JP7590962B2 - 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物 - Google Patents

皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物 Download PDF

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JP7590962B2
JP7590962B2 JP2021523074A JP2021523074A JP7590962B2 JP 7590962 B2 JP7590962 B2 JP 7590962B2 JP 2021523074 A JP2021523074 A JP 2021523074A JP 2021523074 A JP2021523074 A JP 2021523074A JP 7590962 B2 JP7590962 B2 JP 7590962B2
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グレイバック，ルーカス・ティー
タシッチ，ボシリカ
デイグル，ターニャ
ティン，ジョナサン
ゼン，ホンクイ
カルムバック，ブライアン・エドワード
ミッチ，ジョン・ケイ
ヘス，エリック
レイン，エドワード・セバスチャン
リーバイ，ボアズ・ピー
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JP2021523074A 2018-11-05 2019-11-05 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物 Active JP7590962B2 (ja)

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JP2024198841A JP7774695B2 (ja) 2018-11-05 2024-11-14 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
JP2025175276A JP2026021360A (ja) 2018-11-05 2025-10-17 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物

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US201862755988P 2018-11-05 2018-11-05
US62/755,988 2018-11-05
US201962806684P 2019-02-15 2019-02-15
US201962806600P 2019-02-15 2019-02-15
US62/806,684 2019-02-15
US62/806,600 2019-02-15
US201962872021P 2019-07-09 2019-07-09
US62/872,021 2019-07-09
PCT/US2019/059927 WO2020097121A1 (en) 2018-11-05 2019-11-05 Artificial expression constructs for selectively modulating gene expression in excitatory cortical neurons

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JP2022513426A5 JP2022513426A5 (enExample) 2022-11-10
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JP2024198841A Active JP7774695B2 (ja) 2018-11-05 2024-11-14 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
JP2025175276A Pending JP2026021360A (ja) 2018-11-05 2025-10-17 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物

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JP2025175276A Pending JP2026021360A (ja) 2018-11-05 2025-10-17 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物

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AU (2) AU2019375975B2 (enExample)
CA (1) CA3118689A1 (enExample)
WO (1) WO2020097121A1 (enExample)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7590962B2 (ja) 2018-11-05 2024-11-27 アレン インスティテュート 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
CA3142948A1 (en) 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations
CN113710693B (zh) 2019-02-25 2025-07-15 马萨诸塞大学 Dna结合结构域反式激活因子及其用途
WO2021248085A2 (en) * 2020-06-04 2021-12-09 Allen Institute Artificial expression constructs for selectively modulating gene expression in inhibitory neocortical neurons
EP4185303A4 (en) * 2020-07-24 2024-10-30 University of Massachusetts Dna-binding domain transactivators and uses thereof
US20230302158A1 (en) * 2020-08-14 2023-09-28 Allen Institute Artificial expression constructs for modulating gene expression in striatal neurons
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
EP4244366A4 (en) * 2020-11-10 2024-12-25 Allen Institute Artificial expression constructs for modulating gene expression in chandelier cells
EP4277524A4 (en) * 2021-01-13 2025-06-04 Duke University Compositions for and methods of improving fluid flux in the brain
US20240182923A1 (en) * 2021-03-30 2024-06-06 Allen Institute Artificial expression constructs for modulating gene expression in claustrum neurons
AU2022276538A1 (en) * 2021-05-21 2023-11-09 Allen Institute Artificial expression constructs for modulating gene expression in neurons within the thalamus
CN113736734B (zh) * 2021-09-23 2022-10-25 中山大学中山眼科中心 一种用于灵长类动物神经元分离的试剂盒及方法
WO2023108021A1 (en) * 2021-12-07 2023-06-15 Allen Institute Artificial expression constructs for modulating gene expression in neocortical layer 4 or layer 5 intratelencephalic neurons
AU2023226080A1 (en) * 2022-02-24 2024-10-03 Allen Institute Artificial expression constructs for modulating gene expression in dopaminergic neurons
CN114752624B (zh) * 2022-04-08 2025-04-15 中国海洋大学 一种侏儒蛤卵子的电穿孔方法
WO2024011224A2 (en) * 2022-07-08 2024-01-11 The Trustees Of Columbia University In The City Of New York Regulatory element for cell type specific expression of genes in spinal motor neurons
WO2024238448A2 (en) * 2023-05-12 2024-11-21 Temple University - Of The Commonwealth System Of Higher Education Chimeric capsid recombinant adeno-associated virus with bbb penetration and microglia targeting (aav-bm)
CN117346715B (zh) * 2023-12-04 2024-03-08 成都深瑞同华科技有限公司 一种配电网架空线路弧垂测定方法、系统及存储介质

