SG10201913948PA - Engineered target specific nucleases - Google Patents

Engineered target specific nucleases

Info

Publication number
SG10201913948PA
SG10201913948PA SG10201913948PA SG10201913948PA SG10201913948PA SG 10201913948P A SG10201913948P A SG 10201913948PA SG 10201913948P A SG10201913948P A SG 10201913948PA SG 10201913948P A SG10201913948P A SG 10201913948PA SG 10201913948P A SG10201913948P A SG 10201913948PA
Authority
SG
Singapore
Prior art keywords
target specific
specific nucleases
engineered target
engineered
nucleases
Prior art date
Application number
SG10201913948PA
Other languages
English (en)
Inventor
Jeffrey C Miller
Edward J Rebar
Original Assignee
Sangamo Therapeutics 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 Sangamo Therapeutics Inc filed Critical Sangamo Therapeutics Inc
Publication of SG10201913948PA publication Critical patent/SG10201913948PA/en

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
    • C12N15/87Introduction 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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)
    • 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 RNAses, DNAses
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
SG10201913948PA 2016-08-24 2017-08-24 Engineered target specific nucleases SG10201913948PA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662378978P 2016-08-24 2016-08-24
US201762443981P 2017-01-09 2017-01-09

Publications (1)

Publication Number Publication Date
SG10201913948PA true SG10201913948PA (en) 2020-03-30

Family

ID=61246273

Family Applications (3)

Application Number Title Priority Date Filing Date
SG10201913948PA SG10201913948PA (en) 2016-08-24 2017-08-24 Engineered target specific nucleases
SG10202007392SA SG10202007392SA (en) 2016-08-24 2017-08-24 Engineered target specific nucleases
SG11201901364VA SG11201901364VA (en) 2016-08-24 2017-08-24 Engineered target specific nucleases

Family Applications After (2)

Application Number Title Priority Date Filing Date
SG10202007392SA SG10202007392SA (en) 2016-08-24 2017-08-24 Engineered target specific nucleases
SG11201901364VA SG11201901364VA (en) 2016-08-24 2017-08-24 Engineered target specific nucleases

Country Status (12)

