AU2013251558B2 - Nuclease-mediated targeting with large targeting vectors - Google Patents

Nuclease-mediated targeting with large targeting vectors Download PDF

Info

Publication number
AU2013251558B2
AU2013251558B2 AU2013251558A AU2013251558A AU2013251558B2 AU 2013251558 B2 AU2013251558 B2 AU 2013251558B2 AU 2013251558 A AU2013251558 A AU 2013251558A AU 2013251558 A AU2013251558 A AU 2013251558A AU 2013251558 B2 AU2013251558 B2 AU 2013251558B2
Authority
AU
Australia
Prior art keywords
nucleic acid
acid sequence
cell
human
sequence
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
AU2013251558A
Other languages
English (en)
Other versions
AU2013251558A1 (en
Inventor
Wojtek Auerbach
David Frendewey
Ka-Man Venus Lai
David M. Valenzuela
George D. Yancopoulos
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Regeneron Pharmaceuticals Inc
Original Assignee
Regeneron Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharmaceuticals Inc filed Critical Regeneron Pharmaceuticals Inc
Publication of AU2013251558A1 publication Critical patent/AU2013251558A1/en
Application granted granted Critical
Publication of AU2013251558B2 publication Critical patent/AU2013251558B2/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • C07K14/7155Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons for interleukins [IL]
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2013251558A 2012-04-25 2013-04-25 Nuclease-mediated targeting with large targeting vectors Active AU2013251558B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261638267P 2012-04-25 2012-04-25
US61/638,267 2012-04-25
PCT/US2013/038165 WO2013163394A1 (en) 2012-04-25 2013-04-25 Nuclease-mediated targeting with large targeting vectors

Publications (2)

Publication Number Publication Date
AU2013251558A1 AU2013251558A1 (en) 2014-10-02
AU2013251558B2 true AU2013251558B2 (en) 2019-01-03

Family

ID=48289700

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2013251558A Active AU2013251558B2 (en) 2012-04-25 2013-04-25 Nuclease-mediated targeting with large targeting vectors

Country Status (19)

