JP2013544077A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2013544077A5 JP2013544077A5 JP2013533982A JP2013533982A JP2013544077A5 JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5 JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013533982 A JP2013533982 A JP 2013533982A JP 2013544077 A5 JP2013544077 A5 JP 2013544077A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- cells
- seq
- protein
- composition according
- 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.)
- Granted
Links
- 210000003754 fetus Anatomy 0.000 claims description 2
- 210000001161 mammalian embryo Anatomy 0.000 claims description 2
- 210000004027 cell Anatomy 0.000 claims 12
- 239000008194 pharmaceutical composition Substances 0.000 claims 11
- 108090000623 proteins and genes Proteins 0.000 claims 10
- 102000004169 proteins and genes Human genes 0.000 claims 7
- 230000004568 DNA-binding Effects 0.000 claims 4
- 108010017070 Zinc Finger Nucleases Proteins 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 102100022641 Coagulation factor IX Human genes 0.000 claims 3
- 108010076282 Factor IX Proteins 0.000 claims 3
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims 3
- 239000003795 chemical substances by application Substances 0.000 claims 3
- 210000003494 hepatocyte Anatomy 0.000 claims 3
- 239000011701 zinc Substances 0.000 claims 3
- 229910052725 zinc Inorganic materials 0.000 claims 3
- 125000003275 alpha amino acid group Chemical group 0.000 claims 2
- 238000003776 cleavage reaction Methods 0.000 claims 2
- 229960004222 factor ix Drugs 0.000 claims 2
- 229930182830 galactose Natural products 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 230000007017 scission Effects 0.000 claims 2
- VSNHCAURESNICA-NJFSPNSNSA-N 1-oxidanylurea Chemical compound N[14C](=O)NO VSNHCAURESNICA-NJFSPNSNSA-N 0.000 claims 1
- 101710132601 Capsid protein Proteins 0.000 claims 1
- 101710094648 Coat protein Proteins 0.000 claims 1
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 claims 1
- 101710125418 Major capsid protein Proteins 0.000 claims 1
- 241001465754 Metazoa Species 0.000 claims 1
- 101710141454 Nucleoprotein Proteins 0.000 claims 1
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 1
- 101710083689 Probable capsid protein Proteins 0.000 claims 1
- 241000283984 Rodentia Species 0.000 claims 1
- 241001493546 Suina Species 0.000 claims 1
- YZCKVEUIGOORGS-NJFSPNSNSA-N Tritium Chemical compound [3H] YZCKVEUIGOORGS-NJFSPNSNSA-N 0.000 claims 1
- 101710185494 Zinc finger protein Proteins 0.000 claims 1
- 102100023597 Zinc finger protein 816 Human genes 0.000 claims 1
- NOFOAYPPHIUXJR-APNQCZIXSA-N aphidicolin Chemical compound C1[C@@]23[C@@]4(C)CC[C@@H](O)[C@@](C)(CO)[C@@H]4CC[C@H]3C[C@H]1[C@](CO)(O)CC2 NOFOAYPPHIUXJR-APNQCZIXSA-N 0.000 claims 1
- SEKZNWAQALMJNH-YZUCACDQSA-N aphidicolin Natural products C[C@]1(CO)CC[C@]23C[C@H]1C[C@@H]2CC[C@H]4[C@](C)(CO)[C@H](O)CC[C@]34C SEKZNWAQALMJNH-YZUCACDQSA-N 0.000 claims 1
- 210000000013 bile duct Anatomy 0.000 claims 1
- DQLATGHUWYMOKM-UHFFFAOYSA-L cisplatin Chemical compound N[Pt](N)(Cl)Cl DQLATGHUWYMOKM-UHFFFAOYSA-L 0.000 claims 1
- 229960004316 cisplatin Drugs 0.000 claims 1
- 210000001671 embryonic stem cell Anatomy 0.000 claims 1
- VJJPUSNTGOMMGY-MRVIYFEKSA-N etoposide Chemical compound COC1=C(O)C(OC)=CC([C@@H]2C3=CC=4OCOC=4C=C3[C@@H](O[C@H]3[C@@H]([C@@H](O)[C@@H]4O[C@H](C)OC[C@H]4O3)O)[C@@H]3[C@@H]2C(OC3)=O)=C1 VJJPUSNTGOMMGY-MRVIYFEKSA-N 0.000 claims 1
