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EP1263765A4 - Modification de comportement cellulaire par modulation antisens de la maturation d'arn messager - Google Patents

Modification de comportement cellulaire par modulation antisens de la maturation d'arn messager

Info

Publication number
EP1263765A4
EP1263765A4 EP00919726A EP00919726A EP1263765A4 EP 1263765 A4 EP1263765 A4 EP 1263765A4 EP 00919726 A EP00919726 A EP 00919726A EP 00919726 A EP00919726 A EP 00919726A EP 1263765 A4 EP1263765 A4 EP 1263765A4
Authority
EP
European Patent Office
Prior art keywords
alteration
mrna processing
antisense modulation
cellular behavior
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.)
Withdrawn
Application number
EP00919726A
Other languages
German (de)
English (en)
Other versions
EP1263765A1 (fr
Inventor
C Frank Bennett
Stanley T Crooke
Muthiah Manoharan
Jacqueline R Wyatt
Brenda F Baker
Brett P Monia
Susan M Freier
Robert Mckay
James G Karras
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.)
Ionis Pharmaceuticals Inc
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 filed Critical
Publication of EP1263765A1 publication Critical patent/EP1263765A1/fr
Publication of EP1263765A4 publication Critical patent/EP1263765A4/fr
Withdrawn legal-status Critical Current

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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
    • 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
    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
    • 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
    • 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

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

Abstract

La présente invention concerne des compositions et des procédés permettant de commander le comportement d'une cellule, d'un tissu ou d'un organisme, par modulation antisens de la maturation d'ARN messager, impliquant l'utilisation de composés antisens qui n'assurent pas le clivage de la cible d'ARN messager.
EP00919726A 2000-03-28 2000-03-28 Modification de comportement cellulaire par modulation antisens de la maturation d'arn messager Withdrawn EP1263765A4 (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PCT/US2000/008174 WO2001072765A1 (fr) 2000-03-28 2000-03-28 Modification de comportement cellulaire par modulation antisens de la maturation d'arn messager

Publications (2)

Publication Number Publication Date
EP1263765A1 EP1263765A1 (fr) 2002-12-11
EP1263765A4 true EP1263765A4 (fr) 2005-06-29

Family

ID=21741209

Family Applications (1)

Application Number Title Priority Date Filing Date
EP00919726A Withdrawn EP1263765A4 (fr) 2000-03-28 2000-03-28 Modification de comportement cellulaire par modulation antisens de la maturation d'arn messager

Country Status (5)

Country Link
EP (1) EP1263765A4 (fr)
JP (1) JP2004509604A (fr)
AU (2) AU2000240366B2 (fr)
CA (1) CA2400573A1 (fr)
WO (1) WO2001072765A1 (fr)

