AU2003298574B2 - Use of chimeric nucleases to stimulate gene targeting - Google Patents

Use of chimeric nucleases to stimulate gene targeting Download PDF

Info

Publication number
AU2003298574B2
AU2003298574B2 AU2003298574A AU2003298574A AU2003298574B2 AU 2003298574 B2 AU2003298574 B2 AU 2003298574B2 AU 2003298574 A AU2003298574 A AU 2003298574A AU 2003298574 A AU2003298574 A AU 2003298574A AU 2003298574 B2 AU2003298574 B2 AU 2003298574B2
Authority
AU
Australia
Prior art keywords
cell
vector
target sequence
sequence
nucleic acid
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired
Application number
AU2003298574A
Other languages
English (en)
Other versions
AU2003298574A1 (en
Inventor
David Baltimore
Matthew Porteus
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
California Institute of Technology
Original Assignee
California Institute of Technology
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 California Institute of Technology filed Critical California Institute of Technology
Publication of AU2003298574A1 publication Critical patent/AU2003298574A1/en
Application granted granted Critical
Publication of AU2003298574B2 publication Critical patent/AU2003298574B2/en
Anticipated expiration legal-status Critical
Expired legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A61P31/18—Antivirals for RNA viruses for HIV
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • 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
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/43—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a FLAG-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/60—Vector systems having a special element relevant for transcription from viruses
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/90—Vector systems having a special element relevant for transcription from vertebrates avian
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Communicable Diseases (AREA)
  • Hematology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • AIDS & HIV (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2003298574A 2002-09-05 2003-09-05 Use of chimeric nucleases to stimulate gene targeting Expired AU2003298574B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US40845402P 2002-09-05 2002-09-05
US60/408,454 2002-09-05
US41934102P 2002-10-17 2002-10-17
US60/419,341 2002-10-17
US48478803P 2003-07-03 2003-07-03
US60/484,788 2003-07-03
PCT/US2003/027958 WO2004037977A2 (en) 2002-09-05 2003-09-05 Use of chimeric nucleases to stimulate gene targeting

Publications (2)

Publication Number Publication Date
AU2003298574A1 AU2003298574A1 (en) 2004-05-13
AU2003298574B2 true AU2003298574B2 (en) 2008-04-24

Family

ID=32180488

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2003298574A Expired AU2003298574B2 (en) 2002-09-05 2003-09-05 Use of chimeric nucleases to stimulate gene targeting

Country Status (6)

Country Link
US (2) US9447434B2 (https=)
EP (2) EP2806025B1 (https=)
JP (1) JP2006502748A (https=)
AU (1) AU2003298574B2 (https=)
CA (1) CA2497913C (https=)
WO (1) WO2004037977A2 (https=)

