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WO1998050530A3 - Enzymatic nucleic acids: synthesis, selection and use - Google Patents

Enzymatic nucleic acids: synthesis, selection and use Download PDF

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Publication number
WO1998050530A3
WO1998050530A3 PCT/US1998/009249 US9809249W WO9850530A3 WO 1998050530 A3 WO1998050530 A3 WO 1998050530A3 US 9809249 W US9809249 W US 9809249W WO 9850530 A3 WO9850530 A3 WO 9850530A3
Authority
WO
WIPO (PCT)
Prior art keywords
synthesis
selection
nucleic acids
enzymatic nucleic
nucleic acid
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US1998/009249
Other languages
French (fr)
Other versions
WO1998050530A2 (en
WO1998050530A9 (en
Inventor
Thale Jarvis
Jasenka Matulic-Adamic
Mark Reynolds
Kevin Kisich
Laurent Bellon
Tom Parry
Leonid Beigelman
James A Mcswiggen
Alexander Karpeisky
Alex Burgin
James Thompson
Christopher T Workman
Amber Beaudry
David Sweedler
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Ribozyme Pharmaceuticals 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 Ribozyme Pharmaceuticals Inc filed Critical Ribozyme Pharmaceuticals Inc
Priority to CA002288640A priority Critical patent/CA2288640A1/en
Priority to EP98920299A priority patent/EP0980424A2/en
Priority to JP54844898A priority patent/JP2001525667A/en
Priority to AU72905/98A priority patent/AU749561B2/en
Publication of WO1998050530A2 publication Critical patent/WO1998050530A2/en
Publication of WO1998050530A9 publication Critical patent/WO1998050530A9/en
Publication of WO1998050530A3 publication Critical patent/WO1998050530A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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 against oncogenes or tumor suppressor genes
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/06Antipsoriatics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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 against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/02Pentosyltransferases (2.4.2)
    • C12Y204/02001Purine-nucleoside phosphorylase (2.4.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/01Phosphotransferases with an alcohol group as acceptor (2.7.1)
    • C12Y207/01037Protein kinase (2.7.1.37)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Rheumatology (AREA)
  • Pain & Pain Management (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Nucleic acid catalysts which modulate the expression of Raf gene; method of delivery, screening, identification, synthesis, deprotection, purification, of nucleic acid catalysts and processes for identification of nucleic acid molecules is described.
PCT/US1998/009249 1997-05-09 1998-05-05 Enzymatic nucleic acids: synthesis, selection and use Ceased WO1998050530A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
CA002288640A CA2288640A1 (en) 1997-05-09 1998-05-05 Enzymatic nucleic acids: synthesis, selection and use
EP98920299A EP0980424A2 (en) 1997-05-09 1998-05-05 Enzymatic nucleic acids: synthesis, selection and use
JP54844898A JP2001525667A (en) 1997-05-09 1998-05-05 Enzymatic nucleic acid treatment of diseases or conditions related to the expression level of C-RAF
AU72905/98A AU749561B2 (en) 1997-05-09 1998-05-05 Nucleic acid molecules having endonuclease and/or catalytic activity

Applications Claiming Priority (16)

Application Number Priority Date Filing Date Title
US4605997P 1997-05-09 1997-05-09
US4900297P 1997-06-09 1997-06-09
US5171897P 1997-07-03 1997-07-03
US5680897P 1997-08-22 1997-08-22
US6132197P 1997-10-02 1997-10-02
US6132497P 1997-10-02 1997-10-02
US6486697P 1997-11-05 1997-11-05
US6821297P 1997-12-19 1997-12-19
US60/068,212 1997-12-19
US60/049,002 1997-12-19
US60/046,059 1997-12-19
US60/051,718 1997-12-19
US60/061,324 1997-12-19
US60/056,808 1997-12-19
US60/064,866 1997-12-19
US60/061,321 1997-12-19

Publications (3)

Publication Number Publication Date
WO1998050530A2 WO1998050530A2 (en) 1998-11-12
WO1998050530A9 WO1998050530A9 (en) 1999-03-25
WO1998050530A3 true WO1998050530A3 (en) 1999-07-29

Family

ID=27574318

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1998/009249 Ceased WO1998050530A2 (en) 1997-05-09 1998-05-05 Enzymatic nucleic acids: synthesis, selection and use

Country Status (5)

