KR102942447B1 - 위스콧-알드리치(Wiskott-Aldrich) 증후군에 대한 조혈 줄기 세포-유전자 치료요법 - Google Patents

위스콧-알드리치(Wiskott-Aldrich) 증후군에 대한 조혈 줄기 세포-유전자 치료요법

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KR102942447B1
KR102942447B1 KR1020217023216A KR20217023216A KR102942447B1 KR 102942447 B1 KR102942447 B1 KR 102942447B1 KR 1020217023216 A KR1020217023216 A KR 1020217023216A KR 20217023216 A KR20217023216 A KR 20217023216A KR 102942447 B1 KR102942447 B1 KR 102942447B1
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밍 얀
세드릭 피에르 보나버그
치-린 리
차오-광 천
왈리드 한 아자르
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씨에스엘 베링 엘엘씨
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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
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
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    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2310/50—Physical structure
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    • C12N2740/16011—Human Immunodeficiency Virus, HIV
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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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KR1020217023216A 2018-12-23 2019-12-23 위스콧-알드리치(Wiskott-Aldrich) 증후군에 대한 조혈 줄기 세포-유전자 치료요법 Active KR102942447B1 (ko)

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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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KR102942447B1 true KR102942447B1 (ko) 2026-03-24

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CN (1) CN113518825A (enExample)
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BR (1) BR112021012240A2 (enExample)
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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
AU2024358606A1 (en) * 2023-10-08 2026-04-16 Shanghai Qilu Pharmaceutical Research And Development Centre Ltd. Truncated insulator and use thereof in transient expression of recombinant protein
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
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EP3897745A1 (en) 2021-10-27
US20210316013A1 (en) 2021-10-14
AU2019417697A1 (en) 2021-07-08
BR112021012240A2 (pt) 2022-01-18
KR20210118402A (ko) 2021-09-30
WO2020139796A1 (en) 2020-07-02
JP2022514955A (ja) 2022-02-16

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