JP7033583B2 - ゲノム編集効率を高めるための方法、組成物及びキット - Google Patents

ゲノム編集効率を高めるための方法、組成物及びキット Download PDF

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JP7033583B2
JP7033583B2 JP2019501575A JP2019501575A JP7033583B2 JP 7033583 B2 JP7033583 B2 JP 7033583B2 JP 2019501575 A JP2019501575 A JP 2019501575A JP 2019501575 A JP2019501575 A JP 2019501575A JP 7033583 B2 JP7033583 B2 JP 7033583B2
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ノーゼハン アブドゥル－マナン，
デイビッド エー． ニューサム，
ジャック ズワレン，
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JP2019501575A 2016-07-13 2017-07-13 ゲノム編集効率を高めるための方法、組成物及びキット Active JP7033583B2 (ja)

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US201662361781P 2016-07-13 2016-07-13
US201662361961P 2016-07-13 2016-07-13
US62/361,961 2016-07-13
US62/361,781 2016-07-13
PCT/US2017/041979 WO2018013840A1 (en) 2016-07-13 2017-07-13 Methods, compositions and kits for increasing genome editing efficiency

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Families Citing this family (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9458450B2 (en) 2012-03-15 2016-10-04 Flodesign Sonics, Inc. Acoustophoretic separation technology using multi-dimensional standing waves
US10967298B2 (en) 2012-03-15 2021-04-06 Flodesign Sonics, Inc. Driver and control for variable impedence load
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9950282B2 (en) 2012-03-15 2018-04-24 Flodesign Sonics, Inc. Electronic configuration and control for acoustic standing wave generation
CA2935960C (en) 2014-01-08 2023-01-10 Bart Lipkens Acoustophoresis device with dual acoustophoretic chamber
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11021699B2 (en) 2015-04-29 2021-06-01 FioDesign Sonics, Inc. Separation using angled acoustic waves
US11474085B2 (en) 2015-07-28 2022-10-18 Flodesign Sonics, Inc. Expanded bed affinity selection
US11459540B2 (en) 2015-07-28 2022-10-04 Flodesign Sonics, Inc. Expanded bed affinity selection
EP3353296B1 (en) 2015-09-24 2020-11-04 Editas Medicine, Inc. Use of exonucleases to improve crispr/cas-mediated genome editing
CR20180323A (es) 2015-11-20 2018-08-06 Idorsia Pharmaceuticals Ltd Derivados de indol n-sustituídos como moduladores de los receptores de pge2
US11597924B2 (en) 2016-03-25 2023-03-07 Editas Medicine, Inc. Genome editing systems comprising repair-modulating enzyme molecules and methods of their use
WO2017180694A1 (en) 2016-04-13 2017-10-19 Editas Medicine, Inc. Cas9 fusion molecules gene editing systems, and methods of use thereof
US11085035B2 (en) 2016-05-03 2021-08-10 Flodesign Sonics, Inc. Therapeutic cell washing, concentration, and separation utilizing acoustophoresis
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
EP3484870B1 (en) 2016-07-13 2022-11-16 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
CN110494543A (zh) 2016-10-19 2019-11-22 弗洛设计声能学公司 通过声学的亲和细胞提取
CN110612296A (zh) 2017-05-18 2019-12-24 爱杜西亚药品有限公司 作为pge2受体调节剂的苯基衍生物
TW201900180A (zh) 2017-05-18 2019-01-01 瑞士商愛杜西亞製藥有限公司 嘧啶衍生物
AU2018268311B2 (en) 2017-05-18 2022-02-10 Idorsia Pharmaceuticals Ltd Pyrimidine derivatives as PGE2 receptor modulators
JP7093791B2 (ja) 2017-05-18 2022-06-30 イドーシア ファーマシューティカルズ リミテッド Ｐｇｅ２レセプター調節剤としてのベンゾフラン及びベンゾチオフェン誘導体
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