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JP2003533986A (ja) 2000-05-04 2003-11-18 エイブイアイ バイオファーマ， インコーポレイテッド スプライス領域アンチセンス組成物および方法
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7511131B2 (en) 2002-11-13 2009-03-31 Genzyme Corporation Antisense modulation of apolipoprotein B expression
WO2004044181A2 (fr) * 2002-11-13 2004-05-27 Isis Pharmaceuticals, Inc. Modulation antisens de l'expression d'apolipoproteine b
SI1569695T1 (sl) * 2002-11-13 2013-08-30 Genzyme Corporation Protismiselna modulacija ekspresije apolipoproteina B
US7585967B2 (en) * 2003-03-12 2009-09-08 Vasgene Therapeutics, Inc. Nucleic acid compounds for inhibiting angiogenesis and tumor growth
US20050222068A1 (en) 2003-10-23 2005-10-06 Mourich Dan V Method and antisense composition for selective inhibition of HIV infection in hematopoietic cells
JP2007520222A (ja) * 2004-01-20 2007-07-26 アイシス ファーマシューティカルズ インコーポレイテッド グルココルチコイドレセプター発現の調節
WO2005072527A2 (fr) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Oligomeres antisens et methodes destinees a induire la tolerance immunitaire et l'immunosuppression
US20050287558A1 (en) 2004-05-05 2005-12-29 Crooke Rosanne M SNPs of apolipoprotein B and modulation of their expression
EP3808845A1 (fr) 2004-06-28 2021-04-21 The University Of Western Australia Oligonucléotides antisens pour induire l'omission de l'exon et leurs procédés d'utilisation
WO2006086667A2 (fr) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Composition antisens et methode permettant de traiter une atrophie musculaire
AU2007257094B2 (en) 2006-05-05 2012-10-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of SGLT2
JP2010522245A (ja) 2007-03-24 2010-07-01 ゲンザイム コーポレイション ヒトアポリポタンパク質ｂと相補的なアンチセンスオリゴヌクレオチドの投与
WO2009086469A2 (fr) 2007-12-28 2009-07-09 Avi Biopharma, Inc. Agents immunomodulatoires et procédés d'utilisation
CN102203253B (zh) 2008-10-24 2016-04-06 萨雷普塔治疗公司 用于dmd的多外显子跳跃组合物
EP2376633A1 (fr) 2008-12-17 2011-10-19 AVI BioPharma, Inc. Compositions antisens et méthodes de modulation de l'hypersensibilité de contact ou de la dermatite de contact
US9107933B2 (en) 2009-03-16 2015-08-18 Isis Pharmaceuticals, Inc. Compositions and methods of targeting apolipoprotein B for the reduction of apolipoprotein C-III
CA2780563A1 (fr) 2009-11-12 2011-05-19 The University Of Western Australia Molecules antisens et procedes de traitement de pathologies
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
JP6105041B2 (ja) 2012-03-28 2017-03-29 エシコン・エンド−サージェリィ・インコーポレイテッドＥｔｈｉｃｏｎ Ｅｎｄｏ−Ｓｕｒｇｅｒｙ，Ｉｎｃ． 低圧環境を画定するカプセルを含む組織厚コンペンセーター
AU2013203395A1 (en) 2012-03-30 2013-10-17 Isis Pharmaceuticals, Inc. Compositions and methods for modulating TAU expression for reducing seizure and modifying a neurodegenerative syndrome
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
RU2745324C2 (ru) * 2013-03-14 2021-03-23 Ионис Фармасьютикалз, Инк. Композиции и способы модулирования экспрессии tau
KR102258326B1 (ko) 2013-03-14 2021-06-02 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
EA201591792A1 (ru) 2013-03-15 2016-02-29 Сарепта Терапьютикс, Инк. Улучшенные композиции для лечения мышечной дистрофии
JP2016516804A (ja) 2013-04-17 2016-06-09 ファイザー・インク 心血管疾患を治療するためのｎ−ピペリジン−３−イルベンズアミド誘導体
TWI772856B (zh) 2013-07-19 2022-08-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
JP2016534035A (ja) 2013-10-04 2016-11-04 ラナ セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症を治療するための組成物及び方法
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
JOP20190065A1 (ar) 2016-09-29 2019-03-28 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن tau

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WO2000008044A1 (fr) * 1998-08-07 2000-02-17 Isis Pharmaceuticals, Inc. Oligonucleotides 2'-o-aminoethyloxyethyle-modifies

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BAKER B F ET AL: "2'-O-(2-METHOXY)ETHYL-MODIFIED ANTI-INTERCELLULAR ADHESION MOLECULE 1 (ICAM-1) OLIGONUCLEOTIDES SELECTIVELY INCREASE THE ICAM-1 MRNA LEVEL AND INHIBIT FORMATION OF THE ICAM-1 TRANSLATION INITIATION COMPLEX IN HUMAN UMBILICAL VEIN ENDOTHELIAL CELLS", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, US, vol. 272, no. 18, 2 May 1997 (1997-05-02), pages 11994 - 12000, XP002948752, ISSN: 0021-9258 *
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SIERAKOWSKA HALINA ET AL: "Restoration of beta-globin gene expression in mammalian cells by antisense oligonucleotides that modify the aberrant splicing patterns of thalassemic pre-mRNAs", NUCLEOSIDES AND NUCLEOTIDES, vol. 16, no. 7-9, July 1997 (1997-07-01), pages 1173 - 1182, XP008042383, ISSN: 0732-8311 *

Also Published As

Publication number Publication date
JP2004509604A (ja) 2004-04-02
AU4036600A (en) 2001-10-08
WO2001072765A1 (fr) 2001-10-04
AU2000240366B2 (en) 2005-08-04
EP1263765A1 (fr) 2002-12-11
CA2400573A1 (fr) 2001-10-04

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