JP6059141B2 - 核内繋留ｒｎａの調節 - Google Patents

核内繋留ｒｎａの調節 Download PDF

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Publication number
JP6059141B2
JP6059141B2 JP2013520822A JP2013520822A JP6059141B2 JP 6059141 B2 JP6059141 B2 JP 6059141B2 JP 2013520822 A JP2013520822 A JP 2013520822A JP 2013520822 A JP2013520822 A JP 2013520822A JP 6059141 B2 JP6059141 B2 JP 6059141B2
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rna
oligonucleotide
antisense
modified
pharmaceutical composition
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Japanese (ja)
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JP2013538561A (ja
JP2013538561A5 (enExample
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ベネット，シー・フランク
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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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
    • C12N15/1137—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
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    • A61P35/00—Antineoplastic agents
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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
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    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
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    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/00—Structure or type of the nucleic acid
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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
    • C12N2310/33—Chemical structure of the base
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    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy

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  • 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)
JP2013520822A 2010-07-19 2011-07-19 核内繋留ｒｎａの調節 Active JP6059141B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US36577510P 2010-07-19 2010-07-19
US36576210P 2010-07-19 2010-07-19
US61/365,775 2010-07-19
US61/365,762 2010-07-19
US201161478021P 2011-04-21 2011-04-21
US61/478,021 2011-04-21
PCT/US2011/044583 WO2012012467A2 (en) 2010-07-19 2011-07-19 Modulation of nuclear-retained rna

Publications (3)

Publication Number Publication Date
JP2013538561A JP2013538561A (ja) 2013-10-17
JP2013538561A5 JP2013538561A5 (enExample) 2014-09-04
JP6059141B2 true JP6059141B2 (ja) 2017-01-11

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JP2013520822A Active JP6059141B2 (ja) 2010-07-19 2011-07-19 核内繋留ｒｎａの調節
JP2013520815A Active JP5981428B2 (ja) 2010-07-19 2011-07-19 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2016147984A Active JP6343308B2 (ja) 2010-07-19 2016-07-28 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2018095820A Active JP6698740B2 (ja) 2010-07-19 2018-05-18 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2020078906A Pending JP2020183379A (ja) 2010-07-19 2020-04-28 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整

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JP2013520815A Active JP5981428B2 (ja) 2010-07-19 2011-07-19 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2016147984A Active JP6343308B2 (ja) 2010-07-19 2016-07-28 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2018095820A Active JP6698740B2 (ja) 2010-07-19 2018-05-18 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整
JP2020078906A Pending JP2020183379A (ja) 2010-07-19 2020-04-28 筋緊張性ジストロフィー・プロテインキナーゼ（ｄｍｐｋ）発現の調整

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US (7) US20130225659A1 (enExample)
EP (5) EP2595663A4 (enExample)
JP (5) JP6059141B2 (enExample)
KR (2) KR20180105730A (enExample)
CN (2) CN103167883B (enExample)
AU (6) AU2011282243B2 (enExample)
CA (2) CA2805791A1 (enExample)
DK (1) DK3031920T3 (enExample)
ES (1) ES2756326T3 (enExample)
HR (1) HRP20191753T1 (enExample)
IL (2) IL223962B (enExample)
PL (1) PL3031920T3 (enExample)
WO (2) WO2012012443A2 (enExample)

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