JP6018069B2 - 血友病ｂを治療する方法及び組成物 - Google Patents

血友病ｂを治療する方法及び組成物 Download PDF

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JP6018069B2
JP6018069B2 JP2013533982A JP2013533982A JP6018069B2 JP 6018069 B2 JP6018069 B2 JP 6018069B2 JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013533982 A JP2013533982 A JP 2013533982A JP 6018069 B2 JP6018069 B2 JP 6018069B2
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protein
gene
sequence
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zinc finger
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JP2013544077A5 (enExample
JP2013544077A (ja
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ディー．グレゴリー フィリップ
ディー．グレゴリー フィリップ
エー．ハイ キャサリン
エー．ハイ キャサリン
シー．ホームズ マイケル
シー．ホームズ マイケル
リ ホジュン
リ ホジュン
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Childrens Hospital of Philadelphia CHOP
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    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
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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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1024—In vivo mutagenesis using high mutation rate "mutator" host strains by inserting genetic material, e.g. encoding an error prone polymerase, disrupting a gene for mismatch repair
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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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21002—Chymotrypsin C (3.4.21.2)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N5/00—Radiation therapy
    • A61N5/06—Radiation therapy using light
    • A61N2005/0658—Radiation therapy using light characterised by the wavelength of light used
    • A61N2005/0661—Radiation therapy using light characterised by the wavelength of light used ultraviolet

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  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Microbiology (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Radiology & Medical Imaging (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2013533982A 2010-10-12 2011-10-12 血友病ｂを治療する方法及び組成物 Active JP6018069B2 (ja)

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US39233310P 2010-10-12 2010-10-12
US61/392,333 2010-10-12
PCT/US2011/056020 WO2012051343A1 (en) 2010-10-12 2011-10-12 Methods and compositions for treating hemophilia b

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JP2013544077A JP2013544077A (ja) 2013-12-12
JP2013544077A5 JP2013544077A5 (enExample) 2016-01-07
JP6018069B2 true JP6018069B2 (ja) 2016-11-02

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US (2) US9175280B2 (enExample)
EP (1) EP2627665B1 (enExample)
JP (1) JP6018069B2 (enExample)
KR (1) KR101930711B1 (enExample)
CN (1) CN103270046B (enExample)
AU (1) AU2011316575B2 (enExample)
BR (1) BR112013008881B1 (enExample)
CA (1) CA2814143C (enExample)
ES (1) ES2562421T3 (enExample)
RU (1) RU2608643C2 (enExample)
WO (1) WO2012051343A1 (enExample)
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CA2814143C (en) 2020-09-08
US9629930B2 (en) 2017-04-25
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CN103270046A (zh) 2013-08-28
WO2012051343A1 (en) 2012-04-19
ES2562421T3 (es) 2016-03-04
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CA2814143A1 (en) 2012-04-19
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RU2608643C2 (ru) 2017-01-23
EP2627665B1 (en) 2015-12-16
BR112013008881B1 (pt) 2021-12-07
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US20120128635A1 (en) 2012-05-24

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