JP6006120B2 - 対立遺伝子多様体の選択的低減 - Google Patents

対立遺伝子多様体の選択的低減 Download PDF

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JP6006120B2
JP6006120B2 JP2012552932A JP2012552932A JP6006120B2 JP 6006120 B2 JP6006120 B2 JP 6006120B2 JP 2012552932 A JP2012552932 A JP 2012552932A JP 2012552932 A JP2012552932 A JP 2012552932A JP 6006120 B2 JP6006120 B2 JP 6006120B2
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oligonucleotide
modified
wing
certain embodiments
antisense
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JP2013518604A (ja
JP2013518604A5 (enExample
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ベネット，シー・フランク
フレアー，スーザン・エム
グリーンリー，サラ
スウェイジ，エリック・イー
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Ionis Pharmaceuticals Inc
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    • C—CHEMISTRY; METALLURGY
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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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    • C12N2320/34—Allele or polymorphism specific uses
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    • C12N2320/30—Special therapeutic applications
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  • Animal Behavior & Ethology (AREA)
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  • Pharmacology & Pharmacy (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (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)
JP2012552932A 2010-02-08 2011-02-08 対立遺伝子多様体の選択的低減 Active JP6006120B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US30246910P 2010-02-08 2010-02-08
US61/302,469 2010-02-08
US37163510P 2010-08-06 2010-08-06
US61/371,635 2010-08-06
PCT/US2011/024103 WO2011097643A1 (en) 2010-02-08 2011-02-08 Selective reduction of allelic variants

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JP2016078592A Division JP6316334B2 (ja) 2010-02-08 2016-04-11 対立遺伝子多様体の選択的低減

Publications (3)

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JP2013518604A JP2013518604A (ja) 2013-05-23
JP2013518604A5 JP2013518604A5 (enExample) 2014-03-27
JP6006120B2 true JP6006120B2 (ja) 2016-10-12

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JP2012552932A Active JP6006120B2 (ja) 2010-02-08 2011-02-08 対立遺伝子多様体の選択的低減
JP2016078592A Active JP6316334B2 (ja) 2010-02-08 2016-04-11 対立遺伝子多様体の選択的低減
JP2018055560A Active JP6652984B2 (ja) 2010-02-08 2018-03-23 対立遺伝子多様体の選択的低減
JP2020009595A Pending JP2020089382A (ja) 2010-02-08 2020-01-24 対立遺伝子多様体の選択的低減

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JP2018055560A Active JP6652984B2 (ja) 2010-02-08 2018-03-23 対立遺伝子多様体の選択的低減
JP2020009595A Pending JP2020089382A (ja) 2010-02-08 2020-01-24 対立遺伝子多様体の選択的低減

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US (5) US8957040B2 (enExample)
EP (3) EP3321361B1 (enExample)
JP (4) JP6006120B2 (enExample)
AU (3) AU2011213562B2 (enExample)
CA (1) CA2789005A1 (enExample)
DK (1) DK2534248T3 (enExample)
ES (1) ES2733708T3 (enExample)
IL (2) IL221272A (enExample)
WO (1) WO2011097643A1 (enExample)

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