JP2022514955A5 - - Google Patents

Info

Publication number
JP2022514955A5
JP2022514955A5 JP2021536371A JP2021536371A JP2022514955A5 JP 2022514955 A5 JP2022514955 A5 JP 2022514955A5 JP 2021536371 A JP2021536371 A JP 2021536371A JP 2021536371 A JP2021536371 A JP 2021536371A JP 2022514955 A5 JP2022514955 A5 JP 2022514955A5
Authority
JP
Japan
Prior art keywords
nucleic acid
sequence
acid sequence
expression vector
vector according
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021536371A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022514955A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/068233 external-priority patent/WO2020139796A1/en
Publication of JP2022514955A publication Critical patent/JP2022514955A/ja
Publication of JP2022514955A5 publication Critical patent/JP2022514955A5/ja
Pending legal-status Critical Current

Links

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)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (2)

Publication Number Publication Date
JP2022514955A JP2022514955A (ja) 2022-02-16
JP2022514955A5 true JP2022514955A5 (enExample) 2023-01-05

Family

ID=69326699

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021536371A Pending JP2022514955A (ja) 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

Country Status (8)

Country Link
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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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
WO2026003694A1 (en) * 2024-06-24 2026-01-02 Csl Behring Llc Methods of enveloped virus transduction

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WO2005080581A2 (en) 2004-02-17 2005-09-01 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
PL1766010T3 (pl) 2004-06-28 2011-07-29 Univ Western Australia Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
MX2007010008A (es) * 2005-02-16 2008-01-18 Lentigen Corp Vectores lentivirales y su uso.
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