JP2021521152A5 - - Google Patents
Info
- Publication number
- JP2021521152A5 JP2021521152A5 JP2020555346A JP2020555346A JP2021521152A5 JP 2021521152 A5 JP2021521152 A5 JP 2021521152A5 JP 2020555346 A JP2020555346 A JP 2020555346A JP 2020555346 A JP2020555346 A JP 2020555346A JP 2021521152 A5 JP2021521152 A5 JP 2021521152A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- expression construct
- vector
- construct according
- sequence
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
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 (3)
| Publication Number | Publication Date |
|---|---|
| JP2021521152A JP2021521152A (ja) | 2021-08-26 |
| JP2021521152A5 true JP2021521152A5 (https=) | 2022-03-30 |
| JP7472036B2 JP7472036B2 (ja) | 2024-04-22 |
Family
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 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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=) |
Families Citing this family (22)
| 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 |
Family Cites Families (13)
| 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 |
| CA2474751A1 (en) * | 2002-02-25 | 2003-09-04 | Vanderbilt University | Expression system for human brain-specific voltage-gated sodium channel, type 1 |
| EP2176283B1 (en) * | 2007-07-14 | 2016-11-02 | University of Iowa Research Foundation | Methods and compositions for treating brain diseases |
| 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 |
| DE102014207498A1 (de) * | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| US10682424B2 (en) | 2015-03-31 | 2020-06-16 | 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 |
| BR112017024514A2 (pt) | 2015-05-16 | 2018-07-24 | Genzyme Corporation | edição genética de mutações intrônicas profundas |
| KR102423442B1 (ko) * | 2015-12-11 | 2022-07-20 | 캘리포니아 인스티튜트 오브 테크놀로지 | 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드 |
| 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 |
-
2019
- 2019-04-09 CA CA3096407A patent/CA3096407A1/en active Pending
- 2019-04-09 AU AU2019253700A patent/AU2019253700B2/en active Active
- 2019-04-09 WO PCT/US2019/026638 patent/WO2019199867A1/en not_active Ceased
- 2019-04-09 JP JP2020555346A patent/JP7472036B2/ja active Active
- 2019-04-09 US US17/044,232 patent/US12121563B2/en active Active
- 2019-04-09 EP EP19785199.1A patent/EP3781174A4/en active Pending
-
2024
- 2024-04-10 JP JP2024063287A patent/JP7793675B2/ja active Active
- 2024-10-18 US US18/920,747 patent/US20250041384A1/en active Pending
-
2025
- 2025-11-07 AU AU2025263840A patent/AU2025263840A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021521152A5 (https=) | ||
| JP7655700B2 (ja) | 選択的遺伝子治療発現系 | |
| JP7807825B2 (ja) | 医薬組成物 | |
| CN111902539B (zh) | 杂合调控元件 | |
| ES2768763T3 (es) | Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR | |
| ES2989774T3 (es) | Factor IX modificado, y composiciones, métodos y utilizaciones para la transferencia génica a células, órganos y tejidos | |
| CN120324641A (zh) | 用于治疗达农病的基因治疗载体 | |
| ES2814280T3 (es) | Terapia génica basada en VAA para la esclerosis múltiple | |
| WO2020208032A1 (en) | Hybrid promoters for muscle expression | |
| JP2023500793A5 (https=) | ||
| JP2023544264A (ja) | 核酸構築物、ウイルスベクター及びウイルス粒子 | |
| CN116656740A (zh) | 一种腺相关病毒基因治疗载体及其在制备治疗苯丙酮尿症的药物中的应用 | |
| CN113795504B (zh) | 具有心脏保护活性的蛋白质 | |
| EP4410988A1 (en) | An aav2-vector variant for targeted transfer of genes | |
| EP4602061A1 (en) | Atp7b gene therapy | |
| US20260124322A1 (en) | Atp7b gene therapy | |
| HK40096493A (zh) | Danon病和其它自噬障碍的治疗方法 | |
| RU2777571C2 (ru) | Способы лечения болезни данона и других нарушений аутофагии | |
| WO2025212993A1 (en) | Materials and methods for trangene expression in neural cells | |
| EA052984B1 (ru) | Генотерапия aqp1 для предупреждения гипофункции слюнной железы, вызываемой облучением | |
| BR112018014633B1 (pt) | Vetor de terapia gênica e usos de vetores | |
| BR112017027956B1 (pt) | Vetores de vírus adenoassociado recombinante, uso e método de produção dos mesmos, e composição farmacêutica |