Country Link
US (2) US9834786B2 (enExample)
EP (1) EP2847335B1 (enExample)
JP (1) JP6275120B2 (enExample)
KR (1) KR101904508B1 (enExample)
CN (2) CN104364380B (enExample)
AU (1) AU2013251558B2 (enExample)
BR (1) BR112014026294B1 (enExample)
CA (1) CA2869016C (enExample)
CY (1) CY1120572T1 (enExample)
DK (1) DK2847335T3 (enExample)
ES (1) ES2683071T3 (enExample)
IL (1) IL234905B (enExample)
IN (1) IN2014DN09261A (enExample)
MX (1) MX362523B (enExample)
PL (1) PL2847335T3 (enExample)
PT (1) PT2847335T (enExample)
RU (1) RU2645475C2 (enExample)
SG (2) SG10201702445TA (enExample)
WO (1) WO2013163394A1 (enExample)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104364380B (zh) 2012-04-25 2018-10-09 瑞泽恩制药公司 核酸酶介导的使用大靶向载体的靶向
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
MX369747B (es) 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
WO2015040402A1 (en) * 2013-09-18 2015-03-26 Kymab Limited Methods. cells & organisms
KR101566498B1 (ko) * 2013-11-13 2015-11-06 건국대학교 산학협력단 인터루킨 2 수용체 감마 유전자 적중벡터, 그 벡터가 도입된 면역세포 결핍 형질전환 미니 복제돼지 생산과 그 제조방법 및 활용
US10787684B2 (en) * 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
EP4349980A3 (en) 2013-12-11 2024-06-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
RU2685914C1 (ru) * 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
HUE049776T2 (hu) 2014-06-06 2020-10-28 Regeneron Pharma Módszerek és készítmények egy célzott lókusz módosítására
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
PT3207124T (pt) 2014-10-15 2019-08-27 Regeneron Pharma Métodos e composições para a produção ou conservação de células pluripotentes
RU2734770C2 (ru) 2014-11-21 2020-10-23 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации с использованием парных гидовых рнк
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания
SG11201706809WA (en) 2015-03-16 2017-09-28 Regeneron Pharma Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception
BR112017025507A2 (pt) * 2015-05-29 2018-08-07 Regeneron Pharmaceuticals, Inc. roedor, célula isolada de roedor ou tecido isolado de roedor, célula-tronco embrionária de roedor, embrião de roedor, e, métodos para preparação de um roedor e para identificação de um candidato terapêutico.
RU2760877C2 (ru) 2016-09-30 2021-12-01 Регенерон Фармасьютикалс, Инк. Животные, отличные от человека, характеризующиеся экспансией гексануклеотидных повторов в локусе c9orf72
CN118109477A (zh) 2017-06-27 2024-05-31 瑞泽恩制药公司 包含人源化asgr1基因座的非人动物
AU2018309716B2 (en) 2017-07-31 2024-11-21 Regeneron Pharmaceuticals, Inc. Cas-transgenic mouse embryonic stem cells and mice and uses thereof
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
RU2019143568A (ru) 2017-07-31 2021-09-02 Регенерон Фармасьютикалс, Инк. Способы и композиции для оценки crispr/cas-индуцированной рекомбинации с экзогенной донорной нуклеиновой кислотой in vivo
EP4276185A3 (en) 2017-09-29 2024-02-21 Regeneron Pharmaceuticals, Inc. Rodents comprising a humanized ttr locus and methods of use
WO2019094735A1 (en) 2017-11-10 2019-05-16 Regeneron Pharmaceuticals, Inc. Non-human animals comprising slc30a8 mutation and methods of use
CN111655031B (zh) 2017-11-30 2022-11-18 瑞泽恩制药公司 包含人源化trkb基因座的非人动物
WO2019183123A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
EP3849304B1 (en) 2018-09-13 2024-01-10 Regeneron Pharmaceuticals, Inc. Complement factor h gene knockout rat as a model of c3 glomerulopathy
JP7449291B2 (ja) 2018-12-20 2024-03-13 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 ヌクレアーゼ媒介リピート伸長
IL286905B2 (en) 2019-04-04 2024-06-01 Regeneron Pharma Non-human animals containing the human coagulation factor 12 locus
KR20220016869A (ko) 2019-06-04 2022-02-10 리제너론 파마슈티칼스 인코포레이티드 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
BR112021022722A2 (pt) 2019-06-07 2022-01-04 Regeneron Pharma Animal não humano, célula de animal não humana, genoma de animal não humano, gene de albumina animal não humana humanizada, vetor de alvejamento, método de avaliação da atividade de um reagente, e, método de otimização da atividade de um reagente
RU2722933C1 (ru) * 2019-06-11 2020-06-05 Автономная некоммерческая образовательная организация высшего образования Сколковский институт науки и технологий Средство разрезания днк на основе cas9 белка из бактерии demequina sediminicola
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
KR20220133248A (ko) 2020-01-28 2022-10-04 리제너론 파마슈티칼스 인코포레이티드 인간화 pnpla3 좌위를 포함하는 비-인간 동물 및 사용 방법
EP4099821A1 (en) 2020-02-07 2022-12-14 Regeneron Pharmaceuticals, Inc. <smallcaps/>? ? ?klkb1? ? ? ? ?non-human animals comprising a humanizedlocus and methods of use
EP4125348A1 (en) 2020-03-23 2023-02-08 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
WO2021263146A2 (en) 2020-06-26 2021-12-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
CA3212642A1 (en) 2021-03-05 2022-09-09 The Board Of Trustees Of The Leland Stanford Junior University In vivo dna assembly and analysis
WO2023081847A1 (en) 2021-11-04 2023-05-11 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified cacng1 locus
AU2022408167A1 (en) 2021-12-08 2024-06-06 Regeneron Pharmaceuticals, Inc. Mutant myocilin disease model and uses thereof
EP4451863A1 (en) 2021-12-20 2024-10-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising humanized ace2 and tmprss loci
EP4475671A1 (en) 2022-02-07 2024-12-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
US20230257432A1 (en) 2022-02-11 2023-08-17 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
WO2024026488A2 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified transferrin receptor locus
CN119562964A (zh) 2022-08-05 2025-03-04 瑞泽恩制药公司 tdp-43的抗聚集变体
WO2024073679A1 (en) 2022-09-29 2024-04-04 Regeneron Pharmaceuticals, Inc. Correction of hepatosteatosis in humanized liver animals through restoration of il6/il6r/gp130 signaling in human hepatocytes
AU2024214456A1 (en) 2023-02-01 2025-07-03 Regeneron Pharmaceuticals, Inc. Animals comprising a modified klhdc7b locus
WO2024189098A1 (en) 2023-03-13 2024-09-19 Iomx Therapeutics Ag Platform technology for the identification of modulators of immune effector cell function
WO2025122754A1 (en) 2023-12-07 2025-06-12 Regeneron Pharmaceuticals, Inc. Gaa knockout non-human animals
WO2025250495A1 (en) 2024-05-28 2025-12-04 Regeneron Pharmaceuticals, Inc. Acceleration of human hepatocyte engraftment in humanized liver animals by supplementing paracrine ligands or agonists that activate human liver regeneration signals

