ZA201302625B - Methods and compositions for treating hemophilia b - Google Patents

Methods and compositions for treating hemophilia b

Info

Publication number
ZA201302625B
ZA201302625B ZA2013/02625A ZA201302625A ZA201302625B ZA 201302625 B ZA201302625 B ZA 201302625B ZA 2013/02625 A ZA2013/02625 A ZA 2013/02625A ZA 201302625 A ZA201302625 A ZA 201302625A ZA 201302625 B ZA201302625 B ZA 201302625B
Authority
ZA
South Africa
Prior art keywords
compositions
methods
treating hemophilia
hemophilia
treating
Prior art date
Application number
ZA2013/02625A
Other languages
English (en)
Inventor
Philip D Gregory
Michael C Holmes
Katherine A High
Hojun Li
Original Assignee
The Children's Hospital Of Philadelphia
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 The Children's Hospital Of Philadelphia, Sangamo Biosciences Inc filed Critical The Children's Hospital Of Philadelphia
Publication of ZA201302625B publication Critical patent/ZA201302625B/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N5/00—Radiation therapy
    • A61N5/10—X-ray therapy; Gamma-ray therapy; Particle-irradiation therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A61P7/02—Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1024—In vivo mutagenesis using high mutation rate "mutator" host strains by inserting genetic material, e.g. encoding an error prone polymerase, disrupting a gene for mismatch repair
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/50—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25)
    • C12N9/64—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue
    • C12N9/6421—Proteinases, e.g. Endopeptidases (3.4.21-3.4.25) derived from animal tissue from mammals
    • C12N9/6424—Serine endopeptidases (3.4.21)
    • C12N9/644—Coagulation factor IXa (3.4.21.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21002—Chymotrypsin C (3.4.21.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61N—ELECTROTHERAPY; MAGNETOTHERAPY; RADIATION THERAPY; ULTRASOUND THERAPY
    • A61N5/00—Radiation therapy
    • A61N5/06—Radiation therapy using light
    • A61N2005/0658—Radiation therapy using light characterised by the wavelength of light used
    • A61N2005/0661—Radiation therapy using light characterised by the wavelength of light used ultraviolet

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hematology (AREA)
  • Pathology (AREA)
  • Radiology & Medical Imaging (AREA)
  • Immunology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ZA2013/02625A 2010-10-12 2013-04-11 Methods and compositions for treating hemophilia b ZA201302625B (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US39233310P 2010-10-12 2010-10-12
PCT/US2011/056020 WO2012051343A1 (en) 2010-10-12 2011-10-12 Methods and compositions for treating hemophilia b

Publications (1)

Publication Number Publication Date
ZA201302625B true ZA201302625B (en) 2014-05-28

Family

ID=45938707

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2013/02625A ZA201302625B (en) 2010-10-12 2013-04-11 Methods and compositions for treating hemophilia b

Country Status (12)

