ATE313625T1 - ßFUNCTIONAL GENOMICSß UNTER VERWENDUNG VON ZINK FINGER PROTEINEN - Google Patents

ßFUNCTIONAL GENOMICSß UNTER VERWENDUNG VON ZINK FINGER PROTEINEN

Info

Publication number
ATE313625T1
ATE313625T1 AT00963362T AT00963362T ATE313625T1 AT E313625 T1 ATE313625 T1 AT E313625T1 AT 00963362 T AT00963362 T AT 00963362T AT 00963362 T AT00963362 T AT 00963362T AT E313625 T1 ATE313625 T1 AT E313625T1
Authority
AT
Austria
Prior art keywords
zinc finger
finger proteins
ßfunctional
genomicsß
gene expression
Prior art date
Application number
AT00963362T
Other languages
English (en)
Inventor
Casey C Case
Lei Zhang
Original Assignee
Sangamo Biosciences 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 Biosciences Inc filed Critical Sangamo Biosciences Inc
Application granted granted Critical
Publication of ATE313625T1 publication Critical patent/ATE313625T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or 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
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034Isolating an individual clone by screening libraries
    • C12N15/1055Protein x Protein interaction, e.g. two hybrid selection
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/1034Isolating an individual clone by screening libraries
    • C12N15/1079Screening libraries by altering the phenotype or phenotypic trait of the host
    • 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
    • C12N15/82Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8216Methods for controlling, regulating or enhancing expression of transgenes in plant 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
    • 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
    • C12N15/82Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8241Phenotypically and genetically modified plants via recombinant DNA technology
    • 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
    • C12N15/82Vectors or expression systems specially adapted for eukaryotic hosts for plant cells, e.g. plant artificial chromosomes (PACs)
    • C12N15/8241Phenotypically and genetically modified plants via recombinant DNA technology
    • C12N15/8242Phenotypically and genetically modified plants via recombinant DNA technology with non-agronomic quality (output) traits, e.g. for industrial processing; Value added, non-agronomic traits
    • C12N15/8243Phenotypically and genetically modified plants via recombinant DNA technology with non-agronomic quality (output) traits, e.g. for industrial processing; Value added, non-agronomic traits involving biosynthetic or metabolic pathways, i.e. metabolic engineering, e.g. nicotine, caffeine
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Bioinformatics & Computational Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Nutrition Science (AREA)
  • Food Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
AT00963362T 1999-09-14 2000-09-12 ßFUNCTIONAL GENOMICSß UNTER VERWENDUNG VON ZINK FINGER PROTEINEN ATE313625T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US09/395,448 US6599692B1 (en) 1999-09-14 1999-09-14 Functional genomics using zinc finger proteins
PCT/US2000/024897 WO2001019981A2 (en) 1999-09-14 2000-09-12 Functional genomics using zinc finger proteins

Publications (1)

Publication Number Publication Date
ATE313625T1 true ATE313625T1 (de) 2006-01-15

Family

ID=23563088

Family Applications (1)

Application Number Title Priority Date Filing Date
AT00963362T ATE313625T1 (de) 1999-09-14 2000-09-12 ßFUNCTIONAL GENOMICSß UNTER VERWENDUNG VON ZINK FINGER PROTEINEN

Country Status (10)

Country Link
US (3) US6599692B1 (de)
EP (1) EP1238067B1 (de)
JP (3) JP2003527093A (de)
KR (1) KR20020065473A (de)
AT (1) ATE313625T1 (de)
AU (1) AU778964B2 (de)
CA (1) CA2383926C (de)
DE (1) DE60025037T2 (de)
HK (1) HK1049501B (de)
WO (1) WO2001019981A2 (de)