Country Link
EP (1) EP0980424A2 (en)
JP (1) JP2001525667A (en)
AU (1) AU749561B2 (en)
CA (1) CA2288640A1 (en)
WO (1) WO1998050530A2 (en)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6602713B1 (en) * 2001-02-09 2003-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of protein phosphatase 2 catalytic subunit beta expression
WO1999032618A1 (en) * 1997-12-19 1999-07-01 Strata Biosciences Incorporated Non-bacterial cloning in delivery and expression of nucleic acids
DE69933856D1 (en) 1998-02-13 2006-12-14 Koester Hubert USE OF RIBOZYMES FOR DETERMINING THE FUNCTION OF GENES
EP1493818A3 (en) * 1998-04-29 2006-02-15 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
EP1073732A2 (en) * 1998-04-29 2001-02-07 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
JP4719356B2 (en) 1998-07-23 2011-07-06 サザン・リサーチ・インスティテュート Anticancer agent and DNA replication inhibitor
AUPP795198A0 (en) * 1998-12-24 1999-01-28 Commonwealth Scientific And Industrial Research Organisation Miniribozymes active at low magnesium ion concentrations
US7125660B2 (en) 2000-09-13 2006-10-24 Archemix Corp. Nucleic acid sensor molecules and methods of using same
US6566135B1 (en) * 2000-10-04 2003-05-20 Isis Pharmaceuticals, Inc. Antisense modulation of caspase 6 expression
CN101321775B (en) 2005-10-03 2012-05-23 大学健康网络 ODCASE inhibitors for the treatment of malaria
US20100056468A1 (en) * 2007-01-08 2010-03-04 University Health Network Pyrimidine Derivatives As Anticancer Agents
WO2008109365A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting raf1 gene expression and uses thereof
US20090318380A1 (en) * 2007-11-20 2009-12-24 Pharmasset, Inc. 2',4'-substituted nucleosides as antiviral agents
WO2009143372A2 (en) * 2008-05-21 2009-11-26 Intradigm Corporation Compositions comprising a-raf, b-raf, and c-raf sirna and methods of use thereof
JP7016511B2 (en) * 2017-06-20 2022-02-07 学校法人甲南学園 Nucleic acid synthesis method

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993023569A1 (en) * 1992-05-11 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
DE4424762C1 (en) * 1994-07-04 1995-07-27 Max Planck Gesellschaft Ribozyme library in optimised expression cassette
WO1995023225A2 (en) * 1994-02-23 1995-08-31 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes
WO1996018736A2 (en) * 1994-12-13 1996-06-20 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
US5616459A (en) * 1990-07-16 1997-04-01 The Public Health Research Institute Of The City Of New York, Inc. Selection of ribozymes that efficiently cleave target RNA
WO1998032880A1 (en) * 1997-01-23 1998-07-30 Immusol Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5616459A (en) * 1990-07-16 1997-04-01 The Public Health Research Institute Of The City Of New York, Inc. Selection of ribozymes that efficiently cleave target RNA
WO1993023569A1 (en) * 1992-05-11 1993-11-25 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
WO1995023225A2 (en) * 1994-02-23 1995-08-31 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes
DE4424762C1 (en) * 1994-07-04 1995-07-27 Max Planck Gesellschaft Ribozyme library in optimised expression cassette
WO1996018736A2 (en) * 1994-12-13 1996-06-20 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of arthritic conditions, induction of graft tolerance and reversal of immune responses
WO1998032880A1 (en) * 1997-01-23 1998-07-30 Immusol Incorporated Gene functional analysis and discovery using randomized or target-specific ribozyme gene vector libraries

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
COTTEN M: "THE IN VIVO APPLICATION OF RIBOZYMES", TRENDS IN BIOTECHNOLOGY., vol. 8, no. 7, 1 July 1990 (1990-07-01), CAMBRIDGE GB, pages 174 - 178, XP000133091 *
JARVIS T C ET AL: "INHIBITION OF VASCULAR SMOOTH MUSCLE CELL PROLIFERATION BY HAMMERHEAD RIBOZYMES TARGETTING C-MYB", JOURNAL OF CELLULAR BIOCHEMISTRY., vol. 19A, 1995, US, pages 221, XP002024063 *

Also Published As

Publication number Publication date
WO1998050530A2 (en) 1998-11-12
CA2288640A1 (en) 1998-11-12
EP0980424A2 (en) 2000-02-23
AU749561B2 (en) 2002-06-27
AU7290598A (en) 1998-11-27
WO1998050530A9 (en) 1999-03-25
JP2001525667A (en) 2001-12-11

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