CA2385853A1 - Conception d'un oligonucleotide de recrutement de rnase h a haute affinite - Google Patents

Conception d'un oligonucleotide de recrutement de rnase h a haute affinite Download PDF

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Publication number
CA2385853A1
CA2385853A1 CA002385853A CA2385853A CA2385853A1 CA 2385853 A1 CA2385853 A1 CA 2385853A1 CA 002385853 A CA002385853 A CA 002385853A CA 2385853 A CA2385853 A CA 2385853A CA 2385853 A1 CA2385853 A1 CA 2385853A1
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Canada
Prior art keywords
lna
oxy
oligo
high affinity
monomers
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Abandoned
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CA002385853A
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English (en)
Inventor
Mogens Havsteen Jakobsen
Claes Wahlestedt
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Exiqon AS
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Publication of CA2385853A1 publication Critical patent/CA2385853A1/fr
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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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/06—Pyrimidine radicals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/16—Purine radicals
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • 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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications

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

Abstract

La présente invention a trait au domaine des analogues d'ADN bicycliques, par exemple les ALN et les modifications d'ALN, qui sont utilisés pour mettre au point des oligomères formant des duplex à haute affinité avec les ARN complémentaires, les duplex étant des substrats de Rnase H. Les oligonucléotides de l'invention peuvent être partiellement ou entièrement composés d'analogues d'ALN possédant une très haute affinité et une très haute capacité de recrutement de la RNase H. Grâce à la présente invention, on peut utiliser l'oxy-ALN seul pour construire de nouvelles molécules antisens présentant une activité biologique améliorée. Dans un autre mode de réalisation, on peut utiliser l'oxy-ALN en combinaison avec des ALN non oxy, tels que l'ADN, l'ARN ordinaires ou analogues, par exemple, le thio-ALN ou l'amino-ALN, pour créer des composés antisens de recrutement de RNase H à haute affinité sans qu'il soit nécessaire de s'en tenir à une structure rigide.
CA002385853A 1999-10-04 2000-10-03 Conception d'un oligonucleotide de recrutement de rnase h a haute affinite Abandoned CA2385853A1 (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
DKPA199901422 1999-10-04
DKPA199901422 1999-10-04
US15772499P 1999-10-05 1999-10-05
US60/157,724 1999-10-05
PCT/DK2000/000550 WO2001025248A2 (fr) 1999-10-04 2000-10-03 Conception d'un oligonucleotide de recrutement de rnase h a haute affinite

Publications (1)

Publication Number Publication Date
CA2385853A1 true CA2385853A1 (fr) 2001-04-12

Family

ID=26065735

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002385853A Abandoned CA2385853A1 (fr) 1999-10-04 2000-10-03 Conception d'un oligonucleotide de recrutement de rnase h a haute affinite

Country Status (6)

Country Link
EP (1) EP1224280A2 (fr)
JP (1) JP2003511016A (fr)
AU (1) AU7406700A (fr)
CA (1) CA2385853A1 (fr)
IL (1) IL148916A0 (fr)
WO (1) WO2001025248A2 (fr)

Families Citing this family (64)

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ES2576677T3 (es) 2003-03-21 2016-07-08 Roche Innovation Center Copenhagen A/S Análogos de ARN interfirientes cortos
WO2005013901A2 (fr) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Composes oligomeres et compositions utilisables pour moduler des petits arn non-codants
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KR20230050336A (ko) 2020-07-10 2023-04-14 인스티튜트 내셔널 드 라 싼테 에 드 라 리셰르셰 메디칼르 (인 썸) 뇌전증을 치료하기 위한 방법과 조성물
KR20230144588A (ko) 2021-02-12 2023-10-16 머랜드 파마슈티칼스, 인크. 저산소증 및 허혈-관련 장애의 치료를 위한 작용제, 조성물 및 방법
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CN1273476C (zh) * 1997-09-12 2006-09-06 埃克西康有限公司 寡核苷酸类似物

Also Published As

Publication number Publication date
AU7406700A (en) 2001-05-10
WO2001025248A3 (fr) 2001-08-30
WO2001025248A2 (fr) 2001-04-12
JP2003511016A (ja) 2003-03-25
IL148916A0 (en) 2002-09-12
EP1224280A2 (fr) 2002-07-24

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