WO2004016754A3 - ANTISENSE MODULATION OF Nav1.3 EXPRESSION - Google Patents

ANTISENSE MODULATION OF Nav1.3 EXPRESSION Download PDF

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Publication number
WO2004016754A3
WO2004016754A3 PCT/US2003/025465 US0325465W WO2004016754A3 WO 2004016754 A3 WO2004016754 A3 WO 2004016754A3 US 0325465 W US0325465 W US 0325465W WO 2004016754 A3 WO2004016754 A3 WO 2004016754A3
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WO
WIPO (PCT)
Prior art keywords
expression
antisense modulation
methods
compounds
antisense
Prior art date
Application number
PCT/US2003/025465
Other languages
French (fr)
Other versions
WO2004016754A2 (en
Inventor
Steven L Roberds
Original Assignee
Pharmacia Corp
Steven L Roberds
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, Steven L Roberds filed Critical Pharmacia Corp
Priority to EP03749047A priority Critical patent/EP1578927A2/en
Priority to AU2003268096A priority patent/AU2003268096A1/en
Priority to JP2004529403A priority patent/JP2006507808A/en
Priority to MXPA05001836A priority patent/MXPA05001836A/en
Priority to CA002495398A priority patent/CA2495398A1/en
Priority to BRPI0313455-5A priority patent/BR0313455A/en
Publication of WO2004016754A2 publication Critical patent/WO2004016754A2/en
Publication of WO2004016754A3 publication Critical patent/WO2004016754A3/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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/06—Antimigraine agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • 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
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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
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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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/582—Recycling of unreacted starting or intermediate materials

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Genetics & Genomics (AREA)
  • Pain & Pain Management (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Antisense compounds, compositions, and methods are provided for modulating the expression of Type III sodium channel Nav1.3. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding Nav1.3. Methods of using these compounds for modulation of Nav1.3 expression and for treatment of diseases associated with expression of Nav1.3 are provided.
PCT/US2003/025465 2002-08-14 2003-08-14 ANTISENSE MODULATION OF Nav1.3 EXPRESSION WO2004016754A2 (en)

Priority Applications (6)

Application Number Priority Date Filing Date Title
EP03749047A EP1578927A2 (en) 2002-08-14 2003-08-14 Antisense modulation of nav1.3 expression
AU2003268096A AU2003268096A1 (en) 2002-08-14 2003-08-14 ANTISENSE MODULATION OF Nav1.3 EXPRESSION
JP2004529403A JP2006507808A (en) 2002-08-14 2003-08-14 Antisense regulation of Nav1.3 expression
MXPA05001836A MXPA05001836A (en) 2002-08-14 2003-08-14 ANTISENSE MODULATION OF Nav1.3 EXPRESSION.
CA002495398A CA2495398A1 (en) 2002-08-14 2003-08-14 Antisense modulation of nav1.3 expression
BRPI0313455-5A BR0313455A (en) 2002-08-14 2003-08-14 "antisense" modulation of nav1.3 expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US40341602P 2002-08-14 2002-08-14
US60/403,416 2002-08-14

Publications (2)

Publication Number Publication Date
WO2004016754A2 WO2004016754A2 (en) 2004-02-26
WO2004016754A3 true WO2004016754A3 (en) 2005-11-10

Family

ID=31888234

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2003/025465 WO2004016754A2 (en) 2002-08-14 2003-08-14 ANTISENSE MODULATION OF Nav1.3 EXPRESSION

Country Status (7)

Country Link
EP (1) EP1578927A2 (en)
JP (1) JP2006507808A (en)
AU (1) AU2003268096A1 (en)
BR (1) BR0313455A (en)
CA (1) CA2495398A1 (en)
MX (1) MXPA05001836A (en)
WO (1) WO2004016754A2 (en)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102292453A (en) * 2008-10-03 2011-12-21 普罗卡塔生物系统有限公司 Transcription factor decoys
US11852627B2 (en) 2015-11-12 2023-12-26 Hoffmann-La Roche Inc. Oligonucleotides for inducing paternal UBE3A expression

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8258105B2 (en) 2003-10-07 2012-09-04 Isis Pharmaceuticals, Inc. Antisense oligonucleotides optimized for kidney targeting
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
GB0719367D0 (en) 2007-10-03 2007-11-14 Procarta Biosystems Ltd Transcription factor decoys, compositions and methods
US20110059895A1 (en) * 2007-11-09 2011-03-10 Isis Pharmaceuticals, Inc. Modulation of factor 9 expression
JP2011502514A (en) 2007-11-09 2011-01-27 アイシス ファーマシューティカルズ インコーポレイティッド Regulation of factor 7 expression
EP2515947B1 (en) * 2009-12-23 2021-10-06 CuRNA, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
GB201002413D0 (en) 2010-02-12 2010-03-31 Procarta Biosystems Ltd Nucleic acid complexes
GB201005545D0 (en) 2010-04-01 2010-05-19 Procarta Biosystems Ltd Transcription factor decoys
PL2585596T3 (en) 2010-06-23 2021-06-28 Curna, Inc. Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
ES2688831T3 (en) 2012-06-25 2018-11-07 Ionis Pharmaceuticals, Inc. UBE3A-ATS expression modulation
EP2931893A1 (en) * 2012-12-13 2015-10-21 Universität Leipzig T-cell modulation by exon skipping
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
TWI809004B (en) * 2017-11-09 2023-07-21 美商Ionis製藥公司 Compounds and methods for reducing snca expression
EA202190581A1 (en) * 2018-08-20 2021-07-13 Рогкон, Инк. ANTI-SENSE OLIGONUCLEOTIDES TARGETING SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHY
JP7550165B2 (en) 2019-03-29 2024-09-12 アイオーニス ファーマシューティカルズ， インコーポレーテッド Compounds and methods for modulating UBE3A-ATS

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6124133A (en) * 1999-10-15 2000-09-26 Isis Pharmaceuticals Inc. Antisense inhibition of fra-1 expression
US6610539B1 (en) * 1997-07-10 2003-08-26 Genesense Technologies, Inc. Antisense oligonucleotide sequences as inhibitors of microorganisms
US20040170608A1 (en) * 2001-09-14 2004-09-02 Rob Barber Use of a lentiviral vector in the treatment of pain

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6610539B1 (en) * 1997-07-10 2003-08-26 Genesense Technologies, Inc. Antisense oligonucleotide sequences as inhibitors of microorganisms
US6124133A (en) * 1999-10-15 2000-09-26 Isis Pharmaceuticals Inc. Antisense inhibition of fra-1 expression
US20040170608A1 (en) * 2001-09-14 2004-09-02 Rob Barber Use of a lentiviral vector in the treatment of pain

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
BAKER M.D. AND WOOD J.N. ET AL: "Involvement of Na+channels in pain pathways.", TRENDS IN PHARMACOLOGICAL SCIENCE., vol. 22, no. 1, January 2001 (2001-01-01), pages 27 - 31, XP002990543 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102292453A (en) * 2008-10-03 2011-12-21 普罗卡塔生物系统有限公司 Transcription factor decoys
US11852627B2 (en) 2015-11-12 2023-12-26 Hoffmann-La Roche Inc. Oligonucleotides for inducing paternal UBE3A expression

Also Published As

Publication number Publication date
AU2003268096A1 (en) 2004-03-03
WO2004016754A2 (en) 2004-02-26
AU2003268096A8 (en) 2004-03-03
BR0313455A (en) 2007-07-31
CA2495398A1 (en) 2004-02-26
EP1578927A2 (en) 2005-09-28
MXPA05001836A (en) 2005-04-19
JP2006507808A (en) 2006-03-09

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