WO2004003201A3 - Antisense modulation of lrh1 expression - Google Patents

Antisense modulation of lrh1 expression Download PDF

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Publication number
WO2004003201A3
WO2004003201A3 PCT/US2003/020865 US0320865W WO2004003201A3 WO 2004003201 A3 WO2004003201 A3 WO 2004003201A3 US 0320865 W US0320865 W US 0320865W WO 2004003201 A3 WO2004003201 A3 WO 2004003201A3
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WO
WIPO (PCT)
Prior art keywords
lrh1
expression
antisense modulation
methods
compounds
Prior art date
Application number
PCT/US2003/020865
Other languages
French (fr)
Other versions
WO2004003201A2 (en
Inventor
Christopher D Kane
Original Assignee
Pharmacia Corp
Christopher D Kane
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 Pharmacia Corp, Christopher D Kane filed Critical Pharmacia Corp
Priority to JP2004518213A priority Critical patent/JP2005532052A/en
Priority to EP03762317A priority patent/EP1520021A2/en
Publication of WO2004003201A2 publication Critical patent/WO2004003201A2/en
Publication of WO2004003201A3 publication Critical patent/WO2004003201A3/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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N2310/30—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
    • C12N2310/32—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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Genetics & Genomics (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Urology & Nephrology (AREA)
  • Communicable Diseases (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Cardiology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Vascular Medicine (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Biochemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Antisense compounds, compositions and methods are provided for modulating the expression of Liver Related Homolog-1 (LRH1). The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding LRH1. Methods of using these compounds for modulation of LRH1 expression and for treatment of diseases associated with expression of LRH1 are provided.
PCT/US2003/020865 2002-07-01 2003-07-01 Antisense modulation of lrh1 expression WO2004003201A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2004518213A JP2005532052A (en) 2002-07-01 2003-07-01 Antisense regulation of LRH1 expression
EP03762317A EP1520021A2 (en) 2002-07-01 2003-07-01 Antisense modulation of lrh1 expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US39281302P 2002-07-01 2002-07-01
US60/392,813 2002-07-01

Publications (2)

Publication Number Publication Date
WO2004003201A2 WO2004003201A2 (en) 2004-01-08
WO2004003201A3 true WO2004003201A3 (en) 2004-04-22

Family

ID=30000939

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/020865 WO2004003201A2 (en) 2002-07-01 2003-07-01 Antisense modulation of lrh1 expression

Country Status (3)

Country Link
EP (1) EP1520021A2 (en)
JP (1) JP2005532052A (en)
WO (1) WO2004003201A2 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101802536B1 (en) 2009-08-05 2017-11-28 큐알엔에이, 인크. Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
ES2890677T3 (en) 2014-03-19 2022-01-21 Ionis Pharmaceuticals Inc Compositions for Modulating Ataxin 2 Expression
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
CN113645998A (en) 2019-03-26 2021-11-12 富士胶片株式会社 Pharmaceutical composition for inhibiting hepatitis B virus protein production and screening method
CN111334507B (en) * 2019-12-24 2024-01-30 江苏农牧科技职业学院 Sheep Lrh-1 short hairpin RNA and interference vector thereof

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999029727A1 (en) * 1997-12-08 1999-06-17 Tularik Inc. Cyp7 promoter-binding factors
WO2002010769A2 (en) * 2000-07-31 2002-02-07 Glaxo Group Limited Identification of new therapeutic targets for modulating bile acid synthesis
WO2002022635A1 (en) * 2000-09-11 2002-03-21 Isis Pharmaceuticals, Inc. Antisense modulation of clusterin expression

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999029727A1 (en) * 1997-12-08 1999-06-17 Tularik Inc. Cyp7 promoter-binding factors
WO2002010769A2 (en) * 2000-07-31 2002-02-07 Glaxo Group Limited Identification of new therapeutic targets for modulating bile acid synthesis
WO2002022635A1 (en) * 2000-09-11 2002-03-21 Isis Pharmaceuticals, Inc. Antisense modulation of clusterin expression

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ALTMANN K-H ET AL: "NOVEL CHEMISTRY", APPLIED ANTISENSE OLIGONUCLEOTIDE TECHNOLOGY, NEW YORK, WILEY-LISS, US, 1998, pages 73 - 107, XP002119324, ISBN: 0-471-17279-0 *
LI M ET AL: "CLONING AND CHARACTERIZATION OF A NOVEL HUMAN HEPATOCYTE TRANSCRIPTION FACTOR, HBIF, WHICH BINDS AND ACTIVATES ENHANCER II OF HEPATITIS B VIRUS", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 273, no. 44, 30 October 1998 (1998-10-30), pages 29022 - 29031, XP000941573, ISSN: 0021-9258 *
LU TIMOTHY T ET AL: "Molecular basis for feedback regulation of bile acid synthesis by nuclear receptors", MOLECULAR CELL, vol. 6, no. 3, September 2000 (2000-09-01), pages 507 - 515, XP002209572, ISSN: 1097-2765 *

Also Published As

Publication number Publication date
WO2004003201A2 (en) 2004-01-08
EP1520021A2 (en) 2005-04-06
JP2005532052A (en) 2005-10-27

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