JP2022513750A - ホーミングエンドヌクレアーゼバリアント - Google Patents

ホーミングエンドヌクレアーゼバリアント Download PDF

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Publication number
JP2022513750A
JP2022513750A JP2021532870A JP2021532870A JP2022513750A JP 2022513750 A JP2022513750 A JP 2022513750A JP 2021532870 A JP2021532870 A JP 2021532870A JP 2021532870 A JP2021532870 A JP 2021532870A JP 2022513750 A JP2022513750 A JP 2022513750A
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onui
variant
amino acid
group
parent
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JP2022513750A5 (https=
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ジョーダン ジャージョル,
カイル ヘイヴンズ,
コンスタンティン クリュソストモウ,
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2セブンティ バイオ インコーポレイテッド
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Publication of JP2022513750A5 publication Critical patent/JP2022513750A5/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
    • 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
    • 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
    • C12N15/62DNA sequences coding for fusion proteins
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
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  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021532870A 2018-12-10 2019-12-09 ホーミングエンドヌクレアーゼバリアント Pending JP2022513750A (ja)

Priority Applications (1)

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JP2025016020A JP2025072470A (ja) 2018-12-10 2025-02-03 ホーミングエンドヌクレアーゼバリアント

Applications Claiming Priority (3)

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US201862777476P 2018-12-10 2018-12-10
US62/777,476 2018-12-10
PCT/US2019/065211 WO2020123371A2 (en) 2018-12-10 2019-12-09 Homing endonuclease variants

Related Child Applications (1)

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JP2025016020A Division JP2025072470A (ja) 2018-12-10 2025-02-03 ホーミングエンドヌクレアーゼバリアント

Publications (2)

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JP2022513750A true JP2022513750A (ja) 2022-02-09
JP2022513750A5 JP2022513750A5 (https=) 2022-12-16

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JP2021532870A Pending JP2022513750A (ja) 2018-12-10 2019-12-09 ホーミングエンドヌクレアーゼバリアント
JP2025016020A Pending JP2025072470A (ja) 2018-12-10 2025-02-03 ホーミングエンドヌクレアーゼバリアント

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US (2) US12404500B2 (https=)
EP (1) EP3893922A4 (https=)
JP (2) JP2022513750A (https=)
KR (1) KR20210102925A (https=)
CN (1) CN113329760A (https=)
AU (1) AU2019397364B8 (https=)
CA (1) CA3122278A1 (https=)
IL (1) IL283720B2 (https=)
WO (1) WO2020123371A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024503249A (ja) * 2020-12-21 2024-01-25 2セブンティ バイオ インコーポレイテッド 部位特異的変異導入のための組成物及び方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110256607A1 (en) * 2010-04-16 2011-10-20 Georg Hausner Homing endonucleases
US20170298329A1 (en) * 2012-02-24 2017-10-19 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2018022619A1 (en) * 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
WO2018035141A1 (en) * 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018049226A1 (en) * 2016-09-08 2018-03-15 Bluebird Bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use

