CN117737062A - 用于治疗α-1抗胰蛋白酶缺乏症的组合物及方法 - Google Patents
用于治疗α-1抗胰蛋白酶缺乏症的组合物及方法 Download PDFInfo
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| WO2020082047A1 (en) | 2018-10-18 | 2020-04-23 | Intellia Therapeutics, Inc. | Compositions and methods for treating alpha-1 antitrypsin deficiencey |
| US20200270617A1 (en) * | 2018-10-18 | 2020-08-27 | Intellia Therapeutics, Inc. | Compositions and methods for transgene expression from an albumin locus |
| CN120174005A (zh) | 2019-02-13 | 2025-06-20 | 比姆医疗股份有限公司 | 具有用于修饰靶标序列中核碱基的腺苷脱氨酶碱基编辑器的经修饰的免疫细胞 |
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| US20230416725A1 (en) * | 2020-09-15 | 2023-12-28 | Rutgers, The State University Of New Jersey | Systems for gene editing and methods of use thereof |
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| KR102888521B1 (ko) * | 2015-04-06 | 2025-11-19 | 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 | Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna |
| EP4144844B1 (en) * | 2015-10-12 | 2025-09-10 | DuPont US Holding, LLC | Protected dna templates for gene modification and increased homologous recombination in cells and methods of use |
| BR112018008971A2 (pt) * | 2015-11-06 | 2018-11-27 | Crispr Therapeutics Ag | materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a |
| CN109715801B (zh) * | 2015-12-01 | 2022-11-01 | 克里斯普治疗股份公司 | 用于治疗α1抗胰蛋白酶缺乏的材料和方法 |
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| KR20180134412A (ko) | 2016-04-22 | 2018-12-18 | 인텔리아 테라퓨틱스, 인크. | 전사 인자 4에서 트라이뉴클레오타이드 반복부와 연관된 질환의 치료를 위한 조성물 및 방법 |
| SG10202106058WA (en) * | 2016-12-08 | 2021-07-29 | Intellia Therapeutics Inc | Modified guide rnas |
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