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DK2534173T3 - Manipulerede spaltnings-halvdomæner - Google Patents

Manipulerede spaltnings-halvdomæner Download PDF

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Publication number
DK2534173T3
DK2534173T3 DK11740153.9T DK11740153T DK2534173T3 DK 2534173 T3 DK2534173 T3 DK 2534173T3 DK 11740153 T DK11740153 T DK 11740153T DK 2534173 T3 DK2534173 T3 DK 2534173T3
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Denmark
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domains
cleavage half
manipulated
manipulated cleavage
cleavage
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DK11740153.9T
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English (en)
Inventor
Yannick Doyon
Jeffrey C Miller
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Sangamo Therapeutics Inc
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    • 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
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/06Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • 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
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
    • 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
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/50Fusion polypeptide containing protease site
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/21Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004Type II site-specific deoxyribonuclease (3.1.21.4)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
DK11740153.9T 2010-02-08 2011-02-07 Manipulerede spaltnings-halvdomæner DK2534173T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US33776910P 2010-02-08 2010-02-08
US40391610P 2010-09-23 2010-09-23
PCT/US2011/000221 WO2011097036A1 (en) 2010-02-08 2011-02-07 Engineered cleavage half-domains

Publications (1)

Publication Number Publication Date
DK2534173T3 true DK2534173T3 (da) 2019-10-14

Family

ID=44355719

Family Applications (1)

Application Number Title Priority Date Filing Date
DK11740153.9T DK2534173T3 (da) 2010-02-08 2011-02-07 Manipulerede spaltnings-halvdomæner

Country Status (9)

Country Link
US (9) US8962281B2 (da)
EP (4) EP4328304A3 (da)
JP (2) JP6137596B2 (da)
AU (1) AU2011213242B2 (da)
CA (1) CA2788560A1 (da)
DK (1) DK2534173T3 (da)
ES (1) ES2751916T3 (da)
PT (1) PT2534173T (da)
WO (1) WO2011097036A1 (da)

