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MX2016016133A - Métodos y composiciones para modificar un locus dirigido. - Google Patents

Métodos y composiciones para modificar un locus dirigido.

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Publication number
MX2016016133A
MX2016016133A MX2016016133A MX2016016133A MX2016016133A MX 2016016133 A MX2016016133 A MX 2016016133A MX 2016016133 A MX2016016133 A MX 2016016133A MX 2016016133 A MX2016016133 A MX 2016016133A MX 2016016133 A MX2016016133 A MX 2016016133A
Authority
MX
Mexico
Prior art keywords
cell
methods
polynucleotide
compositions
recognition site
Prior art date
Application number
MX2016016133A
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English (en)
Other versions
MX385689B (es
Inventor
Frendewey David
Droguett Gustavo
Auerbach Wojtek
KUNO Junko
M Valenzuela David
Gagliardi Anthony
Original Assignee
Regeneron Pharma
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Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of MX2016016133A publication Critical patent/MX2016016133A/es
Publication of MX385689B publication Critical patent/MX385689B/es

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    • C12N15/09Recombinant DNA-technology
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    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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Abstract

Se proporcionan métodos y composiciones para modificar uno o más loci objetivo en una célula. Tales métodos comprenden proporcionar una célula que comprende un primer polinucleótido que codifica un primer marcador de selección unido operativamente a un primer promotor activo en la célula, en donde el primer polinucleótido comprende, además, un primer sitio de reconocimiento para un primer agente nucleasa. Un primer agente nucleasa se introduce en una célula, en donde el primer agente nucleasa induce una mella o una ruptura de doble hebra en el primer sitio de reconocimiento. En la célula se introduce, además, un primer vector de direccionamiento que comprende un primer polinucleótido inserto flanqueado por un primer y un segunda brazo de homología que corresponden a un primer y un segundo sitio objetivo situados en suficiente proximidad al primer sitio de reconocimiento. Después, se identifica al menos una célula que comprende en su genoma el primer polinucleótido inserto integrado en el locus objetivo.
MX2016016133A 2014-06-06 2015-06-05 Métodos y composiciones para modificar un locus dirigido. MX385689B (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462008832P 2014-06-06 2014-06-06
US201462017916P 2014-06-27 2014-06-27
PCT/US2015/034503 WO2015188109A1 (en) 2014-06-06 2015-06-05 Methods and compositions for modifying a targeted locus

Publications (2)

Publication Number Publication Date
MX2016016133A true MX2016016133A (es) 2017-08-04
MX385689B MX385689B (es) 2025-03-18

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MX2016016133A MX385689B (es) 2014-06-06 2015-06-05 Métodos y composiciones para modificar un locus dirigido.

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US (4) US10106820B2 (es)
EP (2) EP3152312B1 (es)
JP (2) JP6688231B2 (es)
KR (1) KR102374379B1 (es)
CN (2) CN106795521B (es)
AU (1) AU2015269187B2 (es)
BR (1) BR112016028564A2 (es)
CA (1) CA2950173C (es)
CY (1) CY1122897T1 (es)
DK (1) DK3152312T3 (es)
ES (2) ES3019688T3 (es)
HR (1) HRP20200529T1 (es)
HU (1) HUE049776T2 (es)
IL (1) IL249042B (es)
LT (1) LT3152312T (es)
MX (1) MX385689B (es)
NZ (1) NZ727481A (es)
PL (1) PL3152312T3 (es)
PT (1) PT3152312T (es)
RS (1) RS60359B1 (es)
RU (1) RU2704283C9 (es)
SG (2) SG11201609634YA (es)
SI (1) SI3152312T1 (es)
SM (1) SMT202000264T1 (es)
WO (1) WO2015188109A1 (es)

