JP7236398B2 - ドナー修復鋳型多重ゲノム編集 - Google Patents

ドナー修復鋳型多重ゲノム編集 Download PDF

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JP7236398B2
JP7236398B2 JP2019564394A JP2019564394A JP7236398B2 JP 7236398 B2 JP7236398 B2 JP 7236398B2 JP 2019564394 A JP2019564394 A JP 2019564394A JP 2019564394 A JP2019564394 A JP 2019564394A JP 7236398 B2 JP7236398 B2 JP 7236398B2
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dna
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JP2020507342A (ja
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ベクスン リ，
アレクサンダー アストラカーン，
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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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    • C07K16/2878—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
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JP2019564394A 2017-02-15 2018-02-15 ドナー修復鋳型多重ゲノム編集 Active JP7236398B2 (ja)

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US201762459203P 2017-02-15 2017-02-15
US62/459,203 2017-02-15
PCT/US2018/018370 WO2018152325A1 (en) 2017-02-15 2018-02-15 Donor repair templates multiplex genome editing

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JP2020507342A JP2020507342A (ja) 2020-03-12
JP2020507342A5 JP2020507342A5 (https=) 2022-10-17
JP7236398B2 true JP7236398B2 (ja) 2023-03-09

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US (2) US11499149B2 (https=)
EP (2) EP3583203B1 (https=)
JP (1) JP7236398B2 (https=)
CN (1) CN110446781A (https=)
AU (1) AU2018221730B2 (https=)
CA (1) CA3053499A1 (https=)
DK (1) DK3583203T5 (https=)
ES (1) ES2969213T3 (https=)
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Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP4464338A3 (en) 2014-11-07 2025-02-12 Editas Medicine, Inc. Systems for improving crispr/cas-mediated genome-editing
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
US11236313B2 (en) 2016-04-13 2022-02-01 Editas Medicine, Inc. Cas9 fusion molecules, gene editing systems, and methods of use thereof
US11293021B1 (en) 2016-06-23 2022-04-05 Inscripta, Inc. Automated cell processing methods, modules, instruments, and systems
US9982279B1 (en) 2017-06-23 2018-05-29 Inscripta, Inc. Nucleic acid-guided nucleases
US10011849B1 (en) 2017-06-23 2018-07-03 Inscripta, Inc. Nucleic acid-guided nucleases
JP2020530979A (ja) 2017-06-30 2020-11-05 インスクリプタ， インコーポレイテッド 自動細胞処理方法、モジュール、機器及びシステム
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
US12312383B2 (en) 2018-04-18 2025-05-27 Altius Institute For Biomedical Sciences Animal pathogen-derived polypeptides and uses thereof for genetic engineering
US10557216B2 (en) 2018-04-24 2020-02-11 Inscripta, Inc. Automated instrumentation for production of T-cell receptor peptide libraries
US10858761B2 (en) 2018-04-24 2020-12-08 Inscripta, Inc. Nucleic acid-guided editing of exogenous polynucleotides in heterologous cells
US12264181B2 (en) 2018-06-27 2025-04-01 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof
CA3108767A1 (en) 2018-06-30 2020-01-02 Inscripta, Inc. Instruments, modules, and methods for improved detection of edited sequences in live cells
US11142740B2 (en) 2018-08-14 2021-10-12 Inscripta, Inc. Detection of nuclease edited sequences in automated modules and instruments
SG11202103917VA (en) 2018-10-16 2021-05-28 Blueallele Llc Methods for targeted insertion of dna in genes
US11214781B2 (en) 2018-10-22 2022-01-04 Inscripta, Inc. Engineered enzyme
CN113840920A (zh) * 2019-03-14 2021-12-24 加利福尼亚大学董事会 合并的敲入筛选和在内源基因座控制下共表达的异源多肽
US11001831B2 (en) 2019-03-25 2021-05-11 Inscripta, Inc. Simultaneous multiplex genome editing in yeast
AU2020247900A1 (en) 2019-03-25 2021-11-04 Inscripta, Inc. Simultaneous multiplex genome editing in yeast
AU2020288623A1 (en) 2019-06-06 2022-01-06 Inscripta, Inc. Curing for recursive nucleic acid-guided cell editing
CN110358792A (zh) * 2019-07-19 2019-10-22 华中农业大学 外源基因定点整合至actb基因下游的打靶载体构建方法及其应用
US11203762B2 (en) 2019-11-19 2021-12-21 Inscripta, Inc. Methods for increasing observed editing in bacteria
AU2020407048A1 (en) 2019-12-18 2022-06-09 Inscripta, Inc. Cascade/dCas3 complementation assays for in vivo detection of nucleic acid-guided nuclease edited cells
US11225674B2 (en) 2020-01-27 2022-01-18 Inscripta, Inc. Electroporation modules and instrumentation
US20210332388A1 (en) 2020-04-24 2021-10-28 Inscripta, Inc. Compositions, methods, modules and instruments for automated nucleic acid-guided nuclease editing in mammalian cells
US11299731B1 (en) 2020-09-15 2022-04-12 Inscripta, Inc. CRISPR editing to embed nucleic acid landing pads into genomes of live cells
US11512297B2 (en) 2020-11-09 2022-11-29 Inscripta, Inc. Affinity tag for recombination protein recruitment
AU2021415461A1 (en) 2021-01-04 2023-08-17 Inscripta, Inc. Mad nucleases
EP4274890A4 (en) 2021-01-07 2024-11-27 Inscripta, Inc. MAD-NUCLEASES
US11884924B2 (en) 2021-02-16 2024-01-30 Inscripta, Inc. Dual strand nucleic acid-guided nickase editing
JP2024543164A (ja) * 2021-11-26 2024-11-19 イミュネクサス・セラピューティクス・リミテッド 細胞表面上の結合ドメイン分子
EP4493706A4 (en) * 2022-03-14 2026-03-18 Univ California INTEGRATION OF LARGE NUCLEIC ACIDS INTO GENOMES
CN116212022A (zh) * 2022-12-19 2023-06-06 中国医学科学院医学生物学研究所 一种芳香烃受体基因AhR作为治疗靶点在制备抗HSV-1病毒药物中的应用
CN118538425B (zh) * 2024-06-06 2024-10-29 北京大学第一医院(北京大学第一临床医学院) 一种vhl综合征肾癌的诊断模型及其应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015525065A (ja) 2012-05-25 2015-09-03 セレクティスＣｅｌｌｅｃｔｉｓ Ｔｃｒアルファ欠損ｔ細胞を増殖させるためのプレｔアルファまたはその機能性変種の使用
JP2016520308A (ja) 2013-05-13 2016-07-14 セレクティスＣｅｌｌｅｃｔｉｓ 免疫療法のために同種異系かつ高活性のｔ細胞を操作するための方法
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016176690A2 (en) 2015-04-30 2016-11-03 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases

Family Cites Families (46)

* 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
US5061620A (en) 1990-03-30 1991-10-29 Systemix, Inc. Human hematopoietic stem cell
US5635387A (en) 1990-04-23 1997-06-03 Cellpro, Inc. Methods and device for culturing human hematopoietic cells and their precursors
ES2061416T3 (es) 1990-10-12 1997-03-01 Max Planck Gesellschaft Ribozimas modificadas.
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5460964A (en) 1992-04-03 1995-10-24 Regents Of The University Of Minnesota Method for culturing hematopoietic cells
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
US5503022A (en) 1993-01-25 1996-04-02 Barone; Larry A. Marine impeller tester
US5409813A (en) 1993-09-30 1995-04-25 Systemix, Inc. Method for mammalian cell separation from a mixture of cell populations
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
US5677136A (en) 1994-11-14 1997-10-14 Systemix, Inc. Methods of obtaining compositions enriched for hematopoietic stem cells, compositions derived therefrom and methods of use thereof
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6187757B1 (en) 1995-06-07 2001-02-13 Ariad Pharmaceuticals, Inc. Regulation of biological events using novel compounds
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
WO2002088346A2 (en) 2001-05-01 2002-11-07 National Research Council Of Canada A system for inducible expression in eukaryotic cells
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
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
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
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
EP2449135B1 (en) 2009-06-30 2016-01-06 Sangamo BioSciences, Inc. Rapid screening of biologically active nucleases and isolation of nuclease-modified cells
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
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
CN104781789B (zh) 2012-12-20 2018-06-05 三菱电机株式会社 车载装置
EP3925618A1 (en) 2013-07-29 2021-12-22 2seventy bio, Inc. Multipartite signaling proteins and uses thereof
US9108442B2 (en) 2013-08-20 2015-08-18 Ricoh Company, Ltd. Image forming apparatus
BR112016024945A2 (pt) 2014-04-28 2017-10-24 Recombinetics Inc edição de gene multiplex em suínos
KR102531016B1 (ko) 2014-11-21 2023-05-10 리제너론 파마슈티칼스 인코포레이티드 쌍 형성된 가이드 rna를 사용하는 표적화된 유전자 변형을 위한 방법 및 조성물
GB2531454A (en) 2016-01-10 2016-04-20 Snipr Technologies Ltd Recombinogenic nucleic acid strands in situ
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
KR102451510B1 (ko) 2016-09-08 2022-10-07 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015525065A (ja) 2012-05-25 2015-09-03 セレクティスＣｅｌｌｅｃｔｉｓ Ｔｃｒアルファ欠損ｔ細胞を増殖させるためのプレｔアルファまたはその機能性変種の使用
JP2016520308A (ja) 2013-05-13 2016-07-14 セレクティスＣｅｌｌｅｃｔｉｓ 免疫療法のために同種異系かつ高活性のｔ細胞を操作するための方法
WO2016164356A1 (en) 2015-04-06 2016-10-13 The Board Of Trustees Of The Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2016176690A2 (en) 2015-04-30 2016-11-03 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Molecular Therapy-Nucleic Acids，2016年，Vol.5, e352，pp.1-10

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