JP2024138268A5 - - Google Patents
Download PDFInfo
- Publication number
- 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
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- rtt
- cell
- modification
- domain
- 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 (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 |
| 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 |
| 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 |
Family Applications Before (3)
| 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 |
| 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) | EP3790963A4 (enExample) |
| JP (4) | JP7596259B2 (enExample) |
| KR (3) | KR102853088B1 (enExample) |
| CN (3) | CN119587720A (enExample) |
| AU (2) | AU2019266327B2 (enExample) |
| CA (2) | CA3100034A1 (enExample) |
| WO (2) | WO2019217943A1 (enExample) |
Families Citing this family (56)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9944912B2 (en) | 2015-03-03 | 2018-04-17 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| US11932856B2 (en) | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| EP3633034A4 (en) * | 2017-05-31 | 2021-03-24 | The University Of Tokyo | MODIFIED Cas9 PROTEIN AND USE THEREOF |
| CA3073448A1 (en) | 2017-08-23 | 2019-02-28 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| WO2019111258A1 (en) * | 2017-12-07 | 2019-06-13 | Ramot At Tel-Aviv University Ltd. | Treatment for parkinsonian patients with mutations in the lrrk2 gene |
| 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 |
| US12157760B2 (en) * | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12454694B2 (en) | 2018-09-07 | 2025-10-28 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2020221355A1 (en) * | 2019-02-13 | 2021-08-12 | Beam Therapeutics Inc. | Compositions and methods for treating glycogen storage disease type 1a |
| 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 |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CA3140093A1 (en) * | 2019-05-21 | 2020-11-26 | Beam Therapeutics Inc. | Methods of editing a single nucleotide polymorphism using programmable base editor systems |
| WO2020252455A1 (en) | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| 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 |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| WO2021122944A1 (en) * | 2019-12-18 | 2021-06-24 | Alia Therapeutics Srl | Compositions and methods for treating retinitis pigmentosa |
| EP4093864A4 (en) | 2020-01-24 | 2024-04-10 | The General Hospital Corporation | Unconstrained genome targeting with near-pamless engineered crispr-cas9 variants |
| WO2021151085A2 (en) | 2020-01-24 | 2021-07-29 | The General Hospital Corporation | Crispr-cas enzymes with enhanced on-target activity |
| US20230049455A1 (en) * | 2020-01-31 | 2023-02-16 | University Of Massachusetts | A cas9-pdbd base editor platform with improved targeting range and specificity |
| EP4103705A4 (en) | 2020-02-14 | 2024-02-28 | 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 |
| WO2021216623A1 (en) * | 2020-04-21 | 2021-10-28 | Aspen Neuroscience, Inc. | Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom |
| WO2021216622A1 (en) * | 2020-04-21 | 2021-10-28 | Aspen Neuroscience, Inc. | Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom |
| WO2021222318A1 (en) | 2020-04-28 | 2021-11-04 | The Broad Institute, Inc. | Targeted base editing of the ush2a gene |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| US20230190893A1 (en) * | 2020-07-14 | 2023-06-22 | The Regents Of The University Of California | Compositions and methods for treating an inherited retinal disease |
| CN116670154A (zh) | 2020-07-24 | 2023-08-29 | 总医院公司 | 增强的病毒样颗粒及使用其递送至细胞的方法 |
| 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 |
| AU2021350835A1 (en) * | 2020-09-24 | 2023-04-27 | President And Fellows Of Harvard College | Prime editing guide rnas, compositions thereof, and methods of using the same |
| US20220145334A1 (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 |
| EP4308702A4 (en) * | 2021-03-15 | 2025-03-05 | Duke University | GENERATING NEW CRISPR GENOME-EDITING AGENTS USING COMBINATORY CHEMISTRY |
| 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 |
