JP2023537980A - メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 - Google Patents

メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 Download PDF

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JP2023537980A
JP2023537980A JP2023509770A JP2023509770A JP2023537980A JP 2023537980 A JP2023537980 A JP 2023537980A JP 2023509770 A JP2023509770 A JP 2023509770A JP 2023509770 A JP2023509770 A JP 2023509770A JP 2023537980 A JP2023537980 A JP 2023537980A
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nucleotide sequence
seq
nucleic acid
sequence
mecp2
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ダラル、ニキタ
カバディ、アミ
パテル、トシャル、ロヒット
ダウニー、パトリック、マーク
シュリバスタバ、アムールヤ、ニディ
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ユーシービー バイオファルマ エスアールエル
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023509770A 2020-08-12 2021-08-11 メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療 Pending JP2023537980A (ja)

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

Publications (2)

Publication Number Publication Date
JP2023537980A true JP2023537980A (ja) 2023-09-06
JP2023537980A5 JP2023537980A5 (https=) 2024-07-25

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JP2023509770A Pending JP2023537980A (ja) 2020-08-12 2021-08-11 メチルcpg結合タンパク質2(mecp2)プロモータ配列を含む核酸構築体を用いた遺伝子治療

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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=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
CN121335978A (zh) * 2023-06-16 2026-01-13 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
WO2025213097A1 (en) * 2024-04-05 2025-10-09 The Children's Hospital Of Philadelphia Novel gene therapy constructs for stxbp1 haploinsufficiency

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011505859A (ja) * 2007-12-21 2011-03-03 ノバルティス アーゲー 哺乳類発現ベクター
JP2017535266A (ja) * 2014-11-14 2017-11-30 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)を治療する組成物および方法
JP2017536116A (ja) * 2014-11-21 2017-12-07 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 中枢神経系を標的化したaavベクター
JP2018500311A (ja) * 2014-12-16 2018-01-11 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
JP2019511570A (ja) * 2016-03-02 2019-04-25 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 前頭側頭型認知症の治療

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
TWI601821B (zh) * 2016-04-15 2017-10-11 中央研究院 治療神經發展性疾病
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

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2011505859A (ja) * 2007-12-21 2011-03-03 ノバルティス アーゲー 哺乳類発現ベクター
JP2017535266A (ja) * 2014-11-14 2017-11-30 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. 筋萎縮性側索硬化症(als)を治療する組成物および方法
JP2017536116A (ja) * 2014-11-21 2017-12-07 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル 中枢神経系を標的化したaavベクター
JP2018500311A (ja) * 2014-12-16 2018-01-11 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
JP2019511570A (ja) * 2016-03-02 2019-04-25 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアThe Children’S Hospital Of Philadelphia 前頭側頭型認知症の治療

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HUMAN MOLECULAR GENETICS, vol. 14, no. 23, JPN7025002646, 2005, pages 3709 - 3722, ISSN: 0005767187 *
RNA, vol. 9, JPN6025024400, 2003, pages 607 - 617, ISSN: 0005767188 *

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Publication number Publication date
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
CN116113441A (zh) 2023-05-12
PH12023550145A1 (en) 2024-03-18
WO2022034130A1 (en) 2022-02-17
AU2021325717A1 (en) 2023-03-02
ZA202300378B (en) 2024-04-24
CA3188748A1 (en) 2022-02-17
PE20230914A1 (es) 2023-06-02
ECSP23016688A (es) 2023-04-28

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