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)
- C12N9/644—Coagulation factor IXa (3.4.21.22)
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
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| US20100256062A1 (en) | 2004-12-06 | 2010-10-07 | Howard Tommy E | Allelic Variants of Human Factor VIII |
| CN108117586A (zh) | 2010-08-10 | 2018-06-05 | 洛桑聚合联合学院 | 红细胞结合性治疗剂 |
| 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 |
| KR102061557B1 (ko) | 2011-09-21 | 2020-01-03 | 상가모 테라퓨틱스, 인코포레이티드 | 이식 유전자 발현의 조절을 위한 방법 및 조성물 |
| JP6373763B2 (ja) * | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| EP3816281A1 (en) | 2012-07-11 | 2021-05-05 | 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 |
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| 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 |
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| EP3011031B1 (en) | 2013-06-17 | 2020-09-30 | The Broad Institute Inc. | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
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| CA2932479A1 (en) | 2013-12-12 | 2015-06-18 | The Rockefeller University | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for hbv and viral diseases and 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 |
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| KR20250068794A (ko) | 2013-12-12 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
| 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 |
| CN106103705A (zh) | 2013-12-12 | 2016-11-09 | 布罗德研究所有限公司 | 核苷酸重复障碍中crispr‑cas系统的组合物和使用方法 |
| US10046056B2 (en) | 2014-02-21 | 2018-08-14 | École Polytechnique Fédérale De Lausanne (Epfl) | Glycotargeting therapeutics |
| AU2015233084B2 (en) | 2014-02-21 | 2020-12-17 | Anokion Sa | Glycotargeting therapeutics |
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| JP2024511277A (ja) | 2021-02-19 | 2024-03-13 | メビオン・メディカル・システムズ・インコーポレーテッド | 粒子線治療システムのためのガントリー |
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| US9175280B2 (en) | 2015-11-03 |
| US9629930B2 (en) | 2017-04-25 |
| CA2814143A1 (en) | 2012-04-19 |
| CN103270046A (zh) | 2013-08-28 |
| CN103270046B (zh) | 2016-05-11 |
| HK1185623A1 (zh) | 2014-02-21 |
| ZA201302625B (en) | 2014-05-28 |
| RU2013120986A (ru) | 2014-11-20 |
| JP2013544077A (ja) | 2013-12-12 |
| EP2627665B1 (en) | 2015-12-16 |
| BR112013008881B1 (pt) | 2021-12-07 |
| US20160022838A1 (en) | 2016-01-28 |
| EP2627665A4 (en) | 2014-02-26 |
| KR101930711B1 (ko) | 2018-12-19 |
| AU2011316575B2 (en) | 2015-10-29 |
| AU2011316575A1 (en) | 2013-05-02 |
| ES2562421T3 (es) | 2016-03-04 |
| EP2627665A1 (en) | 2013-08-21 |
| US20120128635A1 (en) | 2012-05-24 |
| KR20140042764A (ko) | 2014-04-07 |
| JP6018069B2 (ja) | 2016-11-02 |
| RU2608643C2 (ru) | 2017-01-23 |
| BR112013008881A2 (pt) | 2016-06-28 |
| WO2012051343A1 (en) | 2012-04-19 |
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