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 (enExample
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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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    • 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
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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
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Vascular Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Endocrinology (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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JP2020508056A5 JP2020508056A5 (enExample) 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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US (2) US11920148B2 (enExample)
EP (1) EP3585895A1 (enExample)
JP (3) JP2020508056A (enExample)
CN (2) CN110662838B (enExample)
AU (2) AU2018224387B2 (enExample)
CA (1) CA3054031A1 (enExample)
WO (1) WO2018154387A1 (enExample)

Cited By (2)

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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
US12680096B2 (en) 2019-08-12 2026-07-14 Life Edit Therapeutics, Inc. RNA-guided nucleases and active fragments and variants thereof and methods of use

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US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
AU2016359629B2 (en) 2015-11-23 2023-03-09 Ranjan BATRA Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/Cas9
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2018224387B2 (en) * 2017-02-22 2024-08-08 Crispr Therapeutics Ag Compositions and methods for gene editing
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JP2025526073A (ja) * 2022-08-11 2025-08-07 エピジェニック・セラピューティクス・プライベイト・リミテッド エピジェネティック編集標的部位のための方法およびその使用
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