KR20210053888A - 원발성 고옥살산뇨 1형 (ph1) 치료를 위한 히드록시산 옥시다제 1 (hao1) 유전자 편집을 위한 조성물 및 방법 - Google Patents

원발성 고옥살산뇨 1형 (ph1) 치료를 위한 히드록시산 옥시다제 1 (hao1) 유전자 편집을 위한 조성물 및 방법 Download PDF

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KR20210053888A
KR20210053888A KR1020217005361A KR20217005361A KR20210053888A KR 20210053888 A KR20210053888 A KR 20210053888A KR 1020217005361 A KR1020217005361 A KR 1020217005361A KR 20217005361 A KR20217005361 A KR 20217005361A KR 20210053888 A KR20210053888 A KR 20210053888A
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재커리 윌리엄 디멕
쇼부 오다테
브래들리 앤드류 머래이
레이날드 마이클 레스카르보
아네트 휘브너
월터 스트랩스
세라 베쓰 헤세
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인텔리아 테라퓨틱스, 인크.
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KR1020217005361A 2018-07-31 2019-07-30 원발성 고옥살산뇨 1형 (ph1) 치료를 위한 히드록시산 옥시다제 1 (hao1) 유전자 편집을 위한 조성물 및 방법 Withdrawn KR20210053888A (ko)

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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KR20210053888A true KR20210053888A (ko) 2021-05-12

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KR1020217005361A Withdrawn KR20210053888A (ko) 2018-07-31 2019-07-30 원발성 고옥살산뇨 1형 (ph1) 치료를 위한 히드록시산 옥시다제 1 (hao1) 유전자 편집을 위한 조성물 및 방법

Country Status (12)

Country Link
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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WO2020132659A1 (en) * 2018-12-21 2020-06-25 Precision Biosciences, Inc. Genetic modification of the hydroxyacid oxidase 1 gene for treatment of primary hyperoxaluria
JP7642809B2 (ja) 2020-11-12 2025-03-10 プレシジョン バイオサイエンシズ，インク． ジストロフィン遺伝子内の認識配列に特異性を有する遺伝子操作されたメガヌクレアーゼ
IL303506A (en) 2020-12-11 2023-08-01 Intellia Therapeutics Inc Polynucleotides, compositions, and methods for genome editing involving deamination
US20230034581A1 (en) 2021-06-04 2023-02-02 Arbor Biotechnologies, Inc. Gene editing systems comprising an rna guide targeting lactate dehydrogenase a (ldha) and uses thereof
IL308803A (en) * 2021-06-04 2024-01-01 Arbor Biotechnologies Inc Gene editing systems comprising guide RNA targeting hydroxy acid 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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JPH10500310A (ja) 1994-05-19 1998-01-13 ダコ アクティーゼルスカブ 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ
AU2013266968B2 (en) * 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
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CA3113190A1 (en) 2020-02-06
TW202020156A (zh) 2020-06-01
CO2021002691A2 (es) 2021-04-08
EP3830267A1 (en) 2021-06-09
JP2021531804A (ja) 2021-11-25
BR112021001546A2 (pt) 2021-05-04
US20260043026A1 (en) 2026-02-12
CN112867795A (zh) 2021-05-28
AU2019313348A1 (en) 2021-03-04
US20210163943A1 (en) 2021-06-03
WO2020028327A1 (en) 2020-02-06
MX2021001070A (es) 2021-05-27

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