CA2796440C - Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire - Google Patents

Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire Download PDF

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Publication number
CA2796440C
CA2796440C CA2796440A CA2796440A CA2796440C CA 2796440 C CA2796440 C CA 2796440C CA 2796440 A CA2796440 A CA 2796440A CA 2796440 A CA2796440 A CA 2796440A CA 2796440 C CA2796440 C CA 2796440C
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Prior art keywords
exon
oligonucleotide
mrna
skipping
dystrophin
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Expired - Lifetime
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CA2796440A
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English (en)
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CA2796440A1 (fr
Inventor
Garrit-Jan Boudewijn Van Ommen
Judith Christina Theodora Van Deutekom
Johannes Theodorus Den Dunnen
Annemieke Aartsma-Rus
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Leids Universitair Medisch Centrum LUMC
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Leids Universitair Medisch Centrum LUMC
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Priority to CA2796440A priority Critical patent/CA2796440C/fr
Priority claimed from CA2524255A external-priority patent/CA2524255C/fr
Publication of CA2796440A1 publication Critical patent/CA2796440A1/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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • 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
    • C12N2310/314—Phosphoramidates
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
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  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Linvention fournit un procédé de génération dun oligonucléotide avec lequel un exon peut être sauté dans un pré-ARN et ainsi exclu de son ARNm produit. La présente invention concerne en outre des procédés pour modifier la structure secondaire dun ARNm afin dinterférer avec les processus dépissage et les utilisations des oligonucléotides, ainsi que des procédés de traitement de maladies. Linvention concerne en outre des compositions pharmaceutiques, des procédés et des moyens permettant dinduire le saut de plusieurs exons dans un pré-ARNm.
CA2796440A 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire Expired - Lifetime CA2796440C (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CA2796440A CA2796440C (fr) 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
CA2796440A CA2796440C (fr) 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire
CA2524255A CA2524255C (fr) 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CA2524255A Division CA2524255C (fr) 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire

Publications (2)

Publication Number Publication Date
CA2796440A1 CA2796440A1 (fr) 2004-09-30
CA2796440C true CA2796440C (fr) 2019-08-27

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CA2796440A Expired - Lifetime CA2796440C (fr) 2003-03-21 2003-03-21 Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire

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CA (1) CA2796440C (fr)

Also Published As

Publication number Publication date
CA2796440A1 (fr) 2004-09-30

Similar Documents

Publication Publication Date Title
US11208657B2 (en) Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
CA2796440C (fr) Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire
HK1084694B (en) Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure

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Effective date: 20230321