| CA3220738A1 (en) * | 2021-05-21 | 2022-11-24 | Beam Therapeutics Inc. | Base editing of transthyretin gene |
| CN117729926A (zh) * | 2021-05-28 | 2024-03-19 | 比姆医疗股份有限公司 | 用于使碱基编辑器自失活的组合物和方法 |
| JP2024539652A (ja) * | 2021-10-13 | 2024-10-29 | アペリス・ファーマシューティカルズ・インコーポレイテッド | 新生児ｆｃ受容体をゲノム編集するための組成物および方法 |
| AU2022402249A1 (en) | 2021-12-03 | 2024-07-11 | President And Fellows Of Harvard College | Compositions and methods for efficient in vivo delivery |
| KR20240136988A (ko) * | 2022-01-24 | 2024-09-19 | 주식회사 툴젠 | 스트렙토코커스 피오게네스 유래 Cas9 변이체 |
| 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 |
| US20250304940A1 (en) * | 2022-05-13 | 2025-10-02 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Programmable adenine base editor and uses thereof |
| WO2023225572A2 (en) | 2022-05-17 | 2023-11-23 | Nvelop Therapeutics, Inc. | Compositions and methods for efficient in vivo delivery |
| 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 |
| KR20250067157A (ko) * | 2022-09-08 | 2025-05-14 | 더 유니버시티 코트 오브 더 유니버시티 오브 에딘버그 | 레트 증후군 요법 |
| 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 |
| US20250092426A1 (en) * | 2023-07-25 | 2025-03-20 | 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 |
Family Cites Families (34)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4880635B1 (en) | 1984-08-08 | 1996-07-02 | Liposome Company | Dehydrated liposomes |
| US4797368A (en) | 1985-03-15 | 1989-01-10 | The United States Of America As Represented By The Department Of Health And Human Services | Adeno-associated virus as eukaryotic expression vector |
| US4921757A (en) | 1985-04-26 | 1990-05-01 | Massachusetts Institute Of Technology | System for delayed and pulsed release of biologically active substances |
| US4920016A (en) | 1986-12-24 | 1990-04-24 | Linear Technology, Inc. | Liposomes with enhanced circulation time |
| JPH0825869B2 (ja) | 1987-02-09 | 1996-03-13 | 株式会社ビタミン研究所 | 抗腫瘍剤包埋リポソ−ム製剤 |
| US4917951A (en) | 1987-07-28 | 1990-04-17 | Micro-Pak, Inc. | Lipid vesicles formed of surfactants and steroids |
| US4911928A (en) | 1987-03-13 | 1990-03-27 | Micro-Pak, Inc. | Paucilamellar lipid vesicles |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| US5587308A (en) | 1992-06-02 | 1996-12-24 | The United States Of America As Represented By The Department Of Health & Human Services | Modified adeno-associated virus vector capable of expression from a novel promoter |
| US5846946A (en) | 1996-06-14 | 1998-12-08 | Pasteur Merieux Serums Et Vaccins | Compositions and methods for administering Borrelia DNA |
| US7013219B2 (en) | 1999-01-12 | 2006-03-14 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6534261B1 (en) | 1999-01-12 | 2003-03-18 | Sangamo Biosciences, Inc. | Regulation of endogenous gene expression in cells using zinc finger proteins |
| US6453242B1 (en) | 1999-01-12 | 2002-09-17 | Sangamo Biosciences, Inc. | Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites |
| US6599692B1 (en) | 1999-09-14 | 2003-07-29 | Sangamo Bioscience, Inc. | Functional genomics using zinc finger proteins |
| CA2392490A1 (en) | 1999-11-24 | 2001-05-31 | Mcs Micro Carrier Systems Gmbh | Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells |
| AU776576B2 (en) | 1999-12-06 | 2004-09-16 | Sangamo Biosciences, Inc. | Methods of using randomized libraries of zinc finger proteins for the identification of gene function |
| KR20020086508A (ko) | 2000-02-08 | 2002-11-18 | 상가모 바이오사이언스 인코포레이티드 | 약물 발견용 세포 |
| CN101277971A (zh) | 2004-07-16 | 2008-10-01 | 美国政府健康及人类服务部 | 含cmv/r-核酸构建体的抗aids疫苗 |
| WO2009088786A1 (en) | 2007-12-31 | 2009-07-16 | Nanocor Therapeutics, Inc. | Rna interference for the treatment of heart failure |
| EP3460056B1 (en) | 2009-11-02 | 2020-08-19 | University Of Washington | Therapeutic nuclease compositions and methods |
| US9405700B2 (en) | 2010-11-04 | 2016-08-02 | Sonics, Inc. | Methods and apparatus for virtualization in an integrated circuit |
| KR20250068794A (ko) * | 2013-12-12 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용 |
| WO2015191693A2 (en) * | 2014-06-10 | 2015-12-17 | Massachusetts Institute Of Technology | Method for gene editing |