Families Citing this family (162)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
US8563314B2 (en) * 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
US11235026B2 (en) 2007-09-27 2022-02-01 Sangamo Therapeutics, Inc. Methods and compositions for modulating PD1
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US8956828B2 (en) * 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CA2783351C (en) 2009-12-10 2021-09-07 Regents Of The University Of Minnesota Tal effector-mediated dna modification
ES2751916T3 (es) * 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
BR112012020257A8 (pt) * 2010-02-11 2018-02-14 Recombinetics Inc métodos e aparelhos para produzir artiodátilos transgênicos
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
AU2011312562B2 (en) 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
WO2012051343A1 (en) 2010-10-12 2012-04-19 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
WO2012094132A1 (en) 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
US10920242B2 (en) 2011-02-25 2021-02-16 Recombinetics, Inc. Non-meiotic allele introgression
US9528124B2 (en) 2013-08-27 2016-12-27 Recombinetics, Inc. Efficient non-meiotic allele introgression
EP3461896B1 (en) 2011-07-15 2023-11-29 The General Hospital Corporation Methods of transcription activator like effector assembly
WO2013016446A2 (en) 2011-07-25 2013-01-31 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
CA3186126A1 (en) 2011-09-21 2013-03-28 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA2852955C (en) 2011-10-27 2021-02-16 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
GB201122458D0 (en) * 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
BR112014021104B1 (pt) 2012-02-29 2023-03-28 Sangamo Biosciences, Inc Proteína de fusão de ocorrência não natural compreendendo um domínio de ligação de dna de dedo de zinco manipulado que se liga a um gene htt, seu uso, método in vitro de modificação da expressão de um gene htt em uma célula, e método de geração de um sistema modelo para o estudo da doença de huntington
UA115875C2 (uk) 2012-05-02 2018-01-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Рослина помідора зі зменшеною активністю малатдегідрогенази та спосіб її отримання
BR112014027813A2 (pt) 2012-05-07 2017-08-08 Dow Agrosciences Llc métodos e composições para integração de transgenes direcionada mediada por nuclease
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
US20150225734A1 (en) 2012-06-19 2015-08-13 Regents Of The University Of Minnesota Gene targeting in plants using dna viruses
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
WO2014011901A2 (en) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Methods and compositions for delivery of biologics
ES2697912T3 (es) 2012-07-11 2019-01-29 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de enfermedades monogénicas
US10058078B2 (en) 2012-07-31 2018-08-28 Recombinetics, Inc. Production of FMDV-resistant livestock by allele substitution
AU2013308770B2 (en) * 2012-08-29 2019-01-17 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
CA2884162C (en) 2012-09-07 2020-12-29 Dow Agrosciences Llc Fad3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
UA118090C2 (uk) 2012-09-07 2018-11-26 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб інтегрування послідовності нуклеїнової кислоти, що представляє інтерес, у ген fad2 у клітині сої та специфічний для локусу fad2 білок, що зв'язується, здатний індукувати спрямований розрив
ES2824024T3 (es) 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
EP2906602B1 (en) 2012-10-12 2019-01-16 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
MX367031B (es) 2012-11-01 2019-08-02 Cellectis Plantas para la produccion de proteinas terapeuticas.
AU2013355327A1 (en) * 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
US20140178561A1 (en) 2012-12-21 2014-06-26 Cellectis Potatoes with reduced cold-induced sweetening
US10676749B2 (en) 2013-02-07 2020-06-09 The General Hospital Corporation Tale transcriptional activators
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
HK1220232A1 (zh) 2013-03-15 2017-04-28 怀特黑德生物医学研究院 用於神经变性疾病的细胞性发现平台
US10113162B2 (en) 2013-03-15 2018-10-30 Cellectis Modifying soybean oil composition through targeted knockout of the FAD2-1A/1B genes
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
KR102874079B1 (ko) 2013-03-15 2025-10-22 더 제너럴 하스피탈 코포레이션 Rna-안내 게놈 편집을 위해 특이성을 증가시키기 위한 절단된 안내 rna(tru-grnas)의 이용
EP2975942B1 (en) 2013-03-21 2018-08-08 Sangamo Therapeutics, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2014165612A2 (en) 2013-04-05 2014-10-09 Dow Agrosciences Llc Methods and compositions for integration of an exogenous sequence within the genome of plants
EP2994531B1 (en) * 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10260038B2 (en) 2013-05-10 2019-04-16 Whitehead Institute For Biomedical Research Protein modification of living cells using sortase
AU2014265331B2 (en) 2013-05-15 2019-12-05 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (en) * 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
WO2015031619A1 (en) 2013-08-28 2015-03-05 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
WO2015033293A1 (en) * 2013-09-04 2015-03-12 Csir Site-specific nuclease single-cell assay targeting gene regulatory elements to silence gene expression
