JP7756639B2 - Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略 - Google Patents

Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略

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Publication number
JP7756639B2
JP7756639B2 JP2022524995A JP2022524995A JP7756639B2 JP 7756639 B2 JP7756639 B2 JP 7756639B2 JP 2022524995 A JP2022524995 A JP 2022524995A JP 2022524995 A JP2022524995 A JP 2022524995A JP 7756639 B2 JP7756639 B2 JP 7756639B2
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nucleic acid
sequence
protein
coding sequence
acid construct
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JP2022553573A5 (enExample
JP2022553573A (ja
Inventor
チャーリーン ハント，
ヤン リウ，
グオチュン ゴン，
カーメロ ロマーノ，
ブライアン ザンブロウィッツ，
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Regeneron Pharmaceuticals Inc
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Regeneron Pharmaceuticals Inc
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    • A01K67/0278—Knock-in vertebrates, e.g. humanised vertebrates
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    • C12N9/222—Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
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JP2022524995A 2019-11-08 2020-11-07 Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略 Active JP7756639B2 (ja)

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US201962932608P 2019-11-08 2019-11-08
US62/932,608 2019-11-08
PCT/US2020/059568 WO2021092513A1 (en) 2019-11-08 2020-11-07 Crispr and aav strategies for x-linked juvenile retinoschisis therapy

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JP2022553573A JP2022553573A (ja) 2022-12-23
JP2022553573A5 JP2022553573A5 (enExample) 2023-09-27
JP7756639B2 true JP7756639B2 (ja) 2025-10-20

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US (2) US12521451B2 (enExample)
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JP (1) JP7756639B2 (enExample)
KR (2) KR20260018182A (enExample)
CN (1) CN114746125B (enExample)
AU (1) AU2020379046B2 (enExample)
BR (1) BR112022008861A2 (enExample)
CA (1) CA3156277A1 (enExample)
IL (1) IL292605B2 (enExample)
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JP7756639B2 (ja) 2019-11-08 2025-10-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｘ連鎖性若年網膜分離療法のためのｃｒｉｓｐｒおよびａａｖ戦略
CN113025618B (zh) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
EP4100528A1 (en) * 2020-02-07 2022-12-14 Intellia Therapeutics, Inc. Compositions and methods for kallikrein (klkb1) gene editing
WO2023081739A1 (en) * 2021-11-03 2023-05-11 Teamedon International, Inc. Methods of treating human x-linked retinoschisis using gene therapy
EP4722357A1 (en) 2023-07-07 2026-04-08 Shandong Shunfeng Biotechnology Co., Ltd. Engineered cas protein and use thereof
CN116790559B (zh) * 2023-07-07 2023-11-28 山东舜丰生物科技有限公司 一种融合HNH结构域的V型Cas酶及其应用

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