JP7397488B2 - Ｓｏｄ１二重発現ベクターおよびその使用 - Google Patents
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| WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| DK3119797T3 (da) | 2014-03-18 | 2021-03-15 | Univ Massachusetts | Raav-baserede sammensætninger og fremgangsmåder til behandling af amyotrofisk lateralsklerose |
| JP7327803B2 (ja) | 2017-05-09 | 2023-08-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症（ａｌｓ）を処置する方法 |
| CN111448321A (zh) | 2017-09-22 | 2020-07-24 | 马萨诸塞大学 | Sod1双表达载体及其用途 |
| WO2022060857A1 (en) * | 2020-09-16 | 2022-03-24 | Biogen Ma Inc. | Compositions and methods for treating amyotrophic lateral sclerosis (als) with aav-mir-sod1 |
| EP4291654A2 (en) | 2021-02-12 | 2023-12-20 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| WO2025072224A1 (en) * | 2023-09-25 | 2025-04-03 | California Institute Of Technology | Mirna circuits for controlled gene expression |
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| US20090042828A1 (en) | 2004-04-06 | 2009-02-12 | University Of Massachusetts | Methods and compositions for treating gain-of-function disorders using rna interference |
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| EP3684937A4 (en) | 2021-06-02 |
| EP3684937A1 (en) | 2020-07-29 |
| US20230416757A1 (en) | 2023-12-28 |
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| AU2018338188B2 (en) | 2025-02-20 |
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| CN111448321A (zh) | 2020-07-24 |
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| US12312587B2 (en) | 2025-05-27 |
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| US20200248187A1 (en) | 2020-08-06 |
| WO2019060686A1 (en) | 2019-03-28 |
| CA3075643A1 (en) | 2019-03-28 |
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