ES2683071T3 - Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento - Google Patents

Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento Download PDF

Info

Publication number
ES2683071T3
ES2683071T3 ES13720718.9T ES13720718T ES2683071T3 ES 2683071 T3 ES2683071 T3 ES 2683071T3 ES 13720718 T ES13720718 T ES 13720718T ES 2683071 T3 ES2683071 T3 ES 2683071T3
Authority
ES
Spain
Prior art keywords
nucleic acid
acid sequence
human
sequence
cell
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
ES13720718.9T
Other languages
English (en)
Spanish (es)
Inventor
David Frendewey
Wojtek Auerbach
David M. Valenzuela
George D. Yancopoulos
Ka-Man Venus Lai
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
Application granted granted Critical
Publication of ES2683071T3 publication Critical patent/ES2683071T3/es
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)
ES13720718.9T 2012-04-25 2013-04-25 Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento Active ES2683071T3 (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
ES2683071T3 true ES2683071T3 (es) 2018-09-24

Family

ID=48289700

Family Applications (1)

Application Number Title Priority Date Filing Date
ES13720718.9T Active ES2683071T3 (es) 2012-04-25 2013-04-25 Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento

Country Status (19)

Country Link
US (2) US9834786B2 (cg-RX-API-DMAC7.html)
EP (1) EP2847335B1 (cg-RX-API-DMAC7.html)
JP (1) JP6275120B2 (cg-RX-API-DMAC7.html)
KR (1) KR101904508B1 (cg-RX-API-DMAC7.html)
CN (2) CN104364380B (cg-RX-API-DMAC7.html)
AU (1) AU2013251558B2 (cg-RX-API-DMAC7.html)
BR (1) BR112014026294B1 (cg-RX-API-DMAC7.html)
CA (1) CA2869016C (cg-RX-API-DMAC7.html)
CY (1) CY1120572T1 (cg-RX-API-DMAC7.html)
DK (1) DK2847335T3 (cg-RX-API-DMAC7.html)
ES (1) ES2683071T3 (cg-RX-API-DMAC7.html)
IL (1) IL234905B (cg-RX-API-DMAC7.html)
IN (1) IN2014DN09261A (cg-RX-API-DMAC7.html)
MX (1) MX362523B (cg-RX-API-DMAC7.html)
PL (1) PL2847335T3 (cg-RX-API-DMAC7.html)
PT (1) PT2847335T (cg-RX-API-DMAC7.html)
RU (1) RU2645475C2 (cg-RX-API-DMAC7.html)
SG (2) SG11201406547YA (cg-RX-API-DMAC7.html)
WO (1) WO2013163394A1 (cg-RX-API-DMAC7.html)

