymes with New and Highly Specific Endoribonuclease Activities,”Nature334:585-591 (1988).
Herschlag and Cech, “DNA cleavage catalysed by the ribozyme from Tetrahymena,”Nature344:405-409 (1990).
Hobbs et al., “Polynucleotides Containing 2′-Chloro-2′-deoxyribose,”Biochemistry11:43367-4334 (1972).
Hobbs et al., “Polynucleotides Containing 2′-Amino-2′-deoxyribose and 2′-Azido-2′-deoxyribose,”Biochemistry12:5138-5145 (1973).
Hutchins et al.,“Self-cleavage of Plus and Minus RNA Transcripts of Avocado Sunblotch Viroid,”Nucleic Acids Research14:3627-3640 (1986).
Ikehara and Miki, “Studies of Nucleosides and Nucleotides. LXXXII. Cyclonucleosides. (39). Synthesis and Properties of 2′-Halogeno-2′-deoxyadenosines,”Chem. Pharm. Bull.26:2449-2453 (1978).
Imazawa and Eckstein, “Facile Synthesis of 2′-Amino-2′-deoxyribofuranosyl Purines,”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, “Einfache Unwandlung von 2,2′-Anhydrocytidin in 2′-Desoxy-2′-fluorocytidin durch nucleophile Substitution mit Kaliumfluorid/Kronenether,”Ang. Chem.90:557-558 (1979).
Mungall et al., “Use of the Azido Group in the Synthesis of 5′ Terminal Aminodeoxythymidine Oligonucleotides,”J. Org. Chem.40:1659-1662 (1975).
Nielsen et al., “Sequence-Selective Recognition of DNA by Strand Displacement with a Thymine-Substituted Polyamide,”Science254:1497 (1991).
Paolella et al., “Nuclease Resistant Ribozymes with High Catalytic Activity,”EMBO J.11:1913-1919 (1992).
Perreault et al., “Mixed Deoxyribo- and Ribo-Oligonucleotides with Catalytic Activity,”Nature344:565-567 (1990).
Perreault et al., “Relationship between 2′-Hydroxyls and Magensium Binding in the Hammerhead RNA Domain: A Model for Ribozyme Catalysis,”Biochemistry30:4020-4025 (1991).
Peiken et al., “Kinetic Characterization of Ribonuclease Resistant 2′-Modified Hammerhead Ribozymes,”Science253:314-317 (1991).
Pieken et al., “Influence of 2′-Amino and 2′-Fluoro Modifications on the Catalytic Properties of Hammerhead Ribozymes,”Abstract of the 14th tRNA workshopMay 4-9, 1991, Ridzyna, Poland.
Price and Cech, “Coupling of Tetrahymena Ribosomal RNA Splicing to β-Galactosidase Expression inEscherichia coli,” Science228:719 (1985).
Rossi and Sarver, “RNA enzymes, ribozymes as antiviral therapeutic agents,”TIBTECH8:179-183 (1990).
Ruffner et al., “Thiophosphate interference experiments locate phosphates important for the hammerhead RNA self-cleavage reaction,”Gene82:31-41 (1989).
Sarver et al., “Ribozymes as Potential Anti-HIV-1 Therapeutic Agents”Science247:1222-1225 (1990).
Scaringe et al., “Chemical synthesis of biologically active oligoribonucleotides using β-cyanoethyl protected ribonucleoside phosphoramidites,”Nucl. Acids Res.18:5433-5441 (1990).
Sheldon et al., “RNA Self-Cleavage by the Hammerhead Structure,”Nucleic Acids and Mo

Affiliated with

Also associated with

Rating

Modified ribozymes does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Modified ribozymes, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Modified ribozymes will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-3391630