JP2023516692A - ゲノムを調節するための方法及び組成物 - Google Patents

ゲノムを調節するための方法及び組成物 Download PDF

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JP2023516692A
JP2023516692A JP2022552816A JP2022552816A JP2023516692A JP 2023516692 A JP2023516692 A JP 2023516692A JP 2022552816 A JP2022552816 A JP 2022552816A JP 2022552816 A JP2022552816 A JP 2022552816A JP 2023516692 A JP2023516692 A JP 2023516692A
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polypeptide
sequence
domain
target
dna
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バレット イーサン スタインバーグ，
アン ヘレン ボスマー，
ウィリアム エドワード サロモン，
インナ シェルバコバ，
セシリア ジョバンナ シルビア コッタ－ラムシノ，
ジェイコブ ローゼンブラム ルーベンス，
ロバート ジェイムズ シトリク，
ジー ジュン ワン，
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フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー
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Publication of JP2023516692A publication Critical patent/JP2023516692A/ja
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    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • C12N9/1276—RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N15/11—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
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Cosmetics (AREA)
  • Toys (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Breeding Of Plants And Reproduction By Means Of Culturing (AREA)
JP2022552816A 2020-03-04 2021-03-04 ゲノムを調節するための方法及び組成物 Pending JP2023516692A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202062985291P 2020-03-04 2020-03-04
US62/985,291 2020-03-04
US202063035638P 2020-06-05 2020-06-05
US63/035,638 2020-06-05
PCT/US2021/020933 WO2021178709A1 (en) 2020-03-04 2021-03-04 Methods and compositions for modulating a genome

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JP2023516692A true JP2023516692A (ja) 2023-04-20
JP2023516692A5 JP2023516692A5 (https=) 2024-03-13

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JP2022552816A Pending JP2023516692A (ja) 2020-03-04 2021-03-04 ゲノムを調節するための方法及び組成物

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US (1) US20230242899A1 (https=)
EP (1) EP4114940A4 (https=)
JP (1) JP2023516692A (https=)
AU (1) AU2021232005A1 (https=)
BR (1) BR112022017713A2 (https=)
CA (1) CA3174537A1 (https=)
WO (1) WO2021178709A1 (https=)

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EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
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WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
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US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
RU2724470C1 (ru) * 2019-11-11 2020-06-23 Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий Применение cas9 белка из бактерии pasteurella pneumotropica для модификации геномной днк в клетках
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
US20230203192A1 (en) * 2020-05-20 2023-06-29 Flagship Pioneering, Inc. Compositions and methods for producing human polyclonal antibodies
BR112023023642A2 (pt) 2021-05-11 2024-01-30 Myeloid Therapeutics Inc Métodos e composições para integração genômica
WO2022256294A1 (en) * 2021-06-01 2022-12-08 University Of Massachusetts Cas9 nickase-mediated gene editing
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
US12129495B2 (en) 2021-10-15 2024-10-29 Codexis, Inc. Engineered DNA polymerase variants
CN118103500A (zh) * 2021-10-15 2024-05-28 科德克希思公司 重组逆转录酶变体
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EP4419669A1 (en) * 2021-10-19 2024-08-28 Massachusetts Institute Of Technology Genomic editing with site-specific retrotransposons
CN118556119A (zh) * 2021-11-19 2024-08-27 埃门多生物公司 新型omni crispr核酸酶
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
WO2023141602A2 (en) * 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
US11866728B2 (en) 2022-01-21 2024-01-09 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
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WO2024077267A1 (en) * 2022-10-07 2024-04-11 The Broad Institute, Inc. Prime editing methods and compositions for treating triplet repeat disorders
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EP4114940A1 (en) 2023-01-11
WO2021178709A1 (en) 2021-09-10
CA3174537A1 (en) 2021-09-10
EP4114940A4 (en) 2024-09-04
US20230242899A1 (en) 2023-08-03
BR112022017713A2 (pt) 2022-11-16
AU2021232005A1 (en) 2022-09-29

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