JP6112569B2 - キメラ２重鎖核酸 - Google Patents

キメラ２重鎖核酸 Download PDF

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Publication number
JP6112569B2
JP6112569B2 JP2014528740A JP2014528740A JP6112569B2 JP 6112569 B2 JP6112569 B2 JP 6112569B2 JP 2014528740 A JP2014528740 A JP 2014528740A JP 2014528740 A JP2014528740 A JP 2014528740A JP 6112569 B2 JP6112569 B2 JP 6112569B2
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nucleic acid
pharmaceutical composition
acid strand
nucleotide
nucleotides
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Japanese (ja)
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JP2015502134A5 (enExample
JP2015502134A (ja
Inventor
隆徳 横田
隆徳 横田
一隆 仁科
一隆 仁科
聡 小比賀
聡 小比賀
水澤　英洋
英洋 水澤
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Tokyo Medical and Dental University
The University of Osaka
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Osaka University NUC
Tokyo Medical and Dental University
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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
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • 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
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Peptides Or Proteins (AREA)
JP2014528740A 2011-12-16 2012-12-17 キメラ２重鎖核酸 Active JP6112569B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2014528740A JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
JP2011275488 2011-12-16
JP2011275488 2011-12-16
JP2014528740A JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸
PCT/JP2012/083180 WO2013089283A1 (en) 2011-12-16 2012-12-17 Chimeric double-stranded nucleic acid

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2017044034A Division JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸

Publications (3)

Publication Number Publication Date
JP2015502134A JP2015502134A (ja) 2015-01-22
JP2015502134A5 JP2015502134A5 (enExample) 2016-03-31
JP6112569B2 true JP6112569B2 (ja) 2017-04-12

Family

ID=47559626

Family Applications (7)

Application Number Title Priority Date Filing Date
JP2014528740A Active JP6112569B2 (ja) 2011-12-16 2012-12-17 キメラ２重鎖核酸
JP2017044034A Active JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸
JP2018188022A Active JP6638923B2 (ja) 2011-12-16 2018-10-03 キメラ２重鎖核酸
JP2019224279A Active JP6960123B2 (ja) 2011-12-16 2019-12-12 キメラ２重鎖核酸
JP2020179738A Active JP7174384B2 (ja) 2011-12-16 2020-10-27 キメラ２重鎖核酸
JP2021136038A Pending JP2022000426A (ja) 2011-12-16 2021-08-24 キメラ２重鎖核酸
JP2022146970A Active JP7423015B2 (ja) 2011-12-16 2022-09-15 キメラ２重鎖核酸

Family Applications After (6)

Application Number Title Priority Date Filing Date
JP2017044034A Active JP6416301B2 (ja) 2011-12-16 2017-03-08 キメラ２重鎖核酸
JP2018188022A Active JP6638923B2 (ja) 2011-12-16 2018-10-03 キメラ２重鎖核酸
JP2019224279A Active JP6960123B2 (ja) 2011-12-16 2019-12-12 キメラ２重鎖核酸
JP2020179738A Active JP7174384B2 (ja) 2011-12-16 2020-10-27 キメラ２重鎖核酸
JP2021136038A Pending JP2022000426A (ja) 2011-12-16 2021-08-24 キメラ２重鎖核酸
JP2022146970A Active JP7423015B2 (ja) 2011-12-16 2022-09-15 キメラ２重鎖核酸

Country Status (9)

Country Link
US (6) US9816089B2 (enExample)
EP (1) EP2791335B1 (enExample)
JP (7) JP6112569B2 (enExample)
CN (2) CN104126010B (enExample)
AU (1) AU2012353330B2 (enExample)
CA (2) CA2859581C (enExample)
ES (1) ES2707232T3 (enExample)
IN (1) IN2014CN04590A (enExample)
WO (1) WO2013089283A1 (enExample)

Cited By (2)

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JP2016526529A (ja) * 2013-06-16 2016-09-05 国立大学法人 東京医科歯科大学 エクソンスキッピング効果を有する二本鎖アンチセンス核酸
WO2025063185A1 (ja) 2023-09-19 2025-03-27 国立大学法人東海国立大学機構 １本鎖ポリヌクレオチド

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