JP2024520691A - ヒドロキシ酸オキシダーゼ１（ｈａｏ１）を標的とするｒｎａガイドを含む遺伝子編集システム及びそれらの使用 - Google Patents

ヒドロキシ酸オキシダーゼ１（ｈａｏ１）を標的とするｒｎａガイドを含む遺伝子編集システム及びそれらの使用 Download PDF

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JP2024520691A
JP2024520691A JP2023574534A JP2023574534A JP2024520691A JP 2024520691 A JP2024520691 A JP 2024520691A JP 2023574534 A JP2023574534 A JP 2023574534A JP 2023574534 A JP2023574534 A JP 2023574534A JP 2024520691 A JP2024520691 A JP 2024520691A
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ノーマン ウェッセルズ，クイントン
レイモンド ハスウェル，ジェフリー
マリー ディトマソ，ティア
マイケル ジャキモ，ノア
セングプタ，セジュティ
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アーバー バイオテクノロジーズ， インコーポレイテッド
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JP2023574534A 2021-06-04 2022-06-03 ヒドロキシ酸オキシダーゼ１（ｈａｏ１）を標的とするｒｎａガイドを含む遺伝子編集システム及びそれらの使用 Pending JP2024520691A (ja)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US202163197073P 2021-06-04 2021-06-04
US63/197,073 2021-06-04
US202163225046P 2021-07-23 2021-07-23
US63/225,046 2021-07-23
US202163292889P 2021-12-22 2021-12-22
US63/292,889 2021-12-22
US202263300727P 2022-01-19 2022-01-19
US63/300,727 2022-01-19
PCT/US2022/032144 WO2022256642A2 (en) 2021-06-04 2022-06-03 Gene editing systems comprising an rna guide targeting hydroxyacid oxidase 1 (hao1) and uses thereof

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JP2024520691A true JP2024520691A (ja) 2024-05-24
JP2024520691A5 JP2024520691A5 (https=) 2025-06-11

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US (3) US20230032141A1 (https=)
EP (1) EP4347833A2 (https=)
JP (1) JP2024520691A (https=)
KR (1) KR20240034746A (https=)
AU (1) AU2022283922A1 (https=)
CA (1) CA3222159A1 (https=)
CO (1) CO2023018656A2 (https=)
IL (1) IL308803A (https=)
MX (1) MX2023014406A (https=)
PH (1) PH12023553297A1 (https=)
TW (1) TW202317763A (https=)
WO (1) WO2022256642A2 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4711451A1 (en) * 2023-05-11 2026-03-18 Yoltech Therapeutics Co., Ltd Cas protein, corresponding gene editing system thereof and use thereof
WO2025147604A1 (en) 2024-01-05 2025-07-10 Arbor Biotechnologies, Inc. Methods for treating primary hyperoxaluria via genetic editing of hydroxyacid oxidase 1

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3581654B1 (en) * 2013-12-27 2021-03-31 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (hao1) by double-stranded rna
WO2019178427A1 (en) 2018-03-14 2019-09-19 Arbor Biotechnologies, Inc. Novel crispr dna targeting enzymes and systems
MX2021001070A (es) * 2018-07-31 2021-05-27 Intellia Therapeutics Inc Composiciones y métodos para editar el gen hidroxiácido oxidasa 1 (hao1) para tratar la hiperoxaluria primaria tipo 1 (ph1).
CN121380024A (zh) * 2020-03-31 2026-01-23 阿伯生物技术公司 包含Cas12i2变体多肽的组合物及其用途
WO2021257730A2 (en) * 2020-06-16 2021-12-23 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
CN113308451B (zh) * 2020-12-07 2023-07-25 中国科学院动物研究所 工程化的Cas效应蛋白及其使用方法

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US20240254494A1 (en) 2024-08-01
AU2022283922A9 (en) 2023-12-14
CA3222159A1 (en) 2022-12-08
WO2022256642A3 (en) 2023-01-12
US20230212541A1 (en) 2023-07-06
AU2022283922A1 (en) 2023-12-07
PH12023553297A1 (en) 2024-03-18
CO2023018656A2 (es) 2024-01-25
KR20240034746A (ko) 2024-03-14
IL308803A (en) 2024-01-01
TW202317763A (zh) 2023-05-01
WO2022256642A2 (en) 2022-12-08
EP4347833A2 (en) 2024-04-10
US20230032141A1 (en) 2023-02-02
MX2023014406A (es) 2024-02-12
US11821012B2 (en) 2023-11-21

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