- 229960005420 etoposide Drugs 0.000 claims 1
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 210000002767 hepatic artery Anatomy 0.000 claims 1
- 210000003897 hepatic stem cell Anatomy 0.000 claims 1
- 210000005260 human cell Anatomy 0.000 claims 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 239000002773 nucleotide Substances 0.000 claims 1
- 125000003729 nucleotide group Chemical group 0.000 claims 1
- 238000012753 partial hepatectomy Methods 0.000 claims 1
- OIGNJSKKLXVSLS-VWUMJDOOSA-N prednisolone Chemical compound O=C1C=C[C@]2(C)[C@H]3[C@@H](O)C[C@](C)([C@@](CC4)(O)C(=O)CO)[C@@H]4[C@@H]3CCC2=C1 OIGNJSKKLXVSLS-VWUMJDOOSA-N 0.000 claims 1
- 229960005205 prednisolone Drugs 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 229910052722 tritium Inorganic materials 0.000 claims 1
- 238000009281 ultraviolet germicidal irradiation Methods 0.000 claims 1
- 241000701161 unidentified adenovirus Species 0.000 claims 1
- 150000003751 zinc Chemical class 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US39233310P | 2010-10-12 | 2010-10-12 | |
| US61/392,333 | 2010-10-12 | ||
| PCT/US2011/056020 WO2012051343A1 (en) | 2010-10-12 | 2011-10-12 | Methods and compositions for treating hemophilia b |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013544077A JP2013544077A (ja) | 2013-12-12 |
| JP2013544077A5 true JP2013544077A5 (enExample) | 2016-01-07 |
| JP6018069B2 JP6018069B2 (ja) | 2016-11-02 |
Family
ID=45938707
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013533982A Active JP6018069B2 (ja) | 2010-10-12 | 2011-10-12 | 血友病ｂを治療する方法及び組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (2) | US9175280B2 (enExample) |
| EP (1) | EP2627665B1 (enExample) |
| JP (1) | JP6018069B2 (enExample) |
| KR (1) | KR101930711B1 (enExample) |
| CN (1) | CN103270046B (enExample) |
| AU (1) | AU2011316575B2 (enExample) |
| BR (1) | BR112013008881B1 (enExample) |
| CA (1) | CA2814143C (enExample) |
| ES (1) | ES2562421T3 (enExample) |
| RU (1) | RU2608643C2 (enExample) |
| WO (1) | WO2012051343A1 (enExample) |
| ZA (1) | ZA201302625B (enExample) |
Families Citing this family (64)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006063031A2 (en) | 2004-12-06 | 2006-06-15 | Haplomics | Allelic variants of human factor viii |
| AU2011289579B2 (en) | 2010-08-10 | 2016-11-17 | Ecole Polytechnique Federale De Lausanne | 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 |
| EP2758529B1 (en) | 2011-09-21 | 2019-03-20 | 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 |
| US10648001B2 (en) | 2012-07-11 | 2020-05-12 | Sangamo Therapeutics, Inc. | Method of treating mucopolysaccharidosis type I or II |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| CA2892448A1 (en) * | 2012-12-05 | 2014-06-12 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of metabolic disorders |
| 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 |
| SG10201707569YA (en) | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| 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 |
| EP3011033B1 (en) | 2013-06-17 | 2020-02-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| ES2777217T3 (es) | 2013-06-17 | 2020-08-04 | Broad Inst Inc | Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias |
| KR20250012194A (ko) | 2013-06-17 | 2025-01-23 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
| JP6738728B2 (ja) | 2013-06-17 | 2020-08-19 | ザ・ブロード・インスティテュート・インコーポレイテッド | 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用 |
| WO2014204725A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| EP3058072B1 (en) * | 2013-10-17 | 2021-05-19 | Sangamo Therapeutics, Inc. | Delivery methods and compositions for nuclease-mediated genome engineering |
| CA3229275A1 (en) | 2013-12-09 | 2015-06-18 | Sangamo Biosciences, Inc. | Methods and compositions for genome engineering |
| WO2015089473A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation |
| JP6793547B2 (ja) | 2013-12-12 | 2020-12-02 | ザ・ブロード・インスティテュート・インコーポレイテッド | 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物 |
| CN105899658B (zh) | 2013-12-12 | 2020-02-18 | 布罗德研究所有限公司 | 针对hbv和病毒性疾病以及障碍的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 |
| KR20160097338A (ko) | 2013-12-12 | 2016-08-17 | 더 브로드 인스티튜트, 인코퍼레이티드 | 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도 |
| CN111206032B (zh) | 2013-12-12 | 2024-07-19 | 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 |
| US10046056B2 (en) | 2014-02-21 | 2018-08-14 | École Polytechnique Fédérale De Lausanne (Epfl) | Glycotargeting therapeutics |
| US10953101B2 (en) | 2014-02-21 | 2021-03-23 | École Polytechnique Fédérale De Lausanne (Epfl) | Glycotargeting therapeutics |
| CA2946064C (en) | 2014-02-21 | 2023-09-26 | Ecole Polytechnique Federale De Lausanne (Epfl) | Glycotargeting therapeutics |
| US10946079B2 (en) | 2014-02-21 | 2021-03-16 | Ecole Polytechnique Federale De Lausanne | Glycotargeting therapeutics |
| EP3110454B1 (en) | 2014-02-24 | 2020-11-18 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| EP3929279A1 (en) | 2014-03-18 | 2021-12-29 | Sangamo Therapeutics, Inc. | Methods and compositions for regulation of zinc finger protein expression |
| US10612041B2 (en) | 2014-03-21 | 2020-04-07 | The Board Of Trustees Of The Leland Stanford Junior University | Genome editing without nucleases |
| EP3155098A4 (en) * | 2014-06-11 | 2018-01-03 | Howard, Tom E. | FACTOR VIII MUTATION REPAIR AND TOLERANCE INDUCTION AND RELATED CDNAs, COMPOSITIONS, METHODS AND SYSTEMS |
| WO2016049230A1 (en) * | 2014-09-24 | 2016-03-31 | City Of Hope | Adeno-associated virus vector variants for high efficiency genome editing and methods thereof |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| EP3985115A1 (en) | 2014-12-12 | 2022-04-20 | 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 |
| WO2016106244A1 (en) | 2014-12-24 | 2016-06-30 | The Broad Institute Inc. | Crispr having or associated with destabilization domains |
| US11185596B2 (en) | 2015-01-06 | 2021-11-30 | 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 |
| 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 |
| EP3929287A3 (en) | 2015-06-18 | 2022-04-13 | The Broad Institute, Inc. | Crispr enzyme mutations reducing off-target effects |
| US10143760B2 (en) | 2015-10-28 | 2018-12-04 | Sangamo Therapeutics, Inc. | Liver-specific constructs, factor VIII expression cassettes and methods of use thereof |
| EP3380622A4 (en) | 2015-11-23 | 2019-08-07 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR MODIFYING 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 |
| JP7256739B2 (ja) | 2016-09-07 | 2023-04-12 | サンガモ セラピューティクス， インコーポレイテッド | 肝臓遺伝子のモジュレーション |