Country Link
US (4) US10975393B2 (ko)
EP (2) EP3504327B1 (ko)
JP (2) JP7272948B2 (ko)
KR (2) KR20220145913A (ko)
CN (1) CN110418841A (ko)
AU (1) AU2017315414B2 (ko)
BR (1) BR112019003327A2 (ko)
CA (1) CA3034884A1 (ko)
ES (1) ES2901989T3 (ko)
IL (2) IL300783A (ko)
SG (3) SG10201913948PA (ko)
WO (1) WO2018039448A1 (ko)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
NO2718257T3 (ko) * 2014-05-30 2018-04-14
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US20190225955A1 (en) 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
PE20190568A1 (es) 2016-08-24 2019-04-22 Sangamo Therapeutics Inc Regulacion de la expresion genica usando nucleasas modificadas
US10960085B2 (en) 2016-09-07 2021-03-30 Sangamo Therapeutics, Inc. Modulation of liver genes
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018075736A1 (en) 2016-10-20 2018-04-26 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of fabry disease
CA3041668A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
WO2018102665A1 (en) 2016-12-01 2018-06-07 Sangamo Therapeutics, Inc. Tau modulators and methods and compositions for delivery thereof
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TW201839136A (zh) 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
IL269458B2 (en) 2017-03-23 2024-02-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
CA3061612A1 (en) 2017-04-28 2018-11-01 Acuitas Therapeutics, Inc. Novel carbonyl lipids and lipid nanoparticle formulations for delivery of nucleic acids
RU2019139045A (ru) 2017-05-03 2021-06-03 Сангамо Терапьютикс, Инк. Способы и композиции для модификации гена регулятора трансмембранной проводимости при кистозном фиброзе (cftr)
WO2018209320A1 (en) 2017-05-12 2018-11-15 President And Fellows Of Harvard College Aptazyme-embedded guide rnas for use with crispr-cas9 in genome editing and transcriptional activation
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
EP3697906A1 (en) 2017-10-16 2020-08-26 The Broad Institute, Inc. Uses of adenosine base editors
CA3079748A1 (en) 2017-11-09 2019-05-16 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible sh2-containing protein (cish) gene
EP3749350A4 (en) 2018-02-08 2021-12-01 Sangamo Therapeutics, Inc. SPECIFICALLY MODIFIED TARGET-SPECIFIC NUCLEASES
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
WO2020006131A2 (en) * 2018-06-27 2020-01-02 Altius Institute For Biomedical Sciences Nucleases for genome editing
JP2021536229A (ja) 2018-08-23 2021-12-27 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
KR20210062639A (ko) * 2018-08-27 2021-05-31 고쿠리츠다이가쿠호진 히로시마다이가쿠 신규 뉴클레아제 도메인 및 그 이용
JP2022500052A (ja) * 2018-09-18 2022-01-04 サンガモ セラピューティクス, インコーポレイテッド プログラム細胞死1(pd1)特異的ヌクレアーゼ
WO2020146805A1 (en) 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
MX2021012152A (es) 2019-04-02 2021-11-03 Sangamo Therapeutics Inc Metodos para el tratamiento de beta-talasemia.
CN113766921A (zh) 2019-04-23 2021-12-07 桑格摩生物治疗股份有限公司 染色体9开放阅读框72基因表达的调节物及其用途
WO2020237045A1 (en) 2019-05-21 2020-11-26 Sangamo Therapeutics, Inc. Controlled transgene expression in regulatory t cells
JP2022550608A (ja) 2019-10-02 2022-12-02 サンガモ セラピューティクス, インコーポレイテッド プリオン病の治療のためのジンクフィンガータンパク質転写因子
EP4041754A1 (en) 2019-10-02 2022-08-17 Sangamo Therapeutics, Inc. Zinc finger protein transcription factors for repressing alpha-synuclein expression
US20210130828A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Gin recombinase variants
AU2020376048A1 (en) 2019-11-01 2022-06-02 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
KR20220131273A (ko) 2020-01-22 2022-09-27 상가모 테라퓨틱스, 인코포레이티드 타우 발현을 억제시키기 위한 아연 핑거 단백질 전사 인자
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
CA3189338A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
EP4204545A2 (en) 2020-08-25 2023-07-05 Kite Pharma, Inc. T cells with improved functionality
CA3196599A1 (en) 2020-09-25 2022-03-31 Sangamo Therapeutics, Inc. Zinc finger fusion proteins for nucleobase editing
TW202229320A (zh) 2020-10-02 2022-08-01 美商聖加莫治療股份有限公司 用於抑制α-突觸核蛋白(SYNUCLEIN)表現之新穎鋅指蛋白轉錄因子
CN116406383A (zh) * 2020-11-06 2023-07-07 日商基因编辑力股份有限公司 FokI核酸酶结构域的突变体
CN112195164B (zh) * 2020-12-07 2021-04-23 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法
CN112680431B (zh) * 2021-03-22 2021-06-11 吴江近岸蛋白质科技有限公司 基于片段化酶的dna文库制备方法
CN113293165B (zh) * 2021-06-21 2022-11-15 军事科学院军事医学研究院环境医学与作业医学研究所 一种基于CRISPR-Cas12a技术的HEV特异crRNA、检测试剂盒及其应用
WO2023056291A1 (en) * 2021-09-28 2023-04-06 Acrigen Biosciences Compositions and methods for nucleic acid modifications
AU2022421236A1 (en) 2021-12-22 2024-06-27 Sangamo Therapeutics, Inc. Novel zinc finger fusion proteins for nucleobase editing
WO2024036285A2 (en) * 2022-08-11 2024-02-15 Medisix Therapeutics, Inc. Engineered immune cells expressing a car and a plurality of protein expression blockers and uses thereof
WO2024116373A1 (ja) * 2022-12-01 2024-06-06 国立大学法人広島大学 Dnaを二本鎖切断するヌクレアーゼドメイン

