JP2020508056A - 遺伝子編集のための組成物および方法 - Google Patents

遺伝子編集のための組成物および方法 Download PDF

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JP2020508056A
JP2020508056A JP2019545759A JP2019545759A JP2020508056A JP 2020508056 A JP2020508056 A JP 2020508056A JP 2019545759 A JP2019545759 A JP 2019545759A JP 2019545759 A JP2019545759 A JP 2019545759A JP 2020508056 A JP2020508056 A JP 2020508056A
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cells
sequence
angptl3
gene
rna
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JP2020508056A5 (https=
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アント・スヴェン・ランドバーグ
サマルト・クルカルニ
ローレンス・クライン
ハリ・クマール・パドマナバン
イボンヌ・サラ・アラティン
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CRISPR Therapeutics AG
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CRISPR Therapeutics AG
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Priority to JP2023070182A priority Critical patent/JP7556092B2/ja
Priority to JP2024157136A priority patent/JP2025003976A/ja
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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
    • C12N15/63—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
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/515—Angiogenesic factors; Angiogenin
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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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/1136—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 against growth factors, growth regulators, cytokines, lymphokines or hormones
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
    • C12N9/16—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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Vascular Medicine (AREA)
  • Endocrinology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2019545759A 2017-02-22 2018-02-21 遺伝子編集のための組成物および方法 Ceased JP2020508056A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023070182A JP7556092B2 (ja) 2017-02-22 2023-04-21 遺伝子編集のための組成物および方法
JP2024157136A JP2025003976A (ja) 2017-02-22 2024-09-11 遺伝子編集のための組成物および方法

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762461829P 2017-02-22 2017-02-22
US62/461,829 2017-02-22
US201762526562P 2017-06-29 2017-06-29
US62/526,562 2017-06-29
US201762587832P 2017-11-17 2017-11-17
US62/587,832 2017-11-17
PCT/IB2018/000228 WO2018154387A1 (en) 2017-02-22 2018-02-21 Compositions and methods for gene editing

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JP2020508056A true JP2020508056A (ja) 2020-03-19
JP2020508056A5 JP2020508056A5 (https=) 2021-04-01

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JP2023070182A Active JP7556092B2 (ja) 2017-02-22 2023-04-21 遺伝子編集のための組成物および方法
JP2024157136A Pending JP2025003976A (ja) 2017-02-22 2024-09-11 遺伝子編集のための組成物および方法

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JP2024157136A Pending JP2025003976A (ja) 2017-02-22 2024-09-11 遺伝子編集のための組成物および方法

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US (2) US11920148B2 (https=)
EP (1) EP3585895A1 (https=)
JP (3) JP2020508056A (https=)
CN (2) CN119351474A (https=)
AU (2) AU2018224387B2 (https=)
CA (1) CA3054031A1 (https=)
WO (1) WO2018154387A1 (https=)

Cited By (1)

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JP2023518915A (ja) * 2020-04-09 2023-05-08 ヴァーヴ・セラピューティクス，インコーポレーテッド Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法

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JP7457833B2 (ja) 2020-04-09 2024-03-28 ヴァーヴ・セラピューティクス，インコーポレーテッド Ａｎｇｐｔｌ３の塩基編集および疾患の処置のためにこれを使用する方法
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