JP2022505877A5 - - Google Patents

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Publication number
JP2022505877A5
JP2022505877A5 JP2021522959A JP2021522959A JP2022505877A5 JP 2022505877 A5 JP2022505877 A5 JP 2022505877A5 JP 2021522959 A JP2021522959 A JP 2021522959A JP 2021522959 A JP2021522959 A JP 2021522959A JP 2022505877 A5 JP2022505877 A5 JP 2022505877A5
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JP
Japan
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JP2021522959A
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Japanese (ja)
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JPWO2020084162A5 (https=
JP2022505877A (ja
JP7529286B2 (ja
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JP2021522959A 2018-10-26 2019-10-28 肝特異的核酸調節エレメント並びにその方法及び使用 Active JP7529286B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18202986.8 2018-10-26
EP18202986 2018-10-26
PCT/EP2019/079358 WO2020084162A1 (en) 2018-10-26 2019-10-28 Liver-specific nucleic acid regulatory elements and methods and use thereof

Publications (4)

Publication Number Publication Date
JP2022505877A JP2022505877A (ja) 2022-01-14
JPWO2020084162A5 JPWO2020084162A5 (https=) 2022-11-08
JP2022505877A5 true JP2022505877A5 (https=) 2022-11-08
JP7529286B2 JP7529286B2 (ja) 2024-08-06

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JP2021522959A Active JP7529286B2 (ja) 2018-10-26 2019-10-28 肝特異的核酸調節エレメント並びにその方法及び使用

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US (1) US20210403948A1 (https=)
EP (1) EP3870237A1 (https=)
JP (1) JP7529286B2 (https=)
CN (1) CN112912112B (https=)
WO (1) WO2020084162A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3823985A1 (en) 2018-07-16 2021-05-26 Baxalta Incorporated Gene therapy of hemophilia a using viral vectors encoding recombinant fviii variants with increased expression
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
TWI851647B (zh) * 2019-01-16 2024-08-11 日商武田藥品工業股份有限公司 用於a型血友病基因治療之編碼表現增加之重組fviii變異體的病毒載體
CN110791502B (zh) * 2019-12-04 2022-07-08 陕西理工大学 人源rbp4启动子片段及其应用
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
AU2022242921A1 (en) * 2021-03-25 2023-10-19 The Johns Hopkins University Hydrodynamic gene delivery
JP2025534900A (ja) * 2022-10-08 2025-10-21 リンイ バイオテック カンパニー リミテッド 遺伝子発現を増強するためのコンストラクト
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease

Family Cites Families (15)

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Publication number Priority date Publication date Assignee Title
US7351813B2 (en) 2000-06-20 2008-04-01 The Board Of Trustees Of The Leland Stanford Junior University Liver-specific gene expression cassettes, and methods of use
US20070161031A1 (en) * 2005-12-16 2007-07-12 The Board Of Trustees Of The Leland Stanford Junior University Functional arrays for high throughput characterization of gene expression regulatory elements
US20090018031A1 (en) * 2006-12-07 2009-01-15 Switchgear Genomics Transcriptional regulatory elements of biological pathways tools, and methods
CN102618536B (zh) * 2008-02-14 2013-10-16 财团法人牧岩生命工学研究所 适于表达用于基因治疗的编码序列的表达载体
EP4154903A1 (en) 2008-04-22 2023-03-29 Vib Vzw Liver-specific nucleic acid regulatory elements and methods and use thereof
US20100323376A1 (en) * 2009-06-17 2010-12-23 Maine Standards Company, Llc Method for Measuring Lipoprotein-Specific Apolipoproteins
GB0911870D0 (en) 2009-07-08 2009-08-19 Ucl Business Plc Optimised coding sequence and promoter
US8815779B2 (en) * 2009-09-16 2014-08-26 SwitchGear Genomics, Inc. Transcription biomarkers of biological responses and methods
WO2011051450A1 (en) 2009-10-29 2011-05-05 Vib Vzw Cardiac-specific nucleic acid regulatory elements and methods and use thereof
US20150141320A1 (en) * 2012-05-16 2015-05-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
WO2013186398A1 (en) * 2012-06-15 2013-12-19 Fondazione Telethon Tfeb for use in treating obesity or metabolic syndrome
EP2911687B1 (en) 2012-10-26 2019-02-13 Vrije Universiteit Brussel Vector for liver-directed gene therapy of hemophilia and methods and use thereof
KR101429696B1 (ko) * 2012-11-21 2014-08-13 국립암센터 안전성 및 항암활성이 증가된 재조합 아데노바이러스 및 이의 용도
EP3270944B1 (en) 2015-03-17 2019-10-23 Vrije Universiteit Brussel Optimized liver-specific expression systems for fviii and fix
CA3002524A1 (en) * 2015-10-28 2017-05-04 Sangamo Therapeutics, Inc. Liver-specific constructs, factor viii expression cassettes and methods of use thereof

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