SG11202003907WA (en) 2017-12-14 2020-05-28 Flodesign Sonics Inc Acoustic transducer drive and controller
AU2019209292B2 (en) 2018-01-17 2023-10-05 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
JP7391854B2 (ja) 2018-01-17 2023-12-05 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 Ｄｎａ－ｐｋ阻害剤
BR112020014140A2 (pt) 2018-01-17 2020-12-01 Vertex Pharmaceuticals Incorporated inibidores de dna-pk
CN112867497A (zh) * 2018-05-07 2021-05-28 哈佛学院校长同事会 与体外β细胞分化相关的细胞群和基因表达
US11833225B2 (en) 2018-05-24 2023-12-05 Crispr Therapeutics Ag Methods and compositions for efficient gene deletion
CN110592141B (zh) * 2018-06-13 2023-07-07 中国科学院上海有机化学研究所 用于调控基因编辑效率的化合物及其应用
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
EP3640329A1 (en) * 2018-10-18 2020-04-22 Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) Gene editing based cancer treatment
EP4095259A1 (en) 2018-12-12 2022-11-30 Depixus Method of nucleic acid enrichment using site-specific nucleases followed by capture
WO2020205664A1 (en) * 2019-03-29 2020-10-08 Youhealth Biotech, Limited Compositions and methods for cellular reprogramming to rescue visual function
US20220220472A1 (en) * 2019-05-06 2022-07-14 The Regents Of The University Of Michigan Targeted therapy
KR20220070443A (ko) 2019-08-27 2022-05-31 버텍스 파마슈티칼스 인코포레이티드 반복성 dna와 연관된 장애의 치료용 조성물 및 방법
CN112553330B (zh) * 2019-09-26 2022-07-26 首都医科大学附属北京友谊医院 新型肿瘤相关转录因子zscan16及其在抑制肿瘤中的应用
WO2021062410A2 (en) * 2019-09-27 2021-04-01 The Broad Institute, Inc. Programmable polynucleotide editors for enhanced homologous recombination
TW202132565A (zh) 2019-11-01 2021-09-01 美商聖加莫治療股份有限公司 Ｇｉｎ重組酶變異體
CN110863011A (zh) * 2019-11-28 2020-03-06 浙江大学 干扰猪crtc3表达的质粒及腺病毒的构建方法及其用途
CN112877364B (zh) * 2019-11-29 2023-07-28 中国医学科学院药物研究所 软骨下骨细胞向关节软骨细胞直接转化的重编程诱导方案
CN111088290B (zh) * 2019-12-30 2021-10-08 同济大学 一种杜鹃素在基因编辑中的应用
EP4093731A1 (en) 2020-01-20 2022-11-30 Neophore Limited Isoindoline derivatives which bind to an atp binding site
JP7649562B2 (ja) * 2020-02-19 2025-03-21 国立大学法人京都大学 ゲノム編集された細胞の製造方法及びそのためのキット
EP4153741A1 (en) * 2020-05-21 2023-03-29 Oxford Genetics Limited Hdr enhancers
GB202008201D0 (en) * 2020-06-01 2020-07-15 Neophore Ltd Inhibitor compounds
CN113897357A (zh) * 2020-07-06 2022-01-07 北京大学 Twist1基因编辑系统及其在制备治疗三阴性乳腺癌的产品中的应用
AU2021409729A1 (en) * 2020-12-22 2023-07-13 Nchroma Bio, Inc. Compositions and methods for epigenetic editing
CN114717232A (zh) * 2020-12-22 2022-07-08 未来智人再生医学研究院(广州)有限公司 表达irf-1靶向抑制因子的多能干细胞及其衍生物与应用
CN112750498B (zh) * 2020-12-30 2022-06-24 同济大学 靶向逆转录引物结合位点从而抑制hiv病毒复制的方法
WO2022182959A1 (en) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/slucas9
WO2022182957A1 (en) 2021-02-26 2022-09-01 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/sacas9
CN113373148B (zh) * 2021-06-16 2022-08-26 中国人民解放军军事科学院军事医学研究院 一种调控app表达的靶标位点序列及其在防治ad上的应用
WO2023018637A1 (en) 2021-08-09 2023-02-16 Vertex Pharmaceuticals Incorporated Gene editing of regulatory elements
AU2022375818A1 (en) * 2021-10-29 2024-05-16 Cold Spring Harbor Laboratory Compositions and systems for rna-programable cell editing and methods of making and using same
CN114277119B (zh) * 2021-12-09 2023-05-16 浙江大学医学院附属邵逸夫医院 一种环状RNA circ-Arsb在制备防治骨质疏松产品中的应用
WO2023120530A1 (ja) * 2021-12-24 2023-06-29 国立大学法人大阪大学 相同組換えを利用したゲノム編集細胞の製造方法