| 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 |
| CA3061614A1 (en) | 2017-04-28 | 2018-11-01 | The Regents Of The University Of Colorado, A Body Corporate | Methods of treating rheumatoid arthritis using rna-guided genome editing of hla gene |
| US11046969B2 (en) * | 2017-05-24 | 2021-06-29 | Epiplanta Biotech Ltd. | Transgenic plant and the method for producing the same |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| US11667934B2 (en) * | 2017-06-15 | 2023-06-06 | Toolgen Incorporated | Platform for expressing protein of interest in liver |
| EP3638296A1 (en) | 2017-06-16 | 2020-04-22 | The University Of Chicago | Compositions and methods for inducing immune tolerance |
| EP3716767A4 (en) * | 2017-10-24 | 2021-11-24 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR TREATMENT OF RARE DISEASES |
| EP3790577A4 (en) | 2018-05-09 | 2022-04-27 | The University of Chicago | COMPOSITIONS AND METHODS RELATED TO IMMUNTOLERANCE |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| IL326835A (en) | 2018-10-16 | 2026-04-01 | Blueallele Llc | Methods for targeted insertion of dna genes |
| JP7578590B2 (ja) | 2018-10-18 | 2024-11-06 | インテリア セラピューティクス，インコーポレーテッド | 第ｉｘ因子を発現するための組成物及び方法 |
| 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 |
| WO2020197242A1 (ko) * | 2019-03-26 | 2020-10-01 | 주식회사 툴젠 | 혈우병b 질환 모델 랫드 |
| US12245355B2 (en) | 2021-02-19 | 2025-03-04 | Mevion Medical Systems, Inc. | Gantry for a particle therapy system |
| AU2025209576A1 (en) | 2024-01-16 | 2026-06-11 | Novo Nordisk A/S | Albumin-targeted endonucleases, compositions, and methods of use |
Family Cites Families (66)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US789538A (en) | 1904-11-11 | 1905-05-09 | Colin E Ham | Dumb-bell. |
| 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 |
| US6242568B1 (en) | 1994-01-18 | 2001-06-05 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| JP4012243B2 (ja) | 1994-01-18 | 2007-11-21 | ザ スクリップス リサーチ インスティチュート | 亜鉛フィンガータンパク質誘導体およびそのための方法 |
| US6140466A (en) | 1994-01-18 | 2000-10-31 | The Scripps Research Institute | Zinc finger protein derivatives and methods therefor |
| CA2196419C (en) | 1994-08-20 | 2007-08-21 | Yen Choo | Improvements in or relating to 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 |
| EP0933997A4 (en) | 1996-09-06 | 2000-08-16 | Chiron Corp | METHODS AND COMPOSITIONS FOR LIVER-SPECIFIC ADMINISTRATION OF THERAPEUTIC MOLECULES USING RECOMBINANT 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 |
| GB9703369D0 (en) | 1997-02-18 | 1997-04-09 | Lindqvist Bjorn H | Process |
| GB2338237B (en) | 1997-02-18 | 2001-02-28 | Actinova Ltd | In vitro peptide or protein expression library |
| ES2355588T3 (es) * | 1997-03-14 | 2011-03-29 | The Children's Hospital Of Philadelphia | Composiciones para uso en terapia génica para el tratamiento de hemofilia. |
| 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 |
| DE69942334D1 (de) | 1998-03-02 | 2010-06-17 | Massachusetts Inst Technology | Poly-zinkfinger-proteine mit verbesserten linkern |
| US6140081A (en) | 1998-10-16 | 2000-10-31 | The Scripps Research Institute | Zinc finger binding domains for GNN |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| 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 |