Families Citing this family (283)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US20050084885A1 (en) * 1994-01-18 2005-04-21 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
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
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
US20020164575A1 (en) * 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
US7329728B1 (en) * 1999-10-25 2008-02-12 The Scripps Research Institute Ligand activated transcriptional regulator proteins
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
US20020061512A1 (en) * 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
GB0015090D0 (en) * 2000-06-20 2000-08-09 Implyx Ltd Gene-activating conjugates
US7026462B2 (en) * 2000-12-07 2006-04-11 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
US7067617B2 (en) * 2001-02-21 2006-06-27 The Scripps Research Institute Zinc finger binding domains for nucleotide sequence ANN
US20050235369A1 (en) * 2001-03-28 2005-10-20 Yen Choo Gene regulation II
EP1421177A4 (de) * 2001-08-20 2006-06-07 Scripps Research Inst Zinkfinger-bindungsdomänen für cnn
AU2002365796A1 (en) * 2001-12-07 2003-06-17 Toolgen, Inc. Phenotypic screen of chimeric proteins
US20040259258A1 (en) * 2001-12-07 2004-12-23 Kim Jin-Soo Regulation of prokaryotic gene expression with zinc finger proteins
ATE347593T1 (de) 2002-01-23 2006-12-15 Univ Utah Res Found Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen
WO2004018632A2 (en) * 2002-08-23 2004-03-04 Sangamo Biosciences, Inc. High-throughput screen for modulators of chromatin modifying activity
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
EP1581610A4 (de) 2002-09-05 2009-05-27 California Inst Of Techn Verwendung chimerer nukleasen zur stimulierung des gen-targeting
WO2004099367A2 (en) * 2002-10-23 2004-11-18 The General Hospital Corporation Methods for producing zinc finger proteins that bind to extended dna target sequences
WO2004046387A1 (en) * 2002-11-15 2004-06-03 Sangamo Biosciences, Inc. Methods and compositions for analysis of regulatory sequences
US20060251643A1 (en) * 2002-12-09 2006-11-09 Toolgen, Inc. Regulatory zinc finger proteins
CA2532200C (en) * 2003-07-31 2011-08-16 Kao Corporation Powder composition for paper manufacturing
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
US11311574B2 (en) 2003-08-08 2022-04-26 Sangamo Therapeutics, 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
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, 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
US20090312277A1 (en) * 2004-11-19 2009-12-17 Abdelhadi Rebbaa Compositions And Methods For Reversing Or Preventing Resistance Of A Cancer Cell To A Cytotoxic Agent
JP2009520463A (ja) * 2005-11-28 2009-05-28 ザ スクリプス リサーチ インスティテュート Tnnのための亜鉛フィンガー結合ドメイン
US20070154989A1 (en) * 2006-01-03 2007-07-05 The Scripps Research Institute Zinc finger domains specifically binding agc
US20070178499A1 (en) * 2006-01-06 2007-08-02 The Scripps Research Institute Specific Labeling of Protein with Zinc Finger Tags and Use of Zinc-Finger-Tagged Proteins for Analysis
US20090305977A1 (en) * 2006-02-09 2009-12-10 Sangamo Bioscience, Inc. Method for treating peripheral arterial disease with zinc finger proteins
AU2007254251A1 (en) * 2006-05-19 2007-11-29 Sangamo Therapeutics, Inc. Methods and compositions for inactivation of dihydrofolate reductase
WO2007139898A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Variant foki cleavage half-domains
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
US8148129B2 (en) * 2006-06-30 2012-04-03 The Regents Of The University Of California Generation of potent dominant negative transcriptional inhibitors
CN101883850B (zh) 2007-07-12 2014-11-12 桑格摩生物科学股份有限公司 用于失活α-1,6岩藻糖基转移酶(FUT8)基因表达的方法和组合物
US11235026B2 (en) 2007-09-27 2022-02-01 Sangamo Therapeutics, Inc. Methods and compositions for modulating PD1
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2188384B1 (de) * 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Schnelle in-vivo-identifizierung von biologisch aktiven nukleasen
CN101878307B (zh) * 2007-09-27 2017-07-28 陶氏益农公司 以5‑烯醇式丙酮酰莽草酸‑3‑磷酸合酶基因为靶的改造锌指蛋白
CA2703045C (en) 2007-10-25 2017-02-14 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
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
JP5756016B2 (ja) 2008-10-29 2015-07-29 サンガモ バイオサイエンシーズ, インコーポレイテッド グルタミンシンセターゼ遺伝子の発現を不活性化する方法及び組成物
CA2745031C (en) 2008-12-04 2018-08-14 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
JP5827132B2 (ja) 2009-02-04 2015-12-02 サンガモ バイオサイエンシズ インコーポレイテッド ニューロパチーを治療するための方法および組成物
JP5932632B2 (ja) 2009-03-20 2016-06-15 サンガモ バイオサイエンシーズ, インコーポレイテッド 改変された亜鉛フィンガータンパク質を使用したcxcr4の修飾
US9834787B2 (en) * 2009-04-09 2017-12-05 Sangamo Therapeutics, Inc. Targeted integration into stem cells
EP2727600B1 (de) 2009-07-28 2019-03-27 Sangamo Therapeutics, Inc. Zinkfingerfusionsproteine zur supprimierung eines huntington gens
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011049627A1 (en) 2009-10-22 2011-04-28 Dow Agrosciences Llc Engineered zinc finger proteins targeting plant genes involved in fatty acid biosynthesis
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
AU2011207769B2 (en) * 2010-01-22 2015-05-28 Corteva Agriscience Llc Targeted genomic alteration
EP4328304A2 (de) 2010-02-08 2024-02-28 Sangamo Therapeutics, Inc. Manipulierte spaltungshalbdomänen
EP2660318A1 (de) 2010-02-09 2013-11-06 Sangamo BioSciences, Inc. Gezielte Genommodifikation mit partiell einzelsträngigen Donatormolekülen
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