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
DE69918090T2 (de) 1998-04-24 2005-06-16 University Of Florida, Gainesville Rekombinanter adeno-assoziierter viraler vektor, der alpha-1 antitrypsin kodiert, zur gentherapie
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
EP1430114B1 (en) 2001-09-14 2012-01-18 Stem Cell Therapeutics Inc. Prolactin induced increase in neural stem cell numbers and therapeutical use thereof
EP2028268A1 (en) * 2007-08-20 2009-02-25 Université Libre De Bruxelles Generation of neuronal cells from pluripotent stem cells
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
US20120308530A1 (en) 2011-06-03 2012-12-06 Beijing Allgens Medical Science & Technology Co., Ltd. Materials Composition and Methods to Control Neural Progenitor and Stem Cell Attachment, Proliferation and Guide Cell Differentiation
US9284575B2 (en) 2012-03-06 2016-03-15 Duke University Synthetic regulation of gene expression
WO2013155222A2 (en) 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
US9579399B2 (en) 2012-08-27 2017-02-28 Friedrich Miescher Institute For Biomedical Research Retinal OFF circuit-specific promoter
EP2922972B1 (en) * 2012-11-26 2017-04-26 Ecole Polytechnique Fédérale de Lausanne (EPFL) Colorectal cancer classification with differential prognosis and personalized therapeutic responses
US20150376612A1 (en) 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
CL2014003590A1 (es) * 2014-12-30 2015-07-10 Univ Chile Virus aav/xbp1s-ha, método de tratamiento genético y su uso en la optimizacion y mejoramiento de las capacidades cognitivas, de memoria y de aprendizaje.
US10682424B2 (en) 2015-03-31 2020-06-16 New York University Compositions and method for reducing seizures
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
EP3416663B1 (en) 2016-02-18 2021-04-14 The Penn State Research Foundation Generating gabaergic neurons in brains
CN117821509A (zh) * 2017-04-03 2024-04-05 编码治疗公司 组织选择性转基因表达
JP7590962B2 (ja) 2018-11-05 2024-11-27 アレン インスティテュート 皮質興奮性ニューロンにおける遺伝子発現を選択的に調節するための人工発現構築物
CA3142948A1 (en) 2019-02-15 2020-08-20 Allen Institute Artificial expression constructs for selectively modulating gene expression in selected neuronal cell populations

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Nat Neurosci.，2016年，Vol.19, No.12，p.1743-1749
Neuron，2015年，Vol.86，p.1369-1384
Prospective, brain-wide labeling of neuronal subclasses with enhancer-driven AAVs，bioRxiv，2019年01月31日，doi: https://doi.org/10.1101/525014

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EP3877529A4 (en) 2023-01-25
JP2026021360A (ja) 2026-02-10
EP3877529A1 (en) 2021-09-15
AU2025242185A1 (en) 2025-10-30
JP2022513426A (ja) 2022-02-08
WO2020097121A1 (en) 2020-05-14
AU2019375975B2 (en) 2025-07-10
US20210395780A1 (en) 2021-12-23
CA3118689A1 (en) 2020-05-14
JP2025024092A (ja) 2025-02-19
JP7774695B2 (ja) 2025-11-21
AU2019375975A1 (en) 2021-06-17
US20250236886A1 (en) 2025-07-24
US12703873B2 (en) 2026-08-11

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