Cell ablation using trans-splicing ribozymes

Chemistry: molecular biology and microbiology – Animal cell – per se ; composition thereof; process of... – Primate cell – per se

Patent

Rate now

  [ 0.00 ] – not rated yet Voters 0   Comments 0

Details

435 6, 435 691, 435 9131, 4351723, 4352523, 4353201, 435325, 435348, 435375, 536 231, 536232, 536 245, C12N 1585, C12N 1563, C07H 2104

Patent

active

060109041

ABSTRACT:
The design of new ribozymes capable of self-catalyzed trans-splicing which are based upon the catalytic core of a Group I intron are described. Using this design, it is possible to construct ribozymes capable of efficiently splicing a new 3' exon sequence into any chosen target RNA sequence in a highly precise manner. A method of cell ablation is also described that provides a toxic product to a host cell in vivo in a targetted, regulated manner utilizing novel trans-splicing ribozymes of the invention. Inactive pro-ribozyme forms are also described.

REFERENCES:
patent: 5037746 (1991-08-01), Cech et al.
Agabian, N., "Trans Splicing of Nuclear Pre-mRNAS," Cell 61(7):1157-1160 (Jun. 1990).
Barinaga, M., "Ribozymes: Killing the Messenger," Science 262:1512-1514 (1993).
Been, M.D. et al., "One binding site determines sequence specificity of Tetrahymena pre-mRNA self splicing, trans-splicing, and RNA enzyme activity," Cell 47(2):207-216 (1986).
Been, M.D. et al., "Selection of Circularization Sites in a Group I IVS RNA Requires Multiple Alignments of an Internal Template-Like Sequence," Cell 50:951-961 (1987).
Brand, A. et al., "A General Method for Directed Gene Expression in Drosophila," Presented at The Genome Conference, 13th Annual Conference on the Organisation and Expression of the Genome, Victoria, Australia, Feb. 18-22, 1991, SYM-15-2.
Brand, A.H. et al., "Targeted Gene Expression in Drosophila melanogaster," J. Cell. Biochem. Supp. 14E (Mar.-Apr. 1990), Abstracts, 19th Annual UCLA Symposia, Abst. P201, p. 153.
Breitman, M.L. et al., "Genetic Ablation: Targeted Expression of a Toxin Gene Causes Microphthalmia in Transgenic Mice," Science 238:1563-1565 (1987).
Cech, T.R., "Self-Splicing of Group I Introns," Ann. Rev. Biochem. 59:543-568 (Jul. 1990).
Cech, T.R., "Conserved sequences and structures of group I introns: building an active site for RNA catalysis--a review," Gene 73:259-271 (1988).
Chuat, J.C. et al., "Can Ribozymes Be Used to Regulate Procaryote Gene Expression?," Biochem. Biophys. Res. Commun. 162(3):1025-1029 (1989).
Cotten, M. et al., "Ribozyme Mediated Destruction of RNA in vivo," EMBO J. 8(12):3861-3866 (1989).
Cotten, M., "The in vivo application of ribozymes," Trends Biotech. 8(7):174-178 (Jul. 1990).
Doudna, J.A. et al., "RNA structure, not sequence, determines the 5' splice-site specificity of a group I intron," Proc. Natl. Acad. Sci. 86:7402-7406 (1989).
Evans, G.A., "Dissecting mouse development with toxigenics," Genes and Develop. 3:259-263 (1982).
Fischer, J.A. et al., "GAL4 activates transcription in Drosophila," Nature 332:853-856 (1988).
Flanegan, J.B. et al., "Tetrahymena Ribozyme Catalyzes Trans-Splicing of Model Oligoribonucleotide Substrates," J. Cell. Biochem. Supp. 12D:28 N202 (1988), Presented at the Symposium of the Molecular Biology of RNA Held at the 17th Annual UCLA Symposia on Molecular and Cellular Biology, Keystone, CO (Apr. 1988).
Garriga, G. et al., "Mechanism of recognition of the 5' splice site in self-splicing group I introns," Nature 322:86-89 (1986).
Haseloff, J. et al., "Simple RNA enzymes with new and highly specific endoribonuclease activities," Nature 334:585-591 (1988).
Haseloff, J. et al., "Design of Ribozymes for Trans-Splicing," Presented at The Genome Conference, 13th Annual Conference on the Organisation and Expression of the Genome, Victoria, Australia, Feb. 18-22, 1991, SYM-8-1.
Heus, H.A. et al., "Sequence-dependent structural variations of hammerhead RNA enzymes," Nucl. Acids Res. 18(5):1103-1108 (Mar. 1990).
Hirsh, D. et al., "Trans-Splicing and SL RNAs in C. elegans," Mol. Biol. Rpts. 14(2/3):115 (Mar. 1990).
Hutchins, C.J. et al., "Self-cleavage of plus and minus RNA transcripts of avocado sunblotch viroid," Nucl. Acids Res. 14(9):3627-3640 (1986).