AU2011312562B2 (en) 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
CA2814143C (en) 2010-10-12 2020-09-08 The Children's Hospital Of Philadelphia Methods and compositions for treating hemophilia b
US9267123B2 (en) 2011-01-05 2016-02-23 Sangamo Biosciences, Inc. Methods and compositions for gene correction
EP3461896B1 (de) 2011-07-15 2023-11-29 The General Hospital Corporation Verfahren zur transkription einer aktivatorähnlichen effektoranordnung
EP3613852A3 (de) 2011-07-22 2020-04-22 President and Fellows of Harvard College Beurteilung und verbesserung einer nukleasespaltungsspezifität
CA2841541C (en) 2011-07-25 2019-11-12 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (cftr) gene
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
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
KR102116153B1 (ko) 2012-05-07 2020-05-27 상가모 테라퓨틱스, 인코포레이티드 도입유전자의 뉴클레아제-매개 표적화된 통합을 위한 방법 및 조성물
US9890364B2 (en) 2012-05-29 2018-02-13 The General Hospital Corporation TAL-Tet1 fusion proteins and methods of use thereof
EP2861737B1 (de) 2012-06-19 2019-04-17 Regents Of The University Of Minnesota Gen-targeting in pflanzen durch dns-viren
EP2872154B1 (de) 2012-07-11 2017-05-31 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur freisetzung von biologischen wirkstoffen
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2872625B1 (de) 2012-07-11 2016-11-16 Sangamo BioSciences, Inc. Zusammensetzungen und verfahren zur behandlung von lysosomalen speicherkrankheiten
EP2890780B8 (de) 2012-08-29 2020-08-19 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur behandlung eines genetischen leidens
KR102357105B1 (ko) 2012-09-04 2022-02-08 더 스크립스 리서치 인스티튜트 표적화된 바인딩 특이도를 갖는 키메라 폴리펩타이드들
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
UA119135C2 (uk) 2012-09-07 2019-05-10 ДАУ АГРОСАЙЄНСІЗ ЕлЕлСі Спосіб отримання трансгенної рослини
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
US9597357B2 (en) 2012-10-10 2017-03-21 Sangamo Biosciences, Inc. T cell modifying compounds and uses thereof
US20150267176A1 (en) 2012-10-12 2015-09-24 The General Hospital Corporation Transcription activator-like effector (tale) - lysine-specific demethylase 1 (lsd1) fusion proteins
EP2929017A4 (de) 2012-12-05 2016-09-28 Sangamo Biosciences Inc Verfahren und zusammensetzungen zur regulierung von stoffwechselerkrankungen
WO2014089541A2 (en) 2012-12-07 2014-06-12 Haplomics, Inc. Factor viii mutation repair and tolerance induction
JP6445462B2 (ja) 2013-02-07 2018-12-26 ザ ジェネラル ホスピタル コーポレイション Tale転写活性化因子
ES2904803T3 (es) 2013-02-20 2022-04-06 Regeneron Pharma Modificación genética de ratas
WO2014130955A1 (en) 2013-02-25 2014-08-28 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014153470A2 (en) 2013-03-21 2014-09-25 Sangamo Biosciences, Inc. Targeted disruption of t cell receptor genes using engineered zinc finger protein nucleases
ES2699578T3 (es) 2013-04-16 2019-02-11 Regeneron Pharma Modificación direccionada del genoma de rata
JP2016518142A (ja) 2013-05-10 2016-06-23 サンガモ バイオサイエンシーズ, インコーポレイテッド ヌクレアーゼ媒介ゲノム遺伝子操作のための送達方法および組成物
US9873894B2 (en) 2013-05-15 2018-01-23 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a genetic condition
EP3008186B1 (de) * 2013-06-14 2018-11-28 Cellectis Verfahren zur nichttransgenen genomänderung bei pflanzen
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
EP3988654A1 (de) 2013-08-28 2022-04-27 Sangamo Therapeutics, Inc. Zusammensetzungen zur vernetzung von dna-bindungsdomänen und -spaltungsdomänen
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
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
EP3057432B1 (de) 2013-10-17 2018-11-21 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen für nukleasevermitteltes genom-engineering in hämotopoietischen stammzellen
UA121459C2 (uk) 2013-11-04 2020-06-10 Дау Агросаєнсиз Елелсі Рекомбінантна молекула нуклеїнової кислоти для трансформації рослини сої
EP3066110B1 (de) 2013-11-04 2021-12-29 Corteva Agriscience LLC Optimale maispflanzen
BR102014027436B1 (pt) 2013-11-04 2022-06-28 Dow Agrosciences Llc Molécula de ácido nucleico recombinante, e método para a produção de uma célula vegetal transgênica
JP6633532B2 (ja) 2013-11-04 2020-01-22 ダウ アグロサイエンシィズ エルエルシー 最適なトウモロコシ遺伝子座
CN111218447A (zh) 2013-11-07 2020-06-02 爱迪塔斯医药有限公司 使用统治型gRNA的CRISPR相关方法和组合物
EP3068905A4 (de) 2013-11-11 2017-07-05 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur behandlung der huntington-krankheit
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
KR102170502B1 (ko) 2013-12-11 2020-10-28 리제너론 파마슈티칼스 인코포레이티드 게놈의 표적화된 변형을 위한 방법 및 조성물
PT3080279T (pt) 2013-12-11 2018-12-17 Regeneron Pharma Métodos e composições para a modificação direcionada de um genoma
US20150166985A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting von willebrand factor point mutations
SI3102673T1 (sl) 2014-02-03 2020-11-30 Sangamo Therapeutics, Inc. Postopki in sestavki za zdravljenje beta talasemije
WO2015127439A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
EP3929279A1 (de) 2014-03-18 2021-12-29 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur regulierung der zinkfingerproteinexpression
US9522936B2 (en) 2014-04-24 2016-12-20 Sangamo Biosciences, Inc. Engineered transcription activator like effector (TALE) proteins
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
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
EP3152319A4 (de) 2014-06-05 2017-12-27 Sangamo BioSciences, Inc. Verfahren und zusammensetzungen zur nukleasekonstruktion
LT3152312T (lt) 2014-06-06 2020-04-27 Regeneron Pharmaceuticals, Inc. Tikslinio lokuso modifikavimo būdai ir kompozicijos