Families Citing this family (50)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT2847335T (pt) 2012-04-25 2018-09-28 Regeneron Pharma Alvejamento mediado por nuclease com vetores de alvejamento grandes
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
LT3138910T (lt) 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
EP3561050B1 (en) 2013-02-20 2021-12-08 Regeneron Pharmaceuticals, Inc. Genetic modification of rats
HUE040575T2 (hu) 2013-04-16 2019-03-28 Regeneron Pharma A patkány genom célzott módosítása
DE202014010413U1 (de) 2013-09-18 2015-12-08 Kymab Limited Zellen und Organismen
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
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
SMT201800653T1 (it) 2013-12-11 2019-01-11 Regeneron Pharma Metodi e composizioni per la modificazione indirizzata a bersaglio di un genoma
LT3152312T (lt) * 2014-06-06 2020-04-27 Regeneron Pharmaceuticals, Inc. Tikslinio lokuso modifikavimo būdai ir kompozicijos
EP3461885B1 (en) * 2014-06-26 2021-10-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
JP2017536811A (ja) 2014-10-15 2017-12-14 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. 多能性細胞の樹立又は維持のための方法及び組成物
HUE044907T2 (hu) 2014-11-21 2019-11-28 Regeneron Pharma Módszerek és készítmények célzott genetikai módosításhoz, párosított mutató RNS-ek használatával
NZ732895A (en) * 2014-12-19 2022-05-27 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting
US10285387B2 (en) 2015-03-16 2019-05-14 Regeneron Pharmaceuticals, Inc. Non-human animal exhibiting diminished upper and lower motor neuron function and sensory perception
ES3009538T3 (en) 2015-05-29 2025-03-27 Regeneron Pharma Rodent cells having a disruption in a c9orf72 locus
KR102294755B1 (ko) 2016-09-30 2021-08-31 리제너론 파마슈티칼스 인코포레이티드 C9orf72 유전자좌에서 헥사뉴클레오티드 반복 확장을 갖는 비인간 동물
SG11201911886PA (en) 2017-06-27 2020-01-30 Regeneron Pharma Non-human animals comprising a humanized asgr1 locus
CA3065579A1 (en) 2017-07-31 2019-02-07 Regeneron Pharmaceuticals, Inc. Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
ES3033963T3 (en) 2017-07-31 2025-08-11 Regeneron Pharma Method of testing the ability of a crispr/cas9 nuclease to modify a target genomic locus in vivo, by making use of a cas-transgenic mouse or rat which comprises a cas9 expression cassette in its genome
US11021719B2 (en) 2017-07-31 2021-06-01 Regeneron Pharmaceuticals, Inc. Methods and compositions for assessing CRISPER/Cas-mediated disruption or excision and CRISPR/Cas-induced recombination with an exogenous donor nucleic acid in vivo
WO2019067875A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS AND METHODS OF USE
US20190141966A1 (en) 2017-11-10 2019-05-16 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising SLC30A8 Mutation And Methods Of Use
AU2018375796B2 (en) 2017-11-30 2024-11-21 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized TRKB locus
WO2019183123A1 (en) 2018-03-19 2019-09-26 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
US20220053741A1 (en) 2018-09-13 2022-02-24 Regeneron Pharmaceuticals, Inc. Complement Factor H Gene Knockout Rat as a Model of C3 Glomerulopathy
CN113423831B (zh) 2018-12-20 2023-03-10 瑞泽恩制药公司 核酸酶介导的重复扩增
IL286905B2 (en) 2019-04-04 2024-06-01 Regeneron Pharma Non-human animals containing the human coagulation factor 12 locus
WO2020247452A1 (en) 2019-06-04 2020-12-10 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus with a beta-slip mutation and methods of use
SG11202111256XA (en) 2019-06-07 2021-11-29 Regeneron Pharma Non-human animals comprising a humanized albumin locus
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
US12250931B2 (en) 2020-01-28 2025-03-18 Regeneron Pharmaceuticals, Inc. Genetically modified mouse with a humanized PNPLA3 gene and methods of use
US20230081547A1 (en) 2020-02-07 2023-03-16 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized klkb1 locus 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
US20230232796A1 (en) 2020-06-26 2023-07-27 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ace2 locus
WO2022187697A1 (en) 2021-03-05 2022-09-09 The Board Of Trustees Of The Leland Stanford Junior University In vivo dna assembly and analysis
EP4426107A1 (en) 2021-11-04 2024-09-11 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a modified cacng1 locus
IL312971A (en) 2021-12-08 2024-07-01 Regeneron Pharma Mutant myocilin disease model and uses thereof
WO2023122506A1 (en) 2021-12-20 2023-06-29 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
KR20240144138A (ko) 2022-02-11 2024-10-02 리제너론 파마슈티칼스 인코포레이티드 4r 타우 표적화제를 스크리닝하기 위한 조성물 및 방법
CA3257739A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. NON-HUMAN ANIMALS CONTAINING 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
US20240224964A9 (en) 2022-09-29 2024-07-11 Regeneron Pharmaceuticals, Inc. Correction of hepatosteatosis in humanized liver animals through restoration of il6/il6r/gp130 signaling in human hepatocytes
CN120603491A (zh) 2023-02-01 2025-09-05 瑞泽恩制药公司 包含修饰的klhdc7b基因座的动物
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

Family Cites Families (95)