Family Cites Families (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4751180A (en) 1985-03-28 1988-06-14 Chiron Corporation Expression using fused genes providing for protein product
US4935233A (en) 1985-12-02 1990-06-19 G. D. Searle And Company Covalently linked polypeptide cell modulators
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh Targeted hsv vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
US6692736B2 (en) 2000-03-24 2004-02-17 Cell Genesys, Inc. Cell-specific adenovirus vectors comprising an internal ribosome entry site
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
CN101679959A (zh) * 2007-02-19 2010-03-24 赛莱克蒂斯公司 具有新的底物特异性的laglidadg归巢核酸内切酶变体及其用途
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
EP2524037B1 (en) 2010-01-12 2018-05-16 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
US20140148361A1 (en) 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
US20130337454A1 (en) 2010-10-27 2013-12-19 Philippe Duchateau Method for increasing the efficiency of double-strand break-induced mutagenesis
US8673557B2 (en) 2011-02-28 2014-03-18 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
BR112014029417B1 (pt) 2012-05-25 2023-03-07 Cellectis Método ex vivo para a preparação de células t para imunoterapia
US20140015679A1 (en) 2012-07-15 2014-01-16 Green Badge LLC Specialty Plant Moisture Sensing
AU2014266833B2 (en) 2013-05-13 2020-07-02 Cellectis Methods for engineering highly active T cell for immunotherapy
ES2716867T3 (es) 2013-05-31 2019-06-17 Cellectis Sa Endonucleasa de asentamiento LAGLIDADG que escinde el gen de receptor de células T alfa y usos de la misma
CA2913871C (en) 2013-05-31 2021-07-13 Cellectis A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
EP3925618A1 (en) 2013-07-29 2021-12-22 2seventy bio, Inc. Multipartite signaling proteins and uses thereof
CN112552401B (zh) 2013-09-13 2023-08-25 广州百济神州生物制药有限公司 抗pd1抗体及其作为治疗剂与诊断剂的用途
JP2019509738A (ja) 2016-03-11 2019-04-11 ブルーバード バイオ, インコーポレイテッド ゲノム編集された免疫エフェクター細胞
US20190161530A1 (en) 2016-04-07 2019-05-30 Bluebird Bio, Inc. Chimeric antigen receptor t cell compositions
AU2017312132A1 (en) 2016-08-19 2019-03-21 Bluebird Bio, Inc. Genome editing enhancers
WO2018039333A1 (en) * 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
US11591582B2 (en) * 2016-10-11 2023-02-28 2Seventy Bio, Inc. TCRα homing endonuclease variants
MA46543A (fr) 2016-10-17 2019-08-21 Bluebird Bio Inc Variants de l'endonucléase tgfbéta r2, compositions et procédés d'utilisation
CN106749666B (zh) 2016-12-22 2018-12-25 福州大学 一种人源程序性死亡受体hPD-1单克隆抗体
CN106939049B (zh) 2017-04-20 2019-10-01 苏州思坦维生物技术股份有限公司 拮抗抑制人pd-1抗原与其配体结合的单克隆抗体及其制备方法与应用
MA48797A (fr) 2017-05-25 2020-04-08 Bluebird Bio Inc Variants de l'endonucléase cblb , compositions et procédés d'utilisation
US11779654B2 (en) * 2017-10-04 2023-10-10 2Seventy Bio, Inc. PCSK9 endonuclease variants, compositions, and methods of use
WO2020072059A1 (en) * 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
IL283724B1 (en) 2018-12-10 2026-02-01 Genetix Biotherapeutics Inc pdcd-1 homing endonuclease variants

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110256607A1 (en) * 2010-04-16 2011-10-20 Georg Hausner Homing endonucleases
US20170298329A1 (en) * 2012-02-24 2017-10-19 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
WO2018022619A1 (en) * 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
WO2018035141A1 (en) * 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
WO2018049226A1 (en) * 2016-09-08 2018-03-15 Bluebird Bio, Inc. Pd-1 homing endonuclease variants, compositions, and methods of use

Also Published As

Publication number Publication date
IL283720B1 (en) 2025-01-01
US20250368973A1 (en) 2025-12-04
CN113329760A (zh) 2021-08-31
JP2025072470A (ja) 2025-05-09
AU2019397364B1 (en) 2025-07-17
WO2020123371A2 (en) 2020-06-18
IL283720B2 (en) 2025-05-01
EP3893922A2 (en) 2021-10-20
US12404500B2 (en) 2025-09-02
KR20210102925A (ko) 2021-08-20
US20220119784A1 (en) 2022-04-21
EP3893922A4 (en) 2022-09-14
WO2020123371A3 (en) 2020-08-20
AU2019397364A1 (en) 2021-06-24
CA3122278A1 (en) 2020-06-18
AU2019397364B8 (en) 2025-08-07
IL283720A (en) 2021-07-29

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