JP2021521850A5 - - Google Patents
Info
- Publication number
- JP2021521850A5 JP2021521850A5 JP2020560340A JP2020560340A JP2021521850A5 JP 2021521850 A5 JP2021521850 A5 JP 2021521850A5 JP 2020560340 A JP2020560340 A JP 2020560340A JP 2020560340 A JP2020560340 A JP 2020560340A JP 2021521850 A5 JP2021521850 A5 JP 2021521850A5
- Authority
- JP
- Japan
- Prior art keywords
- nuclease
- cells
- nucleotide sequence
- vector
- seq
- 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 |
|---|---|---|---|
| US201862663485P | 2018-04-27 | 2018-04-27 | |
| US62/663,485 | 2018-04-27 | ||
| PCT/US2019/028858 WO2019209912A2 (en) | 2018-04-27 | 2019-04-24 | Therapeutic genome editing in x-linked hyper igm syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021521850A JP2021521850A (ja) | 2021-08-30 |
| JP2021521850A5 true JP2021521850A5 (enExample) | 2023-03-30 |
Family
ID=68295746
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020560340A Pending JP2021521850A (ja) | 2018-04-27 | 2019-04-24 | X連鎖性高IgM症候群のゲノム編集治療 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210324381A1 (enExample) |
| EP (1) | EP3784292A4 (enExample) |
| JP (1) | JP2021521850A (enExample) |
| KR (1) | KR20210005178A (enExample) |
| CN (1) | CN112312931A (enExample) |
| AU (1) | AU2019261385A1 (enExample) |
| CA (1) | CA3098489A1 (enExample) |
| WO (1) | WO2019209912A2 (enExample) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3060570A1 (en) * | 2017-04-21 | 2018-10-25 | Seattle Children's Hospital (Dba Seattle Children's Research Institute | Therapeutic genome editing in wiskott-aldrich syndrome and x-linked thrombocytopenia |
| WO2019014564A1 (en) | 2017-07-14 | 2019-01-17 | Editas Medicine, Inc. | SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES |
| EP3801640A4 (en) | 2018-05-30 | 2022-03-23 | The Regents Of The University Of California | GENE EDIT OF MONOGENIC DISEASES IN HUMAN HEMATOPOIETIC STEM CELLS - CORRECTION OF X-LINKED AGAMMAGLOBULINEMIA (XLA) |
| WO2025073293A1 (en) * | 2023-10-02 | 2025-04-10 | Consorcio Centro De Investigación Biomédica En Red | Gene editing methods for cd34+ cells to achieve clinically relevant efficacies |
| WO2026024636A1 (en) * | 2024-07-24 | 2026-01-29 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Methods and compositions for editing a cd40lg gene |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU4102099A (en) * | 1998-05-29 | 1999-12-13 | Heska Corporation | Canine Interleukin -4 Proteins, Nucleic Acid Molecules, and Uses Thereof |
| EP3135765A1 (en) * | 2012-12-06 | 2017-03-01 | Sigma-Aldrich Co. LLC | Crispr-based genome modification and regulation |
| US20190275168A1 (en) * | 2015-04-30 | 2019-09-12 | The Trustees Of Columbia University In The City Of New York | Gene therapy for autosomal dominant diseases |
| US10179918B2 (en) * | 2015-05-07 | 2019-01-15 | Sangamo Therapeutics, Inc. | Methods and compositions for increasing transgene activity |
| EP3294343A4 (en) * | 2015-05-13 | 2018-09-26 | Seattle Children's Hospital, dba Seattle Children's Research Institute | Enhancing endonuclease based gene editing in primary cells |
