RU2619184C2 - Антисмысловые нуклеиновые кислоты - Google Patents

Антисмысловые нуклеиновые кислоты Download PDF

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RU2619184C2
RU2619184C2 RU2014130600A RU2014130600A RU2619184C2 RU 2619184 C2 RU2619184 C2 RU 2619184C2 RU 2014130600 A RU2014130600 A RU 2014130600A RU 2014130600 A RU2014130600 A RU 2014130600A RU 2619184 C2 RU2619184 C2 RU 2619184C2
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exon
seq
pmo
nucleotides
nucleic acid
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RU2014130600A
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RU2014130600A (ru
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Наоки ВАТАНАБЕ
Харуна СЕО
Син'ити ТАКЕДА
Тецуя НАГАТА
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Ниппон Синяку Ко., Лтд.
Нэшнл Сентер Оф Ньюролоджи Энд Сайкайэтри
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    • A—HUMAN NECESSITIES
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • C12N2310/3233—Morpholino-type ring
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
RU2014130600A 2011-12-28 2012-12-27 Антисмысловые нуклеиновые кислоты RU2619184C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
JP2011-288040 2011-12-28
JP2011288040 2011-12-28
JP2012-043092 2012-02-29
JP2012043092 2012-02-29
PCT/JP2012/084295 WO2013100190A1 (ja) 2011-12-28 2012-12-27 アンチセンス核酸

Related Child Applications (1)

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RU2017114509A Division RU2651468C1 (ru) 2011-12-28 2012-12-27 Антисмысловые нуклеиновые кислоты

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RU2014130600A RU2014130600A (ru) 2016-02-20
RU2619184C2 true RU2619184C2 (ru) 2017-05-12

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RU2014130600A RU2619184C2 (ru) 2011-12-28 2012-12-27 Антисмысловые нуклеиновые кислоты
RU2017114509A RU2651468C1 (ru) 2011-12-28 2012-12-27 Антисмысловые нуклеиновые кислоты
RU2018109906A RU2681470C1 (ru) 2011-12-28 2018-03-21 Антисмысловые нуклеиновые кислоты

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RU2018109906A RU2681470C1 (ru) 2011-12-28 2018-03-21 Антисмысловые нуклеиновые кислоты

Country Status (12)

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US (5) US9512424B2 (enExample)
EP (2) EP3594347A1 (enExample)
JP (6) JP6141770B2 (enExample)
KR (2) KR102240139B1 (enExample)
CN (10) CN110055243B (enExample)
AU (2) AU2012360702C1 (enExample)
CA (2) CA3132111A1 (enExample)
ES (1) ES2748868T3 (enExample)
IN (1) IN2014DN06220A (enExample)
RU (3) RU2619184C2 (enExample)
TW (1) TWI606059B (enExample)
WO (1) WO2013100190A1 (enExample)

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