JP2013544077A5 - - Google Patents
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- 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
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- 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 |
|---|---|---|---|---|
| US20100256062A1 (en) | 2004-12-06 | 2010-10-07 | Howard Tommy E | Allelic Variants of Human Factor VIII |
| CA2807942C (en) | 2010-08-10 | 2021-07-27 | 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 |
| CA2848417C (en) | 2011-09-21 | 2023-05-02 | Sangamo Biosciences, Inc. | Methods and compositions for regulation of transgene expression |
| WO2013123503A1 (en) * | 2012-02-17 | 2013-08-22 | The Children's Hospital Of Philadelphia | Aav vector compositions and methods for gene transfer to cells, organs and tissues |
| 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 |
| WO2014089541A2 (en) | 2012-12-07 | 2014-06-12 | Haplomics, Inc. | Factor viii mutation repair and tolerance induction |
| 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 |
| WO2014093622A2 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| WO2014093701A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof |
| EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | 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 |
| CA2915837A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
| EP3011033B1 (en) | 2013-06-17 | 2020-02-19 | The Broad Institute, Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
| EP3597755A1 (en) | 2013-06-17 | 2020-01-22 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
| EP3674411A1 (en) | 2013-06-17 | 2020-07-01 | The Broad Institute, Inc. | Delivery, engineering and optimization of tandem guide systems, methods and compositions for sequence manipulation |
| KR20240172759A (ko) | 2013-06-17 | 2024-12-10 | 더 브로드 인스티튜트, 인코퍼레이티드 | 간의 표적화 및 치료를 위한 ＣＲＩＳＰＲＣａｓ 시스템, 벡터 및 조성물의 전달 및 용도 |
| CN105899665B (zh) * | 2013-10-17 | 2019-10-22 | 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 |
| EP3080274B1 (en) | 2013-12-09 | 2020-06-03 | Sangamo Therapeutics, Inc. | Methods and compositions for genome engineering |
| 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 |
| WO2015089364A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Crystal structure of a crispr-cas system, and uses thereof |
| WO2015089354A1 (en) | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Compositions and methods of use of crispr-cas systems in nucleotide repeat disorders |
| EP3080271B1 (en) | 2013-12-12 | 2020-02-12 | The Broad Institute, Inc. | Systems, methods and compositions for sequence manipulation with optimized functional crispr-cas systems |
| 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 |
| EP3653229A1 (en) | 2013-12-12 | 2020-05-20 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for genome editing |
| US10953101B2 (en) | 2014-02-21 | 2021-03-23 | École Polytechnique Fédérale De Lausanne (Epfl) | Glycotargeting therapeutics |
| US10946079B2 (en) | 2014-02-21 | 2021-03-16 | Ecole Polytechnique Federale De Lausanne | Glycotargeting therapeutics |
| CA2946064C (en) | 2014-02-21 | 2023-09-26 | Ecole Polytechnique Federale De Lausanne (Epfl) | Glycotargeting therapeutics |
| US10046056B2 (en) | 2014-02-21 | 2018-08-14 | École Polytechnique Fédérale De Lausanne (Epfl) | Glycotargeting therapeutics |
| AU2015218576B2 (en) | 2014-02-24 | 2020-02-27 | Sangamo Therapeutics, Inc. | Methods and compositions for nuclease-mediated targeted integration |
| ES2879373T3 (es) | 2014-03-18 | 2021-11-22 | Sangamo Therapeutics Inc | Métodos y composiciones para la regulación de la expresión de proteínas de dedo de zinc |
| CN106460009A (zh) | 2014-03-21 | 2017-02-22 | 小利兰·斯坦福大学托管委员会 | 无核酸酶的基因组编辑 |
| CA2951882A1 (en) * | 2014-06-11 | 2015-12-17 | Tom E. HOWARD | Factor viii mutation repair and tolerance induction and related cdnas, compositions, methods and systems |
