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WO1996018733A3 - Ribozyme-mediated inactivation of leukemia-associated rna - Google Patents

Ribozyme-mediated inactivation of leukemia-associated rna Download PDF

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Publication number
WO1996018733A3
WO1996018733A3 PCT/US1995/016451 US9516451W WO9618733A3 WO 1996018733 A3 WO1996018733 A3 WO 1996018733A3 US 9516451 W US9516451 W US 9516451W WO 9618733 A3 WO9618733 A3 WO 9618733A3
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WO
WIPO (PCT)
Prior art keywords
associated rna
leukemia
rna
ribozyme
mediated inactivation
Prior art date
Application number
PCT/US1995/016451
Other languages
French (fr)
Other versions
WO1996018733A2 (en
Inventor
Umberto Pace
Shaji T George
Allan R Goldberg
Original Assignee
Innovir Lab Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Innovir Lab Inc filed Critical Innovir Lab Inc
Priority to AU49619/96A priority Critical patent/AU4961996A/en
Publication of WO1996018733A2 publication Critical patent/WO1996018733A2/en
Publication of WO1996018733A3 publication Critical patent/WO1996018733A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121—Hammerhead
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/126—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes involving RNAse P
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527—Other alkyl chain
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/027—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a retrovirus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/028—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a herpesvirus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

RNA molecules, including ribozymes, external guide sequences for RNAse P, and antisense oligonucleotides have been constructed which promote ribozyme cleavage of, or block transcription of, respectively, specific cancer-associated RNA, for example, acute promyeloleukocytic leukemia-associated RNA, follicular lymphoma-associated RNA and chronic myelocytic leukemia-associated RNA. Methods of producing and using such RNA molecules are also described.
PCT/US1995/016451 1994-12-14 1995-12-14 Ribozyme-mediated inactivation of leukemia-associated rna WO1996018733A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU49619/96A AU4961996A (en) 1994-12-14 1995-12-14 Ribozyme-mediated inactivation of leukemia-associated rna

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US35495694A 1994-12-14 1994-12-14
US354,956 1994-12-14

Publications (2)

Publication Number Publication Date
WO1996018733A2 WO1996018733A2 (en) 1996-06-20
WO1996018733A3 true WO1996018733A3 (en) 1996-12-12

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1995/016451 WO1996018733A2 (en) 1994-12-14 1995-12-14 Ribozyme-mediated inactivation of leukemia-associated rna

Country Status (2)

Country Link
AU (1) AU4961996A (en)
WO (1) WO1996018733A2 (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5869248A (en) * 1994-03-07 1999-02-09 Yale University Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences
US5683873A (en) * 1995-01-13 1997-11-04 Innovir Laboratories, Inc. EGS-mediated inactivation of target RNA
US6057153A (en) * 1995-01-13 2000-05-02 Yale University Stabilized external guide sequences
US20020081733A1 (en) * 1995-11-14 2002-06-27 Catherine M. Verfaillie Method to prepare drug-resistant, non-malignant hematopoietic cells
US5877162A (en) * 1996-03-14 1999-03-02 Innovir Laboratories, Inc. Short external guide sequences
US5976874A (en) * 1996-08-16 1999-11-02 Yale University Phenotypic conversion of drug-resistant bacteria to drug-sensitivity
JP2001505428A (en) * 1996-12-06 2001-04-24 ニールズ パリスガード Detection of chromosomal abnormalities
US6013447A (en) * 1997-11-21 2000-01-11 Innovir Laboratories, Inc. Random intracellular method for obtaining optimally active nucleic acid molecules
EP1032707A2 (en) * 1997-11-21 2000-09-06 Yale University Method for identifying and inhibiting functional nucleic acid molecules in cells
US6248525B1 (en) 1998-03-30 2001-06-19 Yale University Method for identifying essential or functional genes
DE60034564T2 (en) 1999-07-07 2007-12-27 Zymogenetics, Inc., Seattle HUMAN CYTOKIN RECEPTOR
US20030153083A1 (en) 2000-01-31 2003-08-14 Alexei Shir Selective killing of cells by activation of double-stranded rna dependent protein kniase-pkr
FR2832154B1 (en) 2001-11-09 2007-03-16 Centre Nat Rech Scient OLIGONUCLEOTIDES INHIBITORS AND THEIR USE FOR SPECIFICALLY REPRESSING A GENE
EP1356820A1 (en) * 2002-04-26 2003-10-29 Institut National De La Sante Et De La Recherche Medicale (Inserm) DNA vaccine combined with an inducer of tumor cell apoptosis

