JP2021522858A5 - - Google Patents
Info
- Publication number
- JP2021522858A5 JP2021522858A5 JP2021510297A JP2021510297A JP2021522858A5 JP 2021522858 A5 JP2021522858 A5 JP 2021522858A5 JP 2021510297 A JP2021510297 A JP 2021510297A JP 2021510297 A JP2021510297 A JP 2021510297A JP 2021522858 A5 JP2021522858 A5 JP 2021522858A5
- Authority
- JP
- Japan
- Prior art keywords
- composition according
- nuclease
- cells
- donor template
- virus
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024085010A JP2024100980A (ja) | 2018-05-03 | 2024-05-24 | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862666685P | 2018-05-03 | 2018-05-03 | |
| US62/666,685 | 2018-05-03 | ||
| PCT/US2019/030748 WO2019213626A1 (en) | 2018-05-03 | 2019-05-03 | In vivo homology directed repair in heart, skeletal muscle, and muscle stem cells |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024085010A Division JP2024100980A (ja) | 2018-05-03 | 2024-05-24 | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021522858A JP2021522858A (ja) | 2021-09-02 |
| JP2021522858A5 true JP2021522858A5 (enExample) | 2022-05-10 |
| JP7498499B2 JP7498499B2 (ja) | 2024-06-12 |
Family
ID=68386147
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021510297A Active JP7498499B2 (ja) | 2018-05-03 | 2019-05-03 | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 |
| JP2024085010A Pending JP2024100980A (ja) | 2018-05-03 | 2024-05-24 | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024085010A Pending JP2024100980A (ja) | 2018-05-03 | 2024-05-24 | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20210363546A1 (enExample) |
| EP (1) | EP3787692A4 (enExample) |
| JP (2) | JP7498499B2 (enExample) |
| CN (1) | CN112512595A (enExample) |
| AU (1) | AU2019262225B2 (enExample) |
| CA (1) | CA3099332A1 (enExample) |
| WO (1) | WO2019213626A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| JP7490211B2 (ja) | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| KR20210058816A (ko) | 2018-08-18 | 2021-05-24 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 원 위치에 유전자 편집 |
| EP4125349A4 (en) * | 2020-04-27 | 2024-07-10 | Duke University | GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS |
| CN115181722B (zh) * | 2022-08-30 | 2024-06-11 | 江苏农牧科技职业学院 | 一种鹅骨骼肌卫星细胞的体外分离和培养方法 |
| WO2024229028A2 (en) * | 2023-05-01 | 2024-11-07 | University Of Washington | Novel regulatory cassettes for specific expression of genes in muscle stem cells |
| WO2025260086A1 (en) * | 2024-06-14 | 2025-12-18 | President And Fellows Of Harvard College | In vivo site-specific base editing |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3031921B1 (en) * | 2012-12-12 | 2025-03-12 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| CN106714845A (zh) * | 2014-08-11 | 2017-05-24 | 得克萨斯州大学系统董事会 | 通过crispr/cas9介导的基因编辑预防肌营养不良 |
| US20170362635A1 (en) * | 2016-06-20 | 2017-12-21 | University Of Washington | Muscle-specific crispr/cas9 editing of genes |
| EP4321623A3 (en) * | 2016-07-15 | 2024-05-15 | Salk Institute for Biological Studies | Methods and compositions for genome editing in non-dividing cells |
| US20180127786A1 (en) * | 2016-09-23 | 2018-05-10 | Casebia Therapeutics Limited Liability Partnership | Compositions and methods for gene editing |
-
2019
- 2019-05-03 WO PCT/US2019/030748 patent/WO2019213626A1/en not_active Ceased
- 2019-05-03 JP JP2021510297A patent/JP7498499B2/ja active Active
- 2019-05-03 EP EP19796061.0A patent/EP3787692A4/en active Pending
- 2019-05-03 CA CA3099332A patent/CA3099332A1/en active Pending
- 2019-05-03 CN CN201980042149.7A patent/CN112512595A/zh active Pending
- 2019-05-03 US US17/052,798 patent/US20210363546A1/en active Pending
- 2019-05-03 AU AU2019262225A patent/AU2019262225B2/en active Active
-
2024
- 2024-05-24 JP JP2024085010A patent/JP2024100980A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2021522858A5 (enExample) | ||
| Ginn et al. | Gene therapy clinical trials worldwide to 2023—an update | |
| US20230018543A1 (en) | Crispr/cas-mediated gene conversion | |
| JP2024100980A5 (enExample) | ||
| JP7706888B2 (ja) | 異常ヘモグロビン症の治療のためのシステム及び方法 | |
| Grimm et al. | Small but increasingly mighty: latest advances in AAV vector research, design, and evolution | |
| Kay | State-of-the-art gene-based therapies: the road ahead | |
| AU2016276702B2 (en) | CRISPR/CAS-related methods and compositions for improving transplantation | |
| JP2026064992A (ja) | β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物 | |
| JP7069426B2 (ja) | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 | |
| JP7498499B2 (ja) | 心臓、骨格筋、及び筋幹細胞におけるインビボ相同組換え修復 | |
| JP2024045608A (ja) | 制御性ｒｎａを発現させるためのベクターシステム | |
| CN109312339A (zh) | 用于治疗肌萎缩侧索硬化症和/或额颞叶变性的材料和方法 | |
| WO2017191503A1 (en) | Materials and methods for treatment of hemoglobinopathies | |
| CN110214149B (zh) | 用于治疗疼痛相关病症的材料和方法 | |
| US20200172913A1 (en) | Peptides and nanoparticles for intracellular delivery of virus | |
| Kyriakopoulou et al. | Gene editing innovations and their applications in cardiomyopathy research | |
| CN118525089A (zh) | 年龄相关的白质损失的再生治疗 | |
| JP2024528967A (ja) | ヘモグロビン異常症の治療のためのシステム及び方法 | |
| Shin et al. | Effective transduction by self‐complementary adeno‐associated viruses of human dendritic cells with no alteration of their natural characteristics | |
| CN101368180A (zh) | 一类能将间充质干细胞分化为造血细胞的小rna分子及其作用靶点 | |
| Thanaskody et al. | Designing molecules: directing stem cell differentiation | |
| Zhu et al. | Adeno-associated virus vector modification based on directed evolution technology for gene therapy targeting head and neck squamous cell carcinoma | |
| Singh | Role of gene therapy in treatment of cancer for craniofacial regeneration—current molecular strategies, future perspectives, and challenges: a narrative review | |
| Amabile et al. | 729. Inheritable Silencing of Endogenous Gene by Hit-and-Run Targeted Epigenetic Editing |