| KR20230067694A (ko) * | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| 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 |
| CA2970370A1 (en) | 2014-12-24 | 2016-06-30 | Massachusetts Institute Of Technology | Crispr having or associated with destabilization domains |
| CN108064280B (zh) | 2015-01-06 | 2021-10-29 | 延世大学校产学协力团 | 利用以凝血因子viii基因为靶的核酸内切酶的a型血友病治疗用组合物 |
| 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 |
| MX392008B (es) | 2015-06-18 | 2025-03-21 | Broad Inst Inc | Mutaciones de la enzima crispr que reducen los efectos fuera del blanco |
| KR102766526B1 (ko) | 2015-10-28 | 2025-02-13 | 상가모 테라퓨틱스, 인코포레이티드 | 간-특이적 작제물인, 인자 viii 발현 카세트 및 이의 사용 방법 |
| JP6976249B2 (ja) | 2015-11-23 | 2021-12-08 | サンガモ セラピューティクス， インコーポレイテッド | 免疫を工学操作するための方法および組成物 |
| 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 |
| WO2018049009A2 (en) | 2016-09-07 | 2018-03-15 | Sangamo Therapeutics, Inc. | Modulation of liver genes |
| 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 |
| DK3615674T3 (da) | 2017-04-28 | 2024-06-17 | Univ Colorado Regents | 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 |
| KR102738651B1 (ko) * | 2017-06-15 | 2024-12-05 | 주식회사 툴젠 | 간에서 목적하는 단백질 발현하기 위한 플랫폼 |
| US11253579B2 (en) | 2017-06-16 | 2022-02-22 | The University Of Chicago | Compositions and methods for inducing immune tolerance |
| CA3079727A1 (en) * | 2017-10-24 | 2019-05-02 | Sangamo Therapeutics, Inc. | Methods and compositions for the treatment of rare diseases |
| AU2019265734B2 (en) | 2018-05-09 | 2026-03-19 | Anokion Sa | Compositions and methods concerning immune tolerance |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | 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 |
| BR112021007229A2 (pt) | 2018-10-16 | 2021-08-10 | Blueallele, Llc | métodos para inserção dirigida de dna em genes |
| 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 |
| WO2020197242A1 (ko) * | 2019-03-26 | 2020-10-01 | 주식회사 툴젠 | 혈우병b 질환 모델 랫드 |
| CN116917009A (zh) | 2021-02-19 | 2023-10-20 | 美国迈胜医疗系统有限公司 | 用于粒子治疗系统的机架 |
| WO2025153530A1 (en) | 2024-01-16 | 2025-07-24 | 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. |
| 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 |
| US5436150A (en) | 1992-04-03 | 1995-07-25 | The Johns Hopkins University | Functional domains in flavobacterium okeanokoities (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 |
| CA2181548C (en) | 1994-01-18 | 2009-11-03 | Carlos F. Barbas, Iii | Zinc finger protein derivatives and methods therefor |
| DE69535829D1 (de) | 1994-08-20 | 2008-10-16 | Gendaq Ltd | Verbesserung in bezug auf bindungsproteine bei der erkennung von 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 |
| AU5588298A (en) | 1996-12-02 | 1998-06-29 | 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 |
| ATE486614T1 (de) | 1997-03-14 | 2010-11-15 | Philadelphia Children Hospital | Zusammensetzungen zum einsatz in einer gentherapie zur behandlung von hämophilie |
| GB9710807D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| GB9710809D0 (en) | 1997-05-23 | 1997-07-23 | Medical Res Council | Nucleic acid binding proteins |
| US6410248B1 (en) | 1998-01-30 | 2002-06-25 | Massachusetts Institute Of Technology | General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites |
| ES2341926T3 (es) | 1998-03-02 | 2010-06-29 | Massachusetts Institute Of Technology | Poliproteinas con dedos de cinc que tienen enlazadores mejorados. |
| 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 |
| US7070934B2 (en) | 1999-01-12 | 2006-07-04 | Sangamo Biosciences, Inc. | Ligand-controlled regulation of endogenous gene expression |
| 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 |
| 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 |