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993023057A1 (en) * 1992-05-14 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting cancer development
WO1995023225A2 (en) * 1994-02-23 1995-08-31 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993023057A1 (en) * 1992-05-14 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting cancer development
WO1995023225A2 (en) * 1994-02-23 1995-08-31 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes

Non-Patent Citations (11)

* Cited by examiner, † Cited by third party
Title
37TH ANNUAL MEETING OF THE AMERICAN SOCIETY OF HEMATOLOGY, SEATTLE, WASHINGTON, USA, DECEMBER 1-5, 1995. *
85TH ANN.MEET. AM.ASSOC. CANCER RES.; SAN FRANCISCO, 10 April 1994 (1994-04-10) - 13 April 1994 (1994-04-13) *
DE THŸ, H. ET AL.: "The PML-RAR alpha fusion mRNA generated by the t(15;17) translocation in acute promylocytic leukemia encods a functionally altered RAR", CELL, vol. 66, 23 August 1991 (1991-08-23), NA US, pages 675 - 684, XP002008608 *
KEYSTONE SYMPOSIUM ON RIBOZYMES: BASIC SCIENCE AND THERAPEUTIC APPLICATIONS, BRECKENRIDGE, COLORADO, USA, 15 January 1995 (1995-01-15) - 21 January 1995 (1995-01-21) *
NASON-BURCHENAL, K. ET AL.: "Over-expression of ribozymes cleaving PML-RAR-alpha in vitro is cytotoxic to acute promyelocytic leukemia cells in vivo.", BLOOD 86 (10 SUPPL. 1). PAGE 263A, ABSTRACT 1037, 15 November 1995 (1995-11-15), XP002008611 *
NORDSTROM, J. ET AL.: "External guide sequences that direct human ribonuclease P to cleave the fusion junction of the PML-RAR-alpha RNA of acute promyelocytic leukemia.", JOURNAL OF CELLULAR BIOCHEMISTRY SUPPLEMENT 0 (19A).1995. PAGE 223, ABSTRACT A6-317, XP002008610 *
PACE, U. ET AL.: "A ribozyme which discriminates in vitro between PML/RAR alpha, the t(15;17)-associated fusion RNA of acute promyelocytic leukemia, and PML and RAR alpha, the transcripts from the nonrearranged alleles.", CANCER RESEARCH, (1994 DEC 15) 54 (24) 6365-9., XP002008609 *
PACE, U. ET AL.: "A ribozyme which discriminates in vitro between the acute promyelocytic leukemia t(15;17) fusion RNA, PML/RAR alpha, and the normal transcripts from the non-rearranged alleles.", PROC ANNU MEET AM ASSOC CANCER RES, VOL. 35, PAGE 206, ABSTRACT #1227, March 1994 (1994-03-01), XP002008607 *
PAOLELLA, G. ET AL.: "NUCLEASE RESISTANT RIBOZYMES WITH HIGH CATALYTIC ACTIVITY", EMBO JOURNAL, vol. 11, no. 5, 1 May 1992 (1992-05-01), pages 1913 - 1919, XP000268586 *
ROSSI, J. ET AL.: "RNA ENZYMES (RIBOZYMES) AS ANTIVIRAL THERAPEUTIC AGENTS", TRENDS IN BIOTECHNOLOGY, vol. 8, no. 7, 1 July 1990 (1990-07-01), pages 179 - 183, XP000133090 *
SYMONS, R. H.: "RIBOZYMES", CURRENT OPINION IN STRUCTURAL BIOLOGY, vol. 4, no. 3, 1 June 1994 (1994-06-01), pages 322 - 330, XP000523747 *

Also Published As

Publication number Publication date
AU4961996A (en) 1996-07-03
WO1996018733A2 (en) 1996-06-20

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