JPWO2021228944A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021228944A5 JPWO2021228944A5 JP2022568520A JP2022568520A JPWO2021228944A5 JP WO2021228944 A5 JPWO2021228944 A5 JP WO2021228944A5 JP 2022568520 A JP2022568520 A JP 2022568520A JP 2022568520 A JP2022568520 A JP 2022568520A JP WO2021228944 A5 JPWO2021228944 A5 JP WO2021228944A5
- Authority
- JP
- Japan
- Prior art keywords
- hbg1
- promoter
- gene editing
- hbg2
- hbg2 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 |
|---|---|---|---|
| EP20305488.7 | 2020-05-13 | ||
| EP20305488 | 2020-05-13 | ||
| PCT/EP2021/062633 WO2021228944A1 (en) | 2020-05-13 | 2021-05-12 | Base editing approaches for the treatment of betahemoglobinopathies |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2023526007A JP2023526007A (ja) | 2023-06-20 |
| JP2023526007A5 JP2023526007A5 (https=) | 2024-05-01 |
| JPWO2021228944A5 true JPWO2021228944A5 (https=) | 2024-05-01 |
Family
ID=71575241
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022568520A Pending JP2023526007A (ja) | 2020-05-13 | 2021-05-12 | β－ヘモグロビン異常症の処置のための塩基編集アプローチ |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20230279438A1 (https=) |
| EP (1) | EP4150081A1 (https=) |
| JP (1) | JP2023526007A (https=) |
| WO (1) | WO2021228944A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN121555430A (zh) | 2018-05-11 | 2026-02-24 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统取代病原性氨基酸的方法 |
| US20250161492A1 (en) * | 2022-01-25 | 2025-05-22 | Institut National de la Santé et de la Recherche Médicale | Base editing approaches for the treatment of beta-thalassemia |
| WO2025017030A1 (en) | 2023-07-17 | 2025-01-23 | Institut National de la Santé et de la Recherche Médicale | Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell |
| WO2025017033A1 (en) | 2023-07-17 | 2025-01-23 | Institut National de la Santé et de la Recherche Médicale | Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell |
| WO2025065716A1 (en) * | 2023-09-29 | 2025-04-03 | Yoltech Therapeutics Co., Ltd | Lipid nanoparticle formulations and methods of use thereof |
| WO2025120664A1 (en) * | 2023-12-04 | 2025-06-12 | Christian Medical College | Strategies for precision editing of homologous regions |
| WO2025147212A1 (en) * | 2024-01-05 | 2025-07-10 | Singapore Health Services Pte Ltd | Induction of fetal haemoglobin using paired cas9 nickases |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008141248A2 (en) | 2007-05-10 | 2008-11-20 | Agilent Technologies, Inc. | Thiocarbon-protecting groups for rna synthesis |
| RU2650811C2 (ru) * | 2012-02-24 | 2018-04-17 | Фред Хатчинсон Кэнсер Рисерч Сентер | Композиции и способы лечения гемоглобинопатии |
| EP3241902B1 (en) | 2012-05-25 | 2018-02-28 | The Regents of The University of California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| AU2014227653B2 (en) | 2013-03-15 | 2017-04-20 | The General Hospital Corporation | Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing |
| US20140273230A1 (en) | 2013-03-15 | 2014-09-18 | Sigma-Aldrich Co., Llc | Crispr-based genome modification and regulation |
| US9234213B2 (en) | 2013-03-15 | 2016-01-12 | System Biosciences, Llc | Compositions and methods directed to CRISPR/Cas genomic engineering systems |
| WO2019178426A1 (en) * | 2018-03-14 | 2019-09-19 | Editas Medicine, Inc. | Systems and methods for the treatment of hemoglobinopathies |
| CN121555430A (zh) * | 2018-05-11 | 2026-02-24 | 比姆医疗股份有限公司 | 使用可编程碱基编辑器系统取代病原性氨基酸的方法 |
| US20220033856A1 (en) * | 2018-09-11 | 2022-02-03 | Université de Paris | Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies |
-
2021
- 2021-05-12 WO PCT/EP2021/062633 patent/WO2021228944A1/en not_active Ceased
- 2021-05-12 EP EP21724321.1A patent/EP4150081A1/en active Pending
- 2021-05-12 JP JP2022568520A patent/JP2023526007A/ja active Pending
- 2021-05-12 US US17/998,603 patent/US20230279438A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| Liu et al. | Expanding the CRISPR toolbox in zebrafish for studying development and disease | |
| JP2025026840A5 (https=) | ||
| RU2650811C2 (ru) | Композиции и способы лечения гемоглобинопатии | |
| AU2018215726B2 (en) | Compositions and methods for the treatment of hemoglobinopathies | |
| US12385070B2 (en) | Homology directed repair compositions for the treatment of hemoglobinopathies | |
| US20240209332A1 (en) | Enzymes with ruvc domains | |
| WO2016082135A1 (zh) | 一种利用定点切割系统对猪h11位点定点插入的方法 | |
| WO2016201047A1 (en) | Crispr/cas-related methods and compositions for improving transplantation | |
| Wang et al. | CRISPR/Cas9-mediated mutagenesis at microhomologous regions of human mitochondrial genome | |
| EP3765617A1 (en) | Systems and methods for the treatment of hemoglobinopathies | |
| JP2023526007A (ja) | β－ヘモグロビン異常症の処置のための塩基編集アプローチ | |
| JPWO2021228944A5 (https=) | ||
| CN117568321A (zh) | 一种腺嘌呤脱氨酶突变体及其应用 | |
| CN113215193B (zh) | 小分子化合物提高基因敲除和碱基编辑系统活性的方法及其应用方法 | |
| CN113403341B (zh) | 一种基于tale组装的线粒体dna编辑系统 | |
| Zhang et al. | Translating genomic insights into cardiovascular medicine: Opportunities and challenges of CRISPR-Cas9 | |
| CN114560946B (zh) | 无pam限制的腺嘌呤单碱基编辑产品、方法和应用 | |
| EP4337232A1 (en) | Methods for stratifying subjects for fetal hemoglobin reinduction | |
| US20250263692A1 (en) | Curing for iterative nucleic acid-guided nuclease editing | |
| JPWO2022081890A5 (https=) | ||
| Matsuura et al. | Selective transmission of mitochondrial DNA in heteroplasmic lines for intra-and interspecific combinations in Drosophila melanogaster | |
| JP6525342B2 (ja) | タグ付けされたｄｎａ断片の生成 | |
| JPWO2023015307A5 (https=) | ||
| JPWO2023099591A5 (https=) | ||
| JP2021151200A (ja) | 連結ｄｎａの製造方法及びそれに用いるためのベクターの組み合わせ |