JPWO2019199867A5 - - Google Patents

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JPWO2019199867A5
JPWO2019199867A5 JP2020555346A JP2020555346A JPWO2019199867A5 JP WO2019199867 A5 JPWO2019199867 A5 JP WO2019199867A5 JP 2020555346 A JP2020555346 A JP 2020555346A JP 2020555346 A JP2020555346 A JP 2020555346A JP WO2019199867 A5 JPWO2019199867 A5 JP WO2019199867A5
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Japan
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seq
expression construct
vector
composition
construct according
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JP2020555346A
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English (en)
Japanese (ja)
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JP2021521152A5 (https=
JP2021521152A (ja
JP7472036B2 (ja
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Priority claimed from PCT/US2019/026638 external-priority patent/WO2019199867A1/en
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Publication of JP2021521152A5 publication Critical patent/JP2021521152A5/ja
Priority to JP2024063287A priority Critical patent/JP7793675B2/ja
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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)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024063287A JP7793675B2 (ja) 2018-04-09 2024-04-10 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

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024063287A Division JP7793675B2 (ja) 2018-04-09 2024-04-10 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

Publications (4)

Publication Number Publication Date
JP2021521152A JP2021521152A (ja) 2021-08-26
JPWO2019199867A5 true JPWO2019199867A5 (https=) 2022-03-30
JP2021521152A5 JP2021521152A5 (https=) 2022-03-30
JP7472036B2 JP7472036B2 (ja) 2024-04-22

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ID=68164495

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020555346A Active JP7472036B2 (ja) 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
JP2024063287A Active JP7793675B2 (ja) 2018-04-09 2024-04-10 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

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JP2024063287A Active JP7793675B2 (ja) 2018-04-09 2024-04-10 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

Country Status (6)

Country Link
US (2) US12121563B2 (https=)
EP (1) EP3781174A4 (https=)
JP (2) JP7472036B2 (https=)
AU (2) AU2019253700B2 (https=)
CA (1) CA3096407A1 (https=)
WO (1) WO2019199867A1 (https=)

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EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
JP7695192B2 (ja) 2018-10-08 2025-06-18 アレン インスティテュート 介在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
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
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
CA3173609A1 (en) * 2020-03-27 2021-09-30 Allen Institute Artificial expression constructs for selectively modulating gene expression in non-neuronal brain cells
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
EP4162041A4 (en) * 2020-06-04 2024-10-16 Allen Institute ARTIFICIAL EXPRESSION CONSTRUCTS TO SELECTIVELY MODULATE GENE EXPRESSION IN INHIBITORY NEOCORTICAL NEURONS
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
US20240012004A1 (en) * 2020-11-17 2024-01-11 Merck Sharp & Dohme Llc Increased voltage-gated sodium channel alpha protein subunit expression through viral 2a-mediated co-expression of nav beta subunits
US20250325704A1 (en) 2020-12-23 2025-10-23 Boehringer Ingelheim International Gmbh Viral capsid proteins with specificity to heart tissue cells
CA3207224A1 (en) * 2021-02-02 2022-08-11 Bryan GORE Artificial expression constructs for modulating gene expression in gabaergic neurons and astrocytes
TW202313972A (zh) * 2021-06-03 2023-04-01 荷蘭商美勒斯公司 新nrg1融合物、融合接合處及檢測彼等之方法
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
AU2024214841A1 (en) * 2023-02-01 2025-08-28 Allen Institute Intein-mediated reconstitution of voltage-gated sodium channel function
WO2025019685A2 (en) * 2023-07-18 2025-01-23 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

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WO2001036623A2 (en) 1999-11-05 2001-05-25 Avigen, Inc. Ecdysone-inducible adeno-associated virus expression vectors
WO2002064804A2 (en) 2001-02-13 2002-08-22 University Of Florida A bi-directional dual promoter complex with enhanced promoter activity for transgene expression in eukaryotes
WO2003072751A2 (en) 2002-02-25 2003-09-04 Vanderbilt University Expression system for human brain-specific voltage-gated sodium channel, type 1
WO2009012176A2 (en) * 2007-07-14 2009-01-22 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
WO2013142337A1 (en) 2012-03-20 2013-09-26 The Trustees Of The University Of Pennsylvania Human papillomavirus 16 (hpv16) - related epilepsy
EP2888368A1 (en) 2012-08-27 2015-07-01 Friedrich Miescher Institute for Biomedical Research Retinal off circuit-specific promoter
DE102014207498A1 (de) 2014-04-17 2015-10-22 Universitätsklinikum Hamburg-Eppendorf Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark
WO2016161124A1 (en) * 2015-03-31 2016-10-06 New York University Compositions and method for reducing seizures
DK3081263T3 (da) 2015-04-17 2020-03-16 P&X Medical Nv Sammensætninger og redskaber til behandling af glaukom
WO2016186772A2 (en) 2015-05-16 2016-11-24 Genzyme Corporation Gene editing of deep intronic mutations
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
EA201992358A1 (ru) * 2017-04-03 2020-03-24 Инкоудид Терапьютикс, Инк. Тканеселективная экспрессия трансгена
JP7695192B2 (ja) 2018-10-08 2025-06-18 アレン インスティテュート 介在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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