WO2001059102A3 - Nucleozymes with endonuclease activity - Google Patents

Nucleozymes with endonuclease activity Download PDF

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Publication number
WO2001059102A3
WO2001059102A3 PCT/US2001/004223 US0104223W WO0159102A3 WO 2001059102 A3 WO2001059102 A3 WO 2001059102A3 US 0104223 W US0104223 W US 0104223W WO 0159102 A3 WO0159102 A3 WO 0159102A3
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WO
WIPO (PCT)
Prior art keywords
nucleozymes
endonuclease activity
dna
synthesis
nucleic acid
Prior art date
Application number
PCT/US2001/004223
Other languages
French (fr)
Other versions
WO2001059102A2 (en
Inventor
Ronald Breaker
Leonid Beigelman
Gail Emilsson
Original Assignee
Ronald Breaker
Leonid Beigelman
Gail Emilsson
Ribozyme Pharm Inc
Univ Yale
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 Ronald Breaker, Leonid Beigelman, Gail Emilsson, Ribozyme Pharm Inc, Univ Yale filed Critical Ronald Breaker
Priority to EP01907168A priority Critical patent/EP1257639A2/en
Priority to CA002398750A priority patent/CA2398750A1/en
Priority to AU34977/01A priority patent/AU3497701A/en
Priority to JP2001558240A priority patent/JP2003521943A/en
Publication of WO2001059102A2 publication Critical patent/WO2001059102A2/en
Publication of WO2001059102A3 publication Critical patent/WO2001059102A3/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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/332—Abasic residue

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The present invention relates to enzymatic nucleic acid molecules, including ribozymes and nucleozymes (DNA catalysts, DNA enzymes), methods of synthesis, and uses thereof.
PCT/US2001/004223 2000-02-08 2001-02-08 Nucleozymes with endonuclease activity WO2001059102A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
EP01907168A EP1257639A2 (en) 2000-02-08 2001-02-08 Nucleozymes with endonuclease activity
CA002398750A CA2398750A1 (en) 2000-02-08 2001-02-08 Nucleozymes with endonuclease activity
AU34977/01A AU3497701A (en) 2000-02-08 2001-02-08 Nucleozymes with endonuclease activity
JP2001558240A JP2003521943A (en) 2000-02-08 2001-02-08 Nucleozymes having endonuclease activity

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US18136000P 2000-02-08 2000-02-08
US60/181,360 2000-02-08
US19364600P 2000-03-31 2000-03-31
US60/193,646 2000-03-31

Publications (2)

Publication Number Publication Date
WO2001059102A2 WO2001059102A2 (en) 2001-08-16
WO2001059102A3 true WO2001059102A3 (en) 2002-04-25

Family

ID=26877117

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/004223 WO2001059102A2 (en) 2000-02-08 2001-02-08 Nucleozymes with endonuclease activity

Country Status (5)

Country Link
EP (1) EP1257639A2 (en)
JP (1) JP2003521943A (en)
AU (1) AU3497701A (en)
CA (1) CA2398750A1 (en)
WO (1) WO2001059102A2 (en)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6506887B1 (en) 1999-07-29 2003-01-14 Somalogic, Incorporated Conditional-selex
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
JP2015509922A (en) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． RNA regulatory oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
US9856480B2 (en) * 2013-01-14 2018-01-02 National Taiwan University Dnazyme for silencing the expression of EGFR
CN107709555A (en) * 2015-05-15 2018-02-16 达尔马科恩有限公司 The unidirectional of synthesis for the gene editing of Cas9 mediations leads RNA

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999016871A2 (en) * 1997-09-22 1999-04-08 Max-Planck-Gesellschaft Zur Forderung Der Wissensc Nucleic acid catalysts with endonuclease activity
WO1999054459A2 (en) * 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999016871A2 (en) * 1997-09-22 1999-04-08 Max-Planck-Gesellschaft Zur Forderung Der Wissensc Nucleic acid catalysts with endonuclease activity
WO1999054459A2 (en) * 1998-04-20 1999-10-28 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
BREAKER R R: "IN VITRO SELECTION OF CATALYTIC POLYNUCLEOTIDES", CHEMICAL REVIEWS, vol. 97, no. 2, 1 March 1997 (1997-03-01), pages 371 - 390, XP000683274, ISSN: 0009-2665 *
BREAKER, R.: "In vitro selection of self-cleaving ribozymes and deoxyribozymes", 1999, HORIZON SCIENTIFIC PRESS, 'INTRACELLULAR RIBOZYME APPLICATIONS: PRINCIPLES AND PROTOCOLS'; CHAPTER 1, PAGES 1-19; WYMONDHAM, GB, XP002178935 *

Also Published As

Publication number Publication date
EP1257639A2 (en) 2002-11-20
CA2398750A1 (en) 2001-08-16
JP2003521943A (en) 2003-07-22
WO2001059102A2 (en) 2001-08-16
AU3497701A (en) 2001-08-20

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