JP2025504981A (ja) * 2022-02-01 2025-02-19 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド ヒト多能性幹細胞からの褐色脂肪細胞の生成
CN114524811B (zh) * 2022-03-16 2025-01-24 中国人民解放军军事科学院军事医学研究院 Lxh0307、lxh0308作为基因编辑的小分子抑制剂及其应用
CN114685494B (zh) * 2022-03-16 2024-04-16 中国人民解放军军事科学院军事医学研究院 SpCas9抑制剂的合成制备方法及其应用
CN116004542A (zh) * 2022-09-26 2023-04-25 苏州吉纳星辰生物技术有限公司 一种提高基因敲入效率的方法及其应用
CN116286975A (zh) * 2023-02-21 2023-06-23 浙江大学 一种基于dCas9的c-NHEJ定点抑制系统及应用
WO2024238691A2 (en) * 2023-05-15 2024-11-21 The Regents Of The University Of California Andp gene editing system
WO2025024785A2 (en) * 2023-07-26 2025-01-30 University Of Utah Research Foundation Compositions and methods for regulating znf865
CN119215053B (zh) * 2024-12-05 2025-04-01 南昌大学 Ye6144在制备治疗乳腺癌引起的骨骼肌萎缩药物中的应用
CN119752995B (zh) * 2024-12-26 2025-11-28 中国农业科学院棉花研究所 GhNFXL1基因在提高棉属植物耐盐性和/或耐冷性中的应用

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015058067A1 (en) 2013-10-17 2015-04-23 Vertex Pharmaceuticals Incorporated Co-crystals of (s)-n-methyl-8-(1-((2'-methyl-[4,5'-bipyrimidin]-6-yl)amino)propan-2-yl)quinoline-4-carboxamide and deuterated derivatives thereof as dna-pk inhibitors
JP2015514804A (ja) 2012-04-24 2015-05-21 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Ｖｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
WO2016081923A2 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Family Cites Families (92)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
US3773919A (en) 1969-10-23 1973-11-20 Du Pont Polylactide-drug mixtures
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
EP0770129B1 (en) 1994-01-18 2005-11-23 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2186118C (en) 1994-03-23 2010-10-19 Richard W. Hanson Compacted nucleic acids and their delivery to cells
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
CA2196419C (en) 1994-08-20 2007-08-21 Yen Choo Improvements in or relating to binding proteins for recognition of dna
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
AU746454B2 (en) 1998-03-02 2002-05-02 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
HK1049515B (en) 1999-12-06 2006-01-13 Sangamo Biosciences Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
DE60143192D1 (de) 2000-02-08 2010-11-18 Sangamo Biosciences Inc Zellen zur entdeckung von medikamenten
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
AU2003251286B2 (en) 2002-01-23 2007-08-16 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
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
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1737498A2 (en) 2004-04-08 2007-01-03 Sangamo Biosciences Inc. Zinc finger proteins for treatment of neuropathic pain
JP4903689B2 (ja) 2004-04-08 2012-03-28 サンガモ バイオサイエンシズ インコーポレイテッド 神経障害および神経変性症状を治療するための方法と組成物
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
EP2167523B1 (en) 2007-06-19 2014-07-23 Board of Supervisors of Louisiana State University and Agricultural and Mechanical College Synthesis and use of anti-reverse phosphorothioate analogs of the messenger rna cap
WO2010017562A2 (en) 2008-08-08 2010-02-11 Mayo Foundation For Medical Education And Research Induced pluripotent stem cells
WO2011097036A1 (en) 2010-02-08 2011-08-11 Sangamo Biosciences, Inc. Engineered cleavage half-domains
NZ629427A (en) 2012-02-29 2016-04-29 Sangamo Biosciences Inc Methods and compositions for treating huntington’s disease