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011154927A2 (en) * 2010-06-10 2011-12-15 Gene Bridges Gmbh Direct cloning

Family Cites Families (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5830729A (en) 1996-04-18 1998-11-03 Institut Pasteur I Sce I-induced gene replacement and gene conversion in embryonic stem cells
ID24565A (id) 1997-03-14 2000-07-27 Idec Pharma Corp Metoda untuk integrasi gen pada tempat yang spesifik dalam sel mamalia melalui rekombinasi homolog dan vektor untuk pencapaian yang sama
US5830698A (en) * 1997-03-14 1998-11-03 Idec Pharmaceuticals Corporation Method for integrating genes at specific sites in mammalian cells via homologous recombination and vectors for accomplishing the same
US6372956B1 (en) 1998-12-31 2002-04-16 The J. David Gladstone Institutes Transgenic rats and rat cell lines expressing human CD4 and a human chemokine receptor
EP1147209A2 (en) 1999-02-03 2001-10-24 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
ATE309536T1 (de) * 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
US6596541B2 (en) * 2000-10-31 2003-07-22 Regeneron Pharmaceuticals, Inc. Methods of modifying eukaryotic cells
US7105348B2 (en) 2000-10-31 2006-09-12 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
EP1476547B1 (en) 2002-01-23 2006-12-06 The University of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
ATE531796T1 (de) 2002-03-21 2011-11-15 Sangamo Biosciences Inc Verfahren und zusammensetzungen zur verwendung von zinkfinger-endonukleasen zur verbesserung der homologen rekombination
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US20030175968A1 (en) 2002-10-30 2003-09-18 Golic Kent G. Gene targeting method
CA2513226A1 (en) * 2003-01-13 2004-07-29 Mahendra S. Rao 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
WO2005014791A2 (en) 2003-08-08 2005-02-17 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
DK1802193T3 (da) 2004-10-19 2014-06-10 Regeneron Pharma Fremgangsmåde til frembringelse af en homozygot mus til genetisk modifikation
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
US10022457B2 (en) 2005-08-05 2018-07-17 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
GB0615327D0 (en) 2006-03-30 2006-09-13 Univ Edinburgh Culture medium containing kinase inhibitors and uses thereof
AU2007235496B2 (en) 2006-03-31 2013-11-21 E. R. Squibb & Sons, L.L.C. Transgenic animals expressing chimeric antibodies for use in preparing human antibodies
US7771967B2 (en) 2006-12-22 2010-08-10 The J. David Gladstone Institutes Nucleic acid encoding apolipoprotein E-I3
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
AU2008244473B2 (en) 2007-04-26 2013-06-20 Sangamo Therapeutics, Inc. Targeted integration into the PPP1R12C locus
EP2336329B8 (en) * 2007-06-01 2012-11-21 Omt, Inc. Compositions and methods for inhibiting endogenous immunoglobulin genes and producing transgenis human idiotype antibodies
EP2789691B1 (en) 2008-08-22 2018-08-08 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
US20100076057A1 (en) 2008-09-23 2010-03-25 Northwestern University TARGET DNA INTERFERENCE WITH crRNA
EP2180058A1 (en) 2008-10-23 2010-04-28 Cellectis Meganuclease recombination system
ES2627552T3 (es) 2008-12-04 2017-07-28 Sigma Aldrich Company Edición de genoma en ratas usando nucleasas con dedos de cinc
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
WO2010124200A2 (en) 2009-04-23 2010-10-28 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for cancer
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
EP2456876A2 (en) 2009-07-24 2012-05-30 Transposagen Biopharmaceuticals, Inc. Genetically modified rat models for cytokine-cytokine signaling pathways
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.
WO2011053957A2 (en) 2009-11-02 2011-05-05 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
CN106834320B (zh) 2009-12-10 2021-05-25 明尼苏达大学董事会 Tal效应子介导的dna修饰
ES2583060T3 (es) 2009-12-21 2016-09-16 Keygene N.V. Técnicas mejoradas para la transfección de protoplastos
PH12012501467B1 (en) 2010-01-22 2018-07-04 Corteva Agriscience Llc Excision of transgenes in genetically modified organisms
EP2660318A1 (en) * 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
JP6208580B2 (ja) 2010-05-17 2017-10-04 サンガモ セラピューティクス, インコーポレイテッド 新規のdna結合タンパク質及びその使用
CA2800534C (en) 2010-06-11 2019-01-22 Regeneron Pharmaceuticals, Inc. Production of fertile xy female animals from xy es cells