Country Link
US (2) US9175280B2 (cg-RX-API-DMAC7.html)
EP (1) EP2627665B1 (cg-RX-API-DMAC7.html)
JP (1) JP6018069B2 (cg-RX-API-DMAC7.html)
KR (1) KR101930711B1 (cg-RX-API-DMAC7.html)
CN (1) CN103270046B (cg-RX-API-DMAC7.html)
AU (1) AU2011316575B2 (cg-RX-API-DMAC7.html)
BR (1) BR112013008881B1 (cg-RX-API-DMAC7.html)
CA (1) CA2814143C (cg-RX-API-DMAC7.html)
ES (1) ES2562421T3 (cg-RX-API-DMAC7.html)
RU (1) RU2608643C2 (cg-RX-API-DMAC7.html)
WO (1) WO2012051343A1 (cg-RX-API-DMAC7.html)
ZA (1) ZA201302625B (cg-RX-API-DMAC7.html)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006063031A2 (en) 2004-12-06 2006-06-15 Haplomics Allelic variants of human factor viii
US9518087B2 (en) 2010-08-10 2016-12-13 Ecole Polytechnique Federale De Lausanne (Epfl) Erythrocyte-binding therapeutics
US9850296B2 (en) 2010-08-10 2017-12-26 Ecole Polytechnique Federale De Lausanne (Epfl) Erythrocyte-binding therapeutics
US9517257B2 (en) 2010-08-10 2016-12-13 Ecole Polytechnique Federale De Lausanne (Epfl) Erythrocyte-binding therapeutics
IL293650B2 (en) 2011-09-21 2024-10-01 Sangamo Therapeutics Inc Methods and compositions for regulation of transgene expression
BR112014020325A2 (pt) * 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
AU2013289206B2 (en) 2012-07-11 2018-08-09 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
US10648001B2 (en) 2012-07-11 2020-05-12 Sangamo Therapeutics, Inc. Method of treating mucopolysaccharidosis type I or II
EP2929017A4 (en) * 2012-12-05 2016-09-28 Sangamo Biosciences Inc METHOD AND COMPOSITIONS FOR REGULATING METABOLISM DISEASES
US20160045575A1 (en) * 2012-12-07 2016-02-18 Tom E. HOWARD FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED cDNAs, COMPOSITIONS, METHODS AND SYSTEMS
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
WO2014093709A1 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof
IL239317B (en) 2012-12-12 2022-07-01 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
AU2014281028B2 (en) 2013-06-17 2020-09-10 Massachusetts Institute Of Technology Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
EP3011033B1 (en) 2013-06-17 2020-02-19 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof
CN106062197A (zh) 2013-06-17 2016-10-26 布罗德研究所有限公司 用于序列操纵的串联指导系统、方法和组合物的递送、工程化和优化
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
DK3080274T3 (da) 2013-12-09 2020-08-31 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til genom-manipulation
CN118813621A (zh) * 2013-12-12 2024-10-22 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089364A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Crystal structure of a crispr-cas system, and uses thereof
EP3080257A1 (en) 2013-12-12 2016-10-19 The Broad Institute Inc. Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders
WO2015089486A2 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
MX374530B (es) 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de los sistemas y composiciones crispr-cas para actuar sobre hbv y trastornos y enfermedades virales.
US10946079B2 (en) 2014-02-21 2021-03-16 Ecole Polytechnique Federale De Lausanne Glycotargeting therapeutics
US10046056B2 (en) 2014-02-21 2018-08-14 École Polytechnique Fédérale De Lausanne (Epfl) Glycotargeting therapeutics
CN106456743A (zh) 2014-02-21 2017-02-22 洛桑聚合联合学院 糖靶向性治疗剂
US10953101B2 (en) 2014-02-21 2021-03-23 École Polytechnique Fédérale De Lausanne (Epfl) Glycotargeting therapeutics
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
CA2939847C (en) 2014-03-21 2023-09-05 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases
WO2015191899A1 (en) * 2014-06-11 2015-12-17 Howard Tom E FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS
SG11201702073XA (en) 2014-09-24 2017-04-27 Hope City Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
RU2020109343A (ru) 2014-11-05 2020-03-17 Вояджер Терапьютикс, Инк. Полинуклеотиды aadc для лечения болезни паркинсона
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
US10889834B2 (en) 2014-12-15 2021-01-12 Sangamo Therapeutics, Inc. Methods and compositions for enhancing targeted transgene integration
AU2015369725A1 (en) 2014-12-24 2017-06-29 Massachusetts Institute Of Technology CRISPR having or associated with destabilization domains
EP3498843B1 (en) * 2015-01-06 2021-06-23 Industry-Academic Cooperation Foundation, Yonsei University Endonuclease targeting blood coagulation factor viii gene and composition for treating hemophilia comprising same
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
WO2016205613A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzyme mutations reducing off-target effects
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
AU2016343887B2 (en) 2015-10-28 2023-04-06 Sangamo Therapeutics, Inc. Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
AU2016361350B2 (en) 2015-11-23 2023-04-06 Sangamo Therapeutics, Inc. Methods and compositions for engineering immunity
CN105506106A (zh) * 2015-12-30 2016-04-20 杭州艾迪康医学检验中心有限公司 检测fix基因全外显子的方法和引物
EP3509645A4 (en) 2016-09-07 2020-06-17 Sangamo Therapeutics, Inc. MODULATION OF LIVER GENES
HRP20240806T1 (hr) 2017-04-28 2024-12-06 The Regents Of The University Of Colorado, A Body Corporate Metode liječenja reumatoidnog artritisa pomoću rna-vođenog genoma uređivanja hla gena
US11046969B2 (en) * 2017-05-24 2021-06-29 Epiplanta Biotech Ltd. Transgenic plant and the method for producing the same
SG11201911623WA (en) * 2017-06-15 2020-01-30 Toolgen Inc Platform for expressing protein of interest in liver
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3638296A1 (en) 2017-06-16 2020-04-22 The University Of Chicago Compositions and methods for inducing immune tolerance
AU2018355343B2 (en) * 2017-10-24 2024-12-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of rare diseases
KR20210010879A (ko) 2018-05-09 2021-01-28 더 유니버서티 오브 시카고 면역 관용 관여 조성물 및 방법
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
PL3867372T3 (pl) 2018-10-16 2025-06-23 Blueallele Corporation Sposoby ukierunkowanego wprowadzania dna do genów
EP3867380A2 (en) 2018-10-18 2021-08-25 Intellia Therapeutics, Inc. Compositions and methods for expressing factor ix
CN109385425A (zh) * 2018-11-13 2019-02-26 中山大学 一种高特异性ABE碱基编辑系统及其在β血红蛋白病中的应用
EP3954208A4 (en) * 2019-03-26 2022-12-28 Toolgen Incorporated RAT MODEL OF HEMOPHILIA B
WO2022178218A1 (en) 2021-02-19 2022-08-25 Mevion Medical Systems, Inc. Gantry for a particle therapy system
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use

