JP6642943B2 - 血友病を処置するための方法および組成物 - Google Patents

血友病を処置するための方法および組成物 Download PDF

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JP6642943B2
JP6642943B2 JP2016557544A JP2016557544A JP6642943B2 JP 6642943 B2 JP6642943 B2 JP 6642943B2 JP 2016557544 A JP2016557544 A JP 2016557544A JP 2016557544 A JP2016557544 A JP 2016557544A JP 6642943 B2 JP6642943 B2 JP 6642943B2
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zinc finger
seq
nuclease
sequence
protein
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JP2017504354A5 (enExample
JP2017504354A (ja
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ジェフリー シー． ミラー，
ジェフリー シー． ミラー，
デイビッド パッション，
デイビッド パッション，
エドワード ジェイ． リーバー，
エドワード ジェイ． リーバー，
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Sangamo Therapeutics Inc
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Sangamo Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
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    • 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
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
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    • 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
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    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
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  • Plant Pathology (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2016557544A 2013-12-09 2014-12-09 血友病を処置するための方法および組成物 Expired - Fee Related JP6642943B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201361913838P 2013-12-09 2013-12-09
US61/913,838 2013-12-09
US201461943884P 2014-02-24 2014-02-24
US61/943,884 2014-02-24
PCT/US2014/069307 WO2015089046A1 (en) 2013-12-09 2014-12-09 Methods and compositions for treating hemophilia

Publications (3)

Publication Number Publication Date
JP2017504354A JP2017504354A (ja) 2017-02-09
JP2017504354A5 JP2017504354A5 (enExample) 2017-12-21
JP6642943B2 true JP6642943B2 (ja) 2020-02-12

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JP2016557544A Expired - Fee Related JP6642943B2 (ja) 2013-12-09 2014-12-09 血友病を処置するための方法および組成物
JP2016557545A Expired - Fee Related JP6535684B2 (ja) 2013-12-09 2014-12-09 ゲノム操作のための方法および組成物
JP2018236131A Pending JP2019037254A (ja) 2013-12-09 2018-12-18 ゲノム操作のための方法および組成物

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JP2018236131A Pending JP2019037254A (ja) 2013-12-09 2018-12-18 ゲノム操作のための方法および組成物

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US (6) US9771403B2 (enExample)
EP (3) EP3080274B1 (enExample)
JP (3) JP6642943B2 (enExample)
CN (3) CN105874071B (enExample)
AU (3) AU2014364051B2 (enExample)
CA (3) CA3229275A1 (enExample)
DK (1) DK3080274T3 (enExample)
ES (1) ES2813367T3 (enExample)
HU (1) HUE051628T2 (enExample)
IL (3) IL245778B (enExample)
WO (2) WO2015089046A1 (enExample)

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