Families Citing this family (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
CN109913495B (zh) 2013-02-20 2022-11-25 瑞泽恩制药公司 大鼠的遗传修饰
SG10201808935WA (en) 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
JP6174811B2 (ja) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ゲノムの標的改変のための方法及び組成物
SMT201800653T1 (it) 2013-12-11 2019-01-11 Regeneron Pharma Metodi e composizioni per la modificazione indirizzata a bersaglio di un genoma
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
MX385689B (es) 2014-06-06 2025-03-18 Regeneron Pharma Métodos y composiciones para modificar un locus dirigido.
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3561052A1 (en) 2014-10-15 2019-10-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
MX388784B (es) 2014-12-19 2025-03-20 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.
AU2016333898B2 (en) * 2015-10-05 2020-11-12 Precision Biosciences, Inc. Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
DK3359660T3 (en) 2015-10-05 2020-02-17 Prec Biosciences Inc Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene
IL258821B (en) 2015-10-23 2022-07-01 Harvard College Nucleobase editors and their uses
CN109311984A (zh) * 2016-03-11 2019-02-05 蓝鸟生物公司 基因组编辑的免疫效应细胞
EP3440194A1 (en) * 2016-04-04 2019-02-13 ETH Zurich Mammalian cell line for protein production and library generation
SG11201808831TA (en) 2016-04-15 2018-11-29 Memorial Sloan Kettering Cancer Center Transgenic t cell and chimeric antigen receptor t cell compositions and related methods
MX2018014172A (es) 2016-05-20 2019-08-22 Regeneron Pharma Métodos para romper la tolerancia inmunológica usando múltiples arn guías.
CN109640644B (zh) 2016-06-03 2021-10-26 瑞泽恩制药公司 表达外源末端脱氧核苷酸转移酶的非人动物
CN107513538A (zh) * 2016-06-17 2017-12-26 北京大学 基因敲除方法
EP3478852B1 (en) 2016-07-01 2020-08-12 Microsoft Technology Licensing, LLC Storage through iterative dna editing
US11359234B2 (en) 2016-07-01 2022-06-14 Microsoft Technology Licensing, Llc Barcoding sequences for identification of gene expression
US20180004537A1 (en) 2016-07-01 2018-01-04 Microsoft Technology Licensing, Llc Molecular State Machines
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
JP7588390B2 (ja) 2016-10-14 2024-11-22 プレジデント アンド フェローズ オブ ハーバード カレッジ 核酸塩基エディターのaav送達
CA3038720A1 (en) 2016-11-04 2018-07-12 Regeneron Pharmaceuticals, Inc. Non-human animals having an engineered immunoglobulin lambda light chain locus
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CN118140872A (zh) 2017-01-19 2024-06-07 欧莫诺艾比公司 来自具有多个重链免疫球蛋白基因座的转基因啮齿类动物的人抗体
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
WO2018151514A1 (ko) * 2017-02-16 2018-08-23 고려대학교 산학협력단 골재생 효능이 우수한 골질환 예방 또는 치료용 조성물
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
CN110662556A (zh) 2017-03-09 2020-01-07 哈佛大学的校长及成员们 癌症疫苗
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
US20200056190A1 (en) * 2017-03-16 2020-02-20 Pfizer Inc. Tyrosine prototrophy
BR112019019655A2 (pt) 2017-03-23 2020-04-22 Harvard College editores de nucleobase que compreendem proteínas de ligação a dna programáveis por ácido nucleico
