SG10202112057QA - Nuclease-mediated regulation of gene expression - Google Patents

Nuclease-mediated regulation of gene expression

Info

Publication number
SG10202112057QA
SG10202112057QA SG10202112057QA SG10202112057QA SG10202112057QA SG 10202112057Q A SG10202112057Q A SG 10202112057QA SG 10202112057Q A SG10202112057Q A SG 10202112057QA SG 10202112057Q A SG10202112057Q A SG 10202112057QA SG 10202112057Q A SG10202112057Q A SG 10202112057QA
Authority
SG
Singapore
Prior art keywords
nuclease
gene expression
mediated regulation
mediated
regulation
Prior art date
Application number
SG10202112057QA
Other languages
English (en)
Inventor
Andreas Reik
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 SG10202112057QA publication Critical patent/SG10202112057QA/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Materials For Medical Uses (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
SG10202112057QA 2015-05-12 2016-05-12 Nuclease-mediated regulation of gene expression SG10202112057QA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562160396P 2015-05-12 2015-05-12
US201662303595P 2016-03-04 2016-03-04

Publications (1)

Publication Number Publication Date
SG10202112057QA true SG10202112057QA (en) 2021-12-30

Family

ID=57249092

Family Applications (1)

Application Number Title Priority Date Filing Date
SG10202112057QA SG10202112057QA (en) 2015-05-12 2016-05-12 Nuclease-mediated regulation of gene expression

Country Status (17)

Country Link
US (2) US10808020B2 (enrdf_load_stackoverflow)
EP (1) EP3294866A4 (enrdf_load_stackoverflow)
JP (2) JP6873917B2 (enrdf_load_stackoverflow)
KR (1) KR20170141217A (enrdf_load_stackoverflow)
AU (2) AU2016261927B2 (enrdf_load_stackoverflow)
BR (1) BR112017024115A2 (enrdf_load_stackoverflow)
CA (1) CA2984013A1 (enrdf_load_stackoverflow)
CL (1) CL2017002843A1 (enrdf_load_stackoverflow)
CO (1) CO2017012026A2 (enrdf_load_stackoverflow)
HK (1) HK1251011A1 (enrdf_load_stackoverflow)
IL (3) IL284707B2 (enrdf_load_stackoverflow)
MX (2) MX382223B (enrdf_load_stackoverflow)
PH (1) PH12017502026A1 (enrdf_load_stackoverflow)
SA (1) SA517390307B1 (enrdf_load_stackoverflow)
SG (1) SG10202112057QA (enrdf_load_stackoverflow)
WO (1) WO2016183298A2 (enrdf_load_stackoverflow)
ZA (1) ZA201707016B (enrdf_load_stackoverflow)

Families Citing this family (51)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
WO2015073683A2 (en) * 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
CA2984013A1 (en) * 2015-05-12 2016-11-17 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CA2998500A1 (en) 2015-09-23 2017-03-30 Sangamo Therapeutics, Inc. Htt repressors and uses thereof
US10167457B2 (en) 2015-10-23 2019-01-01 President And Fellows Of Harvard College Nucleobase editors and uses thereof
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
NZ750943A (en) 2016-08-24 2025-06-27 Sangamo Therapeutics Inc Engineered target specific nucleases
WO2018039438A1 (en) 2016-08-24 2018-03-01 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
CA3039928A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
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
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
CN110914426A (zh) 2017-03-23 2020-03-24 哈佛大学的校长及成员们 包含核酸可编程dna结合蛋白的核碱基编辑器
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
EP3720473A1 (en) * 2017-12-05 2020-10-14 Vertex Pharmaceuticals Incorporated Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CA3109592A1 (en) 2018-08-23 2020-02-27 Sangamo Therapeutics, Inc. Engineered target specific base editors
EP3852911B1 (en) 2018-09-21 2025-01-22 Acuitas Therapeutics, Inc. Systems and methods for manufacturing lipid nanoparticles and liposomes
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
PL3908568T3 (pl) 2019-01-11 2024-12-02 Acuitas Therapeutics, Inc. Lipidy do dostarczania środków czynnych w nanocząstkach lipidowych
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
EP3946384A1 (en) 2019-04-02 2022-02-09 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
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
WO2022016070A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
WO2022118310A1 (en) * 2020-12-01 2022-06-09 Lepton Pharmaceuticals Ltd. Methods for enhancing therapeutic efficacy of isolated cells for cell therapy
KR20240055084A (ko) 2021-09-13 2024-04-26 라이프 테크놀로지스 코포레이션 유전자 편집 도구
US20250043269A1 (en) 2021-11-03 2025-02-06 The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad Precise Genome Editing Using Retrons
JP2024546952A (ja) 2021-12-16 2024-12-26 アクイタス セラピューティクス インコーポレイテッド 脂質ナノ粒子製剤に用いるための脂質
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2025049959A2 (en) 2023-09-01 2025-03-06 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents

Family Cites Families (114)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994026877A1 (en) * 1993-05-17 1994-11-24 The Regents Of The University Of California Ribozyme gene therapy for hiv infection and aids
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
EP0770129B1 (en) 1994-01-18 2005-11-23 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE45795E1 (en) 1994-08-20 2015-11-10 Gendaq, Ltd. 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
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
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
CA2321938C (en) 1998-03-02 2009-11-24 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
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
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
ATE309536T1 (de) 1999-12-06 2005-11-15 Sangamo Biosciences Inc Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
EP2207032A1 (en) 2000-02-08 2010-07-14 Sangamo BioSciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith 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
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
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
EP1504092B2 (en) 2002-03-21 2014-06-25 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
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 GEN-TARGETING
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
EP1651660B1 (en) * 2003-08-08 2018-01-24 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2562193A1 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
EP1732614B1 (en) 2004-04-08 2008-12-24 Sangamo Biosciences Inc. Compositions for treating neuropathic and neurodegenerative conditions
WO2006033859A2 (en) * 2004-09-16 2006-03-30 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
WO2007014181A2 (en) * 2005-07-25 2007-02-01 Johns Hopkins University Site-specific modification of the human genome using custom-designed zinc finger nucleases
JP2009502170A (ja) * 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
DE102005054628A1 (de) 2005-11-16 2007-05-24 Cevec Pharmaceuticals Gmbh Verfahren zur Herstellung von permanenten humanen Zelllinien
AU2007254251A1 (en) * 2006-05-19 2007-11-29 Sangamo Therapeutics, Inc. Methods and compositions for inactivation of dihydrofolate reductase
ES2378333T3 (es) * 2006-05-25 2012-04-11 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación de genes
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
KR101439568B1 (ko) * 2006-08-11 2014-09-12 다우 아그로사이언시즈 엘엘씨 아연 손가락 뉴클레아제-매개 상동 재조합
EP2137310B1 (en) 2007-04-26 2010-11-24 Sangamo BioSciences, Inc. Targeted integration into the ppp1r12c locus
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
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
AU2008305567B2 (en) 2007-09-27 2014-04-10 Sangamo Therapeutics, Inc. Rapid in vivo identification of biologically active nucleases
CA2700170A1 (en) * 2007-09-27 2009-04-02 Sangamo Biosciences, Inc. Genomic editing in zebrafish using zinc finger nucleases
WO2009131632A1 (en) * 2008-04-14 2009-10-29 Sangamo Biosciences, Inc. Linear donor constructs for targeted integration
WO2009154686A1 (en) 2008-05-28 2009-12-23 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
CN102159722B (zh) 2008-08-22 2014-09-03 桑格摩生物科学股份有限公司 用于靶向单链切割和靶向整合的方法和组合物
EP3208339B1 (en) 2008-09-15 2019-05-01 The Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
EP2344660B1 (en) * 2008-10-29 2018-01-17 Sangamo Therapeutics, Inc. Methods and compositions for inactivating glutamine synthetase gene expression
SG172760A1 (en) * 2008-12-04 2011-08-29 Sangamo Biosciences Inc Genome editing in rats using zinc-finger nucleases
CA2756833C (en) * 2009-04-09 2019-11-19 Sangamo Biosciences, Inc. Targeted integration into stem cells
JP5866283B2 (ja) * 2009-07-28 2016-02-17 サンガモ バイオサイエンシーズ， インコーポレイテッド トリヌクレオチド反復疾患を治療するための方法および組成物
JP5785168B2 (ja) 2009-07-31 2015-09-24 エスリス ゲーエムベーハーｅｔｈｒｉｓ ＧｍｂＨ タンパク質発現用未修飾および修飾ヌクレオチドの組み合わせを有するｒｎａ
BR112012009668B1 (pt) * 2009-10-22 2022-08-16 Dow Agrosciences Llc Proteína de dedo de zinco que não ocorre naturalmente e proteína de fusão compreendendo a mesma
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
CA2787494C (en) * 2010-01-22 2019-09-17 Dow Agrosciences Llc Targeted genomic alteration
EP2534173B1 (en) * 2010-02-08 2019-09-11 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
US9255259B2 (en) 2010-02-09 2016-02-09 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8771985B2 (en) 2010-04-26 2014-07-08 Sangamo Biosciences, Inc. Genome editing of a Rosa locus using zinc-finger nucleases
WO2011139349A1 (en) 2010-05-03 2011-11-10 Sangamo Biosciences, Inc. Compositions for linking zinc finger modules
