BR112020005287A2 - composições e métodos para edição de gene ttr e tratar amiloidose attr - Google Patents
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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Applications Claiming Priority (5)
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| US201762566236P | 2017-09-29 | 2017-09-29 | |
| US62/566,236 | 2017-09-29 | ||
| US201862671902P | 2018-05-15 | 2018-05-15 | |
| US62/671,902 | 2018-05-15 | ||
| PCT/US2018/053382 WO2019067872A1 (en) | 2017-09-29 | 2018-09-28 | COMPOSITIONS AND METHODS FOR EDITING THE TTR GENE AND TREATING AMYLOID DOSE ATTR |
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| BR112020005287A2 true BR112020005287A2 (pt) | 2020-09-24 |
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| TW (1) | TWI904069B (https=) |
| WO (1) | WO2019067872A1 (https=) |
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| JP7543259B2 (ja) | 2018-10-18 | 2024-09-02 | アクイタス セラピューティクス インコーポレイテッド | 活性剤の脂質ナノ粒子送達のための脂質 |
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| EP3946598A1 (en) * | 2019-03-28 | 2022-02-09 | Intellia Therapeutics, Inc. | Compositions and methods comprising a ttr guide rna and a polynucleotide encoding an rna-guided dna binding agent |
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| MX2022006950A (es) * | 2019-12-11 | 2022-11-07 | Intellia Therapeutics Inc | Arn guía modificados para edición de genes. |
| EP4237005A4 (en) * | 2020-10-28 | 2024-10-09 | Novo Nordisk A/S | ANTI-TRANSTHYRETIN ANTIBODIES AND METHODS OF USE THEREOF |
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| CN112795595A (zh) * | 2020-12-24 | 2021-05-14 | 中山大学 | 一种遗传性转甲状腺素蛋白淀粉样变性疾病的基因治疗系统 |
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| US20250127927A1 (en) * | 2021-08-23 | 2025-04-24 | The Board Of Trustees Of The Leland Stanford Junior University | Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells |
| AU2022345881A1 (en) | 2021-09-20 | 2024-03-21 | Alnylam Pharmaceuticals, Inc. | Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof |
| CN116064598B (zh) * | 2021-10-08 | 2024-03-12 | 苏州艾博生物科技有限公司 | 冠状病毒的核酸疫苗 |
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| WO2024003805A1 (en) * | 2022-06-30 | 2024-01-04 | Geneditbio Limited | Methods and compositions for ttr gene editing and therapy using crispr system |
| TW202417017A (zh) * | 2022-06-30 | 2024-05-01 | 香港商正基基因科技有限公司 | 具有化學修飾的導引rna |
| CN118853659A (zh) * | 2023-04-14 | 2024-10-29 | 深圳瑞吉生物科技有限公司 | 提高mRNA翻译蛋白的输出量的核酸及其应用 |
| WO2024259135A1 (en) | 2023-06-13 | 2024-12-19 | Intellia Therapeutics, Inc. | Assays for analysis of ribonucleic acid (rna) molecules |
| CN120265763A (zh) * | 2023-10-25 | 2025-07-04 | 上海津曼特生物科技有限公司 | 靶向ttr的基因编辑组合物 |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
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| WO2018007871A1 (en) * | 2016-07-08 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of transthyretin amyloidosis |
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