JP6480874B2 - Ｔａｌｅｎに基づく遺伝子修正 - Google Patents

Ｔａｌｅｎに基づく遺伝子修正 Download PDF

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Publication number
JP6480874B2
JP6480874B2 JP2015560341A JP2015560341A JP6480874B2 JP 6480874 B2 JP6480874 B2 JP 6480874B2 JP 2015560341 A JP2015560341 A JP 2015560341A JP 2015560341 A JP2015560341 A JP 2015560341A JP 6480874 B2 JP6480874 B2 JP 6480874B2
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cells
talen
nucleic acid
sequence
gene
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JP2016512960A5 (enExample
JP2016512960A (ja
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ジェイ． オズボーン、マーク
ジェイ． オズボーン、マーク
トーラー、ヤクブ
ブレイザー、ブルース
エフ． ボイタス、ダニエル
エフ． ボイタス、ダニエル
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University of Minnesota System
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    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61P17/00—Drugs for dermatological disorders
    • 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
    • A61P19/08—Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Immunology (AREA)
  • Rheumatology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Dermatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2015560341A 2013-03-01 2014-02-28 Ｔａｌｅｎに基づく遺伝子修正 Expired - Fee Related JP6480874B2 (ja)

Priority Applications (1)

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JP2019021260A JP6893945B2 (ja) 2013-03-01 2019-02-08 Ｔａｌｅｎに基づく遺伝子修正

Applications Claiming Priority (3)

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US201361771735P 2013-03-01 2013-03-01
US61/771,735 2013-03-01
PCT/US2014/019322 WO2014134412A1 (en) 2013-03-01 2014-02-28 Talen-based gene correction

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JP2016512960A JP2016512960A (ja) 2016-05-12
JP2016512960A5 JP2016512960A5 (enExample) 2017-03-30
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JP2019021260A Expired - Fee Related JP6893945B2 (ja) 2013-03-01 2019-02-08 Ｔａｌｅｎに基づく遺伝子修正
JP2021015441A Pending JP2021088565A (ja) 2013-03-01 2021-02-03 Ｔａｌｅｎに基づく遺伝子修正

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US (4) US9393257B2 (enExample)
EP (2) EP2961262B1 (enExample)
JP (3) JP6480874B2 (enExample)
AU (2) AU2014223243B2 (enExample)
CA (1) CA2902954A1 (enExample)
ES (1) ES2750550T3 (enExample)
WO (1) WO2014134412A1 (enExample)

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JP2021088565A (ja) 2021-06-10
EP2961262A4 (en) 2016-10-12
JP2019089821A (ja) 2019-06-13
HK1219021A1 (en) 2017-03-24
AU2020200307A1 (en) 2020-02-06
CA2902954A1 (en) 2014-09-04
NZ711254A (en) 2021-02-26
US20190054111A1 (en) 2019-02-21
AU2014223243B2 (en) 2019-10-17
EP3567104A1 (en) 2019-11-13
AU2014223243A1 (en) 2015-09-10
ES2750550T3 (es) 2020-03-26
JP6893945B2 (ja) 2021-06-23
EP2961262B1 (en) 2019-07-24
WO2014134412A1 (en) 2014-09-04
US20160367588A1 (en) 2016-12-22
EP2961262A1 (en) 2016-01-06
JP2016512960A (ja) 2016-05-12
US10973844B2 (en) 2021-04-13
US10172880B2 (en) 2019-01-08
US20140256798A1 (en) 2014-09-11
US20210187006A1 (en) 2021-06-24
US9393257B2 (en) 2016-07-19

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