CA2814143C - Methods and compositions for treating hemophilia b - Google Patents
Methods and compositions for treating hemophilia b Download PDFInfo
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- CA2814143C CA2814143C CA2814143A CA2814143A CA2814143C CA 2814143 C CA2814143 C CA 2814143C CA 2814143 A CA2814143 A CA 2814143A CA 2814143 A CA2814143 A CA 2814143A CA 2814143 C CA2814143 C CA 2814143C
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- C12N9/6424—Serine endopeptidases (3.4.21)
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| US10272163B2 (en) | 2012-12-07 | 2019-04-30 | The Regents Of The University Of California | Factor VIII mutation repair and tolerance induction |
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| 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 |
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| 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. |
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| 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 |
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| 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 |
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| 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 |
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| 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 |
| 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 |
| ZA201302625B (en) | 2014-05-28 |
| KR101930711B1 (ko) | 2018-12-19 |
| CN103270046B (zh) | 2016-05-11 |
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