WO2011146121A1 (en) 2010-05-17 2011-11-24 Sangamo Biosciences, Inc. Novel dna-binding proteins and uses thereof
CA2993567C (en) 2010-07-21 2022-06-28 Sangamo Biosciences, Inc. Methods and compositions for modification of a t-cell receptor gene
US9512444B2 (en) * 2010-07-23 2016-12-06 Sigma-Aldrich Co. Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
US20140127814A1 (en) * 2010-08-10 2014-05-08 Srinivasan Chandrasegaran Generation and use of pluripotent stem cells
US20120214241A1 (en) * 2010-12-22 2012-08-23 Josee Laganiere Zinc finger nuclease modification of leucine rich repeat kinase 2 (lrrk2) mutant fibroblasts and ipscs
KR101556359B1 (ko) * 2011-01-03 2015-10-01 주식회사 툴젠 디자인된 tal 이펙터 뉴클레아제를 통한 게놈 엔지니어링
US9267123B2 (en) 2011-01-05 2016-02-23 Sangamo Biosciences, Inc. Methods and compositions for gene correction
US20120204282A1 (en) * 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
EP2522726A1 (en) * 2011-05-12 2012-11-14 Fundació Privada Centre de Regulació Genòmica (CRG) Zinc finger nucleases for p53 editing
WO2012152912A1 (en) * 2011-05-12 2012-11-15 Newvectys Genetically modified pig as a cancer prone model
AU2012286901B2 (en) * 2011-07-25 2016-10-27 Sangamo Therapeutics, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
WO2013019745A1 (en) * 2011-07-29 2013-02-07 Georgia Health Sciences University Methods and compositions for genetically modifiying cells
ES2961613T3 (es) 2011-09-21 2024-03-12 Sangamo Therapeutics Inc Métodos y composiciones para la regulación de la expresión transgénica
US8895264B2 (en) 2011-10-27 2014-11-25 Sangamo Biosciences, Inc. Methods and compositions for modification of the HPRT locus
JP6144691B2 (ja) 2011-11-16 2017-06-07 サンガモ セラピューティクス， インコーポレイテッド 修飾されたｄｎａ結合タンパク質およびその使用
CN104080462B (zh) * 2011-12-16 2019-08-09 塔尔盖特基因生物技术有限公司 用于修饰预定的靶核酸序列的组合物和方法
WO2013112595A2 (en) * 2012-01-23 2013-08-01 Sangamo Biosciences, Inc. Methods and compositions for gene editing of a pathogen
RU2650811C2 (ru) * 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
DE18200782T1 (de) * 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
US20130280222A1 (en) * 2012-04-18 2013-10-24 Board Of Regents Of The University Of Texas System Non-disruptive gene targeting
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
UA118652C2 (uk) * 2012-05-25 2019-02-25 Селлектіс Спосіб конструювання неалореактивної і стійкої до імуносупресії т-клітини для імунотерапії
US9834782B2 (en) * 2012-05-28 2017-12-05 Evogene Ltd. Isolated polynucleotides and polypeptides, and methods of using same for increasing plant yield and/or agricultural characteristics
DK2890780T3 (da) * 2012-08-29 2020-09-21 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til behandling af en genetisk tilstand
CA2892860C (en) * 2012-11-27 2023-01-03 Children's Medical Center Corporation Targeting bcl11a distal regulatory elements for fetal hemoglobin reinduction
WO2014089212A1 (en) 2012-12-05 2014-06-12 Sangamo Biosciences, Inc. Methods and compositions for regulation of metabolic disorders
DK3138910T3 (en) * 2012-12-06 2017-10-16 Sigma Aldrich Co Llc CRISPR-BASED RE-MODIFICATION AND REGULATION
WO2014089541A2 (en) * 2012-12-07 2014-06-12 Haplomics, Inc. Factor viii mutation repair and tolerance induction
CN116083487A (zh) 2013-05-15 2023-05-09 桑格摩生物治疗股份有限公司 用于治疗遗传病状的方法和组合物
CA2920899C (en) 2013-08-28 2023-02-28 Sangamo Biosciences, Inc. Compositions for linking dna-binding domains and cleavage domains
EP3057432B1 (en) * 2013-10-17 2018-11-21 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
CN105916983A (zh) * 2013-10-25 2016-08-31 塞勒克提斯公司 用于高效且特异性靶向包含高度重复基序的dna序列的稀有切割核酸内切酶的设计
WO2015073683A2 (en) 2013-11-13 2015-05-21 Children's Medical Center Corporation Nuclease-mediated regulation of gene expression
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
JP6722176B2 (ja) * 2014-09-16 2020-07-15 サンガモ セラピューティクス， インコーポレイテッド 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物
AU2016225178B2 (en) * 2015-02-23 2022-05-05 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
JP7288302B2 (ja) * 2015-05-08 2023-06-07 ザ チルドレンズ メディカル センター コーポレーション 胎児型ヘモグロビン再誘導のための、ｂｃｌ１１ａエンハンサー機能性領域を標的とする方法
CA2984013A1 (en) * 2015-05-12 2016-11-17 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
EP3371306B8 (en) * 2015-11-04 2023-02-22 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
IL308706A (en) * 2015-12-28 2024-01-01 Novartis Ag Preparations and methods for the treatment of hemoglobinopathies
DK3445388T3 (da) * 2016-04-18 2024-06-03 Vertex Pharma Materialer og fremgangsmåder til behandling af hæmoglobinopatier
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies

