JP7724157B2 - 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 - Google Patents

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

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Publication number
JP7724157B2
JP7724157B2 JP2021545659A JP2021545659A JP7724157B2 JP 7724157 B2 JP7724157 B2 JP 7724157B2 JP 2021545659 A JP2021545659 A JP 2021545659A JP 2021545659 A JP2021545659 A JP 2021545659A JP 7724157 B2 JP7724157 B2 JP 7724157B2
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vector
enhancer
sequence
cells
viral
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JP2022519623A5 (https=
JP2022519623A (ja
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ジョルデーン ディミッドシュスタイン
ゴードン フィッシェル
オリン デヴィンスキー
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New York University NYU
Broad Institute Inc
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Broad Institute Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30—Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • 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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  • Health & Medical Sciences (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Developmental Biology & Embryology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
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  • Pain & Pain Management (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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

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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

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

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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

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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=)

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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
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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WO2018187363A1 (en) 2017-04-03 2018-10-11 Encoded Therapeutics, Inc. Tissue selective transgene expression

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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

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WO2018187363A1 (en) 2017-04-03 2018-10-11 Encoded Therapeutics, Inc. Tissue selective transgene expression

Non-Patent Citations (1)

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Title
曽根 正光，クロマチン立体構造に基づく遺伝子発現制御のゲノムワイドな解析，科学研究費助成事業 研究成果報告書，2016年

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Publication number Publication date
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
CN113966400A (zh) 2022-01-21
KR20210133227A (ko) 2021-11-05
WO2020163102A1 (en) 2020-08-13

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