WO2001062911A3 - Antisense and catalytically acting nucleic acid molecules targeted to grb2- related with insert domain (grid) proteins and their uses - Google Patents

Antisense and catalytically acting nucleic acid molecules targeted to grb2- related with insert domain (grid) proteins and their uses Download PDF

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Publication number
WO2001062911A3
WO2001062911A3 PCT/US2001/005957 US0105957W WO0162911A3 WO 2001062911 A3 WO2001062911 A3 WO 2001062911A3 US 0105957 W US0105957 W US 0105957W WO 0162911 A3 WO0162911 A3 WO 0162911A3
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WO
WIPO (PCT)
Prior art keywords
nucleic acid
antisense
acid molecules
grb2
grid
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Application number
PCT/US2001/005957
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French (fr)
Other versions
WO2001062911A2 (en
Inventor
Thale Jarvis
Carlowitz Ira Von
James A Mcswiggen
Paul Andrew Hamblin
Jonathon Henry Ellis
Original Assignee
Ribozyme Pharm Inc
Glaxo Group Ltd
Thale Jarvis
Carlowitz Ira Von
James A Mcswiggen
Paul Andrew Hamblin
Jonathon Henry Ellis
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 Pharm Inc, Glaxo Group Ltd, Thale Jarvis, Carlowitz Ira Von, James A Mcswiggen, Paul Andrew Hamblin, Jonathon Henry Ellis filed Critical Ribozyme Pharm Inc
Priority to AU39862/01A priority Critical patent/AU3986201A/en
Priority to CA002397813A priority patent/CA2397813A1/en
Publication of WO2001062911A2 publication Critical patent/WO2001062911A2/en
Publication of WO2001062911A3 publication Critical patent/WO2001062911A3/en

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    • 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
    • A61K31/7105—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
    • A61K31/712—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • 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
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    • 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
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    • 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
    • C12N2310/10—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—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
    • C12N2310/32—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
    • C12N2310/33—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)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Transplantation (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

The present invention relates to nucleic acid molecules, including antisense and enzymatic nucleic acid molecules, such as hammerhead ribozymes, DNAzymes, and antisense, which modulate the expression of the GRID (Grb2-related with Insert Domain) gene.
PCT/US2001/005957 2000-02-24 2001-02-23 Antisense and catalytically acting nucleic acid molecules targeted to grb2- related with insert domain (grid) proteins and their uses WO2001062911A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU39862/01A AU3986201A (en) 2000-02-24 2001-02-23 Method and reagent for the inhibition of grid
CA002397813A CA2397813A1 (en) 2000-02-24 2001-02-23 Method and reagent for the inhibition of grid

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US18459400P 2000-02-24 2000-02-24
US60/184,594 2000-02-24

Publications (2)

Publication Number Publication Date
WO2001062911A2 WO2001062911A2 (en) 2001-08-30
WO2001062911A3 true WO2001062911A3 (en) 2002-02-07

Family

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PCT/US2001/005957 WO2001062911A2 (en) 2000-02-24 2001-02-23 Antisense and catalytically acting nucleic acid molecules targeted to grb2- related with insert domain (grid) proteins and their uses

Country Status (3)

Country Link
AU (1) AU3986201A (en)
CA (1) CA2397813A1 (en)
WO (1) WO2001062911A2 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1833966A2 (en) * 2004-12-14 2007-09-19 National Institute of Immunology Dnazymes for inhibition of japanese encephalitis virus replication
CN109971836A (en) * 2017-12-28 2019-07-05 上海细胞治疗研究院 The method and kit of double fluorescent quantitative PCR measurement CAR copy number
CN109971837A (en) * 2017-12-28 2019-07-05 上海细胞治疗研究院 A kind of detection method and kit of exogenous antibodies gene copy number

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995011304A1 (en) * 1993-10-18 1995-04-27 Ribozyme Pharmaceuticals, Inc. Catalytic dna
WO1997026270A2 (en) * 1996-01-16 1997-07-24 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
WO1998001547A1 (en) * 1996-07-08 1998-01-15 The Board Of Regents, The University Of Texas System INHIBITION OF CHRONIC MYELOGENOUS LEUKEMIC CELL GROWTH BY LIPOSOMAL-ANTISENSE OLIGODEOXY-NUCLEOTIDES TARGETING TO Grb2 OR Crk1
WO1998032846A2 (en) * 1997-01-23 1998-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of c-fos
WO1998040482A1 (en) * 1997-03-11 1998-09-17 Incyte Pharmaceuticals, Inc. Growth factor receptor binding protein
WO1999055857A2 (en) * 1998-04-29 1999-11-04 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
WO2000011160A1 (en) * 1998-08-19 2000-03-02 Glaxo Group Limited Grip, human adapter protein related to the grb2 family member

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1995011304A1 (en) * 1993-10-18 1995-04-27 Ribozyme Pharmaceuticals, Inc. Catalytic dna
WO1997026270A2 (en) * 1996-01-16 1997-07-24 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
WO1998001547A1 (en) * 1996-07-08 1998-01-15 The Board Of Regents, The University Of Texas System INHIBITION OF CHRONIC MYELOGENOUS LEUKEMIC CELL GROWTH BY LIPOSOMAL-ANTISENSE OLIGODEOXY-NUCLEOTIDES TARGETING TO Grb2 OR Crk1
WO1998032846A2 (en) * 1997-01-23 1998-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of c-fos
WO1998040482A1 (en) * 1997-03-11 1998-09-17 Incyte Pharmaceuticals, Inc. Growth factor receptor binding protein
WO1999055857A2 (en) * 1998-04-29 1999-11-04 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
WO2000011160A1 (en) * 1998-08-19 2000-03-02 Glaxo Group Limited Grip, human adapter protein related to the grb2 family member

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
LUDWIG J ET AL: "Extending the cleavage rules for the hammerhead ribozyme: mutating adenosine15.1 to inosine15.1 changes the cleavage site specificity from N16.2U16.1H17 to N16.2C16.1H17", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 26, no. 10, 15 May 1998 (1998-05-15), pages 2279 - 2285, XP002084035, ISSN: 0305-1048 *
VAISH ET AL: "In vitro selection of a purine nucleotide-specific hammerhead-like ribozyme", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE. WASHINGTON, US, vol. 95, no. 5, 1 March 1998 (1998-03-01), pages 2158 - 2162, XP002097465, ISSN: 0027-8424 *

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CA2397813A1 (en) 2001-08-30
AU3986201A (en) 2001-09-03
WO2001062911A2 (en) 2001-08-30

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