DK2548962T3 - Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof - Google Patents

Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof Download PDF

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Publication number
DK2548962T3
DK2548962T3 DK12189270.7T DK12189270T DK2548962T3 DK 2548962 T3 DK2548962 T3 DK 2548962T3 DK 12189270 T DK12189270 T DK 12189270T DK 2548962 T3 DK2548962 T3 DK 2548962T3
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format
sirna
sirnas
modification
formats
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DK12189270.7T
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Nitin Puri
Irudaya Charles
Susan Magdaleno
Alexander Vlassov
Chris Burnett
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Applied Biosystems Llc
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    • 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
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    • 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
    • C12N15/1137—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 enzymes
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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
    • 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
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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 nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/319—Chemical structure of the backbone linked by 2'-5' linkages, i.e. having a free 3'-position
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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
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
DK12189270.7T 2007-09-19 2008-09-17 Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof DK2548962T3 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US97354807P 2007-09-19 2007-09-19
EP08831316.8A EP2195428B1 (en) 2007-09-19 2008-09-17 SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF

Publications (1)

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DK2548962T3 true DK2548962T3 (en) 2016-04-11

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DK12189270.7T DK2548962T3 (en) 2007-09-19 2008-09-17 Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
DK08831316.8T DK2195428T3 (en) 2007-09-19 2008-09-17 SIRNA SEQUENCE-INDEPENDENT MODIFICATION FORMS TO REDUCE TARGET-FAILING PHENOTYPIC EFFECTS OF RNAI, AND STABILIZED FORMS THEREOF

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DK08831316.8T DK2195428T3 (en) 2007-09-19 2008-09-17 SIRNA SEQUENCE-INDEPENDENT MODIFICATION FORMS TO REDUCE TARGET-FAILING PHENOTYPIC EFFECTS OF RNAI, AND STABILIZED FORMS THEREOF

Country Status (6)

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US (6) US8524681B2 (enExample)
EP (2) EP2195428B1 (enExample)
JP (2) JP5723154B2 (enExample)
CN (1) CN101849008A (enExample)
DK (2) DK2548962T3 (enExample)
WO (1) WO2009039173A2 (enExample)

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