JP6336140B2 (ja) 2014-06-23 2018-06-06 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. ヌクレアーゼ媒介dnaアセンブリ
PL3161128T3 (pl) 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Sposoby i kompozycje do celowanych modyfikacji genetycznych i sposoby zastosowania
MX2017000555A (es) 2014-07-14 2017-08-10 Univ Washington State Desactivacion de nanos que produce ablacion de celulas de linea germinal.
US10738278B2 (en) 2014-07-15 2020-08-11 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
US9816074B2 (en) 2014-07-25 2017-11-14 Sangamo Therapeutics, Inc. Methods and compositions for modulating nuclease-mediated genome engineering in hematopoietic stem cells
WO2016014837A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016019144A2 (en) 2014-07-30 2016-02-04 Sangamo Biosciences, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CN113699113A (zh) 2014-09-16 2021-11-26 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
AU2015332451A1 (en) 2014-10-15 2017-04-20 Regeneron Pharmaceuticals, Inc. Methods and compositions for generating or maintaining pluripotent cells
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
MX2017008190A (es) 2014-12-19 2018-03-23 Regeneron Pharma Metodos y composiciones para la modificacion genetica dirigida a traves de direccionamiento multiple de una sola etapa.
EP3247366A4 (de) 2015-01-21 2018-10-31 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zur identifizierung hochspezifischer nukleasen
EP4335918A3 (de) 2015-04-03 2024-04-17 Dana-Farber Cancer Institute, Inc. Zusammensetzung und verfahren zur genomeditierung von b-zellen
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
MX2017014446A (es) 2015-05-12 2018-06-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
CA2991301A1 (en) 2015-07-13 2017-01-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
MA42895A (fr) 2015-07-15 2018-05-23 Juno Therapeutics Inc Cellules modifiées pour thérapie cellulaire adoptive
FI3331355T3 (fi) 2015-08-06 2024-06-17 Univ Missouri Sikojen lisääntymishäiriö- ja keuhkotulehdusoireyhtymävirukselle (PRRSV) resistentti sika ja soluja, joilla on muokattuja CD163-geenejä
US11606940B2 (en) 2015-08-07 2023-03-21 Commonwealth Scientific And Industrial Research Organisation Method for producing an animal comprising a germline genetic modification
IL258030B (en) 2015-09-23 2022-08-01 Sangamo Therapeutics Inc Htt suppressing factors and their uses
US20190225955A1 (en) 2015-10-23 2019-07-25 President And Fellows Of Harvard College Evolved cas9 proteins for gene editing
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
EP3380622A4 (de) 2015-11-23 2019-08-07 Sangamo Therapeutics, Inc. Verfahren und zusammensetzungen zum immunitäts-engineering
US10907133B2 (en) 2015-11-24 2021-02-02 Commonwealth Scientific And Industrial Research Organisation Production of viruses in avian eggs
US11174466B2 (en) 2015-11-24 2021-11-16 Commonwealth Scientific And Industrial Research Organisation Production of viruses in cell culture
BR112018012235A2 (pt) 2015-12-18 2018-12-04 Sangamo Therapeutics Inc rompimento alvejado do receptor de células de mhc
CA3008413A1 (en) 2015-12-18 2017-06-22 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
US20170202931A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
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
AU2017268842B2 (en) 2016-05-27 2022-08-11 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of genome-editing molecules
AU2017295720B2 (en) 2016-07-13 2021-07-22 Vertex Pharmaceuticals Incorporated Methods, compositions and kits for increasing genome editing efficiency
JP2019523009A (ja) 2016-07-29 2019-08-22 リジェネロン・ファーマシューティカルズ・インコーポレイテッドRegeneron Pharmaceuticals, Inc. C末端切断型フィブリリン−1の発現をもたらす変異を有するマウス
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
EP3523422A1 (de) 2016-10-05 2019-08-14 FUJIFILM Cellular Dynamics, Inc. Erzeugung reifer linien aus induzierten pluripotenten stammzellen mit mecp2-unterbrechung
BR112019006652A2 (pt) 2016-10-13 2019-07-02 Juno Therapeutics Inc métodos e composições para imunoterapia envolvendo moduladores da via metabólica do triptofano
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
WO2018102612A1 (en) 2016-12-02 2018-06-07 Juno Therapeutics, Inc. Engineered b cells and related compositions and methods
JP7416406B2 (ja) 2016-12-08 2024-01-17 ケース ウェスタン リザーブ ユニバーシティ 機能的ミエリン産生を増進させるための方法および組成物
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
BR112019014841A2 (pt) 2017-01-23 2020-04-28 Regeneron Pharma rna guia, uso do rna guia, rna antissentido, sirna ou shrna, uso do rna antissentido, do sirna ou do shrna, ácido nucleico isolado, vetor, composição, célula, e, métodos para modificar um gene hsd17b13 em uma célula, para diminuir a expressão de um gene hsd17b13 em uma célula, para modificar uma célula e para tratar um indivíduo que não é portador da variante de hsd17b13
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 (de) 2017-03-09 2020-01-15 President and Fellows of Harvard College Unterdrückung von schmerzen durch geneditierung
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
US11913015B2 (en) 2017-04-17 2024-02-27 University Of Maryland, College Park Embryonic cell cultures and methods of using the same
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
AU2018302098A1 (en) 2017-07-18 2020-02-20 Csl Behring Gene Therapy, Inc. Compositions and methods for treating beta-hemoglobinopathies
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)
AU2018325363A1 (en) 2017-08-29 2020-04-09 Global Crop Innovations Pty. Ltd. Improved BLue Aleurone and other segregation systems