WO2015057976A1 (en) 2013-10-17 2015-04-23 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
US9957526B2 (en) 2013-10-17 2018-05-01 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
EP3060682B1 (en) 2013-10-25 2023-08-30 Livestock Improvement Corporation Limited Genetic markers and uses therefor
WO2015066638A2 (en) 2013-11-04 2015-05-07 Dow Agrosciences Llc Optimal maize loci
BR102014027442B1 (pt) 2013-11-04 2022-09-27 Dow Agrosciences Llc Molécula de ácido nucleico recombinante, uso de uma planta de milho, parte de planta de milho ou célula de planta de milho compreendendo a mesma e método para produzir uma célula vegetal transgênica compreendendo um dna de interesse
US10233465B2 (en) 2013-11-04 2019-03-19 Dow Agrosciences Llc Optimal soybean loci
EP3862434A1 (en) 2013-11-04 2021-08-11 Dow AgroSciences LLC Optimal soybean loci
KR20230054509A (ko) 2013-11-07 2023-04-24 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
CN105934524A (zh) 2013-11-11 2016-09-07 桑格摩生物科学股份有限公司 用于治疗亨廷顿氏病的方法和组合物
DK3492593T3 (da) * 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
CA2930590C (en) * 2013-11-15 2021-02-16 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Engineering neural stem cells using homologous recombination
EP3757116A1 (en) 2013-12-09 2020-12-30 Sangamo Therapeutics, Inc. Methods and compositions for genome engineering
US10072066B2 (en) 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
JP6606088B2 (ja) * 2014-02-24 2019-11-13 サンガモ セラピューティクス, インコーポレイテッド ヌクレアーゼ媒介性標的化組み込みのための方法および組成物
AU2015231353B2 (en) 2014-03-18 2020-11-05 Sangamo Therapeutics, Inc. Methods and compositions for regulation of zinc finger protein expression
US10507232B2 (en) 2014-04-02 2019-12-17 University Of Florida Research Foundation, Incorporated Materials and methods for the treatment of latent viral infection
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
RU2691102C2 (ru) 2014-05-08 2019-06-11 Сангамо Байосайенсиз, Инк. Способы и композиции для лечения болезни хантингтона
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
EP3152319A4 (en) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Methods and compositions for nuclease design
CA2952906A1 (en) 2014-06-20 2015-12-23 Cellectis Potatoes with reduced granule-bound starch synthase
BR112017000925B1 (pt) 2014-07-14 2023-10-10 Washington State University Método de reprodução de porcos, método para gerar um porco sem nenhuma célula espermatogônica funcional, método de produção de um macho receptor e método de produção de porcos
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
US9757420B2 (en) 2014-07-25 2017-09-12 Sangamo Therapeutics, Inc. Gene editing for HIV gene therapy
US9616090B2 (en) 2014-07-30 2017-04-11 Sangamo Biosciences, Inc. Gene correction of SCID-related genes in hematopoietic stem and progenitor cells
US11261453B2 (en) 2014-09-12 2022-03-01 Whitehead Institute For Biomedical Research Cells expressing apolipoprotein E and uses thereof
CN106795488B (zh) 2014-09-16 2021-03-30 桑格摩治疗股份有限公司 用于造血干细胞中核酸酶介导的基因组工程化和校正的方法和组合物
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
WO2016098078A2 (en) 2014-12-19 2016-06-23 Novartis Ag Dimerization switches and uses thereof
CN107109427B (zh) 2014-12-23 2021-06-18 先正达参股股份有限公司 用于鉴定和富集包含位点特异性基因组修饰的细胞的方法和组合物
EP3247366A4 (en) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
EP4335918A3 (en) 2015-04-03 2024-04-17 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
HK1254190A1 (zh) 2015-05-08 2019-07-12 President And Fellows Of Harvard College 通用供体干细胞和相关方法
KR20170141217A (ko) * 2015-05-12 2017-12-22 상가모 테라퓨틱스, 인코포레이티드 유전자 발현의 뉴클레아제-매개된 조절
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017011519A1 (en) 2015-07-13 2017-01-19 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US10837024B2 (en) 2015-09-17 2020-11-17 Cellectis Modifying messenger RNA stability in plant transformations
SI3352776T1 (sl) 2015-09-23 2025-08-29 Sangamo Therapeutics, Inc. Represorji Htt in njihove uporabe
JP2018532403A (ja) * 2015-09-29 2018-11-08 アジェノビア コーポレーション 送達方法および組成物
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
AU2016361350B2 (en) 2015-11-23 2023-04-06 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
CN108699132B (zh) 2015-12-18 2023-08-11 桑格摩生物治疗股份有限公司 Mhc细胞受体的靶向破坏
ES2983043T3 (es) 2015-12-18 2024-10-21 Sangamo Therapeutics Inc Alteración dirigida del receptor de células T
EP3402533B1 (en) 2016-01-15 2021-08-04 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
EP4646936A3 (en) 2016-02-02 2025-11-19 Cellectis Modifying soybean oil composition through targeted knockout of the fad3a/b/c genes
US10724020B2 (en) 2016-02-02 2020-07-28 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
US11421218B2 (en) 2016-03-23 2022-08-23 Dana-Farber Cancer Institute, Inc. Methods for enhancing the efficiency of gene editing
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
CA3030783A1 (en) 2016-07-13 2018-01-18 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
UY40582A (es) 2016-08-17 2024-01-15 Monsanto Technology Llc MÉTODOS, COMPOSICIONES y PLANTAS MEDIANTE LA MANIPULACIÓN DEL METABOLISMO DE GIBERELINA PARA AUMENTAR EL RENDIMIENTO COSECHABLE
US10975393B2 (en) * 2016-08-24 2021-04-13 Sangamo Therapeutics, Inc. Engineered target specific nucleases