JP2015527889A (ja) 2012-07-25 2015-09-24 ザ ブロード インスティテュート， インコーポレイテッド 誘導可能なｄｎａ結合タンパク質およびゲノム撹乱ツール、ならびにそれらの適用
US9181535B2 (en) 2012-09-24 2015-11-10 The Chinese University Of Hong Kong Transcription activator-like effector nucleases (TALENs)
ES2576126T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
CN114634950A (zh) 2012-12-12 2022-06-17 布罗德研究所有限公司 用于序列操纵的crispr-cas组分系统、方法以及组合物
WO2014093701A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
EP2931898B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP2896697B1 (en) 2012-12-12 2015-09-02 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
JP2016504026A (ja) 2012-12-12 2016-02-12 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための系、方法および最適化ガイド組成物のエンジニアリング
EP3434776A1 (en) 2012-12-12 2019-01-30 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
US10378027B2 (en) * 2013-03-15 2019-08-13 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
WO2014172458A1 (en) 2013-04-16 2014-10-23 University Of Washington Through Its Center For Commercialization Activating an alternative pathway for homology-directed repair to stimulate targeted gene correction and genome engineering
EP3725885A1 (en) 2013-06-17 2020-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
JP6665088B2 (ja) 2013-06-17 2020-03-13 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のための最適化されたＣＲＩＳＰＲ−Ｃａｓ二重ニッカーゼ系、方法および組成物
WO2014204723A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Oncogenic models based on delivery and use of the crispr-cas systems, vectors and compositions
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
JP6738729B2 (ja) 2013-06-17 2020-08-12 ザ・ブロード・インスティテュート・インコーポレイテッド 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化
MX2015017312A (es) 2013-06-17 2017-04-10 Broad Inst Inc Suministro y uso de composiciones, vectores y sistemas crispr-cas para la modificación dirigida y terapia hepáticas.
WO2014204724A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation
KR101448678B1 (ko) 2013-08-12 2014-10-08 김형기 단추 재봉실의 뿌리감기 장치
CA2925616A1 (en) 2013-09-27 2015-04-02 Mvpindex Inc. System and apparatus for assessing reach, engagement, conversation or other social metrics based on domain tailored evaluation of social media exposure
WO2015048577A2 (en) 2013-09-27 2015-04-02 Editas Medicine, Inc. Crispr-related methods and compositions
DK3180426T3 (da) 2014-08-17 2020-03-30 Broad Inst Inc Genomredigering ved anvendelse af cas9-nickaser
EP3484870B1 (en) 2016-07-13 2022-11-16 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2015514804A (ja) 2012-04-24 2015-05-21 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Ｖｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｄｎａ−ｐｋ阻害剤
JP2015534817A (ja) 2012-11-01 2015-12-07 ファクター バイオサイエンス インコーポレイテッド 細胞中でタンパク質を発現するための方法および生成物
WO2015058067A1 (en) 2013-10-17 2015-04-23 Vertex Pharmaceuticals Incorporated Co-crystals of (s)-n-methyl-8-(1-((2'-methyl-[4,5'-bipyrimidin]-6-yl)amino)propan-2-yl)quinoline-4-carboxamide and deuterated derivatives thereof as dna-pk inhibitors
WO2016081923A2 (en) 2014-11-21 2016-05-26 Regeneron Pharmaceuticals, Inc. METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Genome Medicine，2015年，7: 93，pp. 1-11

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