JP2020508685A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020508685A5 JP2020508685A5 JP2019547704A JP2019547704A JP2020508685A5 JP 2020508685 A5 JP2020508685 A5 JP 2020508685A5 JP 2019547704 A JP2019547704 A JP 2019547704A JP 2019547704 A JP2019547704 A JP 2019547704A JP 2020508685 A5 JP2020508685 A5 JP 2020508685A5
- Authority
- JP
- Japan
- Prior art keywords
- lentivirus
- aav vector
- polynucleotide sequence
- vector
- aav
- 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
- 102100029791 Double-stranded RNA-specific adenosine deaminase Human genes 0.000 claims description 44
- 101000865408 Homo sapiens Double-stranded RNA-specific adenosine deaminase Proteins 0.000 claims description 44
- 239000013598 vector Substances 0.000 claims description 38
- 230000035772 mutation Effects 0.000 claims description 33
- 239000013607 AAV vector Substances 0.000 claims description 29
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 26
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 23
- 201000010099 disease Diseases 0.000 claims description 16
- 108020001507 fusion proteins Proteins 0.000 claims description 11
- 102000037865 fusion proteins Human genes 0.000 claims description 11
- 108020004485 Nonsense Codon Proteins 0.000 claims description 8
- 230000037434 nonsense mutation Effects 0.000 claims description 8
- 201000006938 muscular dystrophy Diseases 0.000 claims description 7
- 102100038191 Double-stranded RNA-specific editase 1 Human genes 0.000 claims description 6
- 101000742223 Homo sapiens Double-stranded RNA-specific editase 1 Proteins 0.000 claims description 6
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 241000713666 Lentivirus Species 0.000 claims 26
- 108091033319 polynucleotide Proteins 0.000 claims 13
- 239000002157 polynucleotide Substances 0.000 claims 13
- 102000040430 polynucleotide Human genes 0.000 claims 13
- 108090000790 Enzymes Proteins 0.000 claims 6
- 102000004190 Enzymes Human genes 0.000 claims 6
- 230000008685 targeting Effects 0.000 claims 6
- 101000986595 Homo sapiens Ornithine transcarbamylase, mitochondrial Proteins 0.000 claims 3
- 208000000599 Ornithine Carbamoyltransferase Deficiency Disease Diseases 0.000 claims 3
- 206010052450 Ornithine transcarbamoylase deficiency Diseases 0.000 claims 3
- 208000035903 Ornithine transcarbamylase deficiency Diseases 0.000 claims 3
- 102100028200 Ornithine transcarbamylase, mitochondrial Human genes 0.000 claims 3
- 201000011278 ornithine carbamoyltransferase deficiency Diseases 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 2
- 125000003729 nucleotide group Chemical group 0.000 claims 2
- 101710169336 5'-deoxyadenosine deaminase Proteins 0.000 claims 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 1
- 102000055025 Adenosine deaminases Human genes 0.000 claims 1
- 206010003658 Atrial Fibrillation Diseases 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 208000001140 Night Blindness Diseases 0.000 claims 1
- 208000014769 Usher Syndromes Diseases 0.000 claims 1
- 208000018839 Wilson disease Diseases 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 238000007385 chemical modification Methods 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 108091092562 ribozyme Proteins 0.000 claims 1
- 201000011296 tyrosinemia Diseases 0.000 claims 1
- 238000000034 method Methods 0.000 description 66
- 108020004566 Transfer RNA Proteins 0.000 description 33
- 108091028043 Nucleic acid sequence Proteins 0.000 description 24
- 108090000623 proteins and genes Proteins 0.000 description 19
- 238000010357 RNA editing Methods 0.000 description 18
- 230000026279 RNA modification Effects 0.000 description 18
- 238000003259 recombinant expression Methods 0.000 description 18
- 102000004169 proteins and genes Human genes 0.000 description 14
- 108020004705 Codon Proteins 0.000 description 13
- 208000035475 disorder Diseases 0.000 description 10
- 108020005098 Anticodon Proteins 0.000 description 9
- 102000001039 Dystrophin Human genes 0.000 description 8
- 108010069091 Dystrophin Proteins 0.000 description 8
- 102000014150 Interferons Human genes 0.000 description 6
- 108010050904 Interferons Proteins 0.000 description 6
- 101710125418 Major capsid protein Proteins 0.000 description 6
- 150000001413 amino acids Chemical class 0.000 description 6
- 108020001778 catalytic domains Proteins 0.000 description 6
- 229940079322 interferon Drugs 0.000 description 6
