TW202020156A - 用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 - Google Patents

用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 Download PDF

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TW202020156A
TW202020156A TW108127073A TW108127073A TW202020156A TW 202020156 A TW202020156 A TW 202020156A TW 108127073 A TW108127073 A TW 108127073A TW 108127073 A TW108127073 A TW 108127073A TW 202020156 A TW202020156 A TW 202020156A
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composition
seq
sequence selected
sequence
guide
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TW108127073A
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柴克瑞 威廉 岱美克
書布 歐德
布萊德利 安德魯 莫雷
雷納德 麥克 雷斯卡里
安妮特 胡伯納
瓦特 史瑞普斯
莎拉 貝絲 海斯
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美商英特利亞醫療公司
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification

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TW108127073A 2018-07-31 2019-07-30 用於治療原發性高草酸尿症第1型(ph1)之羥酸氧化酶1(hao1)基因編輯之組合物及方法 TW202020156A (zh)

Applications Claiming Priority (8)

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

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TW202020156A true TW202020156A (zh) 2020-06-01

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US (2) US20210163943A1 (https=)
EP (1) EP3830267A1 (https=)
JP (1) JP2021531804A (https=)
KR (1) KR20210053888A (https=)
CN (1) CN112867795A (https=)
AU (1) AU2019313348A1 (https=)
BR (1) BR112021001546A2 (https=)
CA (1) CA3113190A1 (https=)
CO (1) CO2021002691A2 (https=)
MX (1) MX2021001070A (https=)
TW (1) TW202020156A (https=)
WO (1) WO2020028327A1 (https=)

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CR20230305A (es) 2020-12-11 2023-11-10 Intellia Therapeutics Inc Polinucleótidos, composiciones y métodos para la edición del genoma que implican desaminación
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
CA3222159A1 (en) 2021-06-04 2022-12-08 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof
AU2024335327A1 (en) 2023-09-01 2026-03-26 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
CN118086297B (zh) * 2024-02-28 2025-10-03 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
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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US20260043026A1 (en) 2026-02-12
BR112021001546A2 (pt) 2021-05-04
EP3830267A1 (en) 2021-06-09
MX2021001070A (es) 2021-05-27
AU2019313348A1 (en) 2021-03-04
CO2021002691A2 (es) 2021-04-08
CA3113190A1 (en) 2020-02-06
KR20210053888A (ko) 2021-05-12
JP2021531804A (ja) 2021-11-25
CN112867795A (zh) 2021-05-28
WO2020028327A1 (en) 2020-02-06
US20210163943A1 (en) 2021-06-03

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