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MX2009012568A - Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico. - Google Patents

Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico.

Info

Publication number
MX2009012568A
MX2009012568A MX2009012568A MX2009012568A MX2009012568A MX 2009012568 A MX2009012568 A MX 2009012568A MX 2009012568 A MX2009012568 A MX 2009012568A MX 2009012568 A MX2009012568 A MX 2009012568A MX 2009012568 A MX2009012568 A MX 2009012568A
Authority
MX
Mexico
Prior art keywords
rna
complexes
hydroxymethyl
group
strands
Prior art date
Application number
MX2009012568A
Other languages
English (en)
Inventor
Jesper Wengel
Original Assignee
Mdrna 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 by Mdrna Inc filed Critical Mdrna Inc
Publication of MX2009012568A publication Critical patent/MX2009012568A/es

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    • 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
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. 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
    • 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
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • 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
    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
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  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

La presente invención se dirige a los oligonucleótidos de ARN o complejos de oligonucleótidos de ARN, marcados en la presente juntos como complejos de ARN, que contienen al menos uno, pero opcionalmente más de uno, monómeros de nucleótido sustituidos con hidroximetilo. Por un monómero de nucleótido sustituido con hidroximetilo se entiende un monómero de nucleótido que contiene un grupo hidroximetilo (que puede ser no sustituido, sustituido con O por ejemplo, con un grupo de conjugado, o convertido en un grupo aminometilo conjugado u opcionalmente sustituido). El grupo hidroximetilo no participa en la formación de una ligadura de internucleótido y no es el grupo hidroximetilo (que contiene el grupo 5´- hidroxi) de un monómero de ARN natural. Los complejos de ARN de la invención pueden ser útiles para las aplicaciones terapéuticas, aplicaciones de diagnóstico o aplicaciones de investigación. Los complejos incluyen complejos de ARN de interferencia corta (siARN bicatenarios) que comprenden una hebra antisentido y una hebra pasajera discontinua o continua ("hebra sentido") capaz de regular la expresión del gen. Al menos una de estas hebras, posiblemente más de una de estas hebras, se modifican con uno o más monómeros de nucleótido sustituidos con hidroximetilo de esta invención, que pueden colocarse en el extremo 3´, en el extremo 5´ o internamente. Los complejos de ARN de la invención pueden también ser oligonucleótidos de ARN de hebra sencilla ("hebras de ARN") que se modifican con al menos uno, pero opcionalmente más de uno, monómeros de nucleótido sustituidos con hidroximetilo. Tales hebras de ARN de hebra sencilla se consideran que incluyen siempre que el término complejos de ARN se use en esta solicitud de patente. Los complejos de la invención exhiben estabilidad aumentada hacia la degradación nucleolítica con relación a los complejos correspondientes que comprenden completamente los monómeros de ARN naturales.
MX2009012568A 2007-05-22 2008-05-21 Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico. MX2009012568A (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
DKPA200700751 2007-05-22
DKPA200701718 2007-11-30
DKPA200701785 2007-12-14
DKPA200800534 2008-04-11
PCT/US2008/064417 WO2008147824A2 (en) 2007-05-22 2008-05-21 Hydroxymethyl substituted rna oligonucleotides and rna complexes

Publications (1)

Publication Number Publication Date
MX2009012568A true MX2009012568A (es) 2009-12-08

Family

ID=40075730

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2009012568A MX2009012568A (es) 2007-05-22 2008-05-21 Oligonucleotidos de acido ribonucleico sustituidos con hidroximetilo y complejos de acido ribonucleico.

Country Status (18)

Country Link
US (6) US8314227B2 (es)
EP (2) EP3045535B1 (es)
JP (2) JP5726520B2 (es)
KR (2) KR101629017B1 (es)
CN (2) CN101679978B (es)
AU (1) AU2008256871B2 (es)
BR (1) BRPI0811170B8 (es)
CA (1) CA2687850C (es)
DK (1) DK2162538T3 (es)
ES (1) ES2573936T3 (es)
HK (1) HK1138322A1 (es)
IL (1) IL202040A (es)
MX (1) MX2009012568A (es)
MY (1) MY153691A (es)
NZ (1) NZ580712A (es)
PL (1) PL2162538T3 (es)
WO (1) WO2008147824A2 (es)
ZA (1) ZA200907529B (es)

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