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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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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • 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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    • C12N2310/50—Physical structure
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    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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 (enExample) 2023-01-05

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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)
WO (1) WO2020139796A1 (enExample)

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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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CN113518825A (zh) 2021-10-19
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

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