| US6503717B2 (en) | 1999-12-06 | 2003-01-07 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| US7030215B2 (en) | 1999-03-24 | 2006-04-18 | Sangamo Biosciences, Inc. | Position dependent recognition of GNN nucleotide triplets by zinc fingers |
| US6794136B1 (en) | 2000-11-20 | 2004-09-21 | Sangamo Biosciences, Inc. | Iterative optimization in the design of binding proteins |
| DE10005183A1 (de) | 2000-02-05 | 2001-08-09 | Bosch Gmbh Robert | Gleichrichteranordnung |
| AU2001250774B2 (en) | 2000-02-08 | 2005-01-27 | Sangamo Biosciences, Inc. | Cells expressing zinc finger proteins for drug discovery |
| US20020061512A1 (en) | 2000-02-18 | 2002-05-23 | Kim Jin-Soo | Zinc finger domains and methods of identifying same |
| AU2001263155A1 (en) | 2000-05-16 | 2001-11-26 | 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 |
| US7067317B2 (en) | 2000-12-07 | 2006-06-27 | Sangamo Biosciences, Inc. | Regulation of angiogenesis with zinc finger proteins |
| CA2437015A1 (en) * | 2001-02-05 | 2002-08-15 | Novo Nordisk Health Care Ag | Combined use of factor vii polypeptides and factor ix polypeptides |
| GB0108491D0 (en) | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| US20040224385A1 (en) | 2001-08-20 | 2004-11-11 | Barbas Carlos F | Zinc finger binding domains for cnn |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| 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 |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| EP2927318B1 (en) | 2003-08-08 | 2020-05-20 | 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 |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| ATE418346T1 (de) | 2004-04-08 | 2009-01-15 | Sangamo Biosciences Inc | Zusammensetzungen zur behandlung von neuropathischen und neurodegenerativen erkrankungen |
| US7253273B2 (en) * | 2004-04-08 | 2007-08-07 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| KR20070060115A (ko) | 2004-09-16 | 2007-06-12 | 상가모 바이오사이언스 인코포레이티드 | 단백질 생산을 위한 조성물 및 방법 |
| CA2599004C (en) * | 2005-02-28 | 2015-05-26 | Sangamo Biosciences, Inc. | Anti-angiogenic methods and compositions |
| WO2006121866A2 (en) | 2005-05-05 | 2006-11-16 | The Arizona Board Of Regents On Behalf Of The University Of Arizona | Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences |
| EP1913149A4 (en) | 2005-07-26 | 2009-08-05 | Sangamo Biosciences Inc | TARGETED INTEGRATION AND EXPRESSION OF EXOGENOUS NUCLEIC ACID SEQUENCES |
| US7951925B2 (en) | 2006-05-25 | 2011-05-31 | Sangamo Biosciences, Inc. | Methods and compositions for gene inactivation |
| EP2027262B1 (en) | 2006-05-25 | 2010-03-31 | Sangamo Biosciences Inc. | Variant foki cleavage half-domains |
| JP2010500029A (ja) | 2006-08-11 | 2010-01-07 | ダウ アグロサイエンシィズ エルエルシー | ジンクフィンガーヌクレアーゼ媒介相同組換え |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| AU2008275649B2 (en) * | 2007-07-12 | 2013-09-05 | Sangamo Therapeutics, Inc. | Methods and compositions for inactivating alpha 1,6 fucosyltransferase (FUT 8) gene expression |
| JP5763341B2 (ja) | 2007-09-27 | 2015-08-12 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 生物学的活性のあるヌクレアーゼの迅速なインビボ同定法 |
| CA2720903C (en) | 2008-04-14 | 2019-01-15 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| EP3156494B8 (en) | 2008-12-04 | 2018-09-19 | Sangamo Therapeutics, Inc. | Genome editing in rats using zinc-finger nucleases |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| AU2010235161B2 (en) | 2009-04-09 | 2015-01-22 | Sangamo Therapeutics, Inc. | Targeted integration into stem cells |