SG11201901531TA (en) 2016-08-24 2019-03-28 Sangamo Therapeutics Inc Regulation of gene expression using engineered nucleases
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
GB201617559D0 (en) 2016-10-17 2016-11-30 University Court Of The University Of Edinburgh The Swine comprising modified cd163 and associated methods
AU2017345430B2 (en) 2016-10-20 2024-09-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of Fabry disease
JP7108608B2 (ja) 2016-10-31 2022-07-28 サンガモ セラピューティクス, インコーポレイテッド 造血幹細胞および前駆細胞におけるscid関連遺伝子の遺伝子修正
UY37482A (es) 2016-11-16 2018-05-31 Cellectis Métodos para alterar el contenido de aminoácidos en plantas mediante mutaciones de desplazamiento de marco
MX2019006426A (es) 2016-12-01 2019-08-14 Sangamo Therapeutics Inc Reguladores de tau, y composiciones y metodos para su administracion.
MA49610A (fr) 2016-12-08 2020-05-27 Univ Case Western Reserve Procédés et compositions pour améliorer la production de myéline fonctionnelle
AU2018221730B2 (en) 2017-02-15 2024-06-20 Novo Nordisk A/S Donor repair templates multiplex genome editing
EP3615668B1 (en) 2017-04-25 2024-02-28 Cellectis Alfalfa with reduced lignin composition
US11820728B2 (en) 2017-04-28 2023-11-21 Acuitas Therapeutics, Inc. Carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
CN110869497A (zh) 2017-05-03 2020-03-06 桑格摩生物治疗股份有限公司 修饰囊性纤维化跨膜传导调节蛋白(cftr)基因的方法和组合物
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
CN111565730B (zh) 2017-11-09 2024-09-17 桑格摩生物治疗股份有限公司 细胞因子诱导型含sh2蛋白(cish)基因的遗传修饰
RU2763798C1 (ru) 2017-12-23 2022-01-11 Рубиус Терапьютикс, Инк. Искусственные антигенпрезентирующие клетки и способы их применения
EP3740480B1 (en) 2018-01-17 2024-03-06 Vertex Pharmaceuticals Incorporated Dna-pk inhibitors
CN111741955B (zh) 2018-01-17 2024-02-23 沃泰克斯药物股份有限公司 Dna-pk抑制剂
JP7483612B2 (ja) 2018-01-17 2024-05-15 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 ゲノム編集効率を増加するためのキノキサリノン化合物、組成物、方法、およびキット
JP7275152B2 (ja) 2018-02-08 2023-05-17 サンガモ セラピューティクス, インコーポレイテッド 操作された標的特異的なヌクレアーゼ
WO2019161151A1 (en) 2018-02-15 2019-08-22 Monsanto Technology Llc Compositions and methods for improving crop yields through trait stacking
UY38094A (es) 2018-02-15 2019-10-01 Monsanto Technology Llc Métodos mejorados para la producción de semillas de maíz híbridas
WO2019204457A1 (en) 2018-04-18 2019-10-24 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (htt)
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
JP7541508B2 (ja) 2018-08-23 2024-08-28 サンガモ セラピューティクス, インコーポレイテッド 操作された標的特異的な塩基エディター
WO2020061161A1 (en) * 2018-09-18 2020-03-26 Sangamo Therapeutics, Inc. Programmed cell death 1 (pd1) specific nucleases
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
CN113226333A (zh) 2018-10-02 2021-08-06 桑格摩生物治疗股份有限公司 用于调控Tau蛋白的方法和组合物
BR112021007301A2 (pt) 2018-10-18 2021-07-27 Intellia Therapeutics, Inc. composições e métodos para expressar fator ix
WO2020117852A1 (en) 2018-12-03 2020-06-11 Rubius Therapeutics, Inc. Artificial antigen presenting cells including hla-e and hla-g molecules and methods of use
US12274205B2 (en) 2018-12-12 2025-04-15 Monsanto Technology Llc Delayed harvest of short stature corn plants
US11453639B2 (en) 2019-01-11 2022-09-27 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
JP2022519410A (ja) 2019-02-06 2022-03-24 サンガモ セラピューティクス, インコーポレイテッド ムコ多糖症i型の処置のための方法
CA3132167A1 (en) 2019-04-02 2020-10-08 Weston P. MILLER IV Methods for the treatment of beta-thalassemia
PE20212332A1 (es) 2019-04-23 2021-12-14 Sangamo Therapeutics Inc Moduladores de la expresioon del gen de marco de lectura abierto 72 del cromosoma 9 y usos de los mismos
AU2020376048A1 (en) 2019-11-01 2022-06-02 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
US20230279440A1 (en) 2020-05-06 2023-09-07 Cellectis S.A. Methods to genetically modify cells for delivery of therapeutic proteins
MX2023000614A (es) 2020-07-16 2023-02-13 Acuitas Therapeutics Inc Lipidos cationicos para usarse en nanoparticulas lipidicas.
US12152251B2 (en) 2020-08-25 2024-11-26 Kite Pharma, Inc. T cells with improved functionality
EP4217479A1 (en) 2020-09-25 2023-08-02 Sangamo Therapeutics, Inc. Zinc finger fusion proteins for nucleobase editing
US20230416709A1 (en) * 2020-11-06 2023-12-28 Editforce, Inc. Foki nuclease domain mutant
JP2024545942A (ja) 2021-11-12 2024-12-16 モンサント テクノロジー エルエルシー 植物の有限性を変えるための組成物及び方法
WO2023105244A1 (en) 2021-12-10 2023-06-15 Pig Improvement Company Uk Limited Editing tmprss2/4 for disease resistance in livestock
GB202118058D0 (en) 2021-12-14 2022-01-26 Univ Warwick Methods to increase yields in crops
IL313486A (en) 2021-12-16 2024-08-01 Acuitas Therapeutics Inc Lipids for use in lipid nanoparticle formulations
IL313758A (en) 2021-12-22 2024-08-01 Sangamo Therapeutics Inc Novel zinc finger fusion proteins for nuclear base editing
GB2621813A (en) 2022-06-30 2024-02-28 Univ Newcastle Preventing disease recurrence in Mitochondrial replacement therapy
US12446503B2 (en) 2022-11-08 2025-10-21 Ohalo Genetics, Inc. Polyploid hybrid potato breeding
US12365908B2 (en) 2022-11-08 2025-07-22 Ohalo Genetics, Inc. Polyploid hybrid maize breeding
TW202448516A (zh) 2023-02-03 2024-12-16 美商健臻公司 接合hsc特異性抗體的脂質奈米粒子及其用途
GB202314578D0 (en) 2023-09-22 2023-11-08 Univ Manchester Methods of producing homoplasmic modified plants or parts thereof