- ZFOMKMMPBOQKMC-KXUCPTDWSA-N L-pyrrolysine Chemical compound C[C@@H]1CC=N[C@H]1C(=O)NCCCC[C@H]([NH3+])C([O-])=O ZFOMKMMPBOQKMC-KXUCPTDWSA-N 0.000 description 5
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 description 4
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 description 4
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 3
- 108091033409 CRISPR Proteins 0.000 description 3
- 101710132601 Capsid protein Proteins 0.000 description 3
- 101710094648 Coat protein Proteins 0.000 description 3
- 102220610160 DBH-like monooxygenase protein 1_E488Q_mutation Human genes 0.000 description 3
- 102100021181 Golgi phosphoprotein 3 Human genes 0.000 description 3
- 108020005004 Guide RNA Proteins 0.000 description 3
- 102000006992 Interferon-alpha Human genes 0.000 description 3
- 108010047761 Interferon-alpha Proteins 0.000 description 3
- 101710141454 Nucleoprotein Proteins 0.000 description 3
- 101710083689 Probable capsid protein Proteins 0.000 description 3
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 description 3
- 108020005038 Terminator Codon Proteins 0.000 description 3
- 102220362718 c.298G>C Human genes 0.000 description 3
- 102000004196 processed proteins & peptides Human genes 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 description 3
- 241001515965 unidentified phage Species 0.000 description 3
- 102000052866 Amino Acyl-tRNA Synthetases Human genes 0.000 description 2
- 108700028939 Amino Acyl-tRNA Synthetases Proteins 0.000 description 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 description 2
- 230000037213 diet Effects 0.000 description 2
- 235000005911 diet Nutrition 0.000 description 2
- 102000003960 Ligases Human genes 0.000 description 1
- 108090000364 Ligases Proteins 0.000 description 1
- 230000002708 enhancing effect Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023023308A JP2023075118A (ja) | 2017-03-03 | 2023-02-17 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| JP2025008232A JP2025066771A (ja) | 2017-03-03 | 2025-01-21 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762466961P | 2017-03-03 | 2017-03-03 | |
| US62/466,961 | 2017-03-03 | ||
| US201762551732P | 2017-08-29 | 2017-08-29 | |
| US62/551,732 | 2017-08-29 | ||
| PCT/US2018/020762 WO2018161032A1 (en) | 2017-03-03 | 2018-03-02 | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023308A Division JP2023075118A (ja) | 2017-03-03 | 2023-02-17 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020508685A JP2020508685A (ja) | 2020-03-26 |
| JP2020508685A5 true JP2020508685A5 (cg-RX-API-DMAC7.html) | 2021-07-26 |
Family
ID=63370240
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019547704A Pending JP2020508685A (ja) | 2017-03-03 | 2018-03-02 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| JP2023023308A Pending JP2023075118A (ja) | 2017-03-03 | 2023-02-17 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| JP2025008232A Pending JP2025066771A (ja) | 2017-03-03 | 2025-01-21 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023023308A Pending JP2023075118A (ja) | 2017-03-03 | 2023-02-17 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
| JP2025008232A Pending JP2025066771A (ja) | 2017-03-03 | 2025-01-21 | サプレッサーｔＲＮＡ及びデアミナーゼによる変異のＲＮＡターゲティング |
Country Status (7)
| Country | Link |
|---|---|
| US (8) | US11932856B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3589751A4 (cg-RX-API-DMAC7.html) |
| JP (3) | JP2020508685A (cg-RX-API-DMAC7.html) |
| CN (2) | CN118416088A (cg-RX-API-DMAC7.html) |
| GB (1) | GB2574769A (cg-RX-API-DMAC7.html) |
| IL (1) | IL268763A (cg-RX-API-DMAC7.html) |
| WO (1) | WO2018161032A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| WO2016097212A1 (en) * | 2014-12-17 | 2016-06-23 | Proqr Therapeutics Ii B.V. | Targeted rna editing |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| GB2574769A (en) * | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| US20200332272A1 (en) * | 2017-10-23 | 2020-10-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| WO2019090154A1 (en) | 2017-11-02 | 2019-05-09 | University Of Iowa Research Foundation | Methods of rescuing stop codons via genetic reassignment with ace-trna |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| WO2019126709A1 (en) * | 2017-12-22 | 2019-06-27 | The Broad Institute, Inc. | Cas12b systems, methods, and compositions for targeted dna base editing |
| WO2020001793A1 (en) * | 2018-06-29 | 2020-01-02 | Eberhard-Karls-Universität Tübingen | Artificial nucleic acids for rna editing |
