JP2022514955A - ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療 - Google Patents

ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療 Download PDF

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JP2022514955A
JP2022514955A JP2021536371A JP2021536371A JP2022514955A JP 2022514955 A JP2022514955 A JP 2022514955A JP 2021536371 A JP2021536371 A JP 2021536371A JP 2021536371 A JP2021536371 A JP 2021536371A JP 2022514955 A JP2022514955 A JP 2022514955A
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nucleic acid
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expression
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ミン・ヤン
セドリック・ピエール・フォナーブルク
チー－リン・リー
チャオ・グアン・チェン
ワリッド・ジャン・アザール
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シーエスエル・ベーリング・エルエルシー
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
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    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)

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JP2021536371A 2018-12-23 2019-12-23 ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療 Pending JP2022514955A (ja)

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US201862784508P 2018-12-23 2018-12-23
US62/784,508 2018-12-23
PCT/US2019/068233 WO2020139796A1 (en) 2018-12-23 2019-12-23 Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome

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JP2022514955A true JP2022514955A (ja) 2022-02-16
JP2022514955A5 JP2022514955A5 (https=) 2023-01-05

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US (1) US20210316013A1 (https=)
EP (1) EP3897745A1 (https=)
JP (1) JP2022514955A (https=)
KR (1) KR102942447B1 (https=)
CN (1) CN113518825A (https=)
AU (1) AU2019417697A1 (https=)
BR (1) BR112021012240A2 (https=)
WO (1) WO2020139796A1 (https=)

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EP4329822A1 (en) 2021-04-26 2024-03-06 CSL Behring L.L.C. Lentiviral vectors useful for the treatment of disease
EP4402264A4 (en) * 2021-09-16 2025-10-15 Agency Science Tech & Res MODULATION OF TJP1 EXPRESSION TO TREAT LIVER DISEASES
CN115838765A (zh) * 2021-09-18 2023-03-24 济南赛尔生物科技股份有限公司 一种治疗异染性脑白质营养不良的慢病毒载体
CN114990163A (zh) * 2022-03-31 2022-09-02 中海峡(福建)细胞生物科技有限公司 用于干细胞基因修饰的慢病毒载体及其构建方法和应用
WO2024171034A2 (en) * 2023-02-13 2024-08-22 Csl Behring Llc Optimised polynucleotides
EP4720100A1 (en) 2023-05-25 2026-04-08 CSL Behring LLC Modified vectors
WO2025077575A1 (zh) * 2023-10-08 2025-04-17 上海齐鲁制药研究中心有限公司 截短的绝缘子及其用于重组蛋白的瞬时表达的用途
WO2025215591A1 (en) 2024-04-12 2025-10-16 Csl Behring L.L.C. Modified vectors for xla gene therapy
CN120536433A (zh) * 2024-05-20 2025-08-26 上海本导基因技术有限公司 一种能够调节基因表达的调控元件及应用
WO2026003694A1 (en) * 2024-06-24 2026-01-02 Csl Behring Llc Methods of enveloped virus transduction

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WO2020139796A1 (en) 2020-07-02
KR102942447B1 (ko) 2026-03-24
AU2019417697A1 (en) 2021-07-08
US20210316013A1 (en) 2021-10-14
CN113518825A (zh) 2021-10-19
BR112021012240A2 (pt) 2022-01-18
EP3897745A1 (en) 2021-10-27
KR20210118402A (ko) 2021-09-30

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