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 (enExample) | 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 (enExample) |
| EP (1) | EP3953484A4 (enExample) |
| JP (1) | JP2022528010A (enExample) |
| CN (1) | CN114008209A (enExample) |
| WO (1) | WO2020210595A1 (enExample) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3953484A4 (en) | 2019-04-12 | 2023-04-12 | University of Massachusetts | Aav-mediated gene therapy for maple syrup urine disease (msud) |
| US20220370638A1 (en) * | 2019-06-20 | 2022-11-24 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of maple syrup urine disease |
| US20230136699A1 (en) * | 2020-02-28 | 2023-05-04 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Gene therapy for maple syrup urine disease |
| EP4330410A4 (en) * | 2021-04-26 | 2025-04-23 | University of Massachusetts | Gene therapy for BCAA modulation in maple syrup urine disease (MSUD) |
| US12285497B2 (en) * | 2021-10-15 | 2025-04-29 | Research Institute At Nationwide Children's Hospital | Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy |
| US20230279431A1 (en) * | 2021-11-30 | 2023-09-07 | Research Institute At Nationwide Children's Hospital | Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy |
| 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 |
| CN103502458B (zh) * | 2011-02-17 | 2016-11-16 | 宾夕法尼亚大学托管会 | 用于改变组织特异性和改善aav9-介导的基因转移的组合物和方法 |
| 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 |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| EP3247328B1 (en) | 2015-01-21 | 2026-01-07 | Genevant Sciences Gmbh | Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells |
| HRP20231451T1 (hr) * | 2016-02-05 | 2024-03-01 | Emory University | Ubrizgavanje jednolančanog ili samo-komplementarnog adeno-povezanog virusa 9 u cerebrospinalnu tekućinu |
| 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 |
| KR20200074104A (ko) * | 2017-10-04 | 2020-06-24 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물 |
| EP3953484A4 (en) | 2019-04-12 | 2023-04-12 | University of Massachusetts | Aav-mediated gene therapy for maple syrup urine disease (msud) |
| EP4330410A4 (en) | 2021-04-26 | 2025-04-23 | University of Massachusetts | Gene therapy for BCAA modulation in maple syrup urine disease (MSUD) |
-
2020
- 2020-04-10 EP EP20787676.4A patent/EP3953484A4/en active Pending
- 2020-04-10 JP JP2021560728A patent/JP2022528010A/ja active Pending
- 2020-04-10 CN CN202080042483.5A patent/CN114008209A/zh active Pending
- 2020-04-10 US US17/602,353 patent/US12398378B2/en active Active
- 2020-04-10 WO PCT/US2020/027622 patent/WO2020210595A1/en not_active Ceased
-
2025
- 2025-07-29 US US19/283,401 patent/US20260062682A1/en active Pending
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