JP7472036B2 - 抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー - Google Patents

抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー Download PDF

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JP7472036B2
JP7472036B2 JP2020555346A JP2020555346A JP7472036B2 JP 7472036 B2 JP7472036 B2 JP 7472036B2 JP 2020555346 A JP2020555346 A JP 2020555346A JP 2020555346 A JP2020555346 A JP 2020555346A JP 7472036 B2 JP7472036 B2 JP 7472036B2
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expression construct
scn1a
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vector
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ミッチ，ジョン・ケイ
レイン，エドワード・セバスチャン
ティン，ジョナサン
リーバイ，ボアズ・ピー
ヘス，エリク
カルム，フランク
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アレン インスティテュート
シアトル チルドレンズ ホスピタル ドゥーイング ビジネス アズ シアトル チルドレンズ リサーチ インスティテュート
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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JP2020555346A 2018-04-09 2019-04-09 抑制性ニューロンにおける電位開口型ナトリウムチャネルのレスキュー Active JP7472036B2 (ja)

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US201862655043P 2018-04-09 2018-04-09
US62/655,043 2018-04-09
US201862742835P 2018-10-08 2018-10-08
US62/742,835 2018-10-08
US201962810281P 2019-02-25 2019-02-25
US62/810,281 2019-02-25
PCT/US2019/026638 WO2019199867A1 (en) 2018-04-09 2019-04-09 Rescuing voltage-gated sodium channel function in inhibitory neurons

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Families Citing this family (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2020076614A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons
JP7624728B2 (ja) 2019-02-25 2025-01-31 ユニバーシティ オブ マサチューセッツ Ｄｎａ結合ドメイントランスアクチベーター及びその使用
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CA3173609A1 (en) * 2020-03-27 2021-09-30 Allen Institute Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
JP7767324B2 (ja) * 2020-06-04 2025-11-11 アレン インスティテュート 抑制性新皮質ニューロンにおいて遺伝子発現を選択的に調節する人工発現構築物
WO2022020706A1 (en) * 2020-07-24 2022-01-27 University Of Massachusetts Dna-binding domain transactivators and uses thereof
GB202013940D0 (en) 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences
WO2022108832A1 (en) * 2020-11-17 2022-05-27 Merck Sharp & Dohme Corp. Increased voltage-gated sodium channel alpha protein subunit expression through viral 2a-mediated co-expression of nav beta subunits
JP2024501821A (ja) 2020-12-23 2024-01-16 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング 心臓組織細胞に対する特異性を有するウイルスカプシドタンパク質
AU2022217778A1 (en) * 2021-02-02 2023-08-31 Allen Institute Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
JP2024523001A (ja) * 2021-06-03 2024-06-25 メルス ナムローゼ フェンノートシャップ 新たなｎｒｇ１融合体、融合接合部及びそれらを検出するための方法
JP2025520385A (ja) * 2022-06-13 2025-07-03 アレン インスティテュート 中枢神経系の非神経細胞において遺伝子発現を調節するための人工発現コンストラクト
JP2026504390A (ja) * 2023-02-01 2026-02-05 アレン インスティテュート インテインを介した電位依存性ナトリウムチャネルの機能の再構築
AU2024293430A1 (en) * 2023-07-18 2025-12-11 Allen Institute Antisense oligonucleotides and artificial expression constructs for expressing rna in inhibitory neurons
WO2025038955A1 (en) * 2023-08-16 2025-02-20 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
WO2025092951A1 (en) * 2023-11-03 2025-05-08 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating fabry disease
WO2026015848A1 (en) * 2024-07-11 2026-01-15 Bionic Sight, Llc Depolarizing ion channel therapeutics for treating neurodegenerative diseases

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050260576A1 (en) 2002-02-25 2005-11-24 Vanderbilt University Expression system for human brain-specific voltage-gated sodium channel, type 1
US20090162332A1 (en) 2007-07-14 2009-06-25 Davidson Beverly L Methods and compositions for treating brain diseases
US20170029464A1 (en) 2014-04-17 2017-02-02 Universitätsklinikum Hamburg-Eppendorf Viral vector for the targeted transfer of genes in the brain and spinal cord
US20170166926A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
US20180078658A1 (en) 2015-03-31 2018-03-22 New York University Compositions and method for reducing seizures

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU3642601A (en) 1999-11-05 2001-05-30 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
EP1360310A2 (en) * 2001-02-13 2003-11-12 University Of Florida A bi-directional dual promoter complex with enhanced promoter activity for transgene expression in eukaryotes
US20150044667A1 (en) 2012-03-20 2015-02-12 The Trustees Of The University Of Pennsylvania Human papillomavirus 16 (hpv16) - related epilepsy
WO2014033095A1 (en) 2012-08-27 2014-03-06 Friedrich Miescher Institute For Biomedical Research Retinal off circuit-specific promoter
DK3081263T3 (da) * 2015-04-17 2020-03-16 P&X Medical Nv Sammensætninger og redskaber til behandling af glaukom
BR112017024514A2 (pt) 2015-05-16 2018-07-24 Genzyme Corporation edição genética de mutações intrônicas profundas
MX2019011772A (es) * 2017-04-03 2020-01-09 Encoded Therapeutics Inc Expresion transgenica selectiva de tejidos.
WO2020076614A1 (en) 2018-10-08 2020-04-16 Allen Institute Artificial expression constructs for selectively modulating gene expression in interneurons

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050260576A1 (en) 2002-02-25 2005-11-24 Vanderbilt University Expression system for human brain-specific voltage-gated sodium channel, type 1
US20090162332A1 (en) 2007-07-14 2009-06-25 Davidson Beverly L Methods and compositions for treating brain diseases
US20170029464A1 (en) 2014-04-17 2017-02-02 Universitätsklinikum Hamburg-Eppendorf Viral vector for the targeted transfer of genes in the brain and spinal cord
US20180078658A1 (en) 2015-03-31 2018-03-22 New York University Compositions and method for reducing seizures
US20170166926A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)
WO2017100671A1 (en) 2015-12-11 2017-06-15 California Institute Of Technology TARGETING PEPTIDES FOR DIRECTING ADENO-ASSOCIATED VIRUSES (AAVs)

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Progress in Retinal and Eye Research，2013年，Vol.32，p.22-47

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JP7793675B2 (ja) 2026-01-05
AU2025263840A1 (en) 2026-01-15
AU2019253700A2 (en) 2020-12-03
CA3096407A1 (en) 2019-10-17
JP2024095779A (ja) 2024-07-10
EP3781174A4 (en) 2022-01-26
WO2019199867A1 (en) 2019-10-17
AU2019253700A1 (en) 2020-11-26
US12121563B2 (en) 2024-10-22
JP2021521152A (ja) 2021-08-26
AU2019253700B2 (en) 2025-08-14
US20250041384A1 (en) 2025-02-06
EP3781174A1 (en) 2021-02-24
US20210015898A1 (en) 2021-01-21

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