James, W., "Towards gene-inhibition therapy: a review of progress and prospects in the field of antiviral antisense nucleic acids and ribozymes," Antiviral Chem. & Chemotherapy 2(4):191-214 (Nov. 1991).
Joyce, G.F., "Amplification, mutation and selection of catalytic RNA," Gene 82:83-87 (1989).
Kan, N.C. et al., "The intervening sequence of the ribosomal RNA gene is highly conserved between Two Tetrahymena species," Nucl. Acids Res. 10(9):2809-2822 (1982).
Keese, P. et al., "The Structure of Viroids and Virusoids," in Viroids and Viroid-like Pathogens J.S. Semancik, ed., CRC Press, Boca Raton, Fla. 1987, pp. 1-47.
Koizumi, M. et al., "Construction of a series of several self-cleaving RNA duplexes using synthetic 21-mers," FEBS Letters 228(2):228-230 (1988).
Koizumi, M. et al., "Cleavage of specific sites of RNA by designed ribozymes," FEBS Letters 239(2):285-288 (1988).
Mariani, C. et al., "Induction of male sterility in plants by a chimaeric ribonuclease gene," Nature 347(6295):737-741 (Oct. 1990).
Maxwell, I.H. et al., "Regulated Expression of a Diphtheria Toxin A-Chain Gene Transfected into Human Cells: Possible Strategy for Inducing Cancer Cell Suicide," Cancer Research 46:4660-4664 (1986).
Michel, F. et al., "The guanosine binding site of the Tetrahymena ribozyme," Nature 342:391-395 (1989).
Mulligan, R.C. et al., "The Basic Science of Gene Therapy," Science 260:926-932 (1993).
Palmiter, R.D. et al., "Cell Lineage Ablation in Transgenic Mice by Cell-Specific Expression of a Toxin Gene," Cell 50:435-443 (1987).
Qian, S. et al., "Antisense ribosomal protein gene expression specifically disrupts oogenesis in Drosophila melanogaster," Proc. Natl. Acad. Sci. USA 85:9601-9605 (1988).
Reiss, B. et al., "Protein fusions with the kanamycin resistance gene from transposon Tn5," EMBO J. 3(13):3317-3322 (1984).
Riedel, C.J. et al., "Diphtheria toxin mutant selectively kills cerebellar purkinje neurons," Proc. Natl. Acad. Sci. USA 87:5051-5055 (Jul. 1990).
Salvo, J.L.G. et al., "Deletion-tolerance and Trans-splicing of the Bacteriophage T4 TD Intron," J. Mol. Biol. 211(3):537-549 (Feb. 1990).
Sarver, N. et al., "Ribozymes as Potential Anti-HIV-1 Therapeutic Agents," Science 247(4947):1222-1225 (Mar. 1990).
Suh, E.R. et al., "Base Pairing between the 3' Exon and an Internal Guide Sequence Increase 3' Splice Site Specificity in the Tetrahymena Self-Splicing rRNA Intron," Mol. Cell. Biol. 10(6):2960-2965 (Jun. 1990).
Szostak, J.W., "Enzymatic activity of the conserved core of a group I self-splicing intron," Nature 322:83-86 (1986).
Taira, K. et al., "Construction of a novel artificial-ribozyme-releasing plasmid," Protein Engineering 3(8):733-737 (Aug. 1990).
Walbot, V. et al., "Plant development and ribozymes for pathogens," Nature 334:196-197 (1988).
Waring, R.B. et al., "The Tetrahymena rRNA Intron Self-Splices in E. coli: In Vivo Evidence for the Importance of Key Base-Paired Regions of RNA for Rna Enzyme Function,"Cell 40:371-380 (1985).
Wert, S.E. et al., "Morphological Analysis of Lung Cell Ablation in Transgenic Mice Expressing a Toxic Fusion Gene," Am. Rev. Respir. Dis. 141(No.4, Pt2):A695 (Apr. 1990).
Winter, A.J. et al., "The mechanism of Group I self-splicing: an internal guide sequence can be provided in Trans," EMBO J. 9(6):1923-1928 (Jun. 1990).
Yagi, T. et al., "Homologous Recombination at c-fyn locus of mouse embryonic stem cells with use of diphtheria toxin A-fragment gene in negative selection," Proc. Natl. Acad. Sci. USA 87:9918-9922 (Dec. 1990).
Zaug, A.J. et al., "Intervening Sequence RNA of Tetrahymena Is an Enzyme," Science 231:470-475 (1986).

LandOfFree

Say what you really think

Search LandOfFree.com for the USA inventors and patents. Rate them and share your experience with other people.

Rating

Cell ablation using trans-splicing 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 Cell ablation using trans-splicing ribozymes, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Cell ablation using trans-splicing ribozymes will most certainly appreciate the feedback.

Rate now

     

Profile ID: LFUS-PAI-O-1072038

  Search
All data on this website is collected from public sources. Our data reflects the most accurate information available at the time of publication.