* 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
BR9808584A (pt) 1997-03-14 2000-05-23 Idec Pharma Corp Processo para integrar genes em sìtios especìficos em células de mamìferos via recombinação homóloga e vetores para obter os mesmos
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
WO2000039316A1 (en) 1998-12-31 2000-07-06 The J. David Gladstone Institutes Transgenic rodents and rodent cell lines expressing hiv co-receptors
US6503717B2 (en) * 1999-12-06 2003-01-07 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
WO2000046386A2 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
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
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
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
JP2006502748A (ja) 2002-09-05 2006-01-26 カリフォルニア インスティテュート オブ テクノロジー 遺伝子ターゲッティングを誘発するキメラヌクレアーゼの使用方法
US20030175968A1 (en) 2002-10-30 2003-09-18 Golic Kent G. Gene targeting method
WO2004063356A2 (en) * 2003-01-13 2004-07-29 Rao Mahendra S 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
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1789095A2 (en) 2004-09-16 2007-05-30 Sangamo Biosciences Inc. Compositions and methods for protein production
PL1802193T3 (pl) 2004-10-19 2014-09-30 Regeneron Pharma Sposób wytwarzania myszy homozygotycznej 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
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
EP2003960B1 (en) 2006-03-31 2015-06-10 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
EP2137310B1 (en) 2007-04-26 2010-11-24 Sangamo BioSciences, Inc. Targeted integration into the ppp1r12c locus
SI2602323T1 (en) 2007-06-01 2018-05-31 Open Monoclonal Technology, Inc. Compositions and procedures for inhibiting endogenous immunoglobin gene and the formation of transgenic human idiopathic antibodies
KR20160015400A (ko) 2008-08-22 2016-02-12 상가모 바이오사이언스 인코포레이티드 표적화된 단일가닥 분할 및 표적화된 통합을 위한 방법 및 조성물
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
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
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
US20120276537A1 (en) 2009-10-28 2012-11-01 Kuehn Ralf Homologous recombination in the oocyte
JP5908405B2 (ja) 2009-10-29 2016-04-26 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. 多機能性対立遺伝子
US20120315670A1 (en) 2009-11-02 2012-12-13 Gen9, Inc. Compositions and Methods for the Regulation of Multiple Genes of Interest in a Cell
EP2508595B1 (en) 2009-12-01 2016-11-23 National Cancer Center Method for constructing chimeric rat using rat embryonic stem cells
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
AU2010335107B2 (en) 2009-12-21 2014-07-03 Keygene N.V. Improved techniques for transfecting protoplasts
KR101951489B1 (ko) 2010-01-22 2019-04-22 다우 아그로사이언시즈 엘엘씨 유전적으로 변형된 유기체에서 도입유전자의 절제
JP2013518602A (ja) * 2010-02-09 2013-05-23 サンガモ バイオサイエンシーズ, インコーポレイテッド 部分的に一本鎖のドナー分子による標的化ゲノム改変
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
GB201009732D0 (en) * 2010-06-10 2010-07-21 Gene Bridges Gmbh Direct cloning
CN102933074B (zh) 2010-06-11 2015-04-08 瑞泽恩制药公司 由xy es细胞制备能育的xy雌性动物
CA2805442C (en) * 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an 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
WO2012168307A2 (en) 2011-06-07 2012-12-13 Helmholtz Zentrum München - Deutsches Forschungszentrum für Gesundheit und Umwelt (GmbH) Improved recombination efficiency by inhibition of nhej dna repair
IN2014CN03890A (cg-RX-API-DMAC7.html) 2011-10-28 2015-10-16 Regeneron Pharma
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
PT2847335T (pt) 2012-04-25 2018-09-28 Regeneron Pharma Alvejamento mediado por nuclease com vetores de alvejamento grandes
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
MX2014015204A (es) 2012-06-12 2015-08-07 Genentech Inc Metodos y composiciones para generar alelos con inactivacion condicional.
EP3808844A1 (en) 2012-07-25 2021-04-21 The Broad Institute, Inc. Inducible dna binding proteins and genome perturbation tools and applications thereof
CN110643600A (zh) 2012-10-23 2020-01-03 基因工具股份有限公司 用于切割靶dna的系统及其用途
LT3138910T (lt) 2012-12-06 2017-11-10 Sigma-Aldrich Co. Llc Crispr pagrįstas genomo modifikavimas ir reguliavimas
WO2014093479A1 (en) 2012-12-11 2014-06-19 Montana State University Crispr (clustered regularly interspaced short palindromic repeats) rna-guided control of gene regulation
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
US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
EP2921557B1 (en) 2012-12-12 2016-07-13 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions for sequence manipulation
EP2840140B2 (en) 2012-12-12 2023-02-22 The Broad Institute, Inc. Crispr-Cas based method for mutation of prokaryotic cells
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
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
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
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
ES2786193T3 (es) 2012-12-12 2020-10-09 Broad Inst Inc Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias
PL2771468T3 (pl) 2012-12-12 2015-07-31 Broad Inst Inc Inżynieria systemów, metod i zoptymalizowanych kompozycji kierujących dla manipulacji sekwencjami
SG10201704932UA (en) 2012-12-17 2017-07-28 Harvard College Rna-guided human genome engineering
CN105025701B (zh) 2012-12-27 2018-09-25 凯津公司 去除植物中遗传连锁的方法
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
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014131833A1 (en) 2013-02-27 2014-09-04 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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
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
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
CN115261411A (zh) 2013-04-04 2022-11-01 哈佛学院校长同事会 利用CRISPR/Cas系统的基因组编辑的治疗性用途
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
HUE040575T2 (hu) 2013-04-16 2019-03-28 Regeneron Pharma A patkány genom célzott módosítása
WO2014172470A2 (en) 2013-04-16 2014-10-23 Whitehead Institute For Biomedical Research Methods of mutating, modifying or modulating nucleic acid in a cell or nonhuman mammal
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
US9546384B2 (en) 2013-12-11 2017-01-17 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a mouse genome
LT3152312T (lt) 2014-06-06 2020-04-27 Regeneron Pharmaceuticals, Inc. Tikslinio lokuso modifikavimo būdai ir kompozicijos
EP3461885B1 (en) 2014-06-26 2021-10-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for targeted genetic modifications and methods of use
CA2954414A1 (en) 2014-07-15 2016-01-21 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
NZ732895A (en) 2014-12-19 2022-05-27 Regeneron Pharma Methods and compositions for targeted genetic modification through single-step multiple targeting

