JP2023539631A5 - - Google Patents
Info
- Publication number
- JP2023539631A5 JP2023539631A5 JP2023513722A JP2023513722A JP2023539631A5 JP 2023539631 A5 JP2023539631 A5 JP 2023539631A5 JP 2023513722 A JP2023513722 A JP 2023513722A JP 2023513722 A JP2023513722 A JP 2023513722A JP 2023539631 A5 JP2023539631 A5 JP 2023539631A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- polynucleotide according
- vector
- sequence encoding
- base 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063072327P | 2020-08-31 | 2020-08-31 | |
| US63/072,327 | 2020-08-31 | ||
| US202163235359P | 2021-08-20 | 2021-08-20 | |
| US63/235,359 | 2021-08-20 | ||
| PCT/JP2021/031973 WO2022045366A1 (en) | 2020-08-31 | 2021-08-31 | Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023539631A JP2023539631A (ja) | 2023-09-15 |
| JP2023539631A5 true JP2023539631A5 (https=) | 2024-09-05 |
| JP7736329B2 JP7736329B2 (ja) | 2025-09-09 |
Family
ID=80353460
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023513722A Active JP7736329B2 (ja) | 2020-08-31 | 2021-08-31 | Ｄｕｘ４遺伝子を標的とした顔面肩甲上腕型筋ジストロフィーの治療方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20230323456A1 (https=) |
| EP (1) | EP4204556A4 (https=) |
| JP (1) | JP7736329B2 (https=) |
| CN (1) | CN116194578A (https=) |
| WO (1) | WO2022045366A1 (https=) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20230023612A (ko) | 2020-04-02 | 2023-02-17 | 마이레큘, 인크. | 조작된 올리고뉴클레오티드를 사용한 표적화된 억제 |
| CA3189065A1 (en) | 2020-09-11 | 2022-03-17 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
| CN121127598A (zh) * | 2023-03-16 | 2025-12-12 | 埃皮克里斯皮尔生物技术公司 | 用于调控异常基因表达的系统和方法 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3515506B1 (en) | 2016-09-23 | 2023-09-13 | University of Massachusetts | Silencing of dux4 by recombinant gene editing complexes |
| US11160823B2 (en) * | 2016-11-07 | 2021-11-02 | University Of Massachusetts | Therapeutic targets for facioscapulohumeral muscular dystrophy |
| JP7412001B2 (ja) * | 2018-06-08 | 2024-01-12 | 株式会社モダリス | 改変されたＣａｓ９タンパク質及びその用途 |
| CN113166738A (zh) * | 2018-10-24 | 2021-07-23 | 摩大力斯医疗株式会社 | 经改造的Cas9蛋白及其用途 |
| AU2021257213A1 (en) * | 2020-04-17 | 2022-11-03 | Nevada Research & Innovation Corporation | CRISPR-inhibition for facioscapulohumeral muscular dystrophy |
-
2021
- 2021-08-31 WO PCT/JP2021/031973 patent/WO2022045366A1/en not_active Ceased
- 2021-08-31 EP EP21861786.8A patent/EP4204556A4/en active Pending
- 2021-08-31 JP JP2023513722A patent/JP7736329B2/ja active Active
- 2021-08-31 CN CN202180050873.1A patent/CN116194578A/zh active Pending
- 2021-08-31 US US18/043,124 patent/US20230323456A1/en active Pending
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