| WO2018035387A1 (en) * | 2016-08-17 | 2018-02-22 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| WO2018073393A2 (en) * | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| US20180245065A1 (en) * | 2016-11-01 | 2018-08-30 | Novartis Ag | Methods and compositions for enhancing gene editing |
| GB201618414D0 (en) * | 2016-11-01 | 2016-12-14 | Patterson James | Regulated cell lines and methods of use thereof |
| EP3622056A1 (en) * | 2017-05-10 | 2020-03-18 | Aarhus Universitet | Interferon primed plasmacytoid dendritic cells |
| WO2019079527A1 (en) * | 2017-10-17 | 2019-04-25 | Casebia Therapeutics Limited Liability Partnership | COMPOSITIONS AND METHODS FOR GENETIC EDITION FOR HEMOPHILIA A |
| ES2998052T3 (en) * | 2017-10-24 | 2025-02-18 | Editas Medicine Inc | Systems and methods for treating hyper-igm syndrome |
| US20200263206A1 (en) * | 2017-11-07 | 2020-08-20 | Editas Medicine, Inc. | Targeted integration systems and methods for the treatment of hemoglobinopathies |
| EP3710016A4 (en) * | 2017-11-17 | 2021-12-01 | Memorial Sloan-Kettering Cancer Center | METHODS AND COMPOSITIONS FOR RELIEVING CYTOCINE RELEASE SYNDROME |
-
2019
- 2019-04-24 US US17/050,601 patent/US20210324381A1/en active Pending
- 2019-04-24 CN CN201980042296.4A patent/CN112312931A/zh active Pending
- 2019-04-24 AU AU2019261385A patent/AU2019261385A1/en not_active Abandoned
- 2019-04-24 KR KR1020207034098A patent/KR20210005178A/ko not_active Ceased
- 2019-04-24 JP JP2020560340A patent/JP2021521850A/ja active Pending
- 2019-04-24 WO PCT/US2019/028858 patent/WO2019209912A2/en not_active Ceased
- 2019-04-24 EP EP19793005.0A patent/EP3784292A4/en active Pending
- 2019-04-24 CA CA3098489A patent/CA3098489A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7252328B2 (ja) | Rnaを編集する方法および組成物 | |
| JP7698828B2 (ja) | Rnaを編集する方法および組成物 | |
| KR102796744B1 (ko) | 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물 | |
| JP2024073596A5 (enExample) | ||
| 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 | |
| Canver et al. | Customizing the genome as therapy for the β-hemoglobinopathies | |
| CN105331607A (zh) | 嗜热链球菌CRISPR-Cas9系统识别的人CCR5基因的靶序列和sgRNA及其应用 | |
| JP2021521855A5 (enExample) | ||
| JP2016500262A5 (enExample) | ||
| JP2019524140A5 (enExample) | ||
| JP2015533786A5 (enExample) | ||
| CN105316324A (zh) | 嗜热链球菌CRISPR-Cas9系统识别的人CXCR4基因的靶序列和sgRNA及其应用 | |
| JP2016504026A5 (enExample) | ||
| JP2019508051A5 (enExample) | ||
| CN105400779A (zh) | 嗜热链球菌CRISPR-Cas9系统识别的人CCR5基因的靶序列和sgRNA及其应用 | |
| JP2020517270A5 (enExample) | ||
| WO2021083183A1 (zh) | 一种造血干细胞hbb基因修复的方法及产品 | |
| CN114752629B (zh) | 一种针对细胞中单个或多个基因进行基因编辑的方法、产品和应用 | |
| JP2020511931A5 (enExample) | ||
| CN110577969B (zh) | CRISPR/Sa-SlugCas9基因编辑系统及其应用 | |
| JP2023544633A (ja) | 治療における使用のためのrag1の置き換え | |
| JP7416745B2 (ja) | 改変細胞、調製方法、及び構築物 | |
| CN110499334A (zh) | CRISPR/SlugCas9基因编辑系统及其应用 | |
| FI3615674T3 (fi) | Menetelmiä reumatoidin artriitin hoitoon HLA-geenin RNA-ohjattua genomin muokkausta käyttämällä | |
| Osamu et al. | A rat brain-derived neurotrophic factor-encoding gene generates multiple transcripts through alternative use of 5′ exons and polyadenylation sites |