JP2024505027A - Ｇａｂａ作動性ニューロン及び星状膠細胞における遺伝子発現の調節のための人工発現構築物 - Google Patents

Ｇａｂａ作動性ニューロン及び星状膠細胞における遺伝子発現の調節のための人工発現構築物 Download PDF

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JP2024505027A
JP2024505027A JP2023545204A JP2023545204A JP2024505027A JP 2024505027 A JP2024505027 A JP 2024505027A JP 2023545204 A JP2023545204 A JP 2023545204A JP 2023545204 A JP2023545204 A JP 2023545204A JP 2024505027 A JP2024505027 A JP 2024505027A
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seq
ehgt
expression construct
enhancer
artificial expression
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JPWO2022169884A5 (https=
JP2024505027A5 (https=
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ゴア，ブライアン
セバスティアン レイン，エドワード
ピー． レヴィ，ボアズ
メイチェン，デジャ
マーティネス，レフュジオ
ケー． ミック，ジョン
ティン，ジョナサン
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アレン インスティテュート
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JP2023545204A 2021-02-02 2022-02-02 Ｇａｂａ作動性ニューロン及び星状膠細胞における遺伝子発現の調節のための人工発現構築物 Pending JP2024505027A (ja)

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US202163144743P 2021-02-02 2021-02-02
US63/144,743 2021-02-02
PCT/US2022/014956 WO2022169884A2 (en) 2021-02-02 2022-02-02 Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes

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JP2024505027A true JP2024505027A (ja) 2024-02-02
JPWO2022169884A5 JPWO2022169884A5 (https=) 2025-02-10
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US (1) US20240117377A1 (https=)
EP (1) EP4288064A4 (https=)
JP (1) JP2024505027A (https=)
AU (1) AU2022217778A1 (https=)
CA (1) CA3207224A1 (https=)
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WO2025019685A2 (en) * 2023-07-18 2025-01-23 Allen Institute Antisense oligonucleotides and artificial expression constructs for expressing rna in inhibitory neurons
AU2024346721A1 (en) * 2023-09-18 2026-04-09 Allen Institute Artificial expression constructs for modulating gene expression in neurons and astrocytes for the assessment and/or treatment of slc6a1-related disorders

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013155222A2 (en) * 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
WO2019199867A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2020076614A1 (en) * 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2021195591A2 (en) * 2020-03-27 2021-09-30 Allen Institute Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells

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* Cited by examiner, † Cited by third party
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JP4955540B2 (ja) * 2004-06-04 2012-06-20 フラクスオーム サイエンセズ エイ／エス 多価不飽和脂肪酸生産のための代謝改変細胞
EP1859060A2 (en) * 2005-02-28 2007-11-28 Integragen Human autism susceptibility genes encoding a neurotransmitter transporter and uses thereof
EP3260540A1 (en) * 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
PE20170261A1 (es) * 2014-05-13 2017-04-12 Univ Pennsylvania Composiciones que comprenden virus adenoasociado (aav) que expresa constructos de anticuerpos duales y sus usos
CN110546257B (zh) * 2017-03-17 2024-03-01 阿德夫拉姆生物技术股份有限公司 用于增强基因表达的组合物和方法

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013155222A2 (en) * 2012-04-10 2013-10-17 The Regents Of The University Of California Brain-specific enhancers for cell-based therapy
WO2019199867A1 (en) * 2018-04-09 2019-10-17 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
WO2020076614A1 (en) * 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
WO2021195591A2 (en) * 2020-03-27 2021-09-30 Allen Institute Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells

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WO2022169884A3 (en) 2022-09-15
CA3207224A1 (en) 2022-08-11
US20240117377A1 (en) 2024-04-11
EP4288064A4 (en) 2025-01-15
AU2022217778A1 (en) 2023-08-31
WO2022169884A2 (en) 2022-08-11
AU2022217778A9 (en) 2023-09-28

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