CN111417728A - 用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法 - Google Patents

用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法 Download PDF

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Publication number
CN111417728A
CN111417728A CN201880076628.6A CN201880076628A CN111417728A CN 111417728 A CN111417728 A CN 111417728A CN 201880076628 A CN201880076628 A CN 201880076628A CN 111417728 A CN111417728 A CN 111417728A
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composition
seq
nos
sequence selected
amino acid
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A·M·P·坎乔利亚
S·奥达特
J·L·塞泽
R·M·莱斯卡博
W·斯特雷普斯
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Intellia Therapeutics Inc
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CN201880076628.6A 2017-09-29 2018-09-28 用于ttr基因编辑及治疗attr淀粉样变性的组合物及方法 Pending CN111417728A (zh)

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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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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
CN116064598A (zh) * 2021-10-08 2023-05-05 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024213137A1 (zh) * 2023-04-14 2024-10-17 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
WO2025087413A1 (zh) * 2023-10-25 2025-05-01 上海津曼特生物科技有限公司 靶向ttr的基因编辑组合物

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TW202102529A (zh) * 2019-03-28 2021-01-16 美商英特利亞醫療公司 用於多肽表現之多核苷酸、組合物及方法
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WO2023028469A2 (en) * 2021-08-23 2023-03-02 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at beta-globin locus in human hematopoietic stem and progenitor cells
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Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN116064598A (zh) * 2021-10-08 2023-05-05 苏州艾博生物科技有限公司 冠状病毒的核酸疫苗
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
WO2024003810A1 (en) * 2022-06-30 2024-01-04 Geneditbio Limited Guide rna with chemical modifications
WO2024213137A1 (zh) * 2023-04-14 2024-10-17 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
WO2025087413A1 (zh) * 2023-10-25 2025-05-01 上海津曼特生物科技有限公司 靶向ttr的基因编辑组合物

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