| US9944912B2 (en) * | 2015-03-03 | 2018-04-17 | The General Hospital Corporation | Engineered CRISPR-Cas9 nucleases with altered PAM specificity |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| US11851653B2 (en) | 2015-12-01 | 2023-12-26 | Crispr Therapeutics Ag | Materials and methods for treatment of alpha-1 antitrypsin deficiency |
| EP3433364A1 (en) * | 2016-03-25 | 2019-01-30 | Editas Medicine, Inc. | Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency |
| CA3026110A1 (en) * | 2016-04-19 | 2017-11-02 | The Broad Institute, Inc. | Novel crispr enzymes and systems |
| EP3445853A1 (en) * | 2016-04-19 | 2019-02-27 | The Broad Institute, Inc. | Cpf1 complexes with reduced indel activity |
| US12410416B2 (en) * | 2016-04-19 | 2025-09-09 | The Broad Institute, Inc. | CRISPR enzymes and systems with modified pam specificity |
| 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 |
| NZ751483A (en) | 2016-09-01 | 2022-07-01 | Proqr Therapeutics Ii Bv | Chemically modified single-stranded rna-editing oligonucleotides |
| CN107043779B (zh) * | 2016-12-01 | 2020-05-12 | 中国农业科学院作物科学研究所 | 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用 |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
-
2019
- 2019-05-11 CN CN202411723844.9A patent/CN119587720A/zh active Pending
- 2019-05-11 CA CA3100034A patent/CA3100034A1/en active Pending
- 2019-05-11 AU AU2019266327A patent/AU2019266327B2/en active Active
- 2019-05-11 KR KR1020207035001A patent/KR102853088B1/ko active Active
- 2019-05-11 EP EP19799484.1A patent/EP3790963A4/en active Pending
- 2019-05-11 JP JP2021513765A patent/JP7596259B2/ja active Active
- 2019-05-11 CN CN201980046479.3A patent/CN112469446B/zh active Active
- 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 AU AU2019266326A patent/AU2019266326A1/en active Pending
- 2019-05-11 CN CN201980046538.7A patent/CN112469824A/zh active Pending
- 2019-05-11 JP JP2021513764A patent/JP2021523738A/ja active Pending
- 2019-05-11 US US17/054,324 patent/US20210380955A1/en active Pending
- 2019-05-11 US US17/054,348 patent/US20230159956A1/en active Pending
- 2019-05-11 WO PCT/US2019/031899 patent/WO2019217944A1/en not_active Ceased
- 2019-05-11 EP EP19799854.5A patent/EP3790595A4/en active Pending
- 2019-05-11 CA CA3100037A patent/CA3100037A1/en active Pending
- 2019-05-11 KR KR1020257028616A patent/KR20250133482A/ko active Pending
-
2024
- 2024-06-13 JP JP2024095618A patent/JP2024147545A/ja active Pending
- 2024-06-13 JP JP2024095566A patent/JP2024138268A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2024138268A5 (enExample) | ||
| JP7698828B2 (ja) | Ｒｎａを編集する方法および組成物 | |
| JPWO2019217944A5 (enExample) | ||
| JP7252328B2 (ja) | Ｒｎａを編集する方法および組成物 | |
| US20230018543A1 (en) | Crispr/cas-mediated gene conversion | |
| Hermant et al. | TFs for TEs: the transcription factor repertoire of mammalian transposable elements | |
| Li et al. | Genome-wide analyses reveal a role of Polycomb in promoting hypomethylation of DNA methylation valleys | |
| JP2025026840A5 (enExample) | ||
| AU2018352592C1 (en) | Uses of adenosine base editors | |
| Liang et al. | CRISPR/Cas9-mediated gene editing in human tripronuclear zygotes | |
| JP2024038327A5 (enExample) | ||
| JP2023123561A5 (enExample) | ||
| CN107532182A (zh) | 治疗血红蛋白病的材料和方法 | |
| US20140329697A1 (en) | Constructing method of high-throughput sequencing library and use thereof | |
| CN108351350A (zh) | 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法 | |
| JP2016512691A5 (enExample) | ||
| JPWO2021050571A5 (enExample) | ||
| JP2025102768A5 (enExample) | ||
| Zhang et al. | Comprehensive off-target analysis of dCas9-SAM-mediated HIV reactivation via long noncoding RNA and mRNA profiling | |
| EP3645721A1 (en) | Methods for the treatment of disease with gene editing systems | |
| Badat et al. | Direct correction of haemoglobin E β-thalassaemia using base editors | |
| Shimosuga et al. | Locus-specific hypomethylation of the mouse IAP retrotransposon is associated with transcription factor-binding sites | |
| WO2024240223A1 (en) | Deaminases and variants thereof for use in base editing | |
| EP3924476A1 (en) | Methods for targeted depletion of nucleic acids | |
| JP2023538537A (ja) | 核酸の標的化除去のための方法 |