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DK1713912T3 - Modificerede Korte Interfererende RNA (Modificerede siRNA) - Google Patents

Modificerede Korte Interfererende RNA (Modificerede siRNA)

Info

Publication number
DK1713912T3
DK1713912T3 DK05700612.4T DK05700612T DK1713912T3 DK 1713912 T3 DK1713912 T3 DK 1713912T3 DK 05700612 T DK05700612 T DK 05700612T DK 1713912 T3 DK1713912 T3 DK 1713912T3
Authority
DK
Denmark
Prior art keywords
modified
interfering rna
short interfering
sirna
modified sirna
Prior art date
Application number
DK05700612.4T
Other languages
English (en)
Inventor
Joacim Elmen
Claes Wahlestedt
Henrik Orum
Zicai Liang
Troels Koch
Original Assignee
Santaris Pharma As
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
Priority claimed from PCT/DK2004/000192 external-priority patent/WO2004083430A2/en
Application filed by Santaris Pharma As filed Critical Santaris Pharma As
Application granted granted Critical
Publication of DK1713912T3 publication Critical patent/DK1713912T3/da

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/50Methods for regulating/modulating their activity
    • C12N2320/51Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
DK05700612.4T 2004-01-30 2005-01-28 Modificerede Korte Interfererende RNA (Modificerede siRNA) DK1713912T3 (da)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
DKPA200400145 2004-01-30
PCT/DK2004/000192 WO2004083430A2 (en) 2003-03-21 2004-03-22 SHORT INTERFERING RNA (siRNA) ANALOGUES
DKPA200401079 2004-07-08
PCT/DK2005/000062 WO2005073378A1 (en) 2004-01-30 2005-01-28 MODIFIED SHORT INTERFERING RNA (MODIFIED siRNA)

Publications (1)

Publication Number Publication Date
DK1713912T3 true DK1713912T3 (da) 2013-12-16

Family

ID=36997281

Family Applications (1)

Application Number Title Priority Date Filing Date
DK05700612.4T DK1713912T3 (da) 2004-01-30 2005-01-28 Modificerede Korte Interfererende RNA (Modificerede siRNA)

Country Status (4)

Country Link
US (1) US20080249039A1 (da)
EP (1) EP1713912B1 (da)
DK (1) DK1713912T3 (da)
WO (1) WO2005073378A1 (da)

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AU2007209481B2 (en) * 2006-01-27 2012-01-12 Roche Innovation Center Copenhagen A/S LNA modified phosphorothiolated oligonucleotides
JP5244087B2 (ja) * 2006-03-23 2013-07-24 サンタリス ファーマ アー/エス 低分子内部セグメント化干渉rna
US20100216864A1 (en) 2006-10-09 2010-08-26 Ellen Marie Straarup RNA Antagonist Compounds for the Modulation of PCSK9
WO2009039173A2 (en) 2007-09-19 2009-03-26 Applied Biosystems Inc. SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
US20110223665A1 (en) * 2008-07-25 2011-09-15 Alnylam Pharmaceuticals, Inc. ENHANCEMENT OF siRNA SILENCING ACTIVITY USING UNIVERSAL BASES OR MISMATCHES IN THE SENSE STRAND
US8957038B2 (en) 2009-07-15 2015-02-17 Medtronic, Inc. Treatment of neurological disorders
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EP2638163B1 (en) 2010-11-12 2017-05-17 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2663323B1 (en) 2011-01-14 2017-08-16 The General Hospital Corporation Methods targeting mir-128 for regulating cholesterol/lipid metabolism
BR112014008925A2 (pt) 2011-10-11 2020-10-27 The Brigham And Women's Hospital, Inc. micro rnas em distúrbios de neurodegenerativos
CA2873797A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating utrn expression
BR112014028644A2 (pt) 2012-05-16 2017-08-15 Rana Therapeutics Inc Composições e métodos para modulação da expressão de atp2a2
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING THE EXPRESSION OF THE HEMOGLOBIN GENE FAMILIES
HK1208700A1 (en) 2012-05-16 2016-03-11 Rana Therapeutics Inc. Compositions and methods for modulating smn gene family expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
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US10301624B2 (en) 2014-06-25 2019-05-28 The General Hospital Corporation Targeting human satellite II (HSATII)
JP2017526367A (ja) 2014-08-29 2017-09-14 チルドレンズ メディカル センター コーポレーション 癌の処置のための方法および組成物
US10858650B2 (en) 2014-10-30 2020-12-08 The General Hospital Corporation Methods for modulating ATRX-dependent gene repression
WO2016137937A1 (en) * 2015-02-24 2016-09-01 Dcb-Usa Llc Short interfering rna for treating cancer
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
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US20180161429A1 (en) 2015-06-26 2018-06-14 Beth Israel Deaconess Medical Center Inc. Cancer therapy targeting tetraspanin 33 (tspan33) in myeloid derived suppressor cells
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Also Published As

Publication number Publication date
EP1713912A1 (en) 2006-10-25
EP1713912B1 (en) 2013-09-18
WO2005073378A1 (en) 2005-08-11
US20080249039A1 (en) 2008-10-09

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