Family Cites Families (66)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
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
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
AU704601B2 (en) 1994-01-18 1999-04-29 Scripps Research Institute, The Zinc finger protein derivatives and methods therefor
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
US6521225B1 (en) 1996-09-06 2003-02-18 Chiron Corporation AAV vectors
WO1998024479A1 (en) 1996-12-02 1998-06-11 Cell Genesys, Inc. Adeno-associated viral vector-mediated delivery of dna to cells of the liver
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
EP1005376B1 (en) 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions for use in gene therapy for treatment of hemophilia
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
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
US6479626B1 (en) 1998-03-02 2002-11-12 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
EP1236045B1 (en) 1999-12-06 2005-11-09 Sangamo Biosciences Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
DE10005183A1 (de) 2000-02-05 2001-08-09 Bosch Gmbh Robert Gleichrichteranordnung
CA2398590C (en) 2000-02-08 2012-08-28 Sangamo Biosciences, Inc. Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
ES2301624T3 (es) * 2001-02-05 2008-07-01 Novo Nordisk Health Care Ag Uso combinado de polipeptidos de factor vii y polipeptidos de factor viii.
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
JP4968498B2 (ja) 2002-01-23 2012-07-04 ユニバーシティ オブ ユタ リサーチ ファウンデーション ジンクフィンガーヌクレアーゼを用いる、標的化された染色体変異誘発
EP2368982A3 (en) 2002-03-21 2011-10-12 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
US9447434B2 (en) 2002-09-05 2016-09-20 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
CA2534296C (en) 2003-08-08 2013-03-26 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2562193A1 (en) * 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
CA2579677A1 (en) 2004-09-16 2006-03-30 Sangamo Biosciences, Inc. Compositions and methods for protein production
JP4988606B2 (ja) * 2005-02-28 2012-08-01 サンガモ バイオサイエンシズ インコーポレイテッド 抗血管新生方法及び組成物
MX2007013757A (es) 2005-05-05 2008-01-24 Univ Arizona Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas.
SG10201508995QA (en) 2005-07-26 2015-11-27 Sangamo Biosciences Inc Targeted integration and expression of exogenous nucleic acid sequences
EP2765195A1 (en) 2006-05-25 2014-08-13 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
ATE462783T1 (de) 2006-05-25 2010-04-15 Sangamo Biosciences Inc Variante foki-spaltungshälften-domänen
EP2049663B1 (en) 2006-08-11 2015-02-25 Dow AgroSciences LLC Zinc finger nuclease-mediated homologous recombination
DE602008003684D1 (de) 2007-04-26 2011-01-05 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
EP2171052B1 (en) * 2007-07-12 2014-08-27 Sangamo BioSciences, Inc. Methods and compositions for inactivating alpha 1,6 fucosyltransferase (fut 8) gene expression
US9506120B2 (en) 2007-09-27 2016-11-29 Sangamo Biosciences, Inc. Rapid in vivo identification of biologically active nucleases
EP2281050B1 (en) 2008-04-14 2014-04-02 Sangamo BioSciences, Inc. Linear donor constructs for targeted integration
WO2010065123A1 (en) 2008-12-04 2010-06-10 Sangamo Biosciences, Inc. Genome editing in rats using zinc-finger nucleases
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
CA2756833C (en) 2009-04-09 2019-11-19 Sangamo Biosciences, Inc. Targeted integration into stem cells
US20120192298A1 (en) * 2009-07-24 2012-07-26 Sigma Aldrich Co. Llc Method for genome editing
EP2615106B1 (en) 2010-02-08 2018-04-25 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
EP2571512B1 (en) 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof

Also Published As

Publication number Publication date
RU2013120986A (ru) 2014-11-20
BR112013008881A2 (pt) 2016-06-28
BR112013008881B1 (pt) 2021-12-07
EP2627665A1 (en) 2013-08-21
US9629930B2 (en) 2017-04-25
EP2627665B1 (en) 2015-12-16
CA2814143C (en) 2020-09-08
HK1185623A1 (zh) 2014-02-21
WO2012051343A1 (en) 2012-04-19
AU2011316575B2 (en) 2015-10-29
CN103270046A (zh) 2013-08-28
AU2011316575A1 (en) 2013-05-02
JP6018069B2 (ja) 2016-11-02
ES2562421T3 (es) 2016-03-04
US20160022838A1 (en) 2016-01-28
KR20140042764A (ko) 2014-04-07
US9175280B2 (en) 2015-11-03
US20120128635A1 (en) 2012-05-24
RU2608643C2 (ru) 2017-01-23
EP2627665A4 (en) 2014-02-26
CA2814143A1 (en) 2012-04-19
JP2013544077A (ja) 2013-12-12
KR101930711B1 (ko) 2018-12-19
CN103270046B (zh) 2016-05-11

Similar Documents

Publication Publication Date Title
ZA201302625B (en) Methods and compositions for treating hemophilia b
IL224390B (en) Compositions and methods for targeted thermal modulation
EP2655334A4 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASES
SG2014010706A (en) Method and composition
GB201016001D0 (en) Composition and method
IL238416A0 (en) Preparations and methods for the treatment of diseases caused by protein pathology
EP2603199A4 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF CARDIOVASCULAR DISEASES
PH12013500698A1 (en) Hair-mending compositions and associated methods
SG10201500048SA (en) Compositions and methods for treating glioblastoma gbm
ZA201303423B (en) Compositions and methods for treating myelofibrosis
ZA201404187B (en) Compositions and methods for treating dental conditions
SG10201500124VA (en) Methods and Compositions for Treating Cancer
SI2552484T1 (sl) Postopki in sestavki za znižanje ravni paratiroidnega hormona
EP2590674A4 (en) COMPOSITIONS AND METHODS FOR INFLUENZA TREATMENT
IL232648A0 (en) Preparations and methods for treating glioma
GB201003531D0 (en) Composition and method
GB201018650D0 (en) Methods and compositions
GB201008364D0 (en) Composition and method
ZA201305497B (en) Methods and compositions for preventing and treating osteoarthritis
GB201008843D0 (en) Method and composition
PL2606032T3 (pl) Kompozycja i sposoby terapii glejakaep
EP2537031A4 (en) COMPOSITIONS AND METHODS FOR THE TREATMENT OF CANCER
EP2632432A4 (en) COMPOSITION AND METHOD FOR TREATING LESIONS
ZA201308683B (en) Methods and compositions suitable for preventing and treating hyperleptinemia
IL220796A0 (en) Compositions and methods for treating gliblastoma gbm