Also Published As

Publication number Publication date
AU2013251558B2 (en) 2019-01-03
PL2847335T3 (pl) 2019-01-31
US20180037910A1 (en) 2018-02-08
IL234905B (en) 2018-10-31
JP2015514439A (ja) 2015-05-21
BR112014026294B1 (pt) 2021-11-23
KR20150004816A (ko) 2015-01-13
US20130309670A1 (en) 2013-11-21
EP2847335B1 (en) 2018-06-27
RU2645475C2 (ru) 2018-02-21
KR101904508B1 (ko) 2018-10-04
WO2013163394A1 (en) 2013-10-31
CN109536526A (zh) 2019-03-29
CA2869016C (en) 2020-05-05
IN2014DN09261A (cg-RX-API-DMAC7.html) 2015-07-10
BR112014026294A2 (pt) 2020-07-07
JP6275120B2 (ja) 2018-02-07
CN104364380A (zh) 2015-02-18
SG10201702445TA (en) 2017-04-27
DK2847335T3 (en) 2018-08-13
MX362523B (es) 2019-01-22
CA2869016A1 (en) 2013-10-31
PT2847335T (pt) 2018-09-28
MX2014012994A (es) 2015-02-04
CN109536526B (zh) 2020-06-09
SG11201406547YA (en) 2014-11-27
US9834786B2 (en) 2017-12-05
EP2847335A1 (en) 2015-03-18
CN104364380B (zh) 2018-10-09
CY1120572T1 (el) 2019-07-10
AU2013251558A1 (en) 2014-10-02
US10301646B2 (en) 2019-05-28
HK1207396A1 (en) 2016-01-29
RU2014145942A (ru) 2016-06-10

Similar Documents

Publication Publication Date Title
ES2683071T3 (es) Direccionamiento mediado por nucleasas con grandes vectores de direccionamiento
JP7154248B2 (ja) 標的遺伝子座を修飾するための方法及び組成物
ES2947714T3 (es) Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso
ES3013183T3 (en) Methods for targeted genetic modification using paired guide rnas
ES2888250T3 (es) Modificación direccionada del genoma de rata
ES2629509T3 (es) Integración específica de sitio
RU2017101330A (ru) Способы и композиции для нацеленных генетических модификаций и способы их применения
Jazayeri et al. Vector and cell line engineering technologies toward recombinant protein expression in mammalian cell lines
Lee et al. Method to assemble genomic DNA fragments or genes on human artificial chromosome with regulated kinetochore using a multi-integrase system
CN113999874B (zh) Phb1基因敲除非人动物的构建方法及其应用
Fenina et al. I-Sce I-mediated double-strand break does not increase the frequency of homologous recombination at the Dct locus in mouse embryonic stem cells
KR20130069188A (ko) 마이오스타틴을 표적으로 하는 징크 핑거 뉴클레아제 및 이의 용도
HK1207396B (en) Nuclease-mediated targeting with large targeting vectors
Li et al. Application of oligonucleotides to construct a conditional targeting vector for porcine I κ B α
RU2019132992A (ru) Способы и композиции для модификации целевого локуса
HK1235817B (en) Methods and compositions for modifying a targeted locus
HK1235817A1 (en) Methods and compositions for modifying a targeted locus
JP2014103906A (ja) 部位特異的組換え系を利用した遺伝子導入方法およびそのためのキット