Families Citing this family (355)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ITMI20030821A1 (it) * 2003-04-18 2004-10-19 Internat Ct For Genetic En Gineering And Polipeptidi chimerici e loro uso.
US7888121B2 (en) * 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US20070134796A1 (en) * 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US20120196370A1 (en) 2010-12-03 2012-08-02 Fyodor Urnov Methods and compositions for targeted genomic deletion
AU2004263865B2 (en) * 2003-08-08 2007-05-17 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
US11311574B2 (en) 2003-08-08 2022-04-26 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
EP1732945B1 (en) * 2004-04-08 2014-12-24 Sangamo BioSciences, Inc. Methods and compositions for modulating cardiac contractility
US20100256062A1 (en) 2004-12-06 2010-10-07 Howard Tommy E Allelic Variants of Human Factor VIII
WO2007034262A1 (en) * 2005-09-19 2007-03-29 Cellectis Heterodimeric meganucleases and use thereof
EP1863909B2 (en) * 2005-03-15 2014-09-10 Cellectis I-crei meganuclease variants with modified specificity, method of preparation and uses thereof
AU2012245168B2 (en) * 2005-07-26 2014-12-11 Sangamo Therapeutics, Inc. Targeted Integration and Expression of Exogenous Nucleic Acid Sequences
ES2582091T3 (es) 2005-10-18 2016-09-09 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
WO2007134272A2 (en) * 2006-05-12 2007-11-22 Janssen Pharmaceutica N.V. Humanized models via targeted mtagenesis with zinc finger nuclease
WO2007136685A2 (en) * 2006-05-19 2007-11-29 Sangamo Biosciences, Inc. Methods and compositions for inactivation of dihydrofolate reductase
EP2027262B1 (en) 2006-05-25 2010-03-31 Sangamo Biosciences Inc. Variant foki cleavage half-domains
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
US20080003681A1 (en) * 2006-06-28 2008-01-03 Mahalaxmi Gita Bangera Methods for altering cellular susceptibility to infection
BRPI0716427A2 (pt) * 2006-08-11 2014-03-11 Dow Agrosciences Llc Recombinação homóloga mediada por nuclease de apêndice de zinco
WO2008060510A2 (en) 2006-11-13 2008-05-22 Sangamo Biosciences, Inc. Zinc finger nuclease for targeting the human glucocorticoid receptor locus
ES2586210T3 (es) * 2006-12-14 2016-10-13 Sangamo Biosciences, Inc. Proteínas de dedo de zinc no canónicas optimizadas
EP2155868A2 (en) * 2007-04-19 2010-02-24 Codon Devices, Inc Engineered nucleases and their uses for nucleic acid assembly
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
WO2008153742A2 (en) 2007-05-23 2008-12-18 Sangamo Biosciences, Inc. Methods and compositions for increased transgene expression
EP2171052B1 (en) 2007-07-12 2014-08-27 Sangamo BioSciences, Inc. Methods and compositions for inactivating alpha 1,6 fucosyltransferase (fut 8) gene expression
US8563314B2 (en) 2007-09-27 2013-10-22 Sangamo Biosciences, Inc. Methods and compositions for modulating PD1
EP2188384B1 (en) * 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
HRP20161004T1 (hr) * 2007-09-27 2016-10-21 Dow Agrosciences Llc Tehnološki konstruirani proteini cinkovi prsti koji imaju kao cilj gene za sintazu 5-enolpiruvilshikimat-3-fosfata
US11235026B2 (en) 2007-09-27 2022-02-01 Sangamo Therapeutics, Inc. Methods and compositions for modulating PD1
EP2205752B1 (en) 2007-10-25 2016-08-10 Sangamo BioSciences, Inc. Methods and compositions for targeted integration
JP2011505809A (ja) * 2007-12-07 2011-03-03 プレシジョン バイオサイエンシズ，インク． ヒトゲノムのＤＮａｓｅ高感受性領域に見出される認識配列を有する合理的に設計されたメガヌクレアーゼ
WO2009114321A2 (en) * 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
US20100071083A1 (en) * 2008-03-12 2010-03-18 Smith James J Temperature-dependent meganuclease activity
AU2009239333A1 (en) * 2008-04-21 2009-10-29 Danziger Innovations Ltd. Plant viral expression vectors and use of same for generating genotypic variations in plant genomes
CA2722797A1 (en) * 2008-04-28 2009-11-05 Precision Biosciences, Inc. Fusion molecules of rationally-designed dna-binding proteins and effector domains
EP2313498B1 (en) 2008-07-14 2017-03-15 Precision Biosciences, Inc. Recognition sequences for i-crei-derived meganucleases and uses thereof
SG191561A1 (en) 2008-08-22 2013-07-31 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
US20110023143A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of neurodevelopmental genes in animals
US20110023156A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Feline genome editing with zinc finger nucleases
US20110023140A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Rabbit genome editing with zinc finger nucleases
US20110023154A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Silkworm genome editing with zinc finger nucleases
US20110023147A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of prion disorder-related genes in animals
US20110023148A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of addiction-related genes in animals
US20110023158A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Bovine genome editing with zinc finger nucleases
US20110023144A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in amyotrophyic lateral sclerosis disease
US20110023150A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of genes associated with schizophrenia in animals
US20110023145A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in autism spectrum disorders
US20110016539A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of neurotransmission-related genes in animals
EP2352369B1 (en) 2008-12-04 2017-04-26 Sangamo BioSciences, Inc. Genome editing in rats using zinc-finger nucleases
US20110016540A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of genes associated with trinucleotide repeat expansion disorders in animals
US20110030072A1 (en) * 2008-12-04 2011-02-03 Sigma-Aldrich Co. Genome editing of immunodeficiency genes in animals
US20110023139A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in cardiovascular disease
US20110016546A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Porcine genome editing with zinc finger nucleases
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
US20110023153A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in alzheimer's disease
US20110023146A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in secretase-associated disorders
US20110016543A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genomic editing of genes involved in inflammation
US20110023151A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of abc transporters
US20110023149A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved in tumor suppression in animals
US20110023152A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genome editing of cognition related genes in animals
US20110016541A1 (en) * 2008-12-04 2011-01-20 Sigma-Aldrich Co. Genome editing of sensory-related genes in animals
CN102333868B (zh) * 2008-12-17 2015-01-07 陶氏益农公司 靶向整合入Zp15 基因座
KR20100080068A (ko) * 2008-12-31 2010-07-08 주식회사 툴젠 신규한 징크 핑거 뉴클레아제 및 이의 용도
EP2408921B1 (en) 2009-03-20 2017-04-19 Sangamo BioSciences, Inc. Modification of cxcr4 using engineered zinc finger proteins
EP2419511B1 (en) 2009-04-09 2018-01-17 Sangamo Therapeutics, Inc. Targeted integration into stem cells
US8772008B2 (en) 2009-05-18 2014-07-08 Sangamo Biosciences, Inc. Methods and compositions for increasing nuclease activity
EP2449135B1 (en) 2009-06-30 2016-01-06 Sangamo BioSciences, Inc. Rapid screening of biologically active nucleases and isolation of nuclease-modified cells
EP2451837B1 (en) 2009-07-08 2015-03-25 Cellular Dynamics International, Inc. Modified ips cells having a mutant form of human immunodeficiency virus (hiv) cellular entry gene
CA2769262C (en) 2009-07-28 2019-04-30 Sangamo Biosciences, Inc. Methods and compositions for treating trinucleotide repeat disorders
JP5940977B2 (ja) 2009-08-11 2016-06-29 サンガモ バイオサイエンシーズ， インコーポレイテッド 標的改変によるホモ接合生物
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
WO2011048600A1 (en) 2009-10-21 2011-04-28 Danziger Innovations Ltd. Generating genotypic variations in plant genomes by gamete infection
