CN114040970B - 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗 - Google Patents

使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗 Download PDF

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CN114040970B
CN114040970B CN202080028186.5A CN202080028186A CN114040970B CN 114040970 B CN114040970 B CN 114040970B CN 202080028186 A CN202080028186 A CN 202080028186A CN 114040970 B CN114040970 B CN 114040970B
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base editor
disease
nucleic acid
adenosine
gene
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CN114040970A (zh
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I·斯雷梅克
N·戈代尔利
Y·于
B·蔡澈
D·A·玻恩
S-J·李
M·帕克
J·M·格尔克
N·彼得罗相
A·梅萨纳
S·贝尔科维奇
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Bim Medical Co ltd
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CN202080028186.5A 2019-02-13 2020-02-13 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗 Active CN114040970B (zh)

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CN202411269394.0A CN119280261A (zh) 2019-02-13 2020-02-13 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗

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US201962805271P 2019-02-13 2019-02-13
US62/805,271 2019-02-13
US201962850919P 2019-05-21 2019-05-21
US62/850,919 2019-05-21
US201962852224P 2019-05-23 2019-05-23
US201962852228P 2019-05-23 2019-05-23
US62/852,224 2019-05-23
US62/852,228 2019-05-23
US201962873138P 2019-07-11 2019-07-11
US62/873,138 2019-07-11
US201962888867P 2019-08-19 2019-08-19
US62/888,867 2019-08-19
US201962931722P 2019-11-06 2019-11-06
US62/931,722 2019-11-06
US201962941569P 2019-11-27 2019-11-27
US62/941,569 2019-11-27
US202062966526P 2020-01-27 2020-01-27
US62/966,526 2020-01-27
PCT/US2020/018073 WO2020168051A1 (en) 2019-02-13 2020-02-13 Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease

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CN202411269394.0A Pending CN119280261A (zh) 2019-02-13 2020-02-13 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法，包括遗传性疾病的治疗

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US (1) US20230140953A1 (enExample)
EP (1) EP3924484A4 (enExample)
JP (2) JP7586601B2 (enExample)
KR (1) KR20210127206A (enExample)
CN (2) CN114040970B (enExample)
AU (1) AU2020223306A1 (enExample)
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JP7642531B2 (ja) 2018-05-11 2025-03-10 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法
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WO2022027035A1 (en) * 2020-07-27 2022-02-03 The Children's Hospital Of Philadelphia In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders
EP4232584A4 (en) * 2020-10-21 2025-07-16 Univ Leland Stanford Junior SCREENING PLATFORM FOR RECRUITING GUIDE RNAS
WO2022241270A2 (en) * 2021-05-14 2022-11-17 Beam Therapeutics Inc. Compositions and methods for treating transthyretin amyloidosis
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EP4441219A2 (en) * 2021-12-01 2024-10-09 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides for rna editing targeting lrrk2
CN117187220A (zh) * 2022-03-08 2023-12-08 中国科学院遗传与发育生物学研究所 腺嘌呤脱氨酶及其在碱基编辑中的用途
CN114686456B (zh) 2022-05-10 2023-02-17 中山大学 基于双分子脱氨酶互补的碱基编辑系统及其应用
CN120225674A (zh) * 2022-09-08 2025-06-27 爱丁堡大学董事会 雷特综合征疗法
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WO2024233941A1 (en) * 2023-05-11 2024-11-14 Massachusetts Eye And Ear Infirmary Base editing approaches to treat abca4-associated stargardt disease
WO2024238623A1 (en) * 2023-05-15 2024-11-21 University Of Georgia Research Foundation, Inc. Doubly constrained peptides as allosteric inhibitors of leucine rich repeat kinase 2
CN117965505A (zh) * 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器
CN120536418A (zh) * 2024-02-26 2025-08-26 尧唐(上海)生物科技有限公司 一种脱氨酶变体、包含其的碱基编辑器及其应用
WO2025184515A1 (en) * 2024-02-29 2025-09-04 Vesigen, Inc. Arrdc1-mediated microvesicle-based delivery of therapeutic agents to cells and tissues of the eye

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CA3128876A1 (en) 2020-08-20
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