| EP3833761A1 (en) * | 2018-08-07 | 2021-06-16 | The Broad Institute, Inc. | Novel cas12b enzymes and systems |
| JP2021536239A (ja) | 2018-08-28 | 2021-12-27 | ロシュ イノベーション センター コペンハーゲン エーエス | スプライス調節化合物を使用したネオアンチゲン操作 |
| US10905778B2 (en) | 2018-09-26 | 2021-02-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| JP2022501055A (ja) * | 2018-09-26 | 2022-01-06 | ケース ウエスタン リザーブ ユニバーシティ | 未成熟終止コドン媒介性障害を治療するための方法および組成物 |
| US20210355494A1 (en) * | 2018-10-12 | 2021-11-18 | Peking University | Methods and compositions for editing rnas |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| CN113454216A (zh) * | 2018-11-29 | 2021-09-28 | 旗舰先锋创新V股份有限公司 | 调节rna的方法 |
| CA3125299A1 (en) * | 2019-01-04 | 2020-07-09 | The University Of Chicago | Systems and methods for modulating rna |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| CN111793627A (zh) * | 2019-04-08 | 2020-10-20 | 中国科学院上海生命科学研究院 | 利用人工构建rna编辑酶进行rna定点编辑及相关应用 |
| WO2020208169A1 (en) * | 2019-04-11 | 2020-10-15 | Universität Hamburg | Synthetic transfer rna with extended anticodon loop |
| LU101182B1 (en) * | 2019-04-11 | 2020-10-12 | Univ Hamburg | Synthetic transfer RNA with extended anticodon loop |
| CR20210572A (es) | 2019-04-15 | 2022-04-07 | Univ Beijing | Métodos y composiciones para editar ácido ribonucleicos (arn) |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| 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. |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| WO2021072201A1 (en) * | 2019-10-11 | 2021-04-15 | University Of Massachusetts | Raav-mediated in vivo delivery of suppressor trnas |
| AU2020396868A1 (en) * | 2019-12-02 | 2022-07-14 | Shape Therapeutics Inc. | Targeted transfer RNAS for treatment of diseases |
| EP4069842A1 (en) | 2019-12-02 | 2022-10-12 | Shape Therapeutics Inc. | Therapeutic editing |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| CN111326211B (zh) * | 2020-01-07 | 2023-12-19 | 深圳市早知道科技有限公司 | 一种检测地中海贫血基因变异的方法及检测装置 |
| WO2021142343A1 (en) * | 2020-01-09 | 2021-07-15 | The Regents Of The University Of California | Targeting transfer rna for the suppression of nonsense mutations in messenger rna |
| BR112022017704A2 (pt) * | 2020-03-04 | 2022-11-01 | Suzhou Qi Biodesign Biotechnology Company Ltd | Método e sistema de edição de genoma multiplex |
| WO2021182474A1 (ja) * | 2020-03-12 | 2021-09-16 | 株式会社Ｆｒｅｓｔ | オリゴヌクレオチド及び標的ｒｎａの部位特異的編集方法 |
| IL297243A (en) | 2020-04-14 | 2022-12-01 | Flagship Pioneering Innovations Vi Llc | trem compositions and their uses |
| EP4150076A1 (en) * | 2020-05-15 | 2023-03-22 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2) |
| US20230242906A1 (en) * | 2020-06-29 | 2023-08-03 | Bar-Ilan University | Composition and methods for identifying antisense guide rna for rna editing |
| WO2022078569A1 (en) * | 2020-10-12 | 2022-04-21 | Eberhard Karls Universität Tübingen | Artificial nucleic acids for rna editing |
| US20240150754A1 (en) | 2020-12-25 | 2024-05-09 | Astellas Pharma Inc. | Guide rna for editing polyadenylation signal sequence of target rna |
| JP2024504608A (ja) * | 2021-01-12 | 2024-02-01 | 北京大学 | 遺伝子操作ｒｎａを使用した内因性ａｄａｒの活用による標的化ｒｎａの編集 |
| EP4355906A2 (en) * | 2021-06-15 | 2024-04-24 | Massachusetts Institute Of Technology | Deaminase-based rna sensors |
| WO2023034582A1 (en) | 2021-09-03 | 2023-03-09 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| CN119213136A (zh) * | 2022-03-30 | 2024-12-27 | 匹兹堡大学联邦系统高等教育 | 用于将核酸递送至视网膜细胞的腺相关病毒载体 |
| TW202405173A (zh) | 2022-04-18 | 2024-02-01 | 美商維泰克斯製藥公司 | 用於增強aav療法及降低aav向肝臟之趨性的組合物及方法 |
| WO2023206088A1 (en) * | 2022-04-26 | 2023-11-02 | Huigene Therapeutics Co., Ltd. | Rna base editor for treating dmd-associated diseases |
| CN114908066B (zh) * | 2022-05-17 | 2024-01-23 | 杭州嵌化合生医药科技有限公司 | 一种正交翻译系统及其在再分配密码子恢复ptc疾病中功能蛋白表达方面的应用 |
| WO2024155745A1 (en) * | 2023-01-18 | 2024-07-25 | The Broad Institute, Inc. | Base editing-mediated readthrough of premature termination codons (bert) |
| WO2024155741A1 (en) * | 2023-01-18 | 2024-07-25 | The Broad Institute, Inc. | Prime editing-mediated readthrough of premature termination codons (pert) |
Family Cites Families (38)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5759833A (en) | 1994-05-27 | 1998-06-02 | Cubist Pharmaceuticals, Inc. | Human isoleucyl-tRNA synthetase proteins, nucleic acids and tester strains comprising same |
| JP2002508959A (ja) * | 1998-01-14 | 2002-03-26 | ヒューマン ジーン セラピー リサーチ インスティテュート | ヒトのサプレッサーｔＲＮＡオリゴヌクレオチド