JP2021512649A5 - - Google Patents

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Publication number
JP2021512649A5
JP2021512649A5 JP2020564045A JP2020564045A JP2021512649A5 JP 2021512649 A5 JP2021512649 A5 JP 2021512649A5 JP 2020564045 A JP2020564045 A JP 2020564045A JP 2020564045 A JP2020564045 A JP 2020564045A JP 2021512649 A5 JP2021512649 A5 JP 2021512649A5
Authority
JP
Japan
Prior art keywords
nucleic acid
regulatory element
acid sequence
sequence
segment
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.)
Granted
Application number
JP2020564045A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021512649A (ja
JP7381494B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/016692 external-priority patent/WO2019153009A1/en
Publication of JP2021512649A publication Critical patent/JP2021512649A/ja
Publication of JP2021512649A5 publication Critical patent/JP2021512649A5/ja
Priority to JP2023188066A priority Critical patent/JP7776477B2/ja
Application granted granted Critical
Publication of JP7381494B2 publication Critical patent/JP7381494B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2020564045A 2018-02-05 2019-02-05 転写調節要素及びその使用 Active JP7381494B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023188066A JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862626561P 2018-02-05 2018-02-05
US62/626,561 2018-02-05
PCT/US2019/016692 WO2019153009A1 (en) 2018-02-05 2019-02-05 Transcription regulatory elements and uses thereof

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023188066A Division JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Publications (3)

Publication Number Publication Date
JP2021512649A JP2021512649A (ja) 2021-05-20
JP2021512649A5 true JP2021512649A5 (enExample) 2022-02-09
JP7381494B2 JP7381494B2 (ja) 2023-11-15

Family

ID=67478520

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020564045A Active JP7381494B2 (ja) 2018-02-05 2019-02-05 転写調節要素及びその使用
JP2023188066A Active JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023188066A Active JP7776477B2 (ja) 2018-02-05 2023-11-02 転写調節要素及びその使用

Country Status (6)

Country Link
US (1) US12296026B2 (enExample)
EP (1) EP3749756A4 (enExample)
JP (2) JP7381494B2 (enExample)
CN (1) CN111936621B (enExample)
MA (1) MA51753A (enExample)
WO (1) WO2019153009A1 (enExample)

Families Citing this family (19)

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Publication number Priority date Publication date Assignee Title
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CA3086942A1 (en) * 2018-02-07 2019-08-15 Genethon Hybrid regulatory elements
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
AU2020372429A1 (en) * 2019-10-25 2022-04-28 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
WO2021102107A1 (en) * 2019-11-19 2021-05-27 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus comprising liver-specific promoters for treating pompe disease and lysosomal disorders
EP4107259A1 (en) * 2020-02-18 2022-12-28 Vrije Universiteit Brussel Novel combination of nucleic acid regulatory elements and methods and uses thereof
GB202013194D0 (en) * 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
CA3216419A1 (en) * 2021-04-23 2022-10-27 Greg NACHTRAB Tissue-targeted modified aav capsids and methods of use thereof
AR126660A1 (es) * 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
WO2023012313A1 (en) * 2021-08-04 2023-02-09 Genethon Hybrid promoters for gene expression in muscles and in the cns
TW202400796A (zh) * 2022-03-11 2024-01-01 美商同源醫藥公司 雙向雙重啟動子表現載體及其用途
TW202409290A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
EP4598590A4 (en) * 2022-10-08 2026-05-27 Lingyi Biotech Co Ltd CONSTRUCTION TO IMPROVE GENE EXPRESSION
WO2025004002A2 (en) 2023-06-30 2025-01-02 Takeda Pharmaceutical Company Limited Treatment of pompe disease
CN118064436B (zh) * 2024-04-18 2024-07-26 上海凌医生物科技有限公司 一种增强基因表达的嵌合启动子

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1390490B1 (en) * 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
US8021875B2 (en) 2001-08-27 2011-09-20 Roche Madison Inc. Methods for expression of transgenes
ES2436741T3 (es) 2004-09-22 2014-01-07 St. Jude Children's Research Hospital Expresión mejorada de Factor IX en vectores de terapia génica
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
EP4124346A3 (en) 2012-06-19 2023-04-19 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
CN105229144A (zh) * 2013-02-22 2016-01-06 细胞动力学国际有限公司 通过组合的遗传工程和化学工程经由正向编程产生肝细胞
AU2015208247B2 (en) * 2014-01-21 2021-05-27 Universiteit Gent Muscle-specific nucleic acid regulatory elements and methods and use thereof
US10781459B2 (en) 2014-06-20 2020-09-22 University Of Florida Research Foundation, Incorporated Methods of packaging multiple adeno-associated virus vectors
EP3285788B1 (en) 2015-04-23 2024-12-18 University of Massachusetts Modulation of aav vector transgene expression
JP2018535929A (ja) * 2015-09-24 2018-12-06 バイオマリン ファーマシューティカル インコーポレイテッド アデノ随伴ウイルス第viii因子ベクター、関連ウイルス粒子、及びそれらを含む治療的製剤

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