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
WO2019005957A1 (en) 2017-06-30 2019-01-03 Precision Biosciences, Inc. GENETICALLY MODIFIED T CELLS COMPRISING A MODIFIED INTRON IN THE ALPHA T CELL RECEPTOR GENE
CN111094573A (zh) * 2017-07-12 2020-05-01 梅约医学教育与研究基金会 有效靶向敲入或基因置换的材料和方法
JP2020534795A (ja) 2017-07-28 2020-12-03 プレジデント アンド フェローズ オブ ハーバード カレッジ ファージによって支援される連続的進化(pace)を用いて塩基編集因子を進化させるための方法および組成物
CN111247247A (zh) * 2017-08-21 2020-06-05 国立大学法人德岛大学 使用核苷酸靶识别的靶序列特异性改变技术
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US20190119701A1 (en) * 2017-09-08 2019-04-25 Life Technologies Corporation Methods for improved homologous recombination and compositions thereof
KR20250107288A (ko) 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US20210017544A1 (en) * 2017-12-22 2021-01-21 Novozymes A/S Counter-Selection by Inhibition of Conditionally Essential Genes
CN107904208B (zh) * 2017-12-25 2019-11-01 云舟生物科技(广州)有限公司 细胞表型研究用的细胞克隆及其筛选方法和应用
KR102839528B1 (ko) * 2018-01-23 2025-07-29 기초과학연구원 연장된 단일 가이드 rna 및 그 용도
WO2019152843A1 (en) 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
CA3093850A1 (en) 2018-03-26 2019-10-03 Regeneron Pharmaceuticals, Inc. Humanized rodents for testing therapeutic agents
JP7304888B2 (ja) 2018-04-12 2023-07-07 プレシジョン バイオサイエンシズ,インク. ヒトt細胞受容体アルファ定常領域遺伝子に特異性を有する最適化された操作されたヌクレアーゼ
EP3788152A4 (en) * 2018-05-04 2022-03-09 Locus Biosciences, Inc. METHODS AND COMPOSITIONS FOR KILLING A TARGET BACTERIA
KR20210045360A (ko) 2018-05-16 2021-04-26 신테고 코포레이션 가이드 rna 설계 및 사용을 위한 방법 및 시스템
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN117904208A (zh) * 2018-12-30 2024-04-19 豪夫迈·罗氏有限公司 基于可检测标签与靶蛋白的CRISPR/Cas控制的整合而选择细胞的方法
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
EP3942042A1 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
IL286917B (en) * 2019-04-04 2022-09-01 Regeneron Pharma Methods for scarless introduction of targeted modifications into targeting vectors
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220039653A (ko) * 2019-05-13 2022-03-29 래피드 제노믹스 엘엘씨 표적 게놈 영역의 포집 및 분석
EP4004216A1 (en) * 2019-07-25 2022-06-01 Precision BioSciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021119594A1 (en) * 2019-12-13 2021-06-17 The Regents Of The University Of California Reducing antibiotic resistance in bacteria using pro-active genetics
IL271656A (en) 2019-12-22 2021-06-30 Yeda Res & Dev System and methods for identifying cells that have undergone genome editing
WO2021226558A1 (en) 2020-05-08 2021-11-11 The Broad Institute, Inc. Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
US20240018493A1 (en) * 2020-11-10 2024-01-18 The Board Of Trustees Of The Leland Stanford Junior University Knock-in of large dna for long-term high genomic expression
CN114763559B (zh) * 2021-01-15 2024-07-19 中国农业大学 一种不依赖于同源重组的靶向性基因捕获系统及其应用
JP2024533683A (ja) * 2021-09-24 2024-09-12 イムノカン バイオテック カンパニー リミテッド 大型染色体の導入方法と改変染色体およびそれを用いた生物
CN121079425A (zh) * 2023-05-10 2025-12-05 武田药品工业株式会社 可再编辑的模板、细胞、组合物和制备方法