JP6050230B2 (ja) * 2010-07-21 2016-12-21 サンガモ バイオサイエンシーズ, インコーポレイテッド Hla遺伝子座の修飾のための方法及び組成物
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
EP2718446A2 (en) 2011-06-07 2014-04-16 Helmholtz Zentrum München Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Improved recombination efficiency by inhibition of nhej dna repair
EP2771357B1 (en) 2011-10-28 2018-07-18 Regeneron Pharmaceuticals, Inc. Genetically modified t cell receptor mice
WO2013141680A1 (en) 2012-03-20 2013-09-26 Vilnius University RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX
US9637739B2 (en) 2012-03-20 2017-05-02 Vilnius University RNA-directed DNA cleavage by the Cas9-crRNA complex
CN104364380B (zh) 2012-04-25 2018-10-09 瑞泽恩制药公司 核酸酶介导的使用大靶向载体的靶向
WO2013169802A1 (en) 2012-05-07 2013-11-14 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
US20150128300A1 (en) 2012-06-12 2015-05-07 Genentech, Inc. Methods and compositions for generating conditional knock-out alleles
EP3494997B1 (en) 2012-07-25 2019-09-18 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
JP6517143B2 (ja) 2012-10-23 2019-05-22 ツールゲン インコーポレイテッド 標的dnaに特異的なガイドrnaおよびcasタンパク質コード核酸またはcasタンパク質を含む、標的dnaを切断するための組成物、ならびにその使用
KR102479178B1 (ko) 2012-12-06 2022-12-19 시그마-알드리치 컴퍼니., 엘엘씨 Crispr-기초된 유전체 변형과 조절
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
ES2701749T3 (es) 2012-12-12 2019-02-25 Broad Inst Inc Métodos, modelos, sistemas y aparatos para identificar secuencias diana para enzimas Cas o sistemas CRISPR-Cas para secuencias diana y transmitir resultados de los mismos
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
IL239344B2 (en) 2012-12-12 2024-06-01 Broad Inst Inc Systems engineering, methods and optimal guiding components for sequence manipulation
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
PL2896697T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Projektowanie systemów, sposoby i optymalizowane kompozycje kierujące do manipulacji sekwencją
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP4299741A3 (en) 2012-12-12 2024-02-28 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP2898075B1 (en) 2012-12-12 2016-03-09 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
JP2016505256A (ja) 2012-12-12 2016-02-25 ザ・ブロード・インスティテュート・インコーポレイテッ 配列操作のためのCRISPR−Cas成分系、方法および組成物
JP6700788B2 (ja) 2012-12-17 2020-05-27 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 Rna誘導性ヒトゲノム改変
EP3491915B1 (en) 2012-12-27 2023-05-31 Keygene N.V. Method for inducing a targeted translocation in a plant.
WO2014127287A1 (en) 2013-02-14 2014-08-21 Massachusetts Institute Of Technology Method for in vivo tergated mutagenesis
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
EP2958996B1 (en) 2013-02-25 2019-10-16 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
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
WO2014143381A1 (en) 2013-03-09 2014-09-18 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple crispr/cas selections of recombineering events
NZ712727A (en) 2013-03-14 2017-05-26 Caribou Biosciences Inc Compositions and methods of nucleic acid-targeting nucleic acids
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US9937207B2 (en) 2013-03-21 2018-04-10 Sangamo Therapeutics, Inc. Targeted disruption of T cell receptor genes using talens
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
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
MX369747B (es) 2013-04-16 2019-11-20 Regeneron Pharma Modificación dirigida del genoma de rata.
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
EP2796558A1 (en) 2013-04-23 2014-10-29 Rheinische Friedrich-Wilhelms-Universität Bonn Improved gene targeting and nucleic acid carrier molecule, in particular for use in plants
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
RU2685914C1 (ru) 2013-12-11 2019-04-23 Регенерон Фармасьютикалс, Инк. Способы и композиции для направленной модификации генома
HUE049776T2 (hu) 2014-06-06 2020-10-28 Regeneron Pharma Módszerek és készítmények egy célzott lókusz módosítására
WO2015200805A2 (en) 2014-06-26 2015-12-30 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
WO2016011210A2 (en) 2014-07-15 2016-01-21 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
RU2707137C2 (ru) 2014-12-19 2019-11-22 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленной генетической модификации посредством одноэтапного множественного нацеливания

