JP2021531804A - 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 - Google Patents

原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 Download PDF

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JP2021531804A
JP2021531804A JP2021504770A JP2021504770A JP2021531804A JP 2021531804 A JP2021531804 A JP 2021531804A JP 2021504770 A JP2021504770 A JP 2021504770A JP 2021504770 A JP2021504770 A JP 2021504770A JP 2021531804 A JP2021531804 A JP 2021531804A
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ウィリアム ディメク，ザカリー
オダテ，ショブ
アンドリュー ミュレー，ブラッドリー
マイケル レカルボ，レイノルド
ヒューブナー，アネット
ストラップス，ウォルター
ベス ヘッセ，サラ
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インテリア セラピューティクス，インコーポレイテッド
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JP2021504770A 2018-07-31 2019-07-30 原発性高シュウ酸尿症１型（ｐｈ１）を治療するためのヒドロキシ酸オキシダーゼ１（ｈａｏ１）遺伝子編集のための組成物および方法 Pending JP2021531804A (ja)

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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JP2021531804A true JP2021531804A (ja) 2021-11-25
JP2021531804A5 JP2021531804A5 (enExample) 2022-09-05

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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)
BR (1) BR112021001546A2 (enExample)
CA (1) CA3113190A1 (enExample)
CO (1) CO2021002691A2 (enExample)
MX (1) MX2021001070A (enExample)
TW (1) TW202020156A (enExample)
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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
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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CO2021002691A2 (es) 2021-04-08
US20210163943A1 (en) 2021-06-03
CA3113190A1 (en) 2020-02-06
KR20210053888A (ko) 2021-05-12
MX2021001070A (es) 2021-05-27
CN112867795A (zh) 2021-05-28
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

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