Family Cites Families (103)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU664976B2 (en) 1990-08-29 1995-12-14 Gene Pharming Europe Bv Homologous recombination in mammalian cells
US5830729A (en) 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells
WO1999005266A2 (en) 1997-07-26 1999-02-04 Wisconsin Alumni Research Foundation Trans-species nuclear transfer
DE69830065T2 (de) * 1997-09-16 2006-01-19 Egea Biosciences, LLC, San Diego Methoden zur kompletten chemischen synthese und zusammensetzung von genen und genomen
EP1141360A4 (en) 1998-12-31 2002-11-06 David Gladstone Inst Transgenic rodents and rodent cell lines that express HIV receptors.
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US20030104526A1 (en) 1999-03-24 2003-06-05 Qiang Liu Position dependent recognition of GNN nucleotide triplets by zinc fingers
DE60023936T2 (de) 1999-12-06 2006-05-24 Sangamo Biosciences Inc., Richmond Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
US7105348B2 (en) 2000-10-31 2006-09-12 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US6596541B2 (en) 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US20050144655A1 (en) 2000-10-31 2005-06-30 Economides Aris N. Methods of modifying eukaryotic cells
US6586251B2 (en) 2000-10-31 2003-07-01 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
JP2004537260A (ja) 2000-12-07 2004-12-16 サンガモ バイオサイエンシーズ, インコーポレイテッド ジンクフィンガータンパク質による血管新生の調節
CA2435394C (en) 2001-01-22 2018-01-09 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
WO2002057308A2 (en) 2001-01-22 2002-07-25 Sangamo Biosciences, Inc. Zinc finger polypeptides and their use
AUPR451401A0 (en) 2001-04-20 2001-05-24 Monash University A method of nuclear transfer
AU2002355155A1 (en) 2001-07-24 2003-02-17 Affinium Pharmaceuticals Inc. Methods for gene disruption and uses thereof
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
EP1485475B2 (en) 2002-03-15 2017-09-20 Cellectis Hybrid and single chain meganucleases and use thereof
AU2003218382B2 (en) 2002-03-21 2007-12-13 Sangamo Therapeutics, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7612250B2 (en) 2002-07-29 2009-11-03 Trustees Of Tufts College Nuclear transfer embryo formation method
EP1581610A4 (en) 2002-09-05 2009-05-27 California Inst Of Techn USE OF CHIMERIC NUCLEASES TO STIMULATE GEN-TARGETING
WO2004031346A2 (en) 2002-09-06 2004-04-15 Fred Hutchinson Cancer Research Center Methods and compositions concerning designed highly-specific nucleic acid binding proteins
US20030175968A1 (en) 2002-10-30 2003-09-18 Golic Kent G. Gene targeting method
AU2004204509A1 (en) 2003-01-13 2004-07-29 Mario R. Capecchi Persistent expression of candidate molecule in proliferating stem and progenitor cells for delivery of therapeutic products
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP1591521A1 (en) 2004-04-30 2005-11-02 Cellectis I-Dmo I derivatives with enhanced activity at 37 degrees C and use thereof
US20060063231A1 (en) 2004-09-16 2006-03-23 Sangamo Biosciences, Inc. Compositions and methods for protein production
PL2767161T3 (pl) 2004-10-19 2018-09-28 Regeneron Pharmaceuticals, Inc. Sposób wytwarzania zwierzęcia homozygotycznego pod względem modyfikacji genetycznej
FR2879622B1 (fr) 2004-12-17 2008-02-01 Agronomique Inst Nat Rech Procede in vitro de production d'ovocytes ou d'oeufs presentant une modification genomique ciblee
WO2006097784A1 (en) 2005-03-15 2006-09-21 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
WO2006097854A1 (en) 2005-03-15 2006-09-21 Cellectis Heterodimeric meganucleases and use thereof
US20070004041A1 (en) 2005-06-30 2007-01-04 Codon Devices, Inc. Heirarchical assembly methods for genome engineering
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
WO2007117410A2 (en) 2006-03-31 2007-10-18 Medarex, Inc. Transgenic animals expressing chimeric antibodies for use in preparing human antibodies
CN101117633B (zh) 2006-08-03 2011-07-20 上海交通大学附属儿童医院 一种细胞核移植方法
MX2009006303A (es) 2006-12-14 2009-10-21 Dow Agrosciences Llc Proteinas de dedo de zinc no canonicas optimizadas.
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
SG182144A1 (en) 2007-06-01 2012-07-30 Omt Inc Compositions and methods for inhibiting endogenous immunoglobulin genes and producing transgenic human idiotype antibodies
WO2009076464A2 (en) 2007-12-10 2009-06-18 Aliva Biopharmaceuticals, Inc. Methods for sequential replacement of targeted region by homologous recombination
US9206404B2 (en) 2008-12-04 2015-12-08 Sangamo Biosciences, Inc. Method of deleting an IgM gene in an isolated rat cell
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
WO2010107493A2 (en) 2009-03-20 2010-09-23 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
WO2010127111A1 (en) 2009-04-30 2010-11-04 The Trustees Of Comlumbia University In The City Of New York In vivo assembly of dna via homologous recombination
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
ES2948572T3 (es) * 2009-07-08 2023-09-14 Kymab Ltd Modelos de roedores y moléculas terapéuticas
