JP7550648B2 - 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用 - Google Patents

新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用 Download PDF

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JP7550648B2
JP7550648B2 JP2020550714A JP2020550714A JP7550648B2 JP 7550648 B2 JP7550648 B2 JP 7550648B2 JP 2020550714 A JP2020550714 A JP 2020550714A JP 2020550714 A JP2020550714 A JP 2020550714A JP 7550648 B2 JP7550648 B2 JP 7550648B2
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polypeptide
srgn
seq
sequence
nucleic acid
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JP2021518139A5 (https=
JP2021518139A (ja
JPWO2019183150A5 (https=
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アンドレ コーネン，
モーリッツ シュミット，
ウェイン ココ，
マイケル ビアグ ガマリンダ，
アシシュ グプタ，
クリスティアン ピッツラー，
フロリアン リヒター，
ヤン テッベ，
クリストファー チェン，
リョウ タケウチ，
キャロライン ダブリュー． ライス，
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Bayer Healthcare LLC
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Bayer Healthcare LLC
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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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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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
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  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020550714A 2018-03-19 2019-03-19 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用 Active JP7550648B2 (ja)

Applications Claiming Priority (19)

Application Number Priority Date Filing Date Title
EP18162681.3 2018-03-19
EP18162681 2018-03-19
EP18162683 2018-03-19
EP18162683.9 2018-03-19
EP18172625.8 2018-05-16
EP18172625 2018-05-16
EP18174707.2 2018-05-29
EP18174707 2018-05-29
EP18181680.2 2018-07-04
EP18181680 2018-07-04
US201862745246P 2018-10-12 2018-10-12
US201862745239P 2018-10-12 2018-10-12
US201862745240P 2018-10-12 2018-10-12
US201862745238P 2018-10-12 2018-10-12
US62/745,246 2018-10-12
US62/745,239 2018-10-12
US62/745,238 2018-10-12
US62/745,240 2018-10-12
PCT/US2019/023044 WO2019183150A1 (en) 2018-03-19 2019-03-19 Novel rna-programmable endonuclease systems and uses thereof

Publications (4)

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JP2021518139A JP2021518139A (ja) 2021-08-02
JP2021518139A5 JP2021518139A5 (https=) 2022-03-29
JPWO2019183150A5 JPWO2019183150A5 (https=) 2022-03-29
JP7550648B2 true JP7550648B2 (ja) 2024-09-13

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US (2) US12203110B2 (https=)
EP (1) EP3768834B1 (https=)
JP (1) JP7550648B2 (https=)
CN (1) CN112424348B (https=)
AU (1) AU2019239957A1 (https=)
CA (1) CA3092497A1 (https=)
MA (1) MA52074A (https=)
WO (1) WO2019183150A1 (https=)

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US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
KR20200124702A (ko) 2018-02-23 2020-11-03 파이어니어 하이 부렛드 인터내쇼날 인코포레이팃드 신규한 cas9 오르소로그
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
CA3121191A1 (en) * 2018-11-28 2020-06-04 Crispr Therapeutics Ag Optimized mrna encoding cas9 for use in lnps
US12365888B2 (en) 2018-12-14 2025-07-22 Pioneer Hi-Bred International, Inc. CRISPR-Cas systems for genome editing
WO2020186059A2 (en) * 2019-03-12 2020-09-17 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
EP3990636A1 (en) * 2019-06-28 2022-05-04 CRISPR Therapeutics AG Materials and methods for controlling gene editing
JP7667168B2 (ja) * 2020-02-24 2025-04-22 パイオニア ハイ－ブレッド インターナショナル， インコーポレイテッド ゲノム編集のための新規なｃｒｉｓｐｒ－ｃａｓシステム
WO2022018638A1 (en) 2020-07-21 2022-01-27 Crispr Therapeutics Ag Genome-editing compositions and methods to modulate faah for treatment of neurological disorders
JP2023540783A (ja) * 2020-09-09 2023-09-26 バーテックス ファーマシューティカルズ インコーポレイテッド デュシェンヌ型筋ジストロフィーの治療のための組成物及び方法
US20230392134A1 (en) 2020-09-30 2023-12-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
EP4240854A1 (en) 2020-11-06 2023-09-13 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of dm1 with slucas9 and sacas9
AU2021400745A1 (en) 2020-12-17 2023-07-20 Vertex Pharmaceuticals Incorporated Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
WO2022229851A1 (en) * 2021-04-26 2022-11-03 Crispr Therapeutics Ag Compositions and methods for using slucas9 scaffold sequences
WO2022238958A1 (en) 2021-05-12 2022-11-17 Crispr Therapeutics Ag Multiplex gene editing
EP4512403A1 (en) 2023-08-22 2025-02-26 Friedrich-Schiller-Universität Jena Neuropeptide b and w-receptor as a target for treating mood disorders and/or chronic stress

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