JP2025032080A5 - - Google Patents
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- Publication number
- JP2025032080A5 JP2025032080A5 JP2024193416A JP2024193416A JP2025032080A5 JP 2025032080 A5 JP2025032080 A5 JP 2025032080A5 JP 2024193416 A JP2024193416 A JP 2024193416A JP 2024193416 A JP2024193416 A JP 2024193416A JP 2025032080 A5 JP2025032080 A5 JP 2025032080A5
- Authority
- JP
- Japan
- Prior art keywords
- gene
- base editor
- regulatory element
- snp
- amino acid
- 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 (20)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962805271P | 2019-02-13 | 2019-02-13 | |
| US62/805,271 | 2019-02-13 | ||
| US201962850919P | 2019-05-21 | 2019-05-21 | |
| US62/850,919 | 2019-05-21 | ||
| US201962852228P | 2019-05-23 | 2019-05-23 | |
| US201962852224P | 2019-05-23 | 2019-05-23 | |
| US62/852,224 | 2019-05-23 | ||
| US62/852,228 | 2019-05-23 | ||
| US201962873138P | 2019-07-11 | 2019-07-11 | |
| US62/873,138 | 2019-07-11 | ||
| US201962888867P | 2019-08-19 | 2019-08-19 | |
| US62/888,867 | 2019-08-19 | ||
| US201962931722P | 2019-11-06 | 2019-11-06 | |
| US62/931,722 | 2019-11-06 | ||
| US201962941569P | 2019-11-27 | 2019-11-27 | |
| US62/941,569 | 2019-11-27 | ||
| US202062966526P | 2020-01-27 | 2020-01-27 | |
| US62/966,526 | 2020-01-27 | ||
| PCT/US2020/018073 WO2020168051A1 (en) | 2019-02-13 | 2020-02-13 | Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease |
| JP2021546888A JP7586601B2 (ja) | 2019-02-13 | 2020-02-13 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546888A Division JP7586601B2 (ja) | 2019-02-13 | 2020-02-13 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2025032080A JP2025032080A (ja) | 2025-03-11 |
| JP2025032080A5 true JP2025032080A5 (enExample) | 2025-06-16 |
Family
ID=72045106
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546888A Active JP7586601B2 (ja) | 2019-02-13 | 2020-02-13 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
| JP2024193416A Pending JP2025032080A (ja) | 2019-02-13 | 2024-11-05 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021546888A Active JP7586601B2 (ja) | 2019-02-13 | 2020-02-13 | 遺伝的疾患の治療用を含めアデノシンデアミナーゼ塩基エディターを用いて疾患関連遺伝子を編集する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20230140953A1 (enExample) |
| EP (1) | EP3924484A4 (enExample) |
| JP (2) | JP7586601B2 (enExample) |
| KR (1) | KR20210127206A (enExample) |
| CN (2) | CN114040970B (enExample) |
| AU (1) | AU2020223306A1 (enExample) |
| CA (1) | CA3128876A1 (enExample) |
| WO (1) | WO2020168051A1 (enExample) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019178427A1 (en) | 2018-03-14 | 2019-09-19 | Arbor Biotechnologies, Inc. | Novel crispr dna targeting enzymes and systems |
| US12133884B2 (en) | 2018-05-11 | 2024-11-05 | Beam Therapeutics Inc. | Methods of substituting pathogenic amino acids using programmable base editor systems |
| US12454694B2 (en) | 2018-09-07 | 2025-10-28 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| CN114096666B (zh) | 2019-02-13 | 2025-03-18 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
| CR20210572A (es) | 2019-04-15 | 2022-04-07 | Univ Beijing | Métodos y composiciones para editar ácido ribonucleicos (arn) |
| MX2022000497A (es) | 2019-07-12 | 2022-02-03 | Univ Beijing | Edicion de acido ribonucleico (arn) dirigido aprovechando la adenosina desaminasa que actua sobre acido ribonucleico endogeno (adar) utilizando acidos ribonucleicos (arn) modificados geneticamente. |
| PH12022551595A1 (en) * | 2019-12-30 | 2023-11-29 | Edigene Therapeutics Beijing Inc | Leaper technology based method for treating mps ih and composition |
| AU2021218811A1 (en) * | 2020-02-13 | 2022-09-01 | Beam Therapeutics Inc. | Compositions and methods for engraftment of base edited cells |
| US20230340486A1 (en) * | 2020-07-27 | 2023-10-26 | The Children’S Hospital Of Philadelphia | In utero and postnatal gene editing and therapy for treatment of monogenic diseases, including mucopolysaccharidosis type 1h and other disorders |
| WO2022087272A1 (en) * | 2020-10-21 | 2022-04-28 | The Board Of Trustees Of The Leland Stanford Junior University | A screening platform for adar-recruiting guide rnas |
| CN117729931A (zh) * | 2021-05-14 | 2024-03-19 | 比姆医疗股份有限公司 | 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法 |
| EP4426821A1 (en) * | 2021-11-02 | 2024-09-11 | University of Massachusetts | Nme2cas9 inlaid domain fusion proteins |
| WO2023102449A2 (en) * | 2021-12-01 | 2023-06-08 | Shape Therapeutics Inc. | Engineered guide rnas and polynucleotides |
| CN117187220A (zh) * | 2022-03-08 | 2023-12-08 | 中国科学院遗传与发育生物学研究所 | 腺嘌呤脱氨酶及其在碱基编辑中的用途 |
| CN114686456B (zh) | 2022-05-10 | 2023-02-17 | 中山大学 | 基于双分子脱氨酶互补的碱基编辑系统及其应用 |
| KR20250067157A (ko) * | 2022-09-08 | 2025-05-14 | 더 유니버시티 코트 오브 더 유니버시티 오브 에딘버그 | 레트 증후군 요법 |
| CN118931886A (zh) * | 2023-05-09 | 2024-11-12 | 北京齐禾生科生物科技有限公司 | 一种可作用于dna的腺苷脱氨酶及其应用 |
| WO2024233941A1 (en) * | 2023-05-11 | 2024-11-14 | Massachusetts Eye And Ear Infirmary | Base editing approaches to treat abca4-associated stargardt disease |
| WO2024238623A1 (en) * | 2023-05-15 | 2024-11-21 | University Of Georgia Research Foundation, Inc. | Doubly constrained peptides as allosteric inhibitors of leucine rich repeat kinase 2 |
| CN117965505A (zh) * | 2023-06-28 | 2024-05-03 | 微光基因(苏州)有限公司 | 工程化的腺苷脱氨酶及碱基编辑器 |
| CN120536418A (zh) * | 2024-02-26 | 2025-08-26 | 尧唐(上海)生物科技有限公司 | 一种脱氨酶变体、包含其的碱基编辑器及其应用 |
| WO2025184515A1 (en) * | 2024-02-29 | 2025-09-04 | Vesigen, Inc. | Arrdc1-mediated microvesicle-based delivery of therapeutic agents to cells and tissues of the eye |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2012004778A1 (en) * | 2010-07-09 | 2012-01-12 | Ecole Polytechnique Federale De Lausanne (Epfl) | Method for in-vitro monitoring of neuronal disorders and use thereof |
| JP6625627B2 (ja) * | 2014-10-14 | 2019-12-25 | ハロザイム インコーポレイテッド | アデノシンデアミナーゼ−２（ａｄａ２）、その変異体の組成物およびそれを使用する方法 |
| JP7231935B2 (ja) * | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| EP3526320A1 (en) * | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| US11866697B2 (en) | 2017-05-18 | 2024-01-09 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| EP3645054A4 (en) * | 2017-06-26 | 2021-03-31 | The Broad Institute, Inc. | CRISPR / CAS-ADENINE DESAMINASE COMPOSITIONS, TARGETED NUCLEIC ACID EDITING SYSTEMS AND METHODS |
| CN109306361B (zh) * | 2018-02-11 | 2022-06-28 | 华东师范大学 | 一种新的a/t到g/c碱基定点转换的基因编辑系统 |
| CN109295186B (zh) * | 2018-09-30 | 2023-10-03 | 中山大学 | 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用 |
-
2020
- 2020-02-13 JP JP2021546888A patent/JP7586601B2/ja active Active
- 2020-02-13 WO PCT/US2020/018073 patent/WO2020168051A1/en not_active Ceased
- 2020-02-13 US US17/430,672 patent/US20230140953A1/en active Pending
- 2020-02-13 CA CA3128876A patent/CA3128876A1/en active Pending
- 2020-02-13 CN CN202080028186.5A patent/CN114040970B/zh active Active
- 2020-02-13 CN CN202411269394.0A patent/CN119280261A/zh active Pending
- 2020-02-13 AU AU2020223306A patent/AU2020223306A1/en active Pending
- 2020-02-13 KR KR1020217029268A patent/KR20210127206A/ko active Pending
- 2020-02-13 EP EP20756724.9A patent/EP3924484A4/en active Pending
-
2024
- 2024-11-05 JP JP2024193416A patent/JP2025032080A/ja active Pending
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