Family Cites Families (143)

* 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
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
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
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
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
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2681922C (en) 1994-01-18 2012-05-15 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
WO1995025809A1 (en) 1994-03-23 1995-09-28 Ohio University Compacted nucleic acids and their delivery to cells
ATE407205T1 (de) 1994-08-20 2008-09-15 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der erkennung von dna
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
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
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics 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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
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
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding 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
DE60143192D1 (de) 2000-02-08 2010-11-18 Sangamo Biosciences Inc Zellen zur entdeckung von medikamenten
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
DE60126483T2 (de) 2000-04-28 2007-12-06 Sangamo BioSciences, Inc., Richmond Gezielte Modifikation der Chromatinstruktur
ATE374249T1 (de) 2000-04-28 2007-10-15 Sangamo Biosciences Inc Datenbanken von regulatorischen sequenzen, methoden zu ihrer herstellung und verwendung
US7923542B2 (en) 2000-04-28 2011-04-12 Sangamo Biosciences, Inc. Libraries of regulatory sequences, methods of making and using 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
US6919204B2 (en) 2000-09-29 2005-07-19 Sangamo Biosciences, Inc. Modulation of gene expression using localization domains
WO2002044376A2 (en) 2000-11-28 2002-06-06 Sangamo Biosciences, Inc. Modulation of gene expression using insulator binding proteins
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US7273923B2 (en) 2001-01-22 2007-09-25 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
EP1421177A4 (en) 2001-08-20 2006-06-07 Scripps Research Inst ZINC FINGER FASTENING DOMAINS FOR CNN
US7271002B2 (en) 2001-11-09 2007-09-18 United States Of America, Represented By The Secretary, Department Of Health And Human Services Production of adeno-associated virus in insect cells
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
US20060078552A1 (en) 2002-03-15 2006-04-13 Sylvain Arnould Hybrid and single chain meganucleases and use thereof
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7074596B2 (en) 2002-03-25 2006-07-11 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Synthesis and use of anti-reverse mRNA cap analogues
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP1581610A4 (en) 2002-09-05 2009-05-27 California Inst Of Techn USE OF CHIMERIC NUCLEASES TO STIMULATE GENE TARGETING
JP4966006B2 (ja) 2003-01-28 2012-07-04 セレクティス カスタムメイドメガヌクレアーゼおよびその使用
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
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
EP1737498A2 (en) 2004-04-08 2007-01-03 Sangamo Biosciences Inc. Zinc finger proteins for treatment of neuropathic pain
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US20060063231A1 (en) 2004-09-16 2006-03-23 Sangamo Biosciences, Inc. Compositions and methods for protein production
CA2607104A1 (en) 2005-05-05 2006-11-16 The Arizona Board Of Regents On Behalf Of The University Of Arizona Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
CN101273141B (zh) 2005-07-26 2013-03-27 桑格摩生物科学股份有限公司 外源核酸序列的靶向整合和表达
EP2341135A3 (en) 2005-10-18 2011-10-12 Precision Biosciences Rationally-designed meganucleases with altered sequence specificity and DNA-binding affinity
JP5551432B2 (ja) 2006-05-25 2014-07-16 サンガモ バイオサイエンシーズ, インコーポレイテッド 遺伝子不活性化のための方法と組成物
WO2007139898A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Variant foki cleavage half-domains
CA2660485C (en) * 2006-08-11 2016-04-12 Sangamo Biosciences, Inc. Zinc finger nuclease-mediated homologous recombination
ATE489465T1 (de) 2007-04-26 2010-12-15 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
ES2500515T3 (es) 2007-06-19 2014-09-30 Board Of Supervisors Of Louisiana State University And Agricultural And Mechanical College Síntesis y utilización de análogos de fosforotioato antiinversos de la caperuza de ARN mensajero
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
RU2499599C2 (ru) 2007-09-28 2013-11-27 Интрексон Корпорейшн Конструкции терапевтического переключения генов и биореакторы для экспрессии биотерапевтических молекул и их применение
AU2009238629C1 (en) 2008-04-14 2015-04-30 Sangamo Therapeutics, Inc. Linear donor constructs for targeted integration
