JP7528066B2 - 操作されたプロモータを有するフラタキシン発現コンストラクトおよびその使用方法 - Google Patents
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| CN106232618A (zh) | 2013-10-11 | 2016-12-14 | 马萨诸塞眼科耳科诊所 | 预测祖先病毒序列的方法及其用途 |
| CN108603235A (zh) | 2015-07-30 | 2018-09-28 | 马萨诸塞眼科耳科诊所 | 祖先病毒序列及其用途 |
| EP3808849A1 (en) | 2017-08-03 | 2021-04-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
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| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| US20220308072A1 (en) | 2019-08-12 | 2022-09-29 | Voyager Therapeutics, Inc. | High-sensitivity immunoassay for the detection of frataxin in biofluids |
| EP3901262A1 (en) * | 2020-04-20 | 2021-10-27 | Universität Regensburg | Compositions for use in treating autosomal dominant best1-related retinopathies |
| TWI900553B (zh) * | 2020-05-13 | 2025-10-11 | 美商航海家醫療公司 | Aav蛋白殼之趨性重定向 |
| CN111876432B (zh) * | 2020-07-29 | 2022-10-28 | 舒泰神(北京)生物制药股份有限公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| AU2021329527A1 (en) * | 2020-08-21 | 2023-03-23 | Capsida, Inc. | Adeno-associated virus compositions having preferred expression levels |
| US11926842B2 (en) | 2021-02-02 | 2024-03-12 | University Of Massachusetts | Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors |
| KR20240095539A (ko) | 2021-10-08 | 2024-06-25 | 디노 테라퓨틱스, 인코포레이티드 | 캡시드 변이체 및 이의 사용 방법 |
| EP4426845A4 (en) * | 2021-11-01 | 2025-10-01 | Univ Florida | AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH ATAXIA |
| EP4440696A1 (en) * | 2021-12-01 | 2024-10-09 | Shape Therapeutics Inc. | Functional aav capsids for systemic administration |
| CN114276419B (zh) * | 2021-12-30 | 2023-11-17 | 上海勉亦生物科技有限公司 | 肌肉高亲和性的新型腺相关病毒衣壳蛋白及其应用 |
| JP2025504081A (ja) * | 2022-02-01 | 2025-02-06 | コーネル・ユニバーシティー | フリードライヒ運動失調症に関連する心筋症の治療及び予防のための方法及び医薬組成物 |
| CN114516901B (zh) * | 2022-03-11 | 2023-01-17 | 上海勉亦生物科技有限公司 | 一种神经系统高亲和性的aav载体及其应用 |
| CN116041443B (zh) * | 2022-12-30 | 2023-09-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
| EP4709859A1 (en) * | 2023-05-11 | 2026-03-18 | Sichuan Real&Best Biotech Co., Ltd. | Engineered liver-specific core promoters and their applications |
| WO2024238448A2 (en) * | 2023-05-12 | 2024-11-21 | Temple University - Of The Commonwealth System Of Higher Education | Chimeric capsid recombinant adeno-associated virus with bbb penetration and microglia targeting (aav-bm) |
| WO2025015388A1 (en) * | 2023-07-20 | 2025-01-23 | Children's Medical Research Institute | Modified cardiotropic aav capsid polypeptides and vectors |
| CN117624311B (zh) * | 2023-11-29 | 2024-05-28 | 广州译码基因科技有限公司 | 可提高aav病毒神经靶向性的衣壳蛋白突变体及其应用 |
| TW202540436A (zh) | 2023-12-05 | 2025-10-16 | 美商航海家醫療公司 | 用於治療與共濟蛋白(frataxin)缺陷相關之病症之組合物及方法 |
| CN118421807B (zh) * | 2024-07-01 | 2024-09-20 | 西南民族大学 | 牦牛多胸腰椎性状筛查的引物对、试剂盒及其用途和筛查方法 |
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| CN113383010B (zh) | 2025-09-09 |
| CA3114621A1 (en) | 2020-04-02 |
| JP2025179046A (ja) | 2025-12-09 |
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| WO2020069461A1 (en) | 2020-04-02 |
| CN113383010A (zh) | 2021-09-10 |
| JP2022500066A (ja) | 2022-01-04 |
| US12281321B2 (en) | 2025-04-22 |
| AU2019346655A1 (en) | 2021-05-06 |
| SG11202103151RA (en) | 2021-04-29 |
| KR20210068068A (ko) | 2021-06-08 |
| CN121160803A (zh) | 2025-12-19 |
| JP2024073447A (ja) | 2024-05-29 |
| US20210395776A1 (en) | 2021-12-23 |
| EP3856762A1 (en) | 2021-08-04 |
| AU2019346655B2 (en) | 2025-03-13 |
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