CN113966400A - 用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂 - Google Patents

用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂 Download PDF

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CN113966400A
CN113966400A CN202080027324.8A CN202080027324A CN113966400A CN 113966400 A CN113966400 A CN 113966400A CN 202080027324 A CN202080027324 A CN 202080027324A CN 113966400 A CN113966400 A CN 113966400A
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vector
enhancer
polynucleotide sequence
viral
sequence
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Chinese (zh)
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J·迪米施斯坦
G·菲舍尔
O·德温斯基
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New York University NYU
Broad Institute Inc
Harvard University
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New York University NYU
Broad Institute Inc
Harvard University
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

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CN202080027324.8A 2019-02-05 2020-01-27 用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂 Pending CN113966400A (zh)

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

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CN113966400A true CN113966400A (zh) 2022-01-21

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CN202080027324.8A Pending CN113966400A (zh) 2019-02-05 2020-01-27 用于使神经元细胞兴奋性正常化和治疗德拉韦综合征的中间神经元特异性治疗剂

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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=)

Cited By (1)

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CN116024222A (zh) * 2022-11-30 2023-04-28 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

Families Citing this family (6)

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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

Citations (3)

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CN101255424A (zh) * 2007-11-26 2008-09-03 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
US20130347137A1 (en) * 2010-11-05 2013-12-26 The Board Of Trustees Of The Leland Stanford Junior University Stabilized Step Function Opsin Proteins and Methods of Using the Same
US20190024119A1 (en) * 2017-04-03 2019-01-24 Encoded Therapeutics, Inc. Tissue selective transgene expression

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US10682424B2 (en) * 2015-03-31 2020-06-16 New York University Compositions and method for reducing seizures

Patent Citations (3)

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CN101255424A (zh) * 2007-11-26 2008-09-03 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
US20130347137A1 (en) * 2010-11-05 2013-12-26 The Board Of Trustees Of The Leland Stanford Junior University Stabilized Step Function Opsin Proteins and Methods of Using the Same
US20190024119A1 (en) * 2017-04-03 2019-01-24 Encoded Therapeutics, Inc. Tissue selective transgene expression

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Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116024222A (zh) * 2022-11-30 2023-04-28 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
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

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JP2025118768A (ja) 2025-08-13
EP3921326A4 (en) 2022-12-14
US20220195457A1 (en) 2022-06-23
IL284909A (en) 2021-09-30
SG11202107813RA (en) 2021-08-30
EP3921326A1 (en) 2021-12-15
JP2022519623A (ja) 2022-03-24
CA3128525A1 (en) 2020-08-13
JP7724157B2 (ja) 2025-08-15
KR20210133227A (ko) 2021-11-05
WO2020163102A1 (en) 2020-08-13

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