CN113518825A - Wiskott-aldrich综合征的造血干细胞基因疗法 - Google Patents

Wiskott-aldrich综合征的造血干细胞基因疗法 Download PDF

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CN113518825A
CN113518825A CN201980092125.2A CN201980092125A CN113518825A CN 113518825 A CN113518825 A CN 113518825A CN 201980092125 A CN201980092125 A CN 201980092125A CN 113518825 A CN113518825 A CN 113518825A
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nucleic acid
acid sequence
seq
expression vector
polynucleotide
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M·严
C·P·沃纳堡
C-L·李
C-G·陈
W·J·阿扎
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American Jette Bellin Biological Products Co ltd
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CN201980092125.2A 2018-12-23 2019-12-23 Wiskott-aldrich综合征的造血干细胞基因疗法 Pending CN113518825A (zh)

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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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US (1) US20210316013A1 (enExample)
EP (1) EP3897745A1 (enExample)
JP (1) JP2022514955A (enExample)
KR (1) KR102942447B1 (enExample)
CN (1) CN113518825A (enExample)
AU (1) AU2019417697A1 (enExample)
BR (1) BR112021012240A2 (enExample)
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CN111093715A (zh) * 2017-09-18 2020-05-01 儿童医院医疗中心 强绝缘子和其在基因递送中的用途
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

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CA3217247A1 (en) * 2021-04-26 2022-11-03 Csl Behring L.L.C. Lentiviral vectors useful for the treatment of disease
JP2024535716A (ja) * 2021-09-16 2024-10-02 エージェンシー フォー サイエンス， テクノロジー アンド リサーチ 肝疾患を治療するためのｔｊｐ１発現の調節
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
CN121399150A (zh) 2023-05-25 2026-01-23 美国杰特贝林生物制品有限公司 经修饰载体
WO2025215591A1 (en) 2024-04-12 2025-10-16 Csl Behring L.L.C. Modified vectors for xla gene therapy
WO2025241977A1 (zh) * 2024-05-20 2025-11-27 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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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN111093715A (zh) * 2017-09-18 2020-05-01 儿童医院医疗中心 强绝缘子和其在基因递送中的用途
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

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EP3897745A1 (en) 2021-10-27
US20210316013A1 (en) 2021-10-14
AU2019417697A1 (en) 2021-07-08
BR112021012240A2 (pt) 2022-01-18
KR102942447B1 (ko) 2026-03-24
KR20210118402A (ko) 2021-09-30
WO2020139796A1 (en) 2020-07-02
JP2022514955A (ja) 2022-02-16

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