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
EP3697906A1 (de) 2017-10-16 2020-08-26 The Broad Institute, Inc. Verwendungen von adenosinbasiseditoren
CA3079748A1 (en) 2017-11-09 2019-05-16 Sangamo Therapeutics, Inc. Genetic modification of cytokine inducible sh2-containing protein (cish) gene
US20200361877A1 (en) 2018-01-17 2020-11-19 Vertex Pharmaceuticals Incorporated Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency
AU2019209290B2 (en) 2018-01-17 2024-03-07 Vertex Pharmaceuticals Incorporated DNA-PK inhibitors
BR112020014315A2 (pt) 2018-01-17 2020-12-08 Vertex Pharmaceuticals Incorporated Inibidores de dna-pk
EP3749350A4 (de) 2018-02-08 2021-12-01 Sangamo Therapeutics, Inc. Genetisch manipulierte zielspezifische nukleasen
JP7338893B2 (ja) 2018-03-20 2023-09-05 チンホワ ユニバーシティ アルツハイマー病動物モデルおよびその使用
CN112585276A (zh) 2018-04-05 2021-03-30 朱诺治疗学股份有限公司 产生表达重组受体的细胞的方法和相关组合物
CA3095084A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. T cells expressing a recombinant receptor, related polynucleotides and methods
CN112313246A (zh) 2018-04-18 2021-02-02 桑格摩生物治疗股份有限公司 用于调节亨廷顿蛋白(htt)的锌指蛋白组合物
CA3100034A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
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
US20210348177A1 (en) 2018-09-05 2021-11-11 The Regents Of The University Of California Generation of heritably gene-edited plants without tissue culture
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
US20220389395A1 (en) 2018-10-29 2022-12-08 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US20220282275A1 (en) 2018-11-15 2022-09-08 The Broad Institute, Inc. G-to-t base editors and uses thereof
US20220023348A1 (en) 2018-11-28 2022-01-27 Forty Seven, Inc. Genetically modified hspcs resistant to ablation regime
JP7449291B2 (ja) 2018-12-20 2024-03-13 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ヌクレアーゼ媒介リピート伸長
WO2020146805A1 (en) 2019-01-11 2020-07-16 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
WO2020163017A1 (en) 2019-02-06 2020-08-13 Klogenix Llc Dna binding proteins and uses thereof
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
WO2020181195A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through adenine excision
WO2020181202A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to t:a base editing through adenine deamination and oxidation
WO2020181193A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through adenosine methylation
WO2020181180A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. A:t to c:g base editors and uses thereof
WO2020181178A1 (en) 2019-03-06 2020-09-10 The Broad Institute, Inc. T:a to a:t base editing through thymine alkylation
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.
JP2022527809A (ja) 2019-04-03 2022-06-06 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 抗体コード配列をセーフハーバー遺伝子座に挿入するための方法および組成物
US11737435B2 (en) 2019-04-04 2023-08-29 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized coagulation factor 12 locus
AU2020253531C1 (en) 2019-04-04 2022-06-16 Regeneron Pharmaceuticals, Inc. Methods for scarless introduction of targeted modifications into targeting vectors
CN113966401A (zh) 2019-04-10 2022-01-21 犹他大学研究基金会 用于治疗amd的htra1调节
US20220307003A1 (en) 2019-04-17 2022-09-29 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
BR112021021200A2 (pt) 2019-05-01 2021-12-21 Juno Therapeutics Inc Células expressando um receptor quimérico de um locus cd247 modificado, polinucleotídeos relacionados e métodos
MX2021013219A (es) 2019-05-01 2022-02-17 Juno Therapeutics Inc Celulas que expresan un receptor recombinante de un locus de tgfbr2 modificado, polinucleotidos relacionados y metodos.
JP2022534867A (ja) 2019-06-04 2022-08-04 リジェネロン・ファーマシューティカルズ・インコーポレイテッド ベータスリップ変異を有するヒト化ttr遺伝子座を含む非ヒト動物と使用方法
AU2020289581A1 (en) 2019-06-07 2021-11-18 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized albumin locus
AU2020290509A1 (en) 2019-06-14 2021-11-11 Regeneron Pharmaceuticals, Inc. Models of tauopathy
US20220267732A1 (en) 2019-08-01 2022-08-25 Sana Biotechnology, Inc. Dux4 expressing cells and uses thereof
WO2021030666A1 (en) 2019-08-15 2021-02-18 The Broad Institute, Inc. Base editing by transglycosylation
AU2020336302A1 (en) 2019-08-23 2022-03-03 Sana Biotechnology, Inc. CD24 expressing cells and uses thereof
AU2020376048A1 (en) 2019-11-01 2022-06-02 Sangamo Therapeutics, Inc. Compositions and methods for genome engineering
US20210130828A1 (en) 2019-11-01 2021-05-06 Sangamo Therapeutics, Inc. Gin recombinase variants
MX2022005572A (es) 2019-11-08 2022-06-09 Regeneron Pharma Estrategias crispr y aav para la terapia de retinosquisis juvenil ligada al cromosoma x.
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
US20230086199A1 (en) 2019-11-26 2023-03-23 The Broad Institute, Inc. Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
WO2021158921A2 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Adenine base editors and uses thereof
AU2021232598A1 (en) 2020-03-04 2022-09-08 Regeneron Pharmaceuticals, Inc. Methods and compositions for sensitization of tumor cells to immune therapy
BR112022019060A2 (pt) 2020-03-25 2022-11-29 Sana Biotechnology Inc Células neurais hipoimunogênicas para o tratamento de distúrbios e condições neurológicas
EP4143315A1 (de) 2020-04-28 2023-03-08 The Broad Institute Inc. <smallcaps/>?ush2a? ?gezielte basisbearbeitung eines thegens
CN115803435A (zh) 2020-05-06 2023-03-14 塞勒克提斯公司 用于在细胞基因组中靶向插入外源序列的方法
EP4146284A1 (de) 2020-05-06 2023-03-15 Cellectis S.A. Verfahren zur genetischen modifizierung von zellen zur freisetzung therapeutischer proteine