| US20120192298A1 (en) * | 2009-07-24 | 2012-07-26 | Sigma Aldrich Co. Llc | Method for genome editing |
| JP6137596B2 (ja) | 2010-02-08 | 2017-05-31 | サンガモ セラピューティクス， インコーポレイテッド | 遺伝子操作された切断ハーフドメイン |
| AU2011256838B2 (en) | 2010-05-17 | 2014-10-09 | Sangamo Therapeutics, Inc. | Novel DNA-binding proteins and uses thereof |
-
2011
- 2011-10-12 EP EP11833365.7A patent/EP2627665B1/en active Active
- 2011-10-12 ES ES11833365.7T patent/ES2562421T3/es active Active
- 2011-10-12 AU AU2011316575A patent/AU2011316575B2/en active Active
- 2011-10-12 JP JP2013533982A patent/JP6018069B2/ja active Active
- 2011-10-12 WO PCT/US2011/056020 patent/WO2012051343A1/en not_active Ceased
- 2011-10-12 RU RU2013120986A patent/RU2608643C2/ru active
- 2011-10-12 BR BR112013008881-8A patent/BR112013008881B1/pt active IP Right Grant
- 2011-10-12 CN CN201180059911.6A patent/CN103270046B/zh active Active
- 2011-10-12 CA CA2814143A patent/CA2814143C/en active Active
- 2011-10-12 US US13/272,084 patent/US9175280B2/en active Active
- 2011-10-12 KR KR1020137012086A patent/KR101930711B1/ko active Active
-
2013
- 2013-04-11 ZA ZA2013/02625A patent/ZA201302625B/en unknown
-
2015
- 2015-10-08 US US14/878,703 patent/US9629930B2/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2013120986A (ru) | Способы и композиции для лечения гемофилии в | |
| He et al. | Hepatitis D virus infection of mice expressing human sodium taurocholate co-transporting polypeptide | |
| JP2018520997A5 (enExample) | ||
| JP2017500061A5 (enExample) | ||
| JP2017504354A5 (enExample) | ||
| CL2019002280A1 (es) | Vectores virales, adenovirales y composiciones a base de una secuencia de ornitina transcarbamilasa humana (hotch) modificada genéticamente y su uso en el tratamiento de deficiencia de la otc. (divisional solicitud 201602235) | |
| Li et al. | IFN-lambda 3 mediates antiviral protection against porcine epidemic diarrhea virus by inducing a distinct antiviral transcript profile in porcine intestinal epithelia | |
| ES3038656T3 (en) | Methods of treating mitochondrial disorders | |
| JP2017511135A5 (enExample) | ||
| JP6212659B2 (ja) | ヒト化ジペプチジルペプチダーゼｉｖ（ｄｐｐ４）動物 | |
| JP2018505219A (ja) | エピゲノム編集のための組成物および方法 | |
| Keng et al. | Modeling hepatitis B virus X–induced hepatocellular carcinoma in mice with the sleeping beauty transposon system | |
| JP2008545406A5 (enExample) | ||
| JP2019517803A5 (enExample) | ||
| JP2016520320A5 (enExample) | ||
| HRP20201443T1 (hr) | Postupci i pripravci za liječenje genetskog stanja | |
| JP2013525305A5 (enExample) | ||
| JP2016513115A5 (enExample) | ||
| Calton et al. | Oncolytic viruses for multiple myeloma therapy | |
| Dong et al. | Receptors and host factors: key players in human metapneumovirus infection | |
| Repellin et al. | Lymphoid hyperplasia and lymphoma in transgenic mice expressing the small non-coding RNA, EBER1 of Epstein-Barr virus | |
| JP2019517262A5 (enExample) | ||
| Kolev et al. | A FoxL1-CreERT-2A-tdTomato mouse labels subepithelial telocytes | |
| Burnet et al. | Hemin treatment drives viral reactivation and plasma cell differentiation of EBV latently infected B cells | |
| Geng et al. | Construction of an IFNAR1 knockout MDBK cell line using CRISPR/Cas9 and its effect on bovine virus replication |