US9394531B2 (en) 2008-05-28 2016-07-19 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
US8597912B2 (en) 2008-06-10 2013-12-03 Sangamo Biosciences, Inc. Methods and compositions for generation of Bax-and Bak-deficient cell lines
CA2734235C (en) 2008-08-22 2019-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
ES2615829T3 (es) 2008-09-15 2017-06-08 The Children's Medical Center Corporation Modulación de BCL11A para el tratamiento de hemoglobinopatías
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
CA2769670C (en) 2009-07-31 2018-10-02 Ethris Gmbh Rna with a combination of unmodified and modified nucleotides for protein expression
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
US8961281B2 (en) 2009-09-28 2015-02-24 Entsorgafin S.P.A. Ventilation group for flow reversal
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
DE112010004584T5 (de) * 2009-11-27 2012-11-29 Basf Plant Science Company Gmbh Chimäre Endonukleasen und Anwendungen davon
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
BR112012018899A2 (pt) 2010-01-28 2015-09-15 Philadelphia Children Hospital "método para purificar partículas de vetor de vírus adeno-associado."
EP4328304A2 (en) * 2010-02-08 2024-02-28 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2660318A1 (en) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US9567573B2 (en) 2010-04-26 2017-02-14 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using nucleases
US8772453B2 (en) 2010-05-03 2014-07-08 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
CA2805442C (en) 2010-07-21 2020-05-12 Sangamo Biosciences, Inc. Methods and compositions for modification of an hla locus
US9057057B2 (en) 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
GB201014169D0 (en) 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
KR102209330B1 (ko) 2011-09-21 2021-01-29 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
US9222105B2 (en) 2011-10-27 2015-12-29 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
WO2013074999A1 (en) 2011-11-16 2013-05-23 Sangamo Biosciences, Inc. Modified dna-binding proteins and uses thereof
KR102116153B1 (ko) 2012-05-07 2020-05-27 상가모 테라퓨틱스, 인코포레이티드 도입유전자의 뉴클레아제-매개 표적화된 통합을 위한 방법 및 조성물
EA201492222A1 (ru) * 2012-05-25 2015-05-29 Селлектис Способы конструирования неаллореактивной и устойчивой к иммуносупрессии т-клетки для иммунотерапии
EP2890780B8 (en) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
US9597357B2 (en) 2012-10-10 2017-03-21 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
EP2929017A4 (en) 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHODS AND COMPOSITIONS FOR REGULATION OF METABOLIC DISORDERS
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
JP6392245B2 (ja) 2012-12-18 2018-09-19 ノバルティス アーゲー 遺伝子改変哺乳動物細胞における治療用タンパク質の産生
CA3161835A1 (en) 2013-03-15 2014-09-25 The General Hospital Corporation Rna-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
WO2014153470A2 (en) 2013-03-21 2014-09-25 Sangamo Biosciences, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
JP2016524464A (ja) * 2013-05-13 2016-08-18 セレクティスCellectis 免疫療法のために高活性t細胞を操作するための方法
US9873894B2 (en) 2013-05-15 2018-01-23 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3988654A1 (en) 2013-08-28 2022-04-27 Sangamo Therapeutics, Inc. Compositions for linking dna-binding domains and cleavage domains
CN110713995B (zh) 2013-10-17 2023-08-01 桑格摩生物科学股份有限公司 用于核酸酶介导的基因组工程改造的递送方法和组合物
PT3492593T (pt) 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
CN105940013B (zh) 2013-12-09 2020-03-27 桑格摩生物科学股份有限公司 用于治疗血友病的方法和组合物
US20170037431A1 (en) 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
CN106413760B (zh) 2014-05-08 2020-01-14 桑格摩生物科学股份有限公司 用于治疗亨廷顿病的方法和组合物
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
EP3054007A1 (en) 2015-02-09 2016-08-10 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant adeno-associated virus particle purification comprising an immuno-affinity purification step
MX2017014446A (es) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
CA2991301A1 (en) * 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CA3008413A1 (en) 2015-12-18 2017-06-22 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
JP7012650B2 (ja) 2016-02-02 2022-01-28 サンガモ セラピューティクス, インコーポレイテッド Dna結合ドメインと切断ドメインとを連結するための組成物
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
PE20190568A1 (es) 2016-08-24 2019-04-22 Sangamo Therapeutics Inc Regulacion de la expresion genica usando nucleasas modificadas
WO2018107026A1 (en) 2016-12-09 2018-06-14 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
SG11202001035TA (en) * 2017-08-08 2020-03-30 Sangamo Therapeutics Inc Chimeric antigen receptor mediated cell targeting