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
CN115835873A (zh) 2020-05-13 2023-03-21 朱诺治疗学股份有限公司 用于产生表达重组受体的供体分批细胞的方法
US20230190871A1 (en) 2020-05-20 2023-06-22 Sana Biotechnology, Inc. Methods and compositions for treatment of viral infections
CN116234558A (zh) 2020-06-26 2023-06-06 朱诺治疗学有限公司 条件性地表达重组受体的工程化t细胞、相关多核苷酸和方法
CA3189338A1 (en) 2020-07-16 2022-01-20 Acuitas Therapeutics, Inc. Cationic lipids for use in lipid nanoparticles
AU2021325941A1 (en) 2020-08-13 2023-03-09 Sana Biotechnology, Inc. Methods of treating sensitized patients with hypoimmunogenic cells, and associated methods and compositions
EP4204545A2 (de) 2020-08-25 2023-07-05 Kite Pharma, Inc. T-zellen mit verbesserter funktionalität
KR20230090367A (ko) 2020-11-04 2023-06-21 주노 쎄러퓨티크스 인코퍼레이티드 변형된 불변 cd3 이뮤노글로불린 슈퍼패밀리 쇄 유전자좌로부터 키메라 수용체를 발현하는 세포 및 관련 폴리뉴클레오티드 및 방법
CN117042600A (zh) 2020-11-16 2023-11-10 猪改良英国有限公司 具有经编辑的anp32基因的抗甲型流感动物
KR20230137900A (ko) 2020-12-31 2023-10-05 사나 바이오테크놀로지, 인크. Car-t 활성을 조정하기 위한 방법 및 조성물
EP4263825A1 (de) 2021-02-19 2023-10-25 Beam Therapeutics, Inc. Rekombinante tollwutviren für gentherapie
CN118019546A (zh) 2021-03-23 2024-05-10 艾欧凡斯生物治疗公司 肿瘤浸润淋巴细胞的cish基因编辑及其在免疫疗法中的用途
KR20240013135A (ko) 2021-05-27 2024-01-30 사나 바이오테크놀로지, 인크. 조작된 hla-e 또는 hla-g를 포함하는 저면역원성 세포
EP4370544A2 (de) 2021-07-14 2024-05-22 Sana Biotechnology, Inc. Veränderte expression von y-chromosom-gebundenen antigenen in hypoimmunogenen zellen
KR20240073006A (ko) 2021-08-11 2024-05-24 사나 바이오테크놀로지, 인크. 동종이계 세포 요법을 위한 유전자 변형된 1차 세포
AU2022326565A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy
CA3227613A1 (en) 2021-08-11 2023-02-16 William Dowdle Inducible systems for altering gene expression in hypoimmunogenic cells
AU2022325231A1 (en) 2021-08-11 2024-02-08 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce complement-mediated inflammatory reactions
WO2023019225A2 (en) 2021-08-11 2023-02-16 Sana Biotechnology, Inc. Genetically modified cells for allogeneic cell therapy to reduce instant blood mediated inflammatory reactions
WO2023023553A2 (en) * 2021-08-17 2023-02-23 The Board Of Trustees Of The Leland Stanford Junior University Compositions, systems, and methods for activating and silencing gene expression
WO2023039468A1 (en) 2021-09-08 2023-03-16 Beam Therapeutics Inc. Viral guide rna delivery
WO2023069790A1 (en) 2021-10-22 2023-04-27 Sana Biotechnology, Inc. Methods of engineering allogeneic t cells with a transgene in a tcr locus and associated compositions and methods
WO2023081756A1 (en) 2021-11-03 2023-05-11 The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone Precise genome editing using retrons
WO2023081900A1 (en) 2021-11-08 2023-05-11 Juno Therapeutics, Inc. Engineered t cells expressing a recombinant t cell receptor (tcr) and related systems and methods
WO2023105244A1 (en) 2021-12-10 2023-06-15 Pig Improvement Company Uk Limited Editing tmprss2/4 for disease resistance in livestock
CA3241438A1 (en) 2021-12-23 2023-06-29 Sana Biotechnology, Inc. Chimeric antigen receptor (car) t cells for treating autoimmune disease and associated methods
AU2022424002A1 (en) 2021-12-29 2024-06-13 Bristol-Myers Squibb Company Generation of landing pad cell lines
WO2023141602A2 (en) 2022-01-21 2023-07-27 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2023154578A1 (en) 2022-02-14 2023-08-17 Sana Biotechnology, Inc. Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells
WO2023158836A1 (en) 2022-02-17 2023-08-24 Sana Biotechnology, Inc. Engineered cd47 proteins and uses thereof
WO2023173123A1 (en) 2022-03-11 2023-09-14 Sana Biotechnology, Inc. Genetically modified cells and compositions and uses thereof
WO2023220035A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus compositions and methods for gene therapy
WO2023220040A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified capsid for gene therapy
WO2023220043A1 (en) 2022-05-09 2023-11-16 Synteny Therapeutics, Inc. Erythroparvovirus with a modified genome for gene therapy
WO2023220603A1 (en) 2022-05-09 2023-11-16 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
WO2024013514A2 (en) 2022-07-15 2024-01-18 Pig Improvement Company Uk Limited Gene edited livestock animals having coronavirus resistance
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024031053A1 (en) 2022-08-05 2024-02-08 Regeneron Pharmaceuticals, Inc. Aggregation-resistant variants of tdp-43
WO2024040083A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. Evolved cytosine deaminases and methods of editing dna using same
WO2024044723A1 (en) 2022-08-25 2024-02-29 Renagade Therapeutics Management Inc. Engineered retrons and methods of use
WO2024100604A1 (en) 2022-11-09 2024-05-16 Juno Therapeutics Gmbh Methods for manufacturing engineered immune cells
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE3410650A1 (de) 1984-03-23 1985-10-03 Kernforschungsanlage Jülich GmbH, 5170 Jülich Mit mikroorganismen bewachsene poroese anorganische traeger, verfahren zur immobilisierung von mikroorganismen und dafuer geeignete traegerkoerper
US5455170A (en) 1986-08-22 1995-10-03 Hoffmann-La Roche Inc. Mutated thermostable nucleic acid polymerase enzyme from Thermus species Z05
US5317090A (en) 1987-12-16 1994-05-31 Institut Pasteur Steroid/thyroid hormone receptor-related gene, which is inappropriately expressed in human hepatocellular carcinoma, and which is a retinoic acid receptor
US5324819A (en) 1988-04-08 1994-06-28 Stryker Corporation Osteogenic proteins