US20120178647A1 (en) 2009-08-03 2012-07-12 The General Hospital Corporation Engineering of zinc finger arrays by context-dependent assembly
WO2011051390A1 (en) 2009-10-28 2011-05-05 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Homologous recombination in the oocyte
TR201903376T4 (tr) 2009-10-29 2019-04-22 Regeneron Pharma Çok fonksiyonlu alleller.
US20120315670A1 (en) 2009-11-02 2012-12-13 Gen9, Inc. Compositions and Methods for the Regulation of Multiple Genes of Interest in a Cell
WO2011068103A1 (ja) 2009-12-01 2011-06-09 独立行政法人国立がん研究センター ラット胚性幹細胞を用いたキメララットの作製法
NO2510096T3 (es) 2009-12-10 2015-03-21
CN102791865B (zh) 2009-12-21 2015-07-01 凯津公司 用于转染原生质体的改进技术
US9695432B2 (en) 2010-01-22 2017-07-04 Dow Agrosciences Llc Excision of transgenes in genetically modified organisms
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
GB201009732D0 (en) 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
AU2011265306C1 (en) 2010-06-11 2015-01-22 Regeneron Pharmaceuticals, Inc. Production of fertile XY female animals from XY ES cells
CA2802591A1 (en) * 2010-06-17 2011-12-22 Kymab Limited Animal models and therapeutic molecules
US8945868B2 (en) 2010-07-21 2015-02-03 Sangamo Biosciences, Inc. Methods and compositions for modification of a HLA locus
WO2012018726A1 (en) * 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
WO2012129198A1 (en) 2011-03-23 2012-09-27 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for obesity and diabetes
SMT202100243T1 (it) 2011-10-28 2021-05-07 Regeneron Pharma Topi con recettori delle cellule t geneticamente modificati
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
CA2871524C (en) 2012-05-07 2021-07-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
SI3401400T1 (sl) * 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
SG11201503059XA (en) 2012-10-23 2015-06-29 Toolgen Inc Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
EP3138911B1 (en) 2012-12-06 2018-12-05 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
BR112015013784A2 (pt) 2012-12-12 2017-07-11 Massachusetts Inst Technology aplicação, manipulação e otimização de sistemas, métodos e composições para manipulação de sequência e aplicações terapêuticas
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
DK3553174T3 (da) 2012-12-17 2025-08-04 Harvard College Rna-guided modificering af humant genom
DK3491915T5 (da) 2012-12-27 2024-09-02 Keygene Nv Fremgangsmåde til at inducere en målrettet translokation i en plante
CN109913495B (zh) 2013-02-20 2022-11-25 瑞泽恩制药公司 大鼠的遗传修饰
EP2922393B2 (en) 2013-02-27 2022-12-28 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Gene editing in the oocyte by cas9 nucleases
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
US9234213B2 (en) * 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
AU2014235968B2 (en) * 2013-03-21 2018-05-24 Ospedale San Raffaele Srl Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
JP2016522679A (ja) 2013-04-04 2016-08-04 プレジデント アンド フェローズ オブ ハーバード カレッジ CRISPR/Cas系を用いたゲノム編集の治療的使用
SG10201808935WA (en) 2013-04-16 2018-11-29 Regeneron Pharma Targeted modification of rat genome
HK1223401A1 (zh) 2013-05-15 2017-07-28 桑格摩生物科学股份有限公司 用於治疗遗传病状的方法和组合物
ES2670531T3 (es) 2013-05-29 2018-05-30 Cellectis S.A. Un método para producir una escisión de ADN precisa utilizando la actividad nickasa de Cas9
EP3011011A4 (en) 2013-06-19 2017-05-31 Sigma-Aldrich Co. LLC Targeted integration
ES2844174T3 (es) 2013-09-18 2021-07-21 Kymab Ltd Métodos, células y organismos
WO2015052231A2 (en) 2013-10-08 2015-04-16 Technical University Of Denmark Multiplex editing system
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
JP6174811B2 (ja) 2013-12-11 2017-08-02 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ゲノムの標的改変のための方法及び組成物
EP3080275B1 (en) 2013-12-13 2020-01-15 Cellectis Method of selection of transformed diatoms using nuclease
US10233456B2 (en) 2014-01-30 2019-03-19 The Board Of Trustees Of The University Of Arkansas Method, vectors, cells, seeds and kits for stacking genes into a single genomic site
KR101823661B1 (ko) 2014-04-24 2018-01-30 기초과학연구원 마이크로 상동 기반의 유전자 녹아웃을 위한 뉴클레아제 표적 서열을 확인하는 방법
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
MX385689B (es) 2014-06-06 2025-03-18 Regeneron Pharma Métodos y composiciones para modificar un locus dirigido.
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
EP3561052A1 (en) 2014-10-15 2019-10-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
ES2731437T3 (es) 2014-11-21 2019-11-15 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida mediante el uso de pares de ARN guías
MX388784B (es) 2014-12-19 2025-03-20 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de direccionamiento múltiple de una sola etapa.
GB201504223D0 (en) 2015-03-12 2015-04-29 Genome Res Ltd Biallelic genetic modification
WO2019046350A1 (en) 2017-08-30 2019-03-07 President And Fellows Of Harvard College ITERATIVE GENOMIC ASSEMBLY

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