JP2021513866A5 - - Google Patents
Info
- Publication number
- JP2021513866A5 JP2021513866A5 JP2020544787A JP2020544787A JP2021513866A5 JP 2021513866 A5 JP2021513866 A5 JP 2021513866A5 JP 2020544787 A JP2020544787 A JP 2020544787A JP 2020544787 A JP2020544787 A JP 2020544787A JP 2021513866 A5 JP2021513866 A5 JP 2021513866A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleotide sequence
- ats
- ube3a
- target nucleotide
- guide rna
- 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 |
|---|---|---|---|
| US201862635815P | 2018-02-27 | 2018-02-27 | |
| US62/635,815 | 2018-02-27 | ||
| PCT/US2019/019789 WO2019168950A1 (en) | 2018-02-27 | 2019-02-27 | Methods and compositions for treating angelman syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021513866A JP2021513866A (ja) | 2021-06-03 |
| JP2021513866A5 true JP2021513866A5 (enExample) | 2022-03-09 |
Family
ID=67805564
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020544787A Pending JP2021513866A (ja) | 2018-02-27 | 2019-02-27 | アンジェルマン症候群を処置する方法および組成物 |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20210054370A1 (enExample) |
| EP (1) | EP3758714A4 (enExample) |
| JP (1) | JP2021513866A (enExample) |
| CN (1) | CN111770758A (enExample) |
| CA (1) | CA3091912A1 (enExample) |
| WO (1) | WO2019168950A1 (enExample) |
Families Citing this family (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2019255789B2 (en) | 2018-04-19 | 2025-11-20 | The Regents Of The University Of California | Compositions and methods for gene editing |
| SG11202113253SA (en) | 2019-06-07 | 2021-12-30 | Scribe Therapeutics Inc | Engineered casx systems |
| WO2021126087A1 (en) | 2019-12-18 | 2021-06-24 | National University Of Singapore | Method for treating angelman syndrome and related disorders |
| WO2021222232A1 (en) * | 2020-04-28 | 2021-11-04 | The Trustees Of The University Of Pennsylvania | Compositions and uses thereof for treatment of angelman syndrome |
| AU2021392719B2 (en) | 2020-12-03 | 2025-08-14 | Scribe Therapeutics Inc. | Engineered class 2 type v crispr systems |
| WO2023168000A1 (en) * | 2022-03-03 | 2023-09-07 | The Regents Of The University Of California | Compositions and methods for treatment of angelman syndrome |
| WO2023184108A1 (en) * | 2022-03-28 | 2023-10-05 | Huigene Therapeutics Co., Ltd. | Crispr-cas13 system for treating ube3a-associated diseases |
| CN116004788B (zh) * | 2022-08-01 | 2024-05-31 | 湖南家辉生物技术有限公司 | 一种Angelman综合症致病基因UBE3A突变位点的应用及其诊断试剂 |
| WO2026006542A2 (en) * | 2024-06-26 | 2026-01-02 | Yale University | Compositions and methods for crispr/cas9 based reactivation of human angelman syndrome |
| CN121992041A (zh) * | 2024-11-06 | 2026-05-08 | 中国科学院脑科学与智能技术卓越创新中心 | 靶向ube3a等位基因建立天使综合征动物模型的方法和应用 |
| CN119177283B (zh) * | 2024-11-25 | 2025-03-07 | 嘉检(广州)生物工程技术有限公司 | 用于pws/as快速诊断的引物组、试剂盒及系统 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2809199T3 (es) * | 2012-06-25 | 2021-03-03 | Ionis Pharmaceuticals Inc | Modulación de la expresión de UBE3A-ATS |
| SG10201707569YA (en) * | 2012-12-12 | 2017-10-30 | Broad Inst Inc | Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications |
| WO2016028843A2 (en) * | 2014-08-19 | 2016-02-25 | President And Fellows Of Harvard College | Rna-guided systems for probing and mapping of nucleic acids |
| WO2016086104A1 (en) * | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| US20180346531A1 (en) * | 2015-09-15 | 2018-12-06 | Regents Of The University Of California | Compositions and methods for delivering biotherapeutics |
| EP3443081A4 (en) * | 2016-04-13 | 2019-10-30 | Duke University | CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE |
| EP3716767A4 (en) * | 2017-10-24 | 2021-11-24 | Sangamo Therapeutics, Inc. | METHODS AND COMPOSITIONS FOR TREATMENT OF RARE DISEASES |
| PL3717646T3 (pl) * | 2017-12-01 | 2025-10-27 | The Texas A&M University System | Leczenie antysensowe zespołu angelmana |
| EP3720508A4 (en) * | 2017-12-08 | 2021-09-01 | University of Connecticut | COMPOSITIONS AND METHODS FOR TREATMENT OF GENOMIC FOOTPRINT DISORDERS |
-
2019
- 2019-02-27 EP EP19761013.2A patent/EP3758714A4/en not_active Withdrawn
- 2019-02-27 WO PCT/US2019/019789 patent/WO2019168950A1/en not_active Ceased
- 2019-02-27 CA CA3091912A patent/CA3091912A1/en active Pending
- 2019-02-27 US US16/976,386 patent/US20210054370A1/en not_active Abandoned
- 2019-02-27 JP JP2020544787A patent/JP2021513866A/ja active Pending
- 2019-02-27 CN CN201980015559.2A patent/CN111770758A/zh active Pending
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