US8956828B2 (en) * 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
MX336846B (es) * 2010-01-22 2016-02-03 Sangamo Biosciences Inc Alteracion genomica dirigida.
ES2751916T3 (es) 2010-02-08 2020-04-02 Sangamo Therapeutics Inc Semidominios de escisión genomanipulados
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
AU2011281062B2 (en) 2010-07-21 2015-01-22 Board Of Regents, The University Of Texas System Methods and compositions for modification of a HLA locus
US9512444B2 (en) 2010-07-23 2016-12-06 Sigma-Aldrich Co. Llc Genome editing using targeting endonucleases and single-stranded nucleic acids
US9057057B2 (en) * 2010-07-27 2015-06-16 The Johns Hopkins University Obligate heterodimer variants of foki cleavage domain
WO2012018726A1 (en) * 2010-08-02 2012-02-09 Cellectis Sa Method for increasing double-strand break-induced gene targeting
AU2011312562B2 (en) 2010-09-27 2014-10-09 Sangamo Therapeutics, Inc. Methods and compositions for inhibiting viral entry into cells
US9175280B2 (en) 2010-10-12 2015-11-03 Sangamo Biosciences, Inc. Methods and compositions for treating hemophilia B
WO2012094132A1 (en) 2011-01-05 2012-07-12 Sangamo Biosciences, Inc. Methods and compositions for gene correction
BR112013030652A2 (pt) 2011-06-10 2016-12-13 Basf Plant Science Co Gmbh polinucleotídeo que codifica um polipeptídeo, molécula de ácido nucleico, vetor, organismo não humano, polipeptídeo e método para introduzir um ácido nucleico de interesse em um genoma de um organismo não humano
US9161995B2 (en) 2011-07-25 2015-10-20 Sangamo Biosciences, Inc. Methods and compositions for alteration of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
CA3099582A1 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
HK1200871A1 (en) 2011-11-16 2015-08-14 Sangamo Therapeutics, Inc. Modified dna-binding proteins and uses thereof
UA115772C2 (uk) 2011-12-16 2017-12-26 Таргітджин Байотекнолоджиз Лтд Композиція програмованого нуклеопротеїнового молекулярного комплексу і спосіб модифікування заданої послідовності нуклеїнової кислоти-мішені
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
SG11201406547YA (en) 2012-04-25 2014-11-27 Regeneron Pharma Nuclease-mediated targeting with large targeting vectors
MX369788B (es) 2012-05-02 2019-11-21 Dow Agrosciences Llc Modificacion dirigida de deshidrogenasa de malato.
AU2013259647B2 (en) 2012-05-07 2018-11-08 Corteva Agriscience Llc Methods and compositions for nuclease-mediated targeted integration of transgenes
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
JP6329537B2 (ja) 2012-07-11 2018-05-23 サンガモ セラピューティクス， インコーポレイテッド 生物学的薬剤の送達のための方法および組成物
EP3196301B1 (en) 2012-07-11 2018-10-17 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of monogenic diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
SG10201701601WA (en) 2012-08-29 2017-04-27 Sangamo Biosciences Inc Methods and compositions for treatment of a genetic condition
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
US9914930B2 (en) 2012-09-07 2018-03-13 Dow Agrosciences Llc FAD3 performance loci and corresponding target site specific binding proteins capable of inducing targeted breaks
AU2013329186B2 (en) 2012-10-10 2019-02-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
US9255250B2 (en) 2012-12-05 2016-02-09 Sangamo Bioscience, Inc. Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene
PL3360964T3 (pl) 2012-12-06 2020-03-31 Sigma-Aldrich Co. Llc Modyfikacja i regulacja genomu oparta na crispr
EP2928303A4 (en) * 2012-12-07 2016-07-13 Haplomics Inc FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION
MX2015007574A (es) 2012-12-13 2015-10-22 Dow Agrosciences Llc Gen de precision dirigido a un locus particular en el maiz.
MX384291B (es) 2013-02-20 2025-03-14 Regeneron Pharma Modificación genética de ratas.
US10227610B2 (en) 2013-02-25 2019-03-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
WO2014152832A1 (en) 2013-03-14 2014-09-25 Immusoft Corporation Methods for in vitro memory b cell differentiation and transduction with vsv-g pseudotyped viral vectors
RU2662932C2 (ru) 2013-03-14 2018-07-31 Карибо Биосайенсиз, Инк. Композиции и способы с участием нуклеиновых кислот, нацеленных на нуклеиновые кислоты
US9937207B2 (en) 2013-03-21 2018-04-10 Sangamo Therapeutics, Inc. Targeted disruption of T cell receptor genes using talens
CN105263312A (zh) 2013-04-05 2016-01-20 美国陶氏益农公司 用于在植物基因组内整合外源序列的方法和组合物
DK3456831T3 (da) 2013-04-16 2021-09-06 Regeneron Pharma Målrettet modifikation af rottegenom
EP2994531B1 (en) 2013-05-10 2018-03-28 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN105683376A (zh) 2013-05-15 2016-06-15 桑格摩生物科学股份有限公司 用于治疗遗传病状的方法和组合物
US9944925B2 (en) 2013-08-02 2018-04-17 Enevolv, Inc. Processes and host cells for genome, pathway, and biomolecular engineering
CA3131284C (en) 2013-08-28 2023-09-19 David Paschon Compositions for linking dna-binding domains and cleavage domains
WO2015033343A1 (en) 2013-09-03 2015-03-12 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Compositions and methods for expressing recombinant polypeptides
US9765404B2 (en) 2013-09-04 2017-09-19 Dow Agrosciences Llc Rapid assay for identifying transformants having targeted donor insertion
EP3441468B1 (en) 2013-10-17 2021-05-19 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10117899B2 (en) 2013-10-17 2018-11-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
CN105980395A (zh) 2013-11-04 2016-09-28 美国陶氏益农公司 最优大豆座位
NZ746567A (en) 2013-11-04 2019-09-27 Dow Agrosciences Llc Optimal soybean loci
NZ719494A (en) 2013-11-04 2017-09-29 Dow Agrosciences Llc Optimal maize loci
UY35812A (es) 2013-11-04 2015-05-29 Dow Agrosciences Llc ?loci de maíz óptimos?.
WO2015070212A1 (en) 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
DK3492593T3 (da) 2013-11-13 2021-11-08 Childrens Medical Center Nukleasemedieret regulering af genekspression
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
EP3080279B1 (en) 2013-12-11 2018-09-26 Regeneron Pharmaceuticals, Inc. Methods and compositions for the targeted modification of a genome
WO2015089375A1 (en) 2013-12-13 2015-06-18 The General Hospital Corporation Soluble high molecular weight (hmw) tau species and applications thereof
US10774338B2 (en) 2014-01-16 2020-09-15 The Regents Of The University Of California Generation of heritable chimeric plant traits
US10072066B2 (en) 2014-02-03 2018-09-11 Sangamo Therapeutics, Inc. Methods and compositions for treatment of a beta thalessemia
WO2015127439A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
CN106459894B (zh) 2014-03-18 2020-02-18 桑格摩生物科学股份有限公司 用于调控锌指蛋白表达的方法和组合物
WO2015164748A1 (en) 2014-04-24 2015-10-29 Sangamo Biosciences, Inc. Engineered transcription activator like effector (tale) proteins
WO2015171932A1 (en) 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
WO2015175642A2 (en) 2014-05-13 2015-11-19 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a disease
WO2015188056A1 (en) 2014-06-05 2015-12-10 Sangamo Biosciences, Inc. Methods and compositions for nuclease design
HRP20200529T1 (hr) 2014-06-06 2020-09-04 Regeneron Pharmaceuticals, Inc. Postupci i pripravci za modificiranje ciljnog lokusa
ES2781323T3 (es) 2014-06-23 2020-09-01 Regeneron Pharma Montaje de ADN mediado por nucleasas
SI3161128T1 (sl) 2014-06-26 2019-02-28 Regeneron Pharmaceuticals, Inc. Postopki in sestavki za ciljano genetsko spremembo in postopki uporabe
US20170159065A1 (en) 2014-07-08 2017-06-08 Vib Vzw Means and methods to increase plant yield
WO2016005985A2 (en) 2014-07-09 2016-01-14 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Method for reprogramming cells
WO2016014837A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, Inc. Gene editing for hiv gene therapy
WO2016014794A1 (en) 2014-07-25 2016-01-28 Sangamo Biosciences, 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
IL234638A0 (en) 2014-09-14 2014-12-02 Yeda Res & Dev NMDA receptor antagonists for the treatment of Gaucher disease
DK3194570T3 (da) 2014-09-16 2021-09-13 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller
EP3201340B1 (en) 2014-10-01 2020-12-02 The General Hospital Corporation Methods for increasing efficiency of nuclease-induced homology-directed repair
BR112017007770A2 (pt) 2014-10-15 2018-01-16 Regeneron Pharma cultura in vitro, população de hipscs, método para modificar um locus-alvo genômico, e, hipsc.
GB201421096D0 (en) 2014-11-27 2015-01-14 Imp Innovations Ltd Genome editing methods
KR20170081268A (ko) 2014-11-27 2017-07-11 단지거 이노베이션즈 엘티디. 게놈 편집용 핵산 구조체
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
ES2947714T3 (es) 2014-12-19 2023-08-17 Regeneron Pharma Métodos y composiciones para la modificación genética dirigida a través de múltiples direccionamientos en un solo paso
HK1246690A1 (zh) 2015-01-21 2018-09-14 Sangamo Therapeutics, Inc. 用於鉴定高度特异性核酸酶的方法和组合物
US20180064748A1 (en) 2015-03-27 2018-03-08 Yeda Research And Development Co. Ltd. Methods of treating motor neuron diseases
CA2981077A1 (en) 2015-04-03 2016-10-06 Dana-Farber Cancer Institute, Inc. Composition and methods of genome editing of b-cells
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
AU2016261927B2 (en) * 2015-05-12 2022-04-07 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
AU2016291778B2 (en) 2015-07-13 2021-05-06 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
US10786547B2 (en) 2015-07-16 2020-09-29 Biokine Therapeutics Ltd. Compositions, articles of manufacture and methods for treating cancer
JP6853257B2 (ja) 2015-09-23 2021-03-31 サンガモ セラピューティクス， インコーポレイテッド Ｈｔｔリプレッサー及びその使用
WO2017053729A1 (en) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Nuclease-mediated genome editing of primary cells and enrichment thereof
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
WO2017075538A1 (en) 2015-10-29 2017-05-04 Amyris, Inc. Compositions and methods for production of myrcene
US10639383B2 (en) 2015-11-23 2020-05-05 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
EP3389677B1 (en) 2015-12-18 2024-06-26 Sangamo Therapeutics, Inc. Targeted disruption of the t cell receptor
BR112018012235A2 (pt) 2015-12-18 2018-12-04 Sangamo Therapeutics Inc rompimento alvejado do receptor de células de mhc
US10828318B2 (en) 2016-01-06 2020-11-10 Yeda Research And Development Co. Ltd. Compositions and methods for treating malignant, autoimmune and inflammatory diseases
WO2017123757A1 (en) 2016-01-15 2017-07-20 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of neurologic disease
JP7019580B2 (ja) 2016-01-21 2022-02-15 ザ ステイト オブ イスラエル ミニストリー オブ アグリカルチャー アンド ルーラル ディベロップメント アグリカルチュラル リサーチ オーガニゼイション （エー．アール．オー．） （ボルカニ センター） 単為結実植物およびその製造方法
US11261433B2 (en) 2016-01-31 2022-03-01 Hadasit Medical Research Services And Development Ltd. Autosomal-identical pluripotent stem cell populations having non-identical sex chromosomal composition and uses thereof
KR20180101442A (ko) 2016-02-02 2018-09-12 상가모 테라퓨틱스, 인코포레이티드 Dna-결합 도메인 및 절단 도메인을 연결시키기 위한 조성물
WO2017138008A2 (en) 2016-02-14 2017-08-17 Yeda Research And Development Co. Ltd. Methods of modulating protein exocytosis and uses of same in therapy
US20190216891A1 (en) 2016-03-06 2019-07-18 Yeda Research And Development Co., Ltd. Method for modulating myelination
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
WO2018029034A1 (en) 2016-08-09 2018-02-15 Vib Vzw Cellulose synthase inhibitors and mutant plants
CA3033372A1 (en) 2016-08-15 2018-02-22 Enevolv, Inc. Cell-free sensor systems
IL247368A0 (en) 2016-08-18 2016-11-30 Yeda Res & Dev Diagnostic and therapeutic uses of exosomes
EP3995574A1 (en) 2016-08-24 2022-05-11 Sangamo Therapeutics, Inc. Regulation of gene expression using engineered nucleases
KR102455249B1 (ko) 2016-08-24 2022-10-17 상가모 테라퓨틱스, 인코포레이티드 가공된 표적 특이적 뉴클레아제
CA3035534A1 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
DK3523326T3 (da) 2016-10-04 2020-08-03 Prec Biosciences Inc Samstimulerende domæner til anvendelse i genetisk modificerede celler
KR102712926B1 (ko) 2016-10-20 2024-10-07 상가모 테라퓨틱스, 인코포레이티드 파브리병의 치료를 위한 방법 및 조성물
WO2018081775A1 (en) 2016-10-31 2018-05-03 Sangamo Therapeutics, Inc. Gene correction of scid-related genes in hematopoietic stem and progenitor cells
CN110234765A (zh) 2016-11-28 2019-09-13 耶达研究及发展有限公司 分离的多核苷酸和多肽及使用它们来表达感兴趣的表达产物的方法
EP3551754B1 (en) 2016-12-08 2023-08-30 Case Western Reserve University Methods and compositions for enhancing functional myelin production
US20200124615A1 (en) 2016-12-29 2020-04-23 Ukko Inc. Methods for identifying and de-epitoping allergenic polypeptides
US11597947B2 (en) 2016-12-29 2023-03-07 Asc Therapeutics Inc. Gene editing method using virus
CN108314736B (zh) * 2017-01-18 2021-08-31 李燕强 一种促进rna降解的方法
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
IL250479A0 (en) 2017-02-06 2017-03-30 Sorek Rotem Genetically engineered cells expressing a disarm system that confers resistance to phages and methods for their production
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
WO2018191440A1 (en) * 2017-04-11 2018-10-18 President And Fellows Of Harvard College In vivo gene editing of blood progenitors
CN110799492B (zh) 2017-04-28 2023-06-27 爱康泰生治疗公司 用于递送核酸的新型羰基脂质和脂质纳米颗粒制剂
US11655275B2 (en) 2017-05-03 2023-05-23 Sangamo Therapeutics, Inc. Methods and compositions for modification of a cystic fibrosis transmembrane conductance regulator (CFTR) gene
IL252151A0 (en) 2017-05-07 2017-07-31 Fainzilber Michael Treatment of stress disorders
EP4029943A1 (en) 2017-05-08 2022-07-20 Precision Biosciences, Inc. Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof
US10738284B2 (en) 2017-06-05 2020-08-11 Regeneron Pharmaceuticals, Inc. B4GALT1 cDNA variants and compositions comprising the same
US11512287B2 (en) 2017-06-16 2022-11-29 Sangamo Therapeutics, Inc. Targeted disruption of T cell and/or HLA receptors
EP3645038B1 (en) 2017-06-30 2026-02-18 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
US12178830B2 (en) 2017-06-30 2024-12-31 Memorial Sloan Kettering Cancer Center Compositions and methods for adoptive cell therapy for cancer
AU2018301393B2 (en) 2017-07-11 2025-02-27 Compass Therapeutics Llc Agonist antibodies that bind human CD137 and uses thereof
IL253642A0 (en) 2017-07-24 2017-09-28 Seger Rony Combined treatment for cancer
KR20200033259A (ko) 2017-07-31 2020-03-27 리제너론 파마슈티칼스 인코포레이티드 생체내에서 CRISPR/Cas-매개된 파괴 또는 삭제 및 외인성 도너 핵산과의 CRISPR/Cas-유도된 재조합을 평가하기 위한 방법 및 조성물
EP3585161A1 (en) 2017-07-31 2020-01-01 Regeneron Pharmaceuticals, Inc. Assessment of crispr/cas-induced recombination with an exogenous donor nucleic acid in vivo
CN110891420B (zh) 2017-07-31 2022-06-03 瑞泽恩制药公司 Cas转基因小鼠胚胎干细胞和小鼠及其应用
AU2018313167A1 (en) 2017-08-08 2020-02-27 Sangamo Therapeutics, Inc. Chimeric antigen receptor mediated cell targeting
WO2019038771A1 (en) 2017-08-23 2019-02-28 Technion Research & Development Foundation Limited COMPOSITIONS AND METHODS FOR ENHANCING ALCOHOL TOLERANCE IN YEAST
LT3675623T (lt) 2017-08-29 2025-09-10 KWS SAAT SE & Co. KGaA Patobulintas mėlynasis aleuronas ir kitos segregacijos sistemos
JP2020536580A (ja) 2017-09-19 2020-12-17 ザ ステート オブ イスラエル， ミニストリー オブ アグリカルチャー アンド ルーラル ディヴェロプメント， アグリカルチュラル リサーチ オーガニゼーション （エーアールオー） （ボルカニ センター） ゲノム編集された鳥
US20190098879A1 (en) 2017-09-29 2019-04-04 Regeneron Pharmaceuticals, Inc. Non-Human Animals Comprising A Humanized TTR Locus And Methods Of Use
EP4269560A3 (en) 2017-10-03 2024-01-17 Precision Biosciences, Inc. Modified epidermal growth factor receptor peptides for use in genetically-modified cells
EP3700543A4 (en) 2017-10-24 2021-08-25 Elani, Dalia ISCHEMIC DISEASE TREATMENT METHODS
WO2019089753A2 (en) 2017-10-31 2019-05-09 Compass Therapeutics Llc Cd137 antibodies and pd-1 antagonists and uses thereof
WO2019089913A1 (en) 2017-11-01 2019-05-09 Precision Biosciences, Inc. Engineered nucleases that target human and canine factor viii genes as a treatment for hemophilia a
BR112020008568A2 (pt) 2017-11-09 2020-10-06 Sangamo Therapeutics, Inc. modificação genética de gene de proteína contendo sh2 indutível por citoquina (cish)
IL255664A0 (en) 2017-11-14 2017-12-31 Shachar Idit Hematopoietic stem cells with enhanced properties
