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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| HUE052676T2 (hu) | 2013-10-11 | 2021-05-28 | Massachusetts Eye & Ear Infirmary | Eljárások õsi vírusszekvenciák elõrejelzésére és alkalmazásaik |
| CA2994160C (en) | 2015-07-30 | 2021-08-10 | Massachusetts Eye And Ear Infirmary | Ancestral virus sequences and uses thereof |
| JP7221275B2 (ja) | 2017-08-03 | 2023-02-13 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖを送達するための組成物および方法 |
| US20200263199A1 (en) | 2017-09-29 | 2020-08-20 | Voyager Therapeutics, Inc. | Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| EP4014050A1 (en) | 2019-08-12 | 2022-06-22 | 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 |
| CN116096734A (zh) * | 2020-05-13 | 2023-05-09 | 沃雅戈治疗公司 | Aav衣壳的向性的重定向 |
| CN116121274B (zh) * | 2020-07-29 | 2024-11-19 | 北京三诺佳邑生物技术有限责任公司 | 一组肝靶向新型腺相关病毒的获得及其应用 |
| CA3192600A1 (en) * | 2020-08-21 | 2022-02-24 | 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 |
| MX2024004217A (es) | 2021-10-08 | 2024-06-26 | Dyno Therapeutics Inc | Variantes de cápside y métodos de uso de estas. |
| EP4426845A4 (en) * | 2021-11-01 | 2025-10-01 | Univ Florida | AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH ATAXIA |
| US20250205363A1 (en) * | 2021-12-01 | 2025-06-26 | 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 |
| US20250161483A1 (en) * | 2022-02-01 | 2025-05-22 | Lexeo Therapeutics, Inc. | Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy associated with friedreich ataxia |
| 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) |
| AU2024294136A1 (en) * | 2023-07-20 | 2026-02-05 | 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 |
| CN113383010A (zh) | 2021-09-10 |
| JP2024073447A (ja) | 2024-05-29 |
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| CA3114621A1 (en) | 2020-04-02 |
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| AU2019346655B2 (en) | 2025-03-13 |
| US12281321B2 (en) | 2025-04-22 |
| US20210395776A1 (en) | 2021-12-23 |
| WO2020069461A1 (en) | 2020-04-02 |
| KR20210068068A (ko) | 2021-06-08 |
| AU2019346655A1 (en) | 2021-05-06 |
| JP2022500066A (ja) | 2022-01-04 |
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