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011154927A2 (en) * 2010-06-10 2011-12-15 Gene Bridges Gmbh Direct cloning

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LOMBARDO ANGELO ET AL, "Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery", NATURE BIOTECHNOLOGY, vol. 25, no. 11, pages 1298 - 1306 *

Also Published As

Publication number Publication date
CY1120572T1 (el) 2019-07-10
MX362523B (es) 2019-01-22
CN109536526A (zh) 2019-03-29
CN104364380A (zh) 2015-02-18
CA2869016C (en) 2020-05-05
CN109536526B (zh) 2020-06-09
CN104364380B (zh) 2018-10-09
WO2013163394A1 (en) 2013-10-31
KR101904508B1 (ko) 2018-10-04
SG11201406547YA (en) 2014-11-27
EP2847335A1 (en) 2015-03-18
JP6275120B2 (ja) 2018-02-07
HK1207396A1 (en) 2016-01-29
PL2847335T3 (pl) 2019-01-31
BR112014026294B1 (pt) 2021-11-23
EP2847335B1 (en) 2018-06-27
RU2014145942A (ru) 2016-06-10
US20180037910A1 (en) 2018-02-08
ES2683071T3 (es) 2018-09-24
MX2014012994A (es) 2015-02-04
BR112014026294A2 (pt) 2020-07-07
DK2847335T3 (en) 2018-08-13
JP2015514439A (ja) 2015-05-21
IL234905B (en) 2018-10-31
AU2013251558A1 (en) 2014-10-02
PT2847335T (pt) 2018-09-28
SG10201702445TA (en) 2017-04-27
US9834786B2 (en) 2017-12-05
US20130309670A1 (en) 2013-11-21
RU2645475C2 (ru) 2018-02-21
CA2869016A1 (en) 2013-10-31
US10301646B2 (en) 2019-05-28
IN2014DN09261A (enExample) 2015-07-10
KR20150004816A (ko) 2015-01-13

Similar Documents

Publication Publication Date Title
US10301646B2 (en) Nuclease-mediated targeting with large targeting vectors
JP7154248B2 (ja) 標的遺伝子座を修飾するための方法及び組成物
HK1207396B (en) Nuclease-mediated targeting with large targeting vectors
HK40035693A (en) Methods and compositions for modifying a targeted locus
HK1235817B (en) Methods and compositions for modifying a targeted locus
HK1235817A1 (en) Methods and compositions for modifying a targeted locus

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)