JP2023522020A5 - - Google Patents
Info
- Publication number
- JP2023522020A5 JP2023522020A5 JP2022562629A JP2022562629A JP2023522020A5 JP 2023522020 A5 JP2023522020 A5 JP 2023522020A5 JP 2022562629 A JP2022562629 A JP 2022562629A JP 2022562629 A JP2022562629 A JP 2022562629A JP 2023522020 A5 JP2023522020 A5 JP 2023522020A5
- Authority
- JP
- Japan
- Prior art keywords
- fusion polypeptide
- sgrna
- polynucleotide
- seq
- repressor
- 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.)
- Pending
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063011476P | 2020-04-17 | 2020-04-17 | |
| US63/011,476 | 2020-04-17 | ||
| PCT/US2021/025940 WO2021211325A1 (en) | 2020-04-17 | 2021-04-06 | Crispr-inhibition for facioscapulohumeral muscular dystrophy |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023522020A JP2023522020A (ja) | 2023-05-26 |
| JP2023522020A5 true JP2023522020A5 (https=) | 2024-04-19 |
Family
ID=78084611
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022562629A Pending JP2023522020A (ja) | 2020-04-17 | 2021-04-06 | 顔面肩甲上腕型筋ジストロフィーのためのｃｒｉｓｐｒ阻害 |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230174958A1 (https=) |
| EP (1) | EP4135778A4 (https=) |
| JP (1) | JP2023522020A (https=) |
| KR (1) | KR20230003511A (https=) |
| CN (1) | CN115768487A (https=) |
| AU (1) | AU2021257213A1 (https=) |
| BR (1) | BR112022020945A2 (https=) |
| CA (1) | CA3175625A1 (https=) |
| IL (1) | IL297113A (https=) |
| MX (1) | MX2022012965A (https=) |
| WO (1) | WO2021211325A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20230023612A (ko) | 2020-04-02 | 2023-02-17 | 마이레큘, 인크. | 조작된 올리고뉴클레오티드를 사용한 표적화된 억제 |
| WO2022045366A1 (en) * | 2020-08-31 | 2022-03-03 | Modalis Therapeutics Corporation | Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene |
| WO2024020444A2 (en) * | 2022-07-20 | 2024-01-25 | Nevada Research & Innovation Corporation | Muscle-specific regulatory cassettes |
| WO2025019820A1 (en) * | 2023-07-19 | 2025-01-23 | Nevada Research & Innovation Corporation | All in one vectors for the treatment of facioscapulohumeral muscular dystrophy |
| CN117448380A (zh) * | 2023-12-22 | 2024-01-26 | 上海元戊医学技术有限公司 | iPSC来源的COL10A1蛋白低表达MSC细胞株的构建方法及其应用 |
| WO2026039783A1 (en) * | 2024-08-16 | 2026-02-19 | Sarepta Therapeutics, Inc. | Deficient cas-transcriptional repressor fusions for regulation of gene expression |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3192880B1 (en) * | 2010-08-18 | 2019-10-09 | Fred Hutchinson Cancer Research Center | Nucleic acid agents for use in treating facioscapulohumeral dystrophy (fshd) |
| JP7103750B2 (ja) * | 2013-12-12 | 2022-07-20 | ザ・ブロード・インスティテュート・インコーポレイテッド | ゲノム編集のためのＣＲＩＳＰＲ－Ｃａｓ系及び組成物の送達、使用及び治療適用 |
| PL3097197T3 (pl) * | 2014-01-21 | 2021-06-28 | Vrije Universiteit Brussel | Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie |
| 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 |
| WO2019051290A1 (en) * | 2017-09-07 | 2019-03-14 | The Children's Medical Center Corporation | COMPOSITIONS AND METHODS FOR THE TREATMENT OF FACIO-SCAPULO-HUMERAL DYSTROPHY |
| BR112020006677A2 (pt) * | 2017-10-05 | 2020-10-06 | Fulcrum Therapeutics, Inc. | uso de inibidores p38 para reduzir a expressão de dux4 |
-
2021
- 2021-04-06 AU AU2021257213A patent/AU2021257213A1/en active Pending
- 2021-04-06 CN CN202180041592.XA patent/CN115768487A/zh active Pending
- 2021-04-06 JP JP2022562629A patent/JP2023522020A/ja active Pending
- 2021-04-06 CA CA3175625A patent/CA3175625A1/en active Pending
- 2021-04-06 KR KR1020227039447A patent/KR20230003511A/ko active Pending
- 2021-04-06 WO PCT/US2021/025940 patent/WO2021211325A1/en not_active Ceased
- 2021-04-06 BR BR112022020945A patent/BR112022020945A2/pt unknown
- 2021-04-06 IL IL297113A patent/IL297113A/en unknown
- 2021-04-06 US US17/919,198 patent/US20230174958A1/en active Pending
- 2021-04-06 MX MX2022012965A patent/MX2022012965A/es unknown
- 2021-04-06 EP EP21787712.5A patent/EP4135778A4/en active Pending
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