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
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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
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English (en)
Japanese (ja)
Other versions
JP7381494B2 (ja
JP2021512649A (ja
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Publication date
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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 (https=) 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 (https=)
EP (1) EP3749756A4 (https=)
JP (2) JP7381494B2 (https=)
CN (1) CN111936621B (https=)
MA (1) MA51753A (https=)
WO (1) WO2019153009A1 (https=)

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Publication number Priority date Publication date Assignee Title
DK3635009T3 (da) 2017-06-07 2026-03-30 Regeneron Pharma Sammensætninger og fremgangsmåder til internalisering af enzymer
US12258597B2 (en) 2018-02-07 2025-03-25 Regeneron Pharmaceuticals, Inc. Methods and compositions for therapeutic protein delivery
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件
BR112020023145A2 (pt) 2018-05-17 2021-02-02 Regeneron Pharmaceuticals, Inc. anticorpo anti-cd63 ou fragmento de ligação ao antígeno do mesmo, molécula de ligação ao antígeno biespecífica, proteína terapêutica de múltiplos domínios, polinucleotídeo composição farmacêutica, e, composto
GB201813528D0 (en) 2018-08-20 2018-10-03 Ucl Business Plc Factor IX encoding nucleotides
US10842885B2 (en) 2018-08-20 2020-11-24 Ucl Business Ltd Factor IX encoding nucleotides
KR20210148273A (ko) * 2019-04-08 2021-12-07 제네똥 근육 발현을 위한 하이브리드 프로모터
JP2022554141A (ja) * 2019-10-25 2022-12-28 オーデンツ セラピューティクス,インコーポレーテッド 糖原病を治療するための組成物及び方法
KR20220098384A (ko) * 2019-11-19 2022-07-12 아스클레피오스 바이오파마슈티컬, 인크. 폼페병 및 리소좀 장애를 치료하기 위한 간-특이적 프로모터를 포함하는 치료적 아데노-관련 바이러스
JP7744027B2 (ja) * 2020-02-18 2025-09-25 フレイエ ユニヴェルシテイト ブリュッセル 核酸調節エレメントの新規な組合せ並びにその方法及び使用
GB202013194D0 (en) * 2020-08-24 2020-10-07 Combigene Ab Gene therapy for lipodystrophy
AR126660A1 (es) * 2021-07-28 2023-11-01 Univ Brussel Vrije Mejorando la eficacia de la terapia génica dirigida a músculos
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
WO2024006775A2 (en) * 2022-06-27 2024-01-04 Astellas Gene Therapies, Inc. Compositions and methods for the treatment of myotonic dystrophies
EP4598590A1 (en) * 2022-10-08 2025-08-13 Lingyi Biotech Co., Ltd. Construct for enhancing 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
WO2006036502A2 (en) 2004-09-22 2006-04-06 St. Jude Children's Research Hospital Improved expression of factor ix in gene therapy vectors
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
KR20150020250A (ko) * 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
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
PL3097197T3 (pl) * 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
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
TW202446416A (zh) * 2015-09-24 2024-12-01 美商拜奧馬林製藥公司 腺相關病毒因子viii載體、相關病毒粒子及包含其之治療調配物

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