JP2022528010A5 - - Google Patents
Info
- Publication number
- JP2022528010A5 JP2022528010A5 JP2021560728A JP2021560728A JP2022528010A5 JP 2022528010 A5 JP2022528010 A5 JP 2022528010A5 JP 2021560728 A JP2021560728 A JP 2021560728A JP 2021560728 A JP2021560728 A JP 2021560728A JP 2022528010 A5 JP2022528010 A5 JP 2022528010A5
- Authority
- JP
- Japan
- Prior art keywords
- host cell
- isolated nucleic
- capsid protein
- nucleic acid
- cell according
- 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 (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962833159P | 2019-04-12 | 2019-04-12 | |
| US62/833,159 | 2019-04-12 | ||
| US201962930687P | 2019-11-05 | 2019-11-05 | |
| US62/930,687 | 2019-11-05 | ||
| PCT/US2020/027622 WO2020210595A1 (en) | 2019-04-12 | 2020-04-10 | Aav-mediated gene therapy for maple syrup urine disease (msud) |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022528010A JP2022528010A (ja) | 2022-06-07 |
| JP2022528010A5 true JP2022528010A5 (https=) | 2023-04-18 |
Family
ID=72752067
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021560728A Pending JP2022528010A (ja) | 2019-04-12 | 2020-04-10 | メープルシロップ尿症（ｍｓｕｄ）のａａｖ媒介遺伝子治療 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US12398378B2 (https=) |
| EP (1) | EP3953484A4 (https=) |
| JP (1) | JP2022528010A (https=) |
| CN (1) | CN114008209A (https=) |
| WO (1) | WO2020210595A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022528010A (ja) | 2019-04-12 | 2022-06-07 | ユニバーシティ オブ マサチューセッツ | メープルシロップ尿症（ｍｓｕｄ）のａａｖ媒介遺伝子治療 |
| WO2020257731A1 (en) * | 2019-06-20 | 2020-12-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of maple syrup urine disease |
| JP2023515820A (ja) * | 2020-02-28 | 2023-04-14 | アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル | メープルシロップ尿症の遺伝子治療 |
| US20240207370A1 (en) * | 2021-04-26 | 2024-06-27 | University Of Massachusetts | Gene therapy for bcaa modulation in maple syrup urine disease (msud) |
| CN114561333B (zh) * | 2022-02-23 | 2023-08-25 | 山东第一医科大学(山东省医学科学院) | 一种转化支链氨基酸的工程菌及在制备治疗枫糖尿症产品中的应用 |
| WO2025212609A1 (en) * | 2024-04-02 | 2025-10-09 | University Of Massachusetts | Evolved nucleic acids encoding aspartoacylase proteins |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1331092A (zh) | 2000-06-28 | 2002-01-16 | 上海博德基因开发有限公司 | 一种新的多肽——环指基因mid1蛋白10.56和编码这种多肽的多核苷酸 |
| TR201908020T4 (tr) | 2011-02-17 | 2019-06-21 | Univ Pennsylvania | Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler. |
| CN104099349B (zh) | 2013-04-08 | 2018-07-24 | 深圳华大基因股份有限公司 | 枫糖尿病相关基因突变、其检测方法及其用途 |
| CN107073051B (zh) | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| JP2018509387A (ja) * | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
| EP3411484B1 (en) * | 2016-02-05 | 2023-10-04 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| WO2018016884A1 (ko) | 2016-07-20 | 2018-01-25 | 주식회사 녹십자지놈 | 선천성 기능장애 진단용 조성물 및 이의 용도 |
| WO2018185110A1 (en) | 2017-04-03 | 2018-10-11 | Fit Biotech Oy | Novel expression vectors and uses thereof |
| WO2019071048A1 (en) * | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION |
| JP2022528010A (ja) | 2019-04-12 | 2022-06-07 | ユニバーシティ オブ マサチューセッツ | メープルシロップ尿症（ｍｓｕｄ）のａａｖ媒介遺伝子治療 |
| US20240207370A1 (en) | 2021-04-26 | 2024-06-27 | University Of Massachusetts | Gene therapy for bcaa modulation in maple syrup urine disease (msud) |
-
2020
- 2020-04-10 JP JP2021560728A patent/JP2022528010A/ja active Pending
- 2020-04-10 CN CN202080042483.5A patent/CN114008209A/zh active Pending
- 2020-04-10 WO PCT/US2020/027622 patent/WO2020210595A1/en not_active Ceased
- 2020-04-10 EP EP20787676.4A patent/EP3953484A4/en active Pending
- 2020-04-10 US US17/602,353 patent/US12398378B2/en active Active
-
2025
- 2025-07-29 US US19/283,401 patent/US20260062682A1/en active Pending
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