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
3xha
vector
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JPWO2019199867A5 (https=
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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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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
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    • C12N15/86—Viral vectors
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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
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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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  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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 (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

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
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
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
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
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

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

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