EP3713961A2 (en) 2017-11-20 2020-09-30 Compass Therapeutics LLC Cd137 antibodies and tumor antigen-targeting antibodies and uses thereof
IL257225A (en) 2018-01-29 2018-04-09 Yeda Res & Dev Treatment of sarcoma
CA3089587A1 (en) 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
CN112041432A (zh) 2018-02-15 2020-12-04 纪念斯隆-凯特林癌症中心 Foxp3靶向剂组合物以及用于过继细胞疗法的使用方法
EP3765601A1 (en) 2018-03-16 2021-01-20 Immusoft Corporation B cells genetically engineered to secrete follistatin and methods of using the same to treat follistatin-related diseases, conditions, disorders and to enhance muscle growth and strength
EP3592140A1 (en) 2018-03-19 2020-01-15 Regeneron Pharmaceuticals, Inc. Transcription modulation in animals using crispr/cas systems
CA3094468A1 (en) 2018-04-05 2019-10-10 Juno Therapeutics, Inc. Methods of producing cells expressing a recombinant receptor and related compositions
MA52207A (fr) 2018-04-05 2021-02-17 Editas Medicine Inc Lymphocytes t exprimant un récepteur recombinant, polynucléotides et procédés associés
US11421007B2 (en) 2018-04-18 2022-08-23 Sangamo Therapeutics, Inc. Zinc finger protein compositions for modulation of huntingtin (Htt)
US11690921B2 (en) 2018-05-18 2023-07-04 Sangamo Therapeutics, Inc. Delivery of target specific nucleases
GB201809273D0 (en) 2018-06-06 2018-07-25 Vib Vzw Novel mutant plant cinnamoyl-coa reductase proteins
WO2020008412A1 (en) 2018-07-04 2020-01-09 Ukko Inc. Methods of de-epitoping wheat proteins and use of same for the treatment of celiac disease
KR20210058816A (ko) 2018-08-18 2021-05-24 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 원 위치에 유전자 편집
AU2019326408A1 (en) 2018-08-23 2021-03-11 Sangamo Therapeutics, Inc. Engineered target specific base editors
US11708569B2 (en) 2018-08-29 2023-07-25 University Of Copenhagen Modified recombinant lysosomal alpha-galactosidase A and aspartylglucoaminidase having low mannose-6-phosphate and high sialic acid
KR20210060533A (ko) 2018-09-18 2021-05-26 상가모 테라퓨틱스, 인코포레이티드 프로그램화된 세포 사멸 1 (pd1) 특이적 뉴클레아제
IL281615B2 (en) 2018-09-21 2026-01-01 Acuitas Therapeutics Inc Systems and methods for producing lipid nanoparticles and liposomes
CA3116576A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
IL262658A (en) 2018-10-28 2020-04-30 Memorial Sloan Kettering Cancer Center Prevention of age related clonal hematopoiesis and diseases associated therewith
US11046769B2 (en) 2018-11-13 2021-06-29 Compass Therapeutics Llc Multispecific binding constructs against checkpoint molecules and uses thereof
EP3886869A4 (en) 2018-11-28 2022-07-06 Forty Seven, Inc. GENETICALLY MODIFIED ABLATION-RESISTANT HSPCS
KR20200071198A (ko) 2018-12-10 2020-06-19 네오이뮨텍, 인코퍼레이티드 Nrf2 발현 조절 기반 T 세포 항암면역치료법
GB201820109D0 (en) 2018-12-11 2019-01-23 Vib Vzw Plants with a lignin trait and udp-glycosyltransferase mutation
KR102925054B1 (ko) 2018-12-20 2026-02-10 리제너론 파마슈티칼스 인코포레이티드 뉴클레아제-매개 반복부 팽창
WO2020132659A1 (en) 2018-12-21 2020-06-25 Precision Biosciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
PT3908568T (pt) 2019-01-11 2024-09-30 Acuitas Therapeutics Inc Lípidos para a administração de agentes ativos por nanopartículas lipídicas
AU2019428629A1 (en) 2019-02-06 2021-01-28 Sangamo Therapeutics, Inc. Method for the treatment of mucopolysaccharidosis type I
KR20210137499A (ko) 2019-03-05 2021-11-17 더 스테이트 오브 이스라엘, 미니스트리 오브 애그리컬처 & 루럴 디벨로프먼트, 애그리컬처럴 리서치 오거니제이션, (에이.알.오.), 볼카니 센터 게놈-편집 조류
CA3132167A1 (en) 2019-04-02 2020-10-08 Weston P. MILLER IV Methods for the treatment of beta-thalassemia
KR102691932B1 (ko) 2019-04-03 2024-08-06 프리시젼 바이오사이언시스 인코포레이티드 마이크로RNA-적응 shRNA(shRNAmiR)를 포함하는 유전자-변형 면역 세포
AU2020256225B9 (en) 2019-04-03 2025-04-10 Regeneron Pharmaceuticals, Inc. Methods and compositions for insertion of antibody coding sequences into a safe harbor locus
RU2771374C1 (ru) 2019-04-04 2022-05-04 Редженерон Фармасьютикалс, Инк. Способы для бесшовного внесения целевых модификаций в направленные векторы
KR102661779B1 (ko) 2019-04-04 2024-04-30 리제너론 파마슈티칼스 인코포레이티드 인간화 응고 인자 12 좌위를 포함하는 비-인간 동물
EP3947646A1 (en) 2019-04-05 2022-02-09 Precision BioSciences, Inc. Methods of preparing populations of genetically-modified immune cells
CA3136742A1 (en) 2019-05-01 2020-11-05 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified cd247 locus, related polynucleotides and methods
WO2020223535A1 (en) 2019-05-01 2020-11-05 Juno Therapeutics, Inc. Cells expressing a recombinant receptor from a modified tgfbr2 locus, related polynucleotides and methods
US11891618B2 (en) 2019-06-04 2024-02-06 Regeneron Pharmaceuticals, Inc. Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use
MX2021015122A (es) 2019-06-07 2022-04-06 Regeneron Pharma Animales no humanos que comprenden un locus de albumina humanizado.
WO2020252455A1 (en) 2019-06-13 2020-12-17 The General Hospital Corporation Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells
CN113906134B (zh) 2019-06-14 2025-06-24 瑞泽恩制药公司 Tau蛋白病模型
WO2021001784A1 (en) 2019-07-04 2021-01-07 Ukko Inc. De-epitoped alpha gliadin and use of same for the management of celiac disease and gluten sensitivity
IL268111A (en) 2019-07-16 2021-01-31 Fainzilber Michael Methods of treating pain
US20220273715A1 (en) 2019-07-25 2022-09-01 Precision Biosciences, Inc. Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus
CA3148179A1 (en) 2019-08-20 2021-02-25 Bruce J. Mccreedy Jr. Lymphodepletion dosing regimens for cellular immunotherapies
WO2021035170A1 (en) 2019-08-21 2021-02-25 Precision Biosciences, Inc. Compositions and methods for tcr reprogramming using fusion proteins
US20220411479A1 (en) 2019-10-30 2022-12-29 Precision Biosciences, Inc. Cd20 chimeric antigen receptors and methods of use for immunotherapy
IL270306A (en) 2019-10-30 2021-05-31 Yeda Res & Dev Prevention and treatment of pre-myeloid and myeloid malignancies
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
WO2021108363A1 (en) 2019-11-25 2021-06-03 Regeneron Pharmaceuticals, Inc. Crispr/cas-mediated upregulation of humanized ttr allele
WO2021113543A1 (en) 2019-12-06 2021-06-10 Precision Biosciences, Inc. Methods for cancer immunotherapy, using lymphodepletion regimens and cd19, cd20 or bcma allogeneic car t cells
IL271656A (en) 2019-12-22 2021-06-30 Yeda Res & Dev System and methods for identifying cells that have undergone genome editing
EP4096647A1 (en) 2020-01-30 2022-12-07 Yeda Research and Development Co. Ltd Treating acute liver disease with tlr-mik inhibitors
WO2021158915A1 (en) 2020-02-06 2021-08-12 Precision Biosciences, Inc. Recombinant adeno-associated virus compositions and methods for producing and using the same
KR20230004456A (ko) 2020-03-04 2023-01-06 리제너론 파아마슈티컬스, 인크. 면역 요법에 대한 종양 세포의 감작화를 위한 방법 및 조성물
US20230102342A1 (en) 2020-03-23 2023-03-30 Regeneron Pharmaceuticals, Inc. Non-human animals comprising a humanized ttr locus comprising a v30m mutation and methods of use
US20230263121A1 (en) 2020-03-31 2023-08-24 Elo Life Systems Modulation of endogenous mogroside pathway genes in watermelon and other cucurbits
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 (en) 2020-05-06 2023-03-15 Cellectis S.A. Methods to genetically modify cells for delivery of therapeutic proteins
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
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
JP2023531531A (ja) 2020-06-26 2023-07-24 ジュノ セラピューティクス ゲーエムベーハー 組換え受容体を条件付きで発現する操作されたｔ細胞、関連ポリヌクレオチド、および方法
ES3054438T3 (en) 2020-07-16 2026-02-03 Acuitas Therapeutics Inc Cationic lipids for use in lipid nanoparticles
CA3189601A1 (en) 2020-07-24 2022-01-27 The General Hospital Corporation Enhanced virus-like particles and methods of use thereof for delivery to cells
EP4192875A1 (en) 2020-08-10 2023-06-14 Precision BioSciences, Inc. Antibodies and fragments specific for b-cell maturation antigen and uses thereof
WO2022074646A1 (en) 2020-10-05 2022-04-14 Protalix Ltd. Dicer-like knock-out plant cells
WO2022076547A1 (en) 2020-10-07 2022-04-14 Precision Biosciences, Inc. Lipid nanoparticle compositions