| 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 |
| CA2394850C (en) | 1999-12-06 | 2012-02-07 | 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 |
| EP1254369B1 (en) | 2000-02-08 | 2010-10-06 | 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 |
| 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 |
| CA2436807A1 (en) * | 2001-02-05 | 2002-08-15 | Novo Nordisk Health Care Ag | Combined use of factor vii polypeptides and factor viii polypeptides |
| GB0108491D0 (en) * | 2001-04-04 | 2001-05-23 | Gendaq Ltd | Engineering zinc fingers |
| WO2003016496A2 (en) | 2001-08-20 | 2003-02-27 | The Scripps Research Institute | Zinc finger binding domains for cnn |
| US7262054B2 (en) | 2002-01-22 | 2007-08-28 | Sangamo Biosciences, Inc. | Zinc finger proteins for DNA binding and gene regulation in plants |
| ATE347593T1 (de) | 2002-01-23 | 2006-12-15 | Univ Utah Res Found | Zielgerichtete chromosomale mutagenese mit zinkfingernukleasen |
| EP1504092B2 (en) | 2002-03-21 | 2014-06-25 | Sangamo BioSciences, Inc. | Methods and compositions for using zinc finger endonucleases to enhance homologous recombination |
| US7361635B2 (en) | 2002-08-29 | 2008-04-22 | Sangamo Biosciences, Inc. | Simultaneous modulation of multiple genes |
| WO2004037977A2 (en) | 2002-09-05 | 2004-05-06 | California Institute Of Thechnology | Use of chimeric nucleases to stimulate gene targeting |
| ES2808687T3 (es) | 2003-08-08 | 2021-03-01 | Sangamo Therapeutics Inc | Métodos y composiciones para escisión dirigida y recombinación |
| US8409861B2 (en) | 2003-08-08 | 2013-04-02 | Sangamo Biosciences, Inc. | Targeted deletion of cellular DNA sequences |
| US7888121B2 (en) | 2003-08-08 | 2011-02-15 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| US7972854B2 (en) | 2004-02-05 | 2011-07-05 | Sangamo Biosciences, Inc. | Methods and compositions for targeted cleavage and recombination |
| JP4903689B2 (ja) | 2004-04-08 | 2012-03-28 | サンガモ バイオサイエンシズ インコーポレイテッド | 神経障害および神経変性症状を治療するための方法と組成物 |
| US7253273B2 (en) * | 2004-04-08 | 2007-08-07 | Sangamo Biosciences, Inc. | Treatment of neuropathic pain with zinc finger proteins |
| AU2005287278B2 (en) | 2004-09-16 | 2011-08-04 | Sangamo Biosciences, Inc. | Compositions and methods for protein production |
| US7358085B2 (en) * | 2005-02-28 | 2008-04-15 | 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 |
| US20090263900A1 (en) | 2008-04-14 | 2009-10-22 | Sangamo Biosciences, Inc. | Linear donor constructs for targeted integration |
| DE602007005634D1 (de) | 2006-05-25 | 2010-05-12 | Sangamo Biosciences Inc | Variante foki-spaltungshälften-domänen |
| EP2019839B1 (en) | 2006-05-25 | 2011-12-07 | Sangamo BioSciences, Inc. | Methods and compositions for gene inactivation |
| CA2660485C (en) | 2006-08-11 | 2016-04-12 | Sangamo Biosciences, Inc. | Zinc finger nuclease-mediated homologous recombination |
| US8110379B2 (en) | 2007-04-26 | 2012-02-07 | Sangamo Biosciences, Inc. | Targeted integration into the PPP1R12C locus |
| CN101883850B (zh) * | 2007-07-12 | 2014-11-12 | 桑格摩生物科学股份有限公司 | 用于失活α-1,6岩藻糖基转移酶(FUT8)基因表达的方法和组合物 |
| CN101883863B (zh) | 2007-09-27 | 2018-01-23 | 桑格摩生物科学股份有限公司 | 生物活性核酸酶的快速体内鉴定 |
| JP5681114B2 (ja) | 2008-12-04 | 2015-03-04 | サンガモ バイオサイエンシーズ， インコーポレイテッド | 亜鉛フィンガーヌクレアーゼを使用したラットのゲノム編集 |
| EP2206723A1 (en) | 2009-01-12 | 2010-07-14 | Bonas, Ulla | Modular DNA-binding domains |
| JP6215533B2 (ja) | 2009-04-09 | 2017-10-18 | サンガモ セラピューティクス， インコーポレイテッド | 幹細胞への標的組込み |
| US20120192298A1 (en) * | 2009-07-24 | 2012-07-26 | Sigma Aldrich Co. Llc | Method for genome editing |
| DK2534173T3 (da) | 2010-02-08 | 2019-10-14 | Sangamo Therapeutics Inc | Manipulerede spaltnings-halvdomæner |
| JP6208580B2 (ja) | 2010-05-17 | 2017-10-04 | サンガモ セラピューティクス， インコーポレイテッド | 新規のｄｎａ結合タンパク質及びその使用 |
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