JP2022519623A5 - - Google Patents

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Publication number
JP2022519623A5
JP2022519623A5 JP2021545659A JP2021545659A JP2022519623A5 JP 2022519623 A5 JP2022519623 A5 JP 2022519623A5 JP 2021545659 A JP2021545659 A JP 2021545659A JP 2021545659 A JP2021545659 A JP 2021545659A JP 2022519623 A5 JP2022519623 A5 JP 2022519623A5
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JP
Japan
Prior art keywords
polynucleotide sequence
vector
enhancer
seq
viral
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
JP2021545659A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022519623A (ja
JP7724157B2 (ja
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Priority claimed from PCT/US2020/015183 external-priority patent/WO2020163102A1/en
Publication of JP2022519623A publication Critical patent/JP2022519623A/ja
Publication of JP2022519623A5 publication Critical patent/JP2022519623A5/ja
Priority to JP2025076698A priority Critical patent/JP2025118768A/ja
Application granted granted Critical
Publication of JP7724157B2 publication Critical patent/JP7724157B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2021545659A 2019-02-05 2020-01-27 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤 Active JP7724157B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025076698A JP2025118768A (ja) 2019-02-05 2025-05-02 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962801483P 2019-02-05 2019-02-05
US62/801,483 2019-02-05
US201962823281P 2019-03-25 2019-03-25
US62/823,281 2019-03-25
US201962916477P 2019-10-17 2019-10-17
US62/916,477 2019-10-17
PCT/US2020/015183 WO2020163102A1 (en) 2019-02-05 2020-01-27 Interneuron-specific therapeutics for normalizing neuronal cell excitability and treating dravet syndrome

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025076698A Division JP2025118768A (ja) 2019-02-05 2025-05-02 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤

Publications (3)

Publication Number Publication Date
JP2022519623A JP2022519623A (ja) 2022-03-24
JP2022519623A5 true JP2022519623A5 (https=) 2023-02-03
JP7724157B2 JP7724157B2 (ja) 2025-08-15

Family

ID=71947919

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021545659A Active JP7724157B2 (ja) 2019-02-05 2020-01-27 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤
JP2025076698A Pending JP2025118768A (ja) 2019-02-05 2025-05-02 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025076698A Pending JP2025118768A (ja) 2019-02-05 2025-05-02 ニューロン細胞興奮性の正常化およびドラベ症候群の処置のための介在ニューロン特異的治療剤

Country Status (9)

Country Link
US (1) US20220195457A1 (https=)
EP (1) EP3921326A4 (https=)
JP (2) JP7724157B2 (https=)
KR (1) KR20210133227A (https=)
CN (1) CN113966400A (https=)
CA (1) CA3128525A1 (https=)
IL (1) IL284909A (https=)
SG (1) SG11202107813RA (https=)
WO (1) WO2020163102A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115707773A (zh) * 2021-08-20 2023-02-21 中国科学院深圳先进技术研究院 一种利用交感神经控制血管内皮细胞异质性的方法
US12054486B2 (en) 2021-09-24 2024-08-06 Xenon Pharmaceuticals Inc. Pyridine derivatives and their use as sodium channel activators
WO2023108401A1 (zh) * 2021-12-14 2023-06-22 深圳先进技术研究院 重组载体及其构建方法和应用
JP2024545923A (ja) 2021-12-30 2024-12-13 リーゲル セラピューティクス,インコーポレイティッド ナトリウム電位依存性チャネルアルファサブユニット1の発現を調節するための組成物、及びその使用
GB2621102A (en) * 2022-04-11 2024-02-07 Univ Ramot Treatment and Reversion of Dravet syndrome by CAV-2-Mediated Delivery of an SCN1A Expression Cassette
WO2023200700A2 (en) * 2022-04-11 2023-10-19 The Broad Institute, Inc. Enhancers for directed expression of genes in neuronal cell populations, compositions and methods thereof
CN116024222B (zh) * 2022-11-30 2023-12-22 湖南家辉生物技术有限公司 一种导致婴儿严重肌阵挛性癫痫的nac1基因突变体及其应用

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101255424B (zh) * 2007-11-26 2013-02-27 广州医学院第二附属医院 与癫痫相关scn1a基因的启动子及其构建方法和临床应用
EP2635111B1 (en) * 2010-11-05 2018-05-23 The Board of Trustees of the Leland Stanford Junior University Stabilized step function opsin proteins and methods of using the same
US10682424B2 (en) * 2015-03-31 2020-06-16 New York University Compositions and method for reducing seizures
MX2019011772A (es) * 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.

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