JP2024138268A5 - - Google Patents
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- JP2024138268A5 JP2024138268A5 JP2024095566A JP2024095566A JP2024138268A5 JP 2024138268 A5 JP2024138268 A5 JP 2024138268A5 JP 2024095566 A JP2024095566 A JP 2024095566A JP 2024095566 A JP2024095566 A JP 2024095566A JP 2024138268 A5 JP2024138268 A5 JP 2024138268A5
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- amino acid
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Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862670588P | 2018-05-11 | 2018-05-11 | |
| US62/670,588 | 2018-05-11 | ||
| US201862780838P | 2018-12-17 | 2018-12-17 | |
| US62/780,838 | 2018-12-17 | ||
| US201962817986P | 2019-03-13 | 2019-03-13 | |
| US62/817,986 | 2019-03-13 | ||
| PCT/US2019/031899 WO2019217944A1 (en) | 2018-05-11 | 2019-05-11 | Methods of editing single nucleotide polymorphism using programmable base editor systems |
| JP2021513765A JP7596259B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513765A Division JP7596259B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024138268A JP2024138268A (ja) | 2024-10-08 |
| JP2024138268A5 true JP2024138268A5 (enExample) | 2025-04-25 |
Family
ID=68466849
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513765A Active JP7596259B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
| JP2021513764A Pending JP2021523738A (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
| JP2024095566A Pending JP2024138268A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
| JP2024095618A Pending JP2024147545A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021513765A Active JP7596259B2 (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法 |
| JP2021513764A Pending JP2021523738A (ja) | 2018-05-11 | 2019-05-11 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024095618A Pending JP2024147545A (ja) | 2018-05-11 | 2024-06-13 | プログラム可能な塩基エディターシステムを使用して単一ヌクレオチド多型を編集する方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (2) | US20210380955A1 (enExample) |
| EP (2) | EP3790595A4 (enExample) |
| JP (4) | JP7596259B2 (enExample) |
| KR (3) | KR20210023832A (enExample) |
| CN (3) | CN119587720A (enExample) |
| AU (2) | AU2019266326A1 (enExample) |
| CA (2) | CA3100037A1 (enExample) |
| WO (2) | WO2019217943A1 (enExample) |
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| CN118416088A (zh) | 2017-03-03 | 2024-08-02 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| EP3633034A4 (en) | 2017-05-31 | 2021-03-24 | The University Of Tokyo | MODIFIED Cas9 PROTEIN AND USE THEREOF |
| WO2019040650A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | GENETICALLY MODIFIED CRISPR-CAS9 NUCLEASES HAVING MODIFIED PAM SPECIFICITY |
| EP3720507A4 (en) * | 2017-12-07 | 2021-08-25 | Ramot at Tel-Aviv University Ltd. | TREATMENT OF PARKINSON PATIENTS WITH MUTATIONS IN THE LRRK2 GENE |
| CN112041444A (zh) | 2018-03-14 | 2020-12-04 | 阿伯生物技术公司 | 新型crispr dna靶向酶及系统 |
| JP7642531B2 (ja) | 2018-05-11 | 2025-03-10 | ビーム セラピューティクス インク. | プログラム可能塩基エディターシステムを用いて病原性アミノ酸を置換する方法 |
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| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| CN116497067B (zh) | 2019-02-13 | 2025-01-24 | 比姆医疗股份有限公司 | 治疗血红素病变的组合物和方法 |
| WO2020168088A1 (en) * | 2019-02-13 | 2020-08-20 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| EP3942042A1 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020236936A1 (en) * | 2019-05-21 | 2020-11-26 | Beam Therapeutics Inc. | Methods of editing a single nucleotide polymorphism using programmable base editor systems |
| JP2022536364A (ja) | 2019-06-13 | 2022-08-15 | ザ ジェネラル ホスピタル コーポレイション | 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法 |
| JP7500927B2 (ja) * | 2019-08-06 | 2024-06-18 | 日本製紙株式会社 | プロテイン用マスキング剤 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CA3159944A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Therapeutic editing |
| US20230054569A1 (en) * | 2019-12-18 | 2023-02-23 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| US12264341B2 (en) * | 2020-01-24 | 2025-04-01 | The General Hospital Corporation | CRISPR-Cas enzymes with enhanced on-target activity |
| US12312613B2 (en) | 2020-01-24 | 2025-05-27 | The General Hospital Corporation | Unconstrained genome targeting with near-PAMless engineered CRISPR-Cas9 variants |
| WO2021155166A1 (en) * | 2020-01-31 | 2021-08-05 | University Of Massachusetts | A cas9-pdbd base editor platform with improved targeting range and specificity |
| WO2021163492A1 (en) * | 2020-02-14 | 2021-08-19 | Ohio State Innovation Foundation | Nucleobase editors and methods of use thereof |
| IT202000008014A1 (it) * | 2020-04-15 | 2021-10-15 | Fond Telethon | RNA guida e loro usi |
| US20230165909A1 (en) * | 2020-04-21 | 2023-06-01 | Aspen Neuroscience, Inc. | Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom |
| US20250263752A1 (en) * | 2020-04-21 | 2025-08-21 | Aspen Neuroscience, Inc. | Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom |
| US20230159913A1 (en) | 2020-04-28 | 2023-05-25 | The Broad Institute, Inc. | Targeted base editing of the ush2a gene |
| WO2021226558A1 (en) | 2020-05-08 | 2021-11-11 | The Broad Institute, Inc. | Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence |
| WO2022015856A1 (en) * | 2020-07-14 | 2022-01-20 | The Regents Of The University Of California | Compositions and methods for treating an inherited retinal disease |
| WO2022020800A2 (en) | 2020-07-24 | 2022-01-27 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| WO2022027035A1 (en) * | 2020-07-27 | 2022-02-03 | 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 |
| JP2023543803A (ja) * | 2020-09-24 | 2023-10-18 | ザ ブロード インスティテュート,インコーポレーテッド | プライム編集ガイドrna、その組成物、及びその使用方法 |
| CA3200521A1 (en) * | 2020-11-06 | 2022-05-12 | Pairwise Plants Services, Inc. | Compositions and methods for rna-encoded dna-replacement of alleles |
| AU2021396392A1 (en) * | 2020-12-10 | 2023-07-06 | Apellis Pharmaceuticals, Inc. | Genomic editing of complement |
| WO2022197727A1 (en) * | 2021-03-15 | 2022-09-22 | Duke University | Generation of novel crispr genome editing agents using combinatorial chemistry |
| WO2022241270A2 (en) * | 2021-05-14 | 2022-11-17 | Beam Therapeutics Inc. | Compositions and methods for treating transthyretin amyloidosis |
| WO2022246266A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| KR20240012377A (ko) * | 2021-05-28 | 2024-01-29 | 빔 테라퓨틱스, 인크. | 염기 편집기의 자기-불활성화용 조성물 및 방법 |
| WO2023064858A1 (en) * | 2021-10-13 | 2023-04-20 | Apellis Pharmaceuticals, Inc. | Compositions and methods for genome editing the neonatal fc receptor |
| JP2024544013A (ja) | 2021-12-03 | 2024-11-26 | ザ ブロード インスティテュート,インコーポレーテッド | 有効なin vivo送達のための組成物および方法 |
| US20250154479A1 (en) * | 2022-01-24 | 2025-05-15 | Toolgen Incorporated | Streptococcus pyogenes-derived cas9 variant |
| CN114480445B (zh) * | 2022-01-26 | 2023-06-27 | 西南交通大学 | 一种人源超氧化物歧化酶hSOD1突变体的制备及其应用 |
| WO2023217280A1 (en) * | 2022-05-13 | 2023-11-16 | Huidagene Therapeutics Co., Ltd. | Programmable adenine base editor and uses thereof |
| CN120303407A (zh) | 2022-05-17 | 2025-07-11 | 恩维洛普治疗有限责任公司 | 用于有效体内递送的组合物和方法 |
| CN115148281B (zh) * | 2022-06-29 | 2023-07-14 | 广州源井生物科技有限公司 | 一种基因编辑点突变方案自动设计方法及系统 |
| CN120225674A (zh) * | 2022-09-08 | 2025-06-27 | 爱丁堡大学董事会 | 雷特综合征疗法 |
| WO2024107784A2 (en) * | 2022-11-15 | 2024-05-23 | Vanderbilt University | Repair of disease-associated single nucleotide variants via interallelic gene conversion |
| WO2024259364A2 (en) * | 2023-06-15 | 2024-12-19 | Beam Therapeutics Inc. | Methods and compositions for treating alpha-1 antitrypsin deficiency |
| WO2025024486A2 (en) * | 2023-07-25 | 2025-01-30 | Flagship Pioneering Innovations Vii, Llc | Cas endonucleases and related methods |
| US20250059567A1 (en) * | 2023-08-17 | 2025-02-20 | The Trustees Of The University Of Pennsylvania | Compositions and methods for the management and treatment of phenylketonuria |
| WO2025059520A1 (en) * | 2023-09-15 | 2025-03-20 | Beam Therapeutics Inc. | Compositions and methods for base editing a phenylalanine hydroxylase polynucleotide |
| WO2025061198A1 (zh) * | 2023-09-22 | 2025-03-27 | 北京大学现代农业研究院 | 改进的基因组编辑方法 |
| WO2025122725A1 (en) * | 2023-12-06 | 2025-06-12 | The Broad Institute, Inc. | Methods and compositions for base editing of tpp1 in the treatment of batten disease |
| CN118120704B (zh) * | 2024-01-30 | 2025-10-24 | 江苏集萃药康生物科技股份有限公司 | 一种免高脂饮食诱导的动脉粥样硬化小鼠模型的构建方法 |
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-
2019
- 2019-05-11 AU AU2019266326A patent/AU2019266326A1/en active Pending
- 2019-05-11 CA CA3100037A patent/CA3100037A1/en active Pending
- 2019-05-11 KR KR1020207035000A patent/KR20210023832A/ko active Pending
- 2019-05-11 WO PCT/US2019/031898 patent/WO2019217943A1/en not_active Ceased
- 2019-05-11 EP EP19799854.5A patent/EP3790595A4/en active Pending
- 2019-05-11 CN CN202411723844.9A patent/CN119587720A/zh active Pending
- 2019-05-11 WO PCT/US2019/031899 patent/WO2019217944A1/en not_active Ceased
- 2019-05-11 EP EP19799484.1A patent/EP3790963A4/en active Pending
- 2019-05-11 US US17/054,324 patent/US20210380955A1/en active Pending
- 2019-05-11 CA CA3100034A patent/CA3100034A1/en active Pending
- 2019-05-11 KR KR1020207035001A patent/KR102853088B1/ko active Active
- 2019-05-11 KR KR1020257028616A patent/KR20250133482A/ko active Pending
- 2019-05-11 AU AU2019266327A patent/AU2019266327B2/en active Active
- 2019-05-11 CN CN201980046479.3A patent/CN112469446B/zh active Active
- 2019-05-11 US US17/054,348 patent/US20230159956A1/en active Pending
- 2019-05-11 JP JP2021513765A patent/JP7596259B2/ja active Active
- 2019-05-11 JP JP2021513764A patent/JP2021523738A/ja active Pending
- 2019-05-11 CN CN201980046538.7A patent/CN112469824A/zh active Pending
-
2024
- 2024-06-13 JP JP2024095566A patent/JP2024138268A/ja active Pending
- 2024-06-13 JP JP2024095618A patent/JP2024147545A/ja active Pending
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