CN116113441A - 使用包含甲基cpg结合蛋白2（mecp2）启动子序列的核酸构建体的基因疗法 - Google Patents

使用包含甲基cpg结合蛋白2（mecp2）启动子序列的核酸构建体的基因疗法 Download PDF

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Publication number
CN116113441A
CN116113441A CN202180056525.5A CN202180056525A CN116113441A CN 116113441 A CN116113441 A CN 116113441A CN 202180056525 A CN202180056525 A CN 202180056525A CN 116113441 A CN116113441 A CN 116113441A
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nucleotide sequence
seq
nucleic acid
aav
sequence
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Chinese (zh)
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N·达拉尔
A·卡巴迪
T·R·帕特尔
P·M·窦内
A·N·沙利瓦寺塔瓦
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UCB Biopharma SRL
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UCB Biopharma SRL
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    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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CN202180056525.5A 2020-08-12 2021-08-11 使用包含甲基cpg结合蛋白2（mecp2）启动子序列的核酸构建体的基因疗法 Pending CN116113441A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063064431P 2020-08-12 2020-08-12
US63/064,431 2020-08-12
PCT/EP2021/072365 WO2022034130A1 (en) 2020-08-12 2021-08-11 Gene therapy using nucleic acid constructs comprising methyl cpg binding protein 2 (mecp2) promoter sequences

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CN116113441A true CN116113441A (zh) 2023-05-12

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US (1) US20230295657A1 (https=)
EP (1) EP4196171A1 (https=)
JP (1) JP2023537980A (https=)
KR (1) KR20230044506A (https=)
CN (1) CN116113441A (https=)
AR (1) AR123206A1 (https=)
AU (1) AU2021325717A1 (https=)
BR (1) BR112023002374A2 (https=)
CA (1) CA3188748A1 (https=)
CL (1) CL2023000419A1 (https=)
CO (1) CO2023000444A2 (https=)
EC (1) ECSP23016688A (https=)
IL (1) IL300294A (https=)
MX (1) MX2023001701A (https=)
PE (1) PE20230914A1 (https=)
PH (1) PH12023550145A1 (https=)
TW (1) TWI902867B (https=)
WO (1) WO2022034130A1 (https=)
ZA (1) ZA202300378B (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024255792A1 (zh) * 2023-06-16 2024-12-19 舒泰神（北京）生物制药股份有限公司 一种人源MeCp2启动子及其用途

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US20240033332A1 (en) * 2020-12-17 2024-02-01 Kyoto University Agent for activating a neuron
GB202216168D0 (en) 2022-10-31 2022-12-14 UCB Biopharma SRL Route of administration
WO2024163823A1 (en) * 2023-02-02 2024-08-08 Shape Therapeutics Inc. Tissue-specific enhancers for regulating transcription
JP2026509301A (ja) * 2023-03-14 2026-03-17 ユーシービー バイオファルマ エスアールエル 遺伝子治療
WO2025213097A1 (en) * 2024-04-05 2025-10-09 The Children's Hospital Of Philadelphia Novel gene therapy constructs for stxbp1 haploinsufficiency

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WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
TWI601821B (zh) * 2016-04-15 2017-10-11 中央研究院 治療神經發展性疾病

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PL3075858T3 (pl) * 2007-12-21 2020-07-13 Novartis Ag Ssaczy wektor ekspresyjny
EA201492116A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
WO2016077687A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
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CN113166781A (zh) * 2018-10-15 2021-07-23 沃雅戈治疗公司 在杆状病毒/Sf9系统中大规模生产rAAV的表达载体

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WO2016100575A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
TWI601821B (zh) * 2016-04-15 2017-10-11 中央研究院 治療神經發展性疾病

Non-Patent Citations (2)

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Title
MEGUMI ADACHI等: "A segment of the Mecp2 promoter is sufficient to drive expression in neurons", HUMAN MOLECULAR GENETICS, vol. 14, no. 23, 26 October 2005 (2005-10-26) *
MEGUMI ADACHI等: "A segment of the Mecp2 promoter is sufficient to drive expression in neurons", HUMAN MOLECULAR GENETICS, vol. 14, no. 23, 26 October 2015 (2015-10-26), pages 4 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024255792A1 (zh) * 2023-06-16 2024-12-19 舒泰神（北京）生物制药股份有限公司 一种人源MeCp2启动子及其用途

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KR20230044506A (ko) 2023-04-04
TW202221018A (zh) 2022-06-01
CL2023000419A1 (es) 2023-07-21
US20230295657A1 (en) 2023-09-21
CO2023000444A2 (es) 2023-01-26
EP4196171A1 (en) 2023-06-21
IL300294A (en) 2023-04-01
BR112023002374A2 (pt) 2023-03-21
AR123206A1 (es) 2022-11-09
TWI902867B (zh) 2025-11-01
MX2023001701A (es) 2023-03-09
PH12023550145A1 (en) 2024-03-18
WO2022034130A1 (en) 2022-02-17
AU2021325717A1 (en) 2023-03-02
ZA202300378B (en) 2024-04-24
JP2023537980A (ja) 2023-09-06
CA3188748A1 (en) 2022-02-17
PE20230914A1 (es) 2023-06-02
ECSP23016688A (es) 2023-04-28

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