JP2024518793A - 向上した安定性を有するcas9エフェクタータンパク質 - Google Patents

向上した安定性を有するcas9エフェクタータンパク質 Download PDF

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Publication number
JP2024518793A
JP2024518793A JP2023571310A JP2023571310A JP2024518793A JP 2024518793 A JP2024518793 A JP 2024518793A JP 2023571310 A JP2023571310 A JP 2023571310A JP 2023571310 A JP2023571310 A JP 2023571310A JP 2024518793 A JP2024518793 A JP 2024518793A
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Prior art keywords
nuclear localization
localization signal
protein
effector protein
cas9 effector
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JP2023571310A
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English (en)
Japanese (ja)
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JP2024518793A5 (https=
Inventor
シエンスキ,グジェゴシュ
マレスカ,マルチェッロ
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AstraZeneca AB
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AstraZeneca AB
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Publication of JP2024518793A publication Critical patent/JP2024518793A/ja
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2023571310A 2021-05-27 2022-05-26 向上した安定性を有するcas9エフェクタータンパク質 Pending JP2024518793A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163193620P 2021-05-27 2021-05-27
US63/193,620 2021-05-27
PCT/EP2022/064368 WO2022248645A1 (en) 2021-05-27 2022-05-26 Cas9 effector proteins with enhanced stability

Publications (2)

Publication Number Publication Date
JP2024518793A true JP2024518793A (ja) 2024-05-02
JP2024518793A5 JP2024518793A5 (https=) 2025-07-22

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ID=82117168

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JP2023571310A Pending JP2024518793A (ja) 2021-05-27 2022-05-26 向上した安定性を有するcas9エフェクタータンパク質

Country Status (5)

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US (1) US20240384246A1 (https=)
EP (1) EP4347805A1 (https=)
JP (1) JP2024518793A (https=)
CN (1) CN117396602A (https=)
WO (1) WO2022248645A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115716880A (zh) * 2022-12-07 2023-02-28 云舟生物科技(广州)股份有限公司 一种核定位荧光蛋白及其应用
EP4678742A1 (en) * 2024-07-10 2026-01-14 Astrazeneca AB Improved cas9 proteins

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US6007845A (en) 1994-07-22 1999-12-28 Massachusetts Institute Of Technology Nanoparticles and microparticles of non-linear hydrophilic-hydrophobic multiblock copolymers
US5855913A (en) 1997-01-16 1999-01-05 Massachusetts Instite Of Technology Particles incorporating surfactants for pulmonary drug delivery
US5895309A (en) 1998-02-09 1999-04-20 Spector; Donald Collapsible hula-hoop
JP2005511761A (ja) * 2001-07-10 2005-04-28 ノース・キャロライナ・ステイト・ユニヴァーシティ ナノ粒子送達ビヒクル
JP2008078613A (ja) 2006-08-24 2008-04-03 Rohm Co Ltd 窒化物半導体の製造方法及び窒化物半導体素子
EP3269395A1 (en) 2008-11-07 2018-01-17 Massachusetts Institute Of Technology Aminoalcohol lipidoids and uses thereof
US9193827B2 (en) 2010-08-26 2015-11-24 Massachusetts Institute Of Technology Poly(beta-amino alcohols), their preparation, and uses thereof
EP2691443B1 (en) 2011-03-28 2021-02-17 Massachusetts Institute of Technology Conjugated lipomers and uses thereof
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CA2913234A1 (en) 2013-05-22 2014-11-27 Northwestern University Rna-directed dna cleavage and gene editing by cas9 enzyme from neisseria meningitidis
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
EP4321623A3 (en) * 2016-07-15 2024-05-15 Salk Institute for Biological Studies Methods and compositions for genome editing in non-dividing cells
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20230125856A (ko) 2016-12-23 2023-08-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Pcsk9의 유전자 편집
CN106632693B (zh) * 2017-01-19 2021-05-25 上海科技大学 具有多个核定位序列的SpyCas9蛋白及其应用
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
CN120310773A (zh) 2017-11-16 2025-07-15 阿斯利康(瑞典)有限公司 用于改善基于Cas9的敲入策略的有效性的组合物和方法
WO2019213273A1 (en) * 2018-05-01 2019-11-07 The Children's Medical Center Corporation Enhanced bcl11a rnp / crispr delivery & editing using a 3xnls-cas9
WO2020191233A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences

Also Published As

Publication number Publication date
US20240384246A1 (en) 2024-11-21
CN117396602A (zh) 2024-01-12
WO2022248645A1 (en) 2022-12-01
EP4347805A1 (en) 2024-04-10

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