Also Published As

Publication number Publication date
IL255288A0 (en) 2017-12-31
MX2021005226A (es) 2021-06-18
BR112017024115A2 (pt) 2018-08-07
IL284707B1 (en) 2023-09-01
MX2017014446A (es) 2018-06-13
ZA201707016B (en) 2019-08-28
JP2021106611A (ja) 2021-07-29
JP6873917B2 (ja) 2021-05-19
CO2017012026A2 (es) 2018-02-28
IL255288B (en) 2021-10-31
IL284707A (en) 2021-08-31
WO2016183298A3 (en) 2016-12-29
WO2016183298A2 (en) 2016-11-17
US20210024606A1 (en) 2021-01-28
CL2017002843A1 (es) 2018-05-25
JP2018516556A (ja) 2018-06-28
CA2984013A1 (en) 2016-11-17
AU2022204863B2 (en) 2025-09-04
HK1251011A1 (zh) 2019-01-18
US10808020B2 (en) 2020-10-20
AU2022204863A1 (en) 2022-07-28
JP7365374B2 (ja) 2023-10-19
EP3294866A2 (en) 2018-03-21
SA517390307B1 (ar) 2021-07-13
US20180111975A1 (en) 2018-04-26
PH12017502026A1 (en) 2018-04-02
KR20170141217A (ko) 2017-12-22
AU2016261927B2 (en) 2022-04-07
IL287033A (en) 2021-12-01
EP3294866A4 (en) 2018-12-05
AU2016261927A1 (en) 2017-11-02
IL284707B2 (en) 2024-01-01
MX382223B (es) 2025-03-13

Similar Documents

Publication Publication Date Title
IL287033A (en) Regulation of nuclease-mediated gene expression
IL284796A (en) Control of gene expression through aptamer-mediated modulation of alternative splicing
ZA201900765B (en) Regulation of gene expression using engineered nucleases
LT3492593T (lt) Nukleazės sąlygojama genų raiškos reguliacija
IL255515A (en) Identification of genetic changes
EP3118315A4 (en) Nucleic acid that inhibits expression of irf5
ZA201804397B (en) Biosynthetic gene cluster of carrimycin
PT3167068T (pt) Controlo de processos de conversão de monóxido de carbono em biorreatores
IL249254B (en) Gene expression system
IL260840B1 (en) Regulation of gene expression through aptamer-modulated polyadenylation
IL257500A (en) Altered cullin1 gene