Family Cites Families (62)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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
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
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
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
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
WO1991017424A1 (en) 1990-05-03 1991-11-14 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
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
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The 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
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
EP0781331B1 (en) 1994-08-20 2008-09-03 Gendaq Limited 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
US5928638A (en) 1996-06-17 1999-07-27 Systemix, Inc. Methods for gene transfer
GB9616139D0 (en) 1996-08-01 1996-09-11 Grampian Pharm Ltd Veterinary treatments
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
US6145121A (en) 1997-04-17 2000-11-07 University Of Washington Trace based method for the analysis, benchmarking and tuning of object oriented databases and applications
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
US6800744B1 (en) 1997-07-02 2004-10-05 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to Streptococcus pneumoniae for diagnostics and therapeutics
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US7257541B1 (en) 1999-10-08 2007-08-14 I2 Technologies Us, Inc. System and method for performing a business process in a multi-enterprise, collaborating network
AU776576B2 (en) 1999-12-06 2004-09-16 Sangamo Biosciences, Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
JP5047437B2 (ja) * 2000-02-08 2012-10-10 サンガモ バイオサイエンシーズ, インコーポレイテッド 薬物の発見のための細胞
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology 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
CA2435394C (en) 2001-01-22 2018-01-09 Sangamo Biosciences, Inc. Modified zinc finger binding proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) * 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Cnnについての亜鉛フィンガー結合ドメイン
WO2003087341A2 (en) * 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
US20030232410A1 (en) * 2002-03-21 2003-12-18 Monika Liljedahl 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
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
EP3222715A1 (en) 2003-08-08 2017-09-27 Sangamo BioSciences, Inc. Methods and compositions for targeted cleavage and recombination
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
WO2007014275A2 (en) 2005-07-26 2007-02-01 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
EP2213731B1 (en) * 2006-05-25 2013-12-04 Sangamo BioSciences, Inc. Variant foki cleavage half-domains
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
CA2667414C (en) 2006-11-13 2015-12-29 Sangamo Biosciences, Inc. Methods and compositions for modification of the human glucocorticoid receptor locus
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
CA2755192C (en) * 2009-03-20 2018-09-11 Sangamo Biosciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
ES2751916T3 (es) * 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit

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