TW202039854A - 治療威爾遜氏病的組合物和方法 - Google Patents
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- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2750/00011—Details
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| BR112019013245A2 (pt) | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| DK3906066T5 (da) | 2019-01-04 | 2024-08-05 | Ultragenyx Pharmaceutical Inc | Geneterapikonstruktioner til behandling af wilsons sygdom |
| US20240307553A1 (en) * | 2021-04-01 | 2024-09-19 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| CN115216481A (zh) * | 2021-04-21 | 2022-10-21 | 四川至善唯新生物科技有限公司 | 一种表达量提高的截短型atp7b基因及其应用 |
| CN113699211A (zh) * | 2021-07-20 | 2021-11-26 | 深圳市儿童医院 | 一种用于儿童威尔森氏症快速筛查的参数组合和试剂盒 |
| WO2023038909A1 (en) * | 2021-09-07 | 2023-03-16 | Alexion Pharmaceuticals, Inc. | Methods of treating copper metabolism-associated diseases or disorders |
| TW202323528A (zh) * | 2021-10-18 | 2023-06-16 | 美商邏輯生物療法公司 | 用於治療威爾森氏病之基因療法 |
| JP2025533994A (ja) * | 2022-10-11 | 2025-10-09 | メイラグティーエックス ユーケー アイアイ リミティド | Ａｔｐ７ｂ遺伝子療法 |
| EP4677077A1 (en) * | 2023-03-06 | 2026-01-14 | Lingyi Biotech Co., Ltd. | A truncated atp7b and the use thereof |
| WO2024193638A1 (en) * | 2023-03-22 | 2024-09-26 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of wilson disease |
| WO2025002226A1 (zh) * | 2023-06-28 | 2025-01-02 | 迈威(上海)生物科技股份有限公司 | 编码atp7b的核酸、腺相关病毒载体及其在制备防治肝豆状核变性的药物中的应用 |
| CN120882865A (zh) * | 2023-07-28 | 2025-10-31 | 凌意(杭州)生物科技有限公司 | 用于治疗威尔逊病的多核苷酸 |
| CN117757797B (zh) * | 2023-09-25 | 2024-08-16 | 北京圭特细胞科技有限公司 | 一种编码人III型胶原蛋白α链的核酸及其应用 |
| WO2025110984A1 (en) * | 2023-11-21 | 2025-05-30 | Hydrogene Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| CN117947035A (zh) * | 2024-03-25 | 2024-04-30 | 上海凌医生物科技有限公司 | 一种增强基因表达的启动子 |
| CN121265551A (zh) * | 2024-07-02 | 2026-01-06 | 苏州星核迪赛生物技术有限公司 | 一种靶向肝实质细胞的核酸递送载体及其应用 |
| CN121319202B (zh) * | 2025-12-15 | 2026-04-07 | 凌意(杭州)生物科技有限公司 | Atp7b抗体及其应用 |
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| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
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| US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
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| US8048999B2 (en) | 2005-12-13 | 2011-11-01 | Kyoto University | Nuclear reprogramming factor |
| JP5813321B2 (ja) | 2007-03-23 | 2015-11-17 | ウィスコンシン アラムニ リサーチ ファンデーション | 体細胞の再プログラム化 |
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| KR20180000341A (ko) | 2009-06-16 | 2018-01-02 | 젠자임 코포레이션 | 재조합 ａａｖ 벡터에 대한 개선된 정제 방법 |
| WO2012097826A2 (en) | 2011-01-21 | 2012-07-26 | Aarhus Universitet | Crystal structure of a type ib p-type atpase |
| EP3105334B1 (en) * | 2014-02-11 | 2019-07-03 | Friedrich Miescher Institute for Biomedical Research | Müller cell-specific promoter |
| US11118238B2 (en) * | 2014-12-17 | 2021-09-14 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions |
| US11147887B2 (en) * | 2014-12-17 | 2021-10-19 | Fundación Para La Investigación Mèdica Aplicada | Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease |
| EP3240558A1 (en) * | 2014-12-30 | 2017-11-08 | CureVac AG | Artificial nucleic acid molecules |
| US11007280B2 (en) * | 2015-03-17 | 2021-05-18 | Vrije Universiteit Brussel | Optimized liver-specific expression systems for FVIII and FIX |
| GB201522416D0 (en) | 2015-12-18 | 2016-02-03 | Ucl Business Plc | Wilson's disease gene therapy |
| BR112019013245A2 (pt) * | 2016-12-30 | 2020-02-11 | The Trustees Of The University Of Pennsylvania | Terapia gênica para tratamento da doença de wilson |
| WO2018204764A1 (en) * | 2017-05-05 | 2018-11-08 | Camp4 Therapeutics Corporation | Identification and targeted modulation of gene signaling networks |
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