US5340739A (en) 1988-07-13 1994-08-23 Brigham & Women's Hospital Hematopoietic cell specific transcriptional regulatory elements of serglycin and uses thereof
US5223409A (en) 1988-09-02 1993-06-29 Protein Engineering Corp. Directed evolution of novel binding proteins
EP0451206A4 (en) 1988-12-23 1992-07-22 The Salk Institute For Biological Studies Receptor transcription-repression activity compositions and methods
US5198346A (en) 1989-01-06 1993-03-30 Protein Engineering Corp. Generation and selection of novel DNA-binding proteins and polypeptides
US5096815A (en) 1989-01-06 1992-03-17 Protein Engineering Corporation Generation and selection of novel dna-binding proteins and polypeptides
US4990607A (en) 1989-03-14 1991-02-05 The Rockefeller University Alteration of gene expression in plants
DE69032324T2 (de) 1989-03-17 1998-10-08 The Salk Institute For Biological Studies, La Jolla, Calif. Zusammensetzung und testverfahren eines elementes als antwort auf ein hormon
DE69033127T2 (de) 1989-11-13 1999-10-14 Massachusetts Inst Technology Lokalisation und charakterisierung des wilms-tumor-gens
US5578482A (en) 1990-05-25 1996-11-26 Georgetown University Ligand growth factors that bind to the erbB-2 receptor protein and induce cellular responses
AU655943B2 (en) 1990-09-21 1995-01-19 Salk Institute For Biological Studies, The Functional antagonism between proto-oncoprotein c-jun and hormone receptors
US5348864A (en) 1991-01-25 1994-09-20 E. R. Squibb & Sons, Inc. Mouse vav proto-oncogene DNA and protein sequences
US5871907A (en) 1991-05-15 1999-02-16 Medical Research Council Methods for producing members of specific binding pairs
US5324818A (en) 1991-08-21 1994-06-28 The Regents Of The University Of Michigan Proteins useful in the regulation of κB-containing genes
US5243041A (en) 1991-08-22 1993-09-07 Fernandez Pol Jose A DNA vector with isolated CDNA gene encoding metallopanstimulin
US5302519A (en) 1991-09-09 1994-04-12 Fred Hutchinson Cancer Research Center Method of producing a Mad polypeptide
US5270170A (en) 1991-10-16 1993-12-14 Affymax Technologies N.V. Peptide library and screening method
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792640A (en) 1992-04-03 1998-08-11 The Johns Hopkins University General method to clone hybrid restriction endonucleases using lig gene
US5916794A (en) 1992-04-03 1999-06-29 Johns Hopkins University Methods for inactivating target DNA and for detecting conformational change in a nucleic acid
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5324638A (en) 1992-05-13 1994-06-28 Sloan-Kettering Institute For Cancer Research Brain transcription factor, nucleic acids encoding same and uses thereof
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
US5376630A (en) * 1994-03-17 1994-12-27 International Flavors & Fragrances Inc. Methyl, substituted propyl-substituted pentamethyl indane derivatives, processes for producing same and perfumery uses thereof
WO1996006110A1 (en) 1994-08-18 1996-02-29 Gilman, Michael, Z. Composite dna-binding proteins and materials and methods relating thereto
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
ATE407205T1 (de) * 1994-08-20 2008-09-15 Gendaq Ltd Verbesserung in bezug auf bindungsproteine bei der erkennung von dna
DE4435919C1 (de) 1994-10-07 1995-12-07 Deutsches Krebsforsch Zinkfinger-DNA, -Protein und ihre Verwendung
US5871902A (en) 1994-12-09 1999-02-16 The Gene Pool, Inc. Sequence-specific detection of nucleic acid hybrids using a DNA-binding molecule or assembly capable of discriminating perfect hybrids from non-perfect hybrids
US5702914A (en) 1994-12-21 1997-12-30 The Salk Institute For Biological Studies Use of reporter genes for retinoid receptor screening assays having novel retinoid-associated response elements
CA2209183A1 (en) 1994-12-29 1996-07-11 Joel L. Pomerantz Chimeric dna-binding proteins
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
WO1996032475A2 (en) 1995-04-12 1996-10-17 University Of Washington Methods for preparing dna-binding proteins
US6455247B1 (en) 1996-01-23 2002-09-24 Board Of Trustees Of The Leland Stanford Junior University Methods for screening for transdominant effector peptides and RNA molecules
FR2752734B1 (fr) 1996-09-02 1998-11-06 Cird Galderma Utilisation de retinoides pour la preparation d'un medicament destine a traiter les affections liees a une surexpression de vegf
US5939538A (en) * 1996-10-25 1999-08-17 Immusol Incorporated Methods and compositions for inhibiting HIV infection of cells by cleaving HIV co-receptor RNA
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
EP1046034A1 (de) 1998-01-09 2000-10-25 Cubist Pharmaceuticals, Inc. Verfahren zur identifizierung von ziel und test kombinationen
CA2318402A1 (en) 1998-01-15 1999-07-22 Ariad Gene Therapeutics, Inc. Regulation of biological events using multimeric chimeric proteins
US5972615A (en) 1998-01-21 1999-10-26 Urocor, Inc. Biomarkers and targets for diagnosis, prognosis and management of prostate disease
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
DE69933856D1 (de) 1998-02-13 2006-12-14 Koester Hubert Verwendung von ribozymen zur bestimmung der funktion von genen
IL137952A0 (en) 1998-02-20 2001-10-31 Genome Dynamics Inc Method for designing dna-binding proteins of the zinc-finger class
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
DE69932813D1 (de) 1998-03-17 2006-09-28 Gendaq Ltd Nukleinsäurebindungsproteine
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
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US20020164575A1 (en) * 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification

Also Published As

Publication number Publication date
WO2001019981A2 (en) 2001-03-22
DE60025037T2 (de) 2006-08-17
CA2383926A1 (en) 2001-03-22
US20020081614A1 (en) 2002-06-27
JP5832787B2 (ja) 2015-12-16
HK1049501A1 (en) 2003-05-16
HK1049501B (zh) 2006-04-07
US7235354B2 (en) 2007-06-26
EP1238067B1 (de) 2005-12-21
US6599692B1 (en) 2003-07-29
CA2383926C (en) 2008-09-09
EP1238067A2 (de) 2002-09-11
KR20020065473A (ko) 2002-08-13
JP2011244818A (ja) 2011-12-08
JP2003527093A (ja) 2003-09-16
AU7478700A (en) 2001-04-17
US20040203064A1 (en) 2004-10-14
US6777185B2 (en) 2004-08-17
WO2001019981A3 (en) 2002-07-11
JP2014144000A (ja) 2014-08-14
AU778964B2 (en) 2004-12-23
DE60025037D1 (de) 2006-01-26

Similar Documents

Publication Publication Date Title
ATE313625T1 (de) ßFUNCTIONAL GENOMICSß UNTER VERWENDUNG VON ZINK FINGER PROTEINEN
ATE309536T1 (de) Methoden zur verwendung von randomisierten zinkfingerprotein-bibliotheken zur identifizierung von genfunktionen
ATE320500T1 (de) Nukleotid-sequenzen zur kontrolle der exprimierung von dns-sequenzen in einem zellulärem wirt
ATE310812T1 (de) Derivate von zinkfingerproteinen und methoden
DE60041452D1 (de) CpG REZEPTOR (CpG-R) UND DARAUF BEZOGENE METHODEN
ATE462006T1 (de) Isoform des vegfs
ATE304052T1 (de) Humane checkpointkinase, hcds1, zusammensetzungen und verfahren
DE60213221D1 (de) Neuartige polynukleotide und polypeptide des ifnalpha-21 gens
DE69928395D1 (de) Cyclin-e2 proteine und dafür kodierende gene
ATE363070T1 (de) Auswertung von biologischen mitteln in lebenden zielzellen
WO2003008578A3 (en) Reagents and methods for identifying gene targets for treating cancer
DK1082415T3 (da) Anvendelige fremgangsmåder til modulation af angioge-nese ved anvendelse af tyrosin kinase SRC
ATE380245T1 (de) Gen von alteromonas
WO2004096833A3 (en) Inducers of recombinant protein expression
DE60210970D1 (de) Mutante formen von etxb und ctxb und ihre verwendung als träger
ATE219515T1 (de) Methoden und reagenzien zur regulierung von apoptosis
EP1908835A3 (de) Regulierung von Genexpression durch einkettige, monomere, ligandenabhängige Polypeptidschalter
DE60326640D1 (de) Rekombinante biotincarboxylasedomänen zur identifizierung von acetyl-coa-carboxylase-inhibitoren
ATE273394T1 (de) Wirts-vektor-systeme zur phosphatregulierten überproduktion von polypeptiden in bacillus
DE60330140D1 (de) Gen-silencing-vektor und diesen verwendendes gen-silencing-verfahren
WO2004013634A3 (en) Dna damage repair polypeptide and uses thereof
EP4234570A3 (de) Für programmierten zelltod 1 (pd1) spezifische nukleasen
DE69821102D1 (de) Verwendung von calculus
SE0100412D0 (sv) Novel human poly(A) polymerase

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties