[go: up one dir, main page]

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

ANTISENSE MODULATION OF Nav1.3 EXPRESSION Download PDF

Info

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
Authority
WO
WIPO (PCT)
Prior art keywords
expression
antisense modulation
methods
compounds
antisense
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/US2003/025465
Other languages
French (fr)
Other versions
WO2004016754A2 (en
Inventor
Steven L Roberds
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.)
Pharmacia LLC
Original Assignee
Pharmacia LLC
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 LLC filed Critical Pharmacia LLC
Priority to EP03749047A priority Critical patent/EP1578927A2/en
Priority to BRPI0313455-5A priority patent/BR0313455A/en
Priority to JP2004529403A priority patent/JP2006507808A/en
Priority to AU2003268096A priority patent/AU2003268096A1/en
Priority to CA002495398A priority patent/CA2495398A1/en
Priority to MXPA05001836A priority patent/MXPA05001836A/en
Publication of WO2004016754A2 publication Critical patent/WO2004016754A2/en
Anticipated expiration legal-status Critical
Publication of WO2004016754A3 publication Critical patent/WO2004016754A3/en
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • C12N2310/334—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Genetics & Genomics (AREA)
  • Pain & Pain Management (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (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 Ceased 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
BRPI0313455-5A BR0313455A (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
AU2003268096A AU2003268096A1 (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
MXPA05001836A MXPA05001836A (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 Ceased 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 (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1687410A4 (en) 2003-10-07 2008-04-09 Isis Pharmaceuticals Inc ANTISENSE OLIGONUCLEOTIDES OPTIMIZED TO TARGET THE KIDNEY
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
NZ585250A (en) * 2007-11-09 2012-06-29 Isis Pharmaceuticals Inc Antisense modulation of factor 7 expression
US20110059895A1 (en) * 2007-11-09 2011-03-10 Isis Pharmaceuticals, Inc. Modulation of factor 9 expression
KR101793753B1 (en) * 2009-12-23 2017-11-03 큐알엔에이, 인크. 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
JP6023705B2 (en) 2010-06-23 2016-11-09 カッパーアールエヌエー，インコーポレイテッド Treatment of SCNA-related diseases by inhibition of natural antisense transcripts on sodium channels, voltage-gated, alpha subunit (SCNA)
DK2864479T3 (en) 2012-06-25 2018-10-22 Ionis Pharmaceuticals Inc MODULATION OF UBE3A-ATS EXPRESSION
WO2014090985A1 (en) * 2012-12-13 2014-06-19 Universität Leipzig T-cell modulation by exon skipping
US10400243B2 (en) 2014-11-25 2019-09-03 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
CA3110211A1 (en) * 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
CN117431244A (en) 2019-03-29 2024-01-23 Ionis制药公司 Compounds and methods for modulating UBE3A-ATS
CA3186935A1 (en) 2020-08-07 2022-02-10 Paymaan JAFAR-NEJAD Compounds and methods for modulating scn2a
CA3195722A1 (en) * 2020-09-17 2022-03-24 Q-State Biosciences, Inc. Therapeutic compositions for treating pain via multiple targets

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

Similar Documents

Publication Publication Date Title
WO2004044181A3 (en) Antisense modulation of apolipoprotein b expression
WO2002036743A3 (en) Antisense modulation of calreticulin expression
WO2005049630A3 (en) Antisense modulation of kinesin-like 1 expression
WO2003011887A3 (en) Antisense modulation of apolipoprotein b expression
AU2001290706A1 (en) Antisense modulation of clusterin expression
WO2002092772A3 (en) Antisense modulation of ptp1b expression
WO2003097662A8 (en) Antisense modulation of apolipoprotein b expression
WO2004016754A3 (en) ANTISENSE MODULATION OF Nav1.3 EXPRESSION
WO2003008543A3 (en) Antisense modulation of bcl2-associated x protein expression
WO2003046132A3 (en) Antisense modulation of myd88 expression
WO2003053341A3 (en) Antisense modulation of ship-1 expression
WO2001083513A3 (en) Antisense modulation of inhibitor of dna binding-1 expression
WO2002095053A3 (en) Antisense modulation of src-c expression
WO2002036810A3 (en) Antisense modulation of talin expression
WO2003028636A3 (en) Antisense modulation of il-1 receptor-associated kinase-4 expression
WO2003052062A3 (en) Antisense modulation of cd36l1 expression
WO2003099224A3 (en) Antisense moodulation of kinesin-like 1 expression
WO2003027229A3 (en) Antisense modulation of rip2 expression
WO2003050247A3 (en) Antisense modulation of mhc class ii transactivator expression
WO2004003201A3 (en) Antisense modulation of lrh1 expression
WO2003031576A3 (en) Antisense modulation of inhibitor-kappa b kinase-gamma expression
WO2004014299A3 (en) Antisense modulation of resistin expression
WO2003033659A3 (en) Antisense modulation of matrix metalloproteinase 1 expression
WO2003099204A3 (en) Antisense modulation of inhibitor-kappa b kinase-beta expression
WO2002055535A3 (en) Antisense modulation of cytohesin-1 expression

Legal Events

Date Code Title Description
AK Designated states

Kind code of ref document: A2

Designated state(s): AE AG AL AM AT AU AZ BA BB BG BR BY BZ CA CH CN CO CR CU CZ DE DK DM DZ EC EE ES FI GB GD GE GH GM HR HU ID IL IN IS JP KE KG KP KR KZ LC LK LR LS LT LU LV MA MD MG MK MN MW MX MZ NI NO NZ OM PG PH PL PT RO RU SC SD SE SG SK SL SY TJ TM TN TR TT TZ UA UG US UZ VC VN YU ZA ZM ZW

AL Designated countries for regional patents

Kind code of ref document: A2

Designated state(s): GH GM KE LS MW MZ SD SL SZ TZ UG ZM ZW AM AZ BY KG KZ MD RU TJ TM AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HU IE IT LU MC NL PT RO SE SI SK TR BF BJ CF CG CI CM GA GN GQ GW ML MR NE SN TD TG

121 Ep: the epo has been informed by wipo that ep was designated in this application
ENP Entry into the national phase

Ref document number: 2495398

Country of ref document: CA

WWE Wipo information: entry into national phase

Ref document number: PA/a/2005/001836

Country of ref document: MX

Ref document number: 2004529403

Country of ref document: JP

WWE Wipo information: entry into national phase

Ref document number: 2003749047

Country of ref document: EP

DFPE Request for preliminary examination filed prior to expiration of 19th month from priority date (pct application filed before 20040101)
WWP Wipo information: published in national office

Ref document number: 2003749047

Country of ref document: EP

WWW Wipo information: withdrawn in national office

Ref document number: 2003749047

Country of ref document: EP

ENP Entry into the national phase

Ref document number: PI0313455

Country of ref document: BR