US20240016855A1 (en) * 2020-10-13 2024-01-18 Centre National De La Recherche Scientifique (Cnrs) Targeted-antibacterial-plasmids combining conjugation and crispr/cas systems and uses thereof
EP4228637A1 (en) 2020-10-15 2023-08-23 Yeda Research and Development Co. Ltd Method of treating myeloid malignancies
US20240060079A1 (en) 2020-10-23 2024-02-22 Elo Life Systems Methods for producing vanilla plants with improved flavor and agronomic production
EP4240756A1 (en) 2020-11-04 2023-09-13 Juno Therapeutics, Inc. Cells expressing a chimeric receptor from a modified invariant cd3 immunoglobulin superfamily chain locus and related polynucleotides and methods
IL302707A (en) 2020-11-26 2023-07-01 Ukko Inc Modified high molecular weight glutenin subunit and uses thereof
IL279559A (en) 2020-12-17 2022-07-01 Yeda Res & Dev Controlling ubiquitination of mlkl for treatment of disease
US20250127811A1 (en) 2021-01-28 2025-04-24 Precision Biosciences, Inc. Modulation of tgf beta signaling in genetically-modified eukaryotic cells
CA3213080A1 (en) 2021-03-23 2022-09-29 Krit RITTHIPICHAI Cish gene editing of tumor infiltrating lymphocytes and uses of same in immunotherapy
US20240141311A1 (en) 2021-04-22 2024-05-02 North Carolina State University Compositions and methods for generating male sterile plants
WO2022236010A1 (en) * 2021-05-06 2022-11-10 The University Of North Carolina At Chapel Hill Use of chemical epigenetic modifiers to modulate gene expression from vectors
CA3218511A1 (en) 2021-05-10 2022-11-17 Sqz Biotechnologies Company Methods for delivering genome editing molecules to the nucleus or cytosol of a cell and uses thereof
JP2024517903A (ja) 2021-05-10 2024-04-23 イッサム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティー オブ エルサレム リミテッド 神経学的状態を治療するための薬学的組成物
WO2022251644A1 (en) 2021-05-28 2022-12-01 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
EP4347826A1 (en) 2021-06-02 2024-04-10 Lyell Immunopharma, Inc. Nr4a3-deficient immune cells and uses thereof
IL309333A (en) 2021-06-13 2024-02-01 Yissum Res Dev Co Of Hebrew Univ Jerusalem Ltd A method for reprogramming cells
CA3226947A1 (en) 2021-08-03 2023-02-09 Muhammad YASSIN Engineered tcr complex and methods of using same
EP4130028A1 (en) 2021-08-03 2023-02-08 Rhazes Therapeutics Ltd Engineered tcr complex and methods of using same
WO2023035011A1 (en) 2021-09-03 2023-03-09 North Carolina State University Compositions and methods for conferring resistance to geminivirus
WO2023064872A1 (en) 2021-10-14 2023-04-20 Precision Biosciences, Inc. Combinations of anti-bcma car t cells and gamma secretase inhibitors
US20240407364A1 (en) 2021-10-14 2024-12-12 Weedout Ltd. Methods of weed control
IL311962A (en) 2021-10-14 2024-06-01 Lonza Sales Ag Modified producer cells for extracellular vesicle production
IL312244A (en) 2021-10-19 2024-06-01 Prec Biosciences Inc Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
US20230149563A1 (en) 2021-10-27 2023-05-18 Regeneron Pharmaceuticals, Inc. Compositions and methods for expressing factor ix for hemophilia b therapy
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
AU2022379973A1 (en) 2021-11-08 2024-06-27 Progentos Therapeutics, Inc. Platelet-derived growth factor receptor (pdgfr) alpha inhibitors and uses thereof
WO2023091910A1 (en) 2021-11-16 2023-05-25 Precision Biosciences, Inc. Methods for cancer immunotherapy
KR20240117571A (ko) 2021-12-08 2024-08-01 리제너론 파마슈티칼스 인코포레이티드 돌연변이 마이오실린 질환 모델 및 이의 용도
CA3242402A1 (en) 2021-12-16 2023-06-22 Acuitas Therapeutics, Inc. Lipids for use in lipid nanoparticle formulations
AU2022424002A1 (en) 2021-12-29 2024-06-13 Bristol-Myers Squibb Company Generation of landing pad cell lines
EP4460571A1 (en) 2022-01-05 2024-11-13 Vib Vzw Means and methods to increase abiotic stress tolerance in plants
WO2023131637A1 (en) 2022-01-06 2023-07-13 Vib Vzw Improved silage grasses
WO2023144199A1 (en) 2022-01-26 2023-08-03 Vib Vzw Plants having reduced levels of bitter taste metabolites
CA3242731A1 (en) 2022-02-02 2023-08-10 Regeneron Pharmaceuticals, Inc. Anti-tfr:gaa and anti-cd63:gaa insertions for treatment of pompe disease
WO2023150798A1 (en) 2022-02-07 2023-08-10 Regeneron Pharmaceuticals, Inc. Compositions and methods for defining optimal treatment timeframes in lysosomal disease
AU2023218391A1 (en) 2022-02-11 2024-07-11 Regeneron Pharmaceuticals, Inc. Compositions and methods for screening 4r tau targeting agents
US20250302998A1 (en) 2022-05-09 2025-10-02 Regeneron Pharmaceuticals, Inc. Vectors and methods for in vivo antibody production
JP2025516823A (ja) 2022-05-19 2025-05-30 ライエル・イミュノファーマ・インコーポレイテッド Ｎｒ４ａ３を標的とするポリヌクレオチド及びその使用
AU2023283552A1 (en) 2022-06-10 2025-01-23 Umoja Biopharma, Inc. Engineered stem cells and uses thereof
CN120659627A (zh) 2022-07-29 2025-09-16 瑞泽恩制药公司 用于转铁蛋白受体（tfr）介导的脑和肌肉递送的组合物和方法
JP2025525745A (ja) 2022-08-05 2025-08-07 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 Ｔｄｐ－４３の凝集耐性バリアント
WO2024064958A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
WO2024064952A1 (en) 2022-09-23 2024-03-28 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells overexpressing c-jun
KR20250075694A (ko) 2022-09-28 2025-05-28 리제너론 파마슈티칼스 인코포레이티드 세포 기반 요법을 강화하기 위한 항체 저항성 변형 수용체
WO2024077174A1 (en) 2022-10-05 2024-04-11 Lyell Immunopharma, Inc. Methods for culturing nr4a-deficient cells
EP4612184A1 (en) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024100604A1 (en) 2022-11-09 2024-05-16 Juno Therapeutics Gmbh Methods for manufacturing engineered immune cells
KR20250116795A (ko) 2022-11-14 2025-08-01 리제너론 파마슈티칼스 인코포레이티드 별아교세포로의 섬유아세포 성장 인자 수용체 3-매개된 전달을 위한 조성물 및 방법
JP2026504491A (ja) 2023-02-03 2026-02-05 ツェー３エス２ ゲーエムベーハー 操作された免疫細胞の非ウイルス的製造のための方法
WO2024216116A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
WO2024216118A1 (en) 2023-04-14 2024-10-17 Precision Biosciences, Inc. Muscle-specific expression cassettes
CN121693566A (zh) 2023-07-28 2026-03-17 瑞泽恩制药公司 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达
US20250049896A1 (en) 2023-07-28 2025-02-13 Regeneron Pharmaceuticals, Inc. Anti-tfr:acid sphingomyelinase for treatment of acid sphingomyelinase deficiency
IL326018A (en) 2023-07-28 2026-03-01 Regeneron Pharma Introduction of anti-TFR:GAA and anti-CD63:GAA for the treatment of Pompe disease
WO2025049524A1 (en) 2023-08-28 2025-03-06 Regeneron Pharmaceuticals, Inc. Cxcr4 antibody-resistant modified receptors
WO2025129084A1 (en) 2023-12-13 2025-06-19 Umoja Biopharma, Inc. Engineered induced stem cell derived myeloid cells and methods of differentiating and using same
WO2025147573A2 (en) 2024-01-05 2025-07-10 Immusoft Corporation Glp-1 expressing modified b cells for the treatment of metabolic disease
US20250276092A1 (en) 2024-03-01 2025-09-04 Regeneron Pharmaceuticals, Inc. Methods and compositions for re-dosing aav using anti-cd40 antagonistic antibody to suppress host anti-aav antibody response
WO2025217398A1 (en) 2024-04-10 2025-10-16 Lyell Immunopharma, Inc. Methods for culturing cells with improved culture medium
WO2025235388A1 (en) 2024-05-06 2025-11-13 Regeneron Pharmaceuticals, Inc. Transgene genomic identification by nuclease-mediated long read sequencing
US20250345431A1 (en) 2024-05-10 2025-11-13 Juno Therapeutics, Inc. Genetically engineered t cells expressing a cd19 chimeric antigen receptor (car) and uses thereof for allogeneic cell therapy
WO2025265017A1 (en) 2024-06-20 2025-12-26 Regeneron Pharmaceuticals, Inc. Ass1 gene insertion for the treatment of citrullinemia type i
WO2026009227A1 (en) 2024-07-04 2026-01-08 Yeda Research And Development Co. Ltd. Compositions for downregulating zeb2 in macrophages and uses thereof
WO2026072529A1 (en) 2024-09-24 2026-04-02 University Of Florida Research Foundation, Incorporated Mettl7a for improved embryo competence
WO2026083329A1 (en) 2024-10-18 2026-04-23 Precision Biosciences, Inc. Methods of nuclease-initiated homology directed repair and replacement and compositions and uses thereof
WO2026083328A1 (en) 2024-10-18 2026-04-23 Precision Biosciences, Inc. Base editing nucleotide sequences using homology directed repair