Also Published As

Publication number Publication date
EP3504327A4 (en) 2020-02-12
SG10202007392SA (en) 2020-09-29
EP3504327A1 (en) 2019-07-03
EP3504327B1 (en) 2021-10-06
AU2017315414B2 (en) 2024-02-15
KR20220145913A (ko) 2022-10-31
US20200340016A1 (en) 2020-10-29
US10975393B2 (en) 2021-04-13
RU2019108242A3 (ko) 2021-04-09
IL300783A (en) 2023-04-01
AU2017315414A2 (en) 2020-03-12
JP7272948B2 (ja) 2023-05-12
WO2018039448A9 (en) 2019-04-25
KR102455249B1 (ko) 2022-10-17
US20240132917A1 (en) 2024-04-25
RU2019108242A (ru) 2020-09-25
JP2023100751A (ja) 2023-07-19
WO2018039448A1 (en) 2018-03-01
US11827900B2 (en) 2023-11-28
IL264792B1 (en) 2023-06-01
CN110418841A (zh) 2019-11-05
US20180087072A1 (en) 2018-03-29
US20210024957A1 (en) 2021-01-28
JP2019528073A (ja) 2019-10-10
IL264792B2 (en) 2023-10-01
SG11201901364VA (en) 2019-03-28
EP3964573A1 (en) 2022-03-09
KR20190039955A (ko) 2019-04-16
AU2017315414A9 (en) 2019-07-18
IL264792A (ko) 2019-03-31
ES2901989T3 (es) 2022-03-24
CA3034884A1 (en) 2018-03-01
US20210024958A1 (en) 2021-01-28
BR112019003327A2 (pt) 2019-07-02
AU2017315414A1 (en) 2019-03-14

Similar Documents

Publication Publication Date Title
SG10202007392SA (en) Engineered target specific nucleases
HK1257676A1 (zh) 工程化 crispr-cas 9 核酸酶
IL271342A (en) Nucleases are guided by nucleic acids
IL254187A0 (en) beta-tgf inhibitors
IL276490A (en) Engineered target-specific nucleases
PL3504506T3 (pl) Cel
GB2540441B (en) Amplifiers
GB201501482D0 (en) Cost-effective tracking
EP3353244A4 (en) POLYMER AMINOACRYLATE
GB201611925D0 (en) Cyclotron target
GB2536673B (en) Evaporator
DK3347540T3 (en) Bærer til et lineært loftspanel
GB201515809D0 (en) Actuator
GB201500242D0 (en) Projectile
IL257500A (en) Altered cullin1 gene
GB201509204D0 (en) Improved tracking
GB201517270D0 (en) Target designator
SG11201704051SA (en) Target assembly
FI20160292A (fi) Tarkkuusmaali
GB201520386D0 (en) Actuator
SG11201700721QA (en) Target assembly
GB201503312D0 (en) Targets
GB2537392B (en) Improved evaporator
IL247839B (en) muzzle
SG10201600814XA (en) Nuclease