JP2024513907A5 - - Google Patents
Info
- Publication number
- JP2024513907A5 JP2024513907A5 JP2023561616A JP2023561616A JP2024513907A5 JP 2024513907 A5 JP2024513907 A5 JP 2024513907A5 JP 2023561616 A JP2023561616 A JP 2023561616A JP 2023561616 A JP2023561616 A JP 2023561616A JP 2024513907 A5 JP2024513907 A5 JP 2024513907A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- msec
- enhancer
- miniaturized
- promoter
- 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 |
|---|---|---|---|
| US202163173295P | 2021-04-09 | 2021-04-09 | |
| US63/173,295 | 2021-04-09 | ||
| PCT/US2022/023915 WO2022216988A2 (en) | 2021-04-09 | 2022-04-07 | Artificial regulatory cassettes for muscle-specific gene expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024513907A JP2024513907A (ja) | 2024-03-27 |
| JP2024513907A5 true JP2024513907A5 (https=) | 2025-04-15 |
Family
ID=83546588
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023561616A Pending JP2024513907A (ja) | 2021-04-09 | 2022-04-07 | 筋特異的遺伝子発現用の人工調節カセット |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20240200093A1 (https=) |
| EP (2) | EP4707401A2 (https=) |
| JP (1) | JP2024513907A (https=) |
| WO (1) | WO2022216988A2 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024216116A1 (en) * | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| WO2024216118A1 (en) * | 2023-04-14 | 2024-10-17 | Precision Biosciences, Inc. | Muscle-specific expression cassettes |
| KR20260035966A (ko) * | 2023-07-10 | 2026-03-13 | 케이트 테라퓨틱스, 인크. | Lgmd2b/디스펄린병증을 치료하기 위한 aav 유전자 치료 접근법 |
| WO2025129079A1 (en) * | 2023-12-13 | 2025-06-19 | Tenaya Therapeutics, Inc. | Cardiac-specific promoters for gene therapy |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6183984B1 (en) * | 1991-11-12 | 2001-02-06 | Arch Development Corporation | Sequences for promoting epidermal cell-specific transcription |
| US6967019B2 (en) * | 1999-04-06 | 2005-11-22 | The Regents Of The University Of California | Production of pancreatic islet cells and delivery of insulin |
| ATE373095T1 (de) * | 2000-07-14 | 2007-09-15 | Univ Michigan | Mutierte muskelspezifische enhancer |
| WO2002077161A2 (en) * | 2001-03-12 | 2002-10-03 | Progenetics Llc | Production of high levels of trangenic factor ix without gene rescue, and its therapeutic uses |
| US10196636B2 (en) * | 2011-04-21 | 2019-02-05 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of myotilin |
| US20130136729A1 (en) * | 2011-11-11 | 2013-05-30 | University of Virginia Patent Foundation, d/b/a University of Virginia Licensing & Ventures Group | Compositions and methods for targeting and treating diseases and injuries using adeno-associated virus vectors |
| WO2013102904A1 (en) * | 2012-01-05 | 2013-07-11 | Hadasit Medical Research Services & Development Ltd. | Methods and compositions for gene delivery |
| JP6832280B2 (ja) * | 2015-01-16 | 2021-02-24 | ユニバーシティ オブ ワシントンＵｎｉｖｅｒｓｉｔｙ ｏｆ Ｗａｓｈｉｎｇｔｏｎ | 新規のマイクロジストロフィンおよび使用の関連する方法 |
| EP3405579A1 (en) * | 2016-01-22 | 2018-11-28 | Modernatx, Inc. | Messenger ribonucleic acids for the production of intracellular binding polypeptides and methods of use thereof |
| EP4004214A1 (en) * | 2019-07-26 | 2022-06-01 | RegenxBio Inc. | Engineered nucleic acid regulatory element and methods of uses thereof |
-
2022
- 2022-04-07 EP EP25215724.3A patent/EP4707401A2/en active Pending
- 2022-04-07 US US18/286,215 patent/US20240200093A1/en active Pending
- 2022-04-07 JP JP2023561616A patent/JP2024513907A/ja active Pending
- 2022-04-07 EP EP22785484.1A patent/EP4319874A4/en not_active Withdrawn
- 2022-04-07 WO PCT/US2022/023915 patent/WO2022216988A2/en not_active Ceased
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