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6046036A (en) * 1996-10-25 2000-04-04 Advanced Research And Technology Institute DNA sequences encoding fusions of DNA repair proteins and uses thereof

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4942227A (en) 1982-01-11 1990-07-17 California Institute Of Technology Bifunctional molecules having a DNA intercalator or DNA groove binder linked to ethylene diamine tetraacetic acid, their preparation and use to cleave DNA
US4665184A (en) * 1983-10-12 1987-05-12 California Institute Of Technology Bifunctional molecules having a DNA intercalator or DNA groove binder linked to ethylene diamine tetraacetic acid
US4795700A (en) * 1985-01-25 1989-01-03 California Institute Of Technology Nucleic acid probes and methods of using same
US5789155A (en) * 1987-10-30 1998-08-04 California Institute Of Technology Process for identifying nucleic acids and triple helices formed thereby
US5955341A (en) * 1991-04-10 1999-09-21 The Scripps Research Institute Heterodimeric receptor libraries using phagemids
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
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
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
US5792640A (en) * 1992-04-03 1998-08-11 The Johns Hopkins University General method to clone hybrid restriction endonucleases using lig gene
US5496720A (en) * 1993-02-10 1996-03-05 Susko-Parrish; Joan L. Parthenogenic oocyte activation
US6331658B1 (en) * 1993-04-20 2001-12-18 Integris Baptist Medical Center, Inc. Genetically engineered mammals for use as organ donors
US6140466A (en) * 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) * 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
USRE39229E1 (en) * 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US6326166B1 (en) * 1995-12-29 2001-12-04 Massachusetts Institute Of Technology 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
AU6096296A (en) 1995-06-07 1996-12-30 Ohio State University, The Artificial restriction endonuclease
GB9517780D0 (en) * 1995-08-31 1995-11-01 Roslin Inst Edinburgh Biological manipulation
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US6265196B1 (en) * 1996-05-07 2001-07-24 Johns Hopkins University Methods for inactivating target DNA and for detecting conformational change in a nucleic acid
US5928914A (en) 1996-06-14 1999-07-27 Albert Einstein College Of Medicine Of Yeshiva University, A Division Of Yeshiva University Methods and compositions for transforming cells
US5945577A (en) * 1997-01-10 1999-08-31 University Of Massachusetts As Represented By Its Amherst Campus Cloning using donor nuclei from proliferating somatic cells
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
AU757930B2 (en) 1997-12-01 2003-03-13 Roche Diagnostics Gmbh Optimization of cells for endogenous gene activation
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
ES2341926T3 (es) 1998-03-02 2010-06-29 Massachusetts Institute Of Technology Poliproteinas con dedos de cinc que tienen enlazadores mejorados.
JP2002522096A (ja) 1998-08-12 2002-07-23 パンジーン・コーポレイション ドメイン特異的遺伝子の展開
US6140081A (en) * 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
BR9915191A (pt) 1998-11-10 2001-12-11 Maxygen Inc Métodos para obter um polinucleotìdeo isolado epara produzir uma célula recombinante,protoplasto de célula vegetal, coleção deprotoplastos de célula vegetal, vegetal,polinucleotìdeo, biblioteca, um ou mais membros debiblioteca e célula recombinante alvo
US6534261B1 (en) * 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
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
EP1151124A1 (en) * 1999-02-03 2001-11-07 The Children's Medical Center Corporation Gene repair involving in vivo excision of targeting dna
CA2361191A1 (en) * 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
US6977295B2 (en) 1999-04-21 2005-12-20 Invitrogen Corporation Locked nucleic acid hybrids and methods of use
AU781628B2 (en) 1999-07-14 2005-06-02 Clontech Laboratories, Inc. Recombinase-based methods for producing expression vectors and compositions for use in practicing the same
JP2001086989A (ja) * 1999-09-17 2001-04-03 Univ Osaka 哺乳類型Ｃｒｅリコンビナーゼ遺伝子
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
WO2001059094A2 (en) * 2000-02-11 2001-08-16 The Salk Institute For Biological Studies Method of regulating transcription in a cell by altering remodeling of cromatin
CA2401677A1 (en) 2000-03-03 2001-09-13 University Of Utah Research Foundation Gene targeting method
EP1272630A2 (en) 2000-03-16 2003-01-08 Genetica, Inc. Methods and compositions for rna interference
US20030084471A1 (en) 2000-03-16 2003-05-01 David Beach Methods and compositions for RNA interference
ES2336887T5 (es) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediadores de interferencia por ARN específicos de secuencias de ARN
US6492117B1 (en) 2000-07-12 2002-12-10 Gendaq Limited Zinc finger polypeptides capable of binding DNA quadruplexes
US7091026B2 (en) * 2001-02-16 2006-08-15 University Of Iowa Research Foundation Artificial endonuclease
WO2003087341A2 (en) 2002-01-23 2003-10-23 The University Of Utah Research Foundation Targeted chromosomal mutagenesis using zinc finger nucleases
WO2003080809A2 (en) 2002-03-21 2003-10-02 Sangamo Biosciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6046036A (en) * 1996-10-25 2000-04-04 Advanced Research And Technology Institute DNA sequences encoding fusions of DNA repair proteins and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Giesen K et al, Journal of General Virology, 2000, volume 81, p 2231-2244 *

Also Published As

Publication number Publication date
US20170114368A1 (en) 2017-04-27
AU2003298574A1 (en) 2004-05-13
US9447434B2 (en) 2016-09-20
US10006053B2 (en) 2018-06-26
EP1581610A4 (en) 2009-05-27
CA2497913A1 (en) 2004-05-06
EP2806025B1 (en) 2019-04-03
WO2004037977A3 (en) 2005-07-28
CA2497913C (en) 2014-06-03
US20050026157A1 (en) 2005-02-03
EP2806025A1 (en) 2014-11-26
EP1581610A2 (en) 2005-10-05
JP2006502748A (ja) 2006-01-26
WO2004037977A2 (en) 2004-05-06

Similar Documents

Publication Publication Date Title
AU2003298574B2 (en) Use of chimeric nucleases to stimulate gene targeting
US20230203541A1 (en) Optimized gene editing utilizing a recombinant endonuclease system
EP2539445B1 (en) Use of endonucleases for inserting transgenes into safe harbor loci
JP7763237B2 (ja) ゲノム編集のための新規の天然に存在しないｃｒｉｓｐｒ－ｃａｓヌクレアーゼ
US20170037434A1 (en) Chromosomal modification involving the induction of double-stranded dna cleavage and homologous recombination at the cleavage site
CN111163633A (zh) 包含人源化ttr基因座的非人类动物及其使用方法
CN110997909A (zh) 改进的转座酶多肽及其用途
KR102927708B1 (ko) 베타-슬립 돌연변이를 갖는 인간화 ttr 좌위를 포함하는 비-인간 동물 및 사용 방법
US20090217400A1 (en) Enzymes, cells and methods for site specific recombination at asymmetric sites
AU2982300A (en) Gene repair involving (in vivo) excision of targeting dna
WO2022247873A1 (zh) 工程化的Cas12i核酸酶、效应蛋白及其用途
CA3238939A1 (en) Mutant myocilin disease model and uses thereof
US9816077B2 (en) Use of integrase for targeted gene expression
WO2012007848A2 (en) Meganuclease variants cleaving a dna target sequence in the was gene and uses thereof
AU2004202860B2 (en) Gene repair involving in vivo excision of targeting DNA

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)
MK14 Patent ceased section 143(a) (annual fees not paid) or expired