Chemistry: molecular biology and microbiology – Micro-organism – tissue cell culture or enzyme using process... – Preparing compound containing saccharide radical
Reissue Patent
2010-05-06
2011-12-06
Whiteman, Brian (Department: 1635)
Chemistry: molecular biology and microbiology
Micro-organism, tissue cell culture or enzyme using process...
Preparing compound containing saccharide radical
C435S006120, C435S091310, C536S023100
Reissue Patent
active
RE043000
ABSTRACT:
The present invention relates to riboswitches that have been engineered to regulate pre-mRNA splicing. In particular, the insertion of a high affinity theophylline binding aptamer into the 3′ splice site region, 5′ splice site region, or branchpoint sequence (BPS) of a pre-mRNA modulates RNA splicing in the presence of theophylline. Accordingly, the aspects of the present invention include, but are not limited to, theophylline-dependent riboswitches which modulate RNA splicing, methods of modulating RNA splicing using theophylline and its corresponding riboswitches, methods of improving/identifying theophylline-dependent riboswitches, methods of treating diseases associated with or caused by abnormal RNA splicing.
REFERENCES:
Crooke, S., Ann. Rev. Medicine, vol. 55, pp. 61-95 (2004).
Peracchi et al., Rev. Med. Virol., vol. 14, pp. 47-64 (2004).
Agrawal et al., Molecular Med. Today, vol. 6, pp. 72-81 (2000).
Chirila et al., Biomaterials, vol. 23, pp. 321-342 (2002).
Opalinska et al., Nature Rev., vol. 1, pp. 503-514 (2002).
Jang et al., Expert Rev. Medical Devices, vol. 1, No. 1, pp. 127-138 (2004).
Pillai, S., “Controlling Pre-mRNA Splicing Through Small Molecule-RNA Interactions,” Presentations made by Pillai at M. Rosbash laboratory, Feb. 2004, and at Ribomed Biotechnologies Inc., Mar. 2004.
Opalinska, J.B. et al., Nature Reviews, vol. 1, pp. 503-514 (2002).
Peracchi, A. et al., Rev. Med. Virol., vol. 14, pp. 47-64 (2004).
Chriila, T. et al., Biomaterials, vol. 23, pp. 321-342 (2002).
Agrawal, S. et al., Molecular med. Today, vol. 6, pp. 72-81 (2000).
Branch, A., Trends in Biochem. Sci., vol. 23, pp. 45-40 (1998).
Crooke, S. Antisense Res. & Application, Chapter 1, pp. 1-50 (Springer-Verlag, Publ.) S. Crooke, Ed) (1998).
Aebi, M., Hornig, H., Padgett, R.A., Reiser, J. and Weissmann, C. (1986) Sequence Requirements for splicing of higher eukaryotic nuclear pre-mRNA.Cell47:555-565.
Aurup, H., Williams, D. and Eckstein, F. (1992) 2′-Fluoro- and 2′-amino-2′-deoxynucleoside 5′-triphosphates as substrates for T7 RNA polymerase.Biochemistry31:9636-9641.
Banerjee, H., Rahn, A., Davis, W. and Singh, R.(2003) Sex lethal and U2 small nuclear ribonucleoprotein auxiliary factor (U2AF65) recognize polypyrimidine tracts using multiple modes of binding.RNA9:88-99.
Belshaw, P.J., Ho, S.N., Crabtree, G.R. and Schreiber, S.L. (1996) Controlling protein association and subcellular localization with a synthetic ligand that induces heterodimerization of proteins.Proc. Natl. Acad. Sci. USA 93:4604-4607.
Bevers, S., Ha S.B. and McLaughlin, L.W. (1999) Critical nature of a specific uridineO2-carbonyl for clevage by the hammerhead ribozyme.Biochemistry38:7710-7718.
Black, D. L. (2003) Mechanisms of alternative pre-messenger RNA splicing.Annu. Rev. Biochem. 72:291-336.
Boggs, R.T., Gregor, P., Idriss, S., Belote, J.M. and McKeown, M. (1987) Regulation of sexual differentiation in D. melanogaster via alternative splicing of RNA from the transformer gene.Cell50:739-747.
Bunch, T.A., Grinblat, Y. and Goldstein, L.S. (1988) Characterization and use of theDrosophilametallothionein promoter in culturedDrosophila melanogastercells. Nucleic Acids Res. 16:1043-1061.
Buratti, E., Muro, A.F., Giombi, M., Gherbassi, D., laconcig, A. and Baralle, F.E. (2004) RNA folding affects the recruitment of SR proteins by mouse and human polypurinic enhancer elements in the fibronectin EDA exon.Mol. Cell. Biol. 24:1387-1400.
Burgstaller, P. and Famulok, M. (1994) Isolation of RNA aptamers for biological cofactors by in vitro selection.Angew. Chem. Int. Ed. Engl. 33:1084-1087.
Cartegni, L. and Krainer, A.R. (2003) Correction of disease-associated exon skipping by synthetic exon-specific activators.Nat. Struct. Biol. 10:120-125.
Chen, Z., Monia, B.P. and Corey, D.R. (2002) Telomerase inhibition, telomere shortening, and decreased cell proliferation by cell permeable 2′-O-methoxyethyl oligonucleotides.J Med. Chem. 45:5423-5425.
Chiara, M.D., Palandjian, L., Feld Kramer, R. and Reed, R. (1997) Evidence that U5 snRNP recognizes the 3′ splice site for catalytic step II in mammals.EMBO J16:4746-4756.
Chua, K. and Reed, R. (2001) An upstream AG determines whether a downstream AG is selected during catalytic step II of splicing.Mol. Cell. Biol. 21:1509-1514.
Claverie, J.M. (2001) Gene number. What if there are only 30,000 human genes?Science291:1255-1257.
Cline, T.W. and Meyer, B.J. (1996) Vive la difference: males vs females in flies vs worms.Annu. Rev. Genet. 30:637-702.
Collins, C.A. and Guthrie, C. (1999) Allele -specific genetic interactions between Prp8 and RNA active site residues suggest a function for Prp8 at the catalytic core of the splicesome.Genes Dev. 13:1970-1982.
Collins, C.A. and Guthrie, C. (2001) Genetic interactions between the 5′ and 3′ splice site consensus sequences and U6 snRNA during the second catalytic step of pre-mRNA splicing.RNA7:1845-1854. Das, R. and Reed, R. (1999) Resolution of the mammalian E complex and the ATP-dependent spliceosomal complexes on native agarose mini-gels.RNA5:1504-1508.
Deirdre, A., Scadden, J. and Smith, C.W. (1995) Interactions between the terminal bases of mammalian introns are retained in inosine-containing pre-mRNAs.EMBO J14:3236-3246.
Dignam. J.D., Lebovitz, R.M. and Roeder, R.G. (1983) Accurate transcription initiation by RNA polymerase II in a soluble extract from isolated mammalian nuclei.Nucleic Acids Res. 11:1475-1489.
Dominski, Z. and Kole, R. (1993) Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.Proc. Natl. Acad. Sci. USA 90:8673-8677.
Dredge, B.K., Polydorides, A.D. and Darnell, R.B. (2001) The splice of life: alternative splicing and neurological disease.Nat. Rev. Neurosci. 2:43-50.
Duconge, F. and Toulme, J.J. (1999) In vitro selection identifies key determinants for loop-loop interactions: RNA aptamers selective for the TAR RNA element of HIV-1.RNA5:1605-1614.
Eder, P.S., DeVine, R.J., Dagle, J.M. and Walder, J.A. Substrate specificity and kinetics of degradation of antisense oligonucleotides by a 3′exonuclease in plasma.Antisense Res. Dev., 1:141-151 (1991).
Endo, M., Mitsui, T., Okuni, T., Kimoto, M., Hirao, I. and Yokoyama, S. (2004) Unnatural base pairs mediate the site-specific incorporation of an unnatural hydrophobic component into RNA transcripts.Bioorg. Med. Chem. Lett. 14:2593-2596.
Eperon, I.C. and Muntoni, F. (2003) Can a ‘patch’ in a skipped exon make the pre-mRNA splicing machine run better?Trends Mol. Med. 9:233-234.
Eperon, L. P., Graham, I.R., Griffiths, A.D. and Eperon, I.C. (1988) Effects of RNA secondary structure on alternative splicing of pre-mRNA: is folding limited to a region behind the transcribing RNA polymerase?Cell54:393-401.
Faustino, N.A. and Cooper, T.A. (2003) Pre-mRNA splicing and human disease.Genes Dev. 17:419-437.
Freyer, G.A., Arenas, J., Perkins, K.K., Furneaux, H.M., Pick, L., Young, B., Roberts, R.J. and Hurwitz, J. (1987) In vitro formation of a lariat structure containing a G2′-5′G linkage.J Biol. Chem. 262:4267-4273.
Garcia-Blanco, M.A., Baraniak, A.P. and Lasda, E.L. (2004) Alternative splicing in disease and therapy.Nat. Biotechnol. 22:535-546.
Gaur, R.K., McLaughlin, L.W. and Green, M.R. (1997) Functional group substitutions of the branchpoint adenosine in a nuclear pre-mRNA and a group II intron.RNA3:861-869.
Gaur, R.K., Valcarcel, J. and Green, M.R. (1995) Sequential recognition of the pre-mRNA branch point by U2AF65and a novel spliceo-some-associated 28-kDa protein.RNA1:407-417.
Goguel, V. and Rosbash, M. (1993) Splice site choice and splicing efficiency are positively influenced by the pre-mRNA intramolecular base pairing in yeast.Cell72:893-901.
Goguel, V., Wang, Y. and Rosbash, M. (1993) Short artificial hairpins sequester splicing signals and inhibit yeast pre-mRNA splicing.Mol. Cell. Biol. 13:6841-6848.
Goodall, G.J. and Filipowicz, W. (1991) Different effects of intron nucleotide composition and secondary structure on pre-mRNA splicing in monocot an
Gaur Rajesh K.
Pillai Sailesh Gopalakrishna
City of Hope
Perkins Coie LLP
Wang Yingli
Whiteman Brian
LandOfFree
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