CN112867795A - 用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法 - Google Patents

用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法 Download PDF

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CN112867795A
CN112867795A CN201980064321.9A CN201980064321A CN112867795A CN 112867795 A CN112867795 A CN 112867795A CN 201980064321 A CN201980064321 A CN 201980064321A CN 112867795 A CN112867795 A CN 112867795A
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seq
use according
sequence selected
sequence
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Z·W·迪梅克
S·奥达特
B·A·穆雷
R·M·莱斯卡博
A·许布纳
W·斯特雷普斯
S·B·赫西
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Intellia Therapeutics Inc
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CN201980064321.9A 2018-07-31 2019-07-30 用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法 Pending CN112867795A (zh)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201862712904P 2018-07-31 2018-07-31
US62/712,904 2018-07-31
US201862738936P 2018-09-28 2018-09-28
US62/738,936 2018-09-28
US201962834328P 2019-04-15 2019-04-15
US62/834,328 2019-04-15
US201962841734P 2019-05-01 2019-05-01
US62/841,734 2019-05-01
PCT/US2019/044080 WO2020028327A1 (en) 2018-07-31 2019-07-30 Compositions and methods for hydroxyacid oxidase 1 ( hao1) gene editing for treating primary hyperoxaluria type 1 (ph1)

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Publication Number Publication Date
CN112867795A true CN112867795A (zh) 2021-05-28

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CN201980064321.9A Pending CN112867795A (zh) 2018-07-31 2019-07-30 用于执行羟基酸氧化酶1（hao1）基因编辑以治疗1型原发性高草酸尿症（ph1）的组合物和方法

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US (2) US20210163943A1 (enExample)
EP (1) EP3830267A1 (enExample)
JP (1) JP2021531804A (enExample)
KR (1) KR20210053888A (enExample)
CN (1) CN112867795A (enExample)
AU (1) AU2019313348A1 (enExample)
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MX (1) MX2021001070A (enExample)
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CN117925623A (zh) * 2024-03-20 2024-04-26 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物
CN118086297A (zh) * 2024-02-28 2024-05-28 复旦大学附属儿科医院 用于人HAO1基因编辑的sgRNA、组合物及其应用

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EP3898661A1 (en) * 2018-12-21 2021-10-27 Precision BioSciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
KR20250099412A (ko) 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
EP4259792A1 (en) 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
WO2022256655A2 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
KR20240034746A (ko) * 2021-06-04 2024-03-14 아버 바이오테크놀로지스, 인크. 하이드록시산 산화효소 1(hao1)을 표적으로 하는 rna 가이드를 포함하는 유전자 편집 시스템 및 이의 용도
EP4771154A2 (en) 2023-09-01 2026-07-08 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
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
WO2025174765A1 (en) 2024-02-12 2025-08-21 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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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118086297A (zh) * 2024-02-28 2024-05-28 复旦大学附属儿科医院 用于人HAO1基因编辑的sgRNA、组合物及其应用
CN117925623A (zh) * 2024-03-20 2024-04-26 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物
CN117925623B (zh) * 2024-03-20 2024-06-07 北京引正基因科技有限公司 用于hao1基因编辑和治疗ph1的组合物

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US20210163943A1 (en) 2021-06-03
CA3113190A1 (en) 2020-02-06
KR20210053888A (ko) 2021-05-12
MX2021001070A (es) 2021-05-27
EP3830267A1 (en) 2021-06-09
TW202020156A (zh) 2020-06-01
US20260043026A1 (en) 2026-02-12
AU2019313348A1 (en) 2021-03-04
BR112021001546A2 (pt) 2021-05-04
WO2020028327A1 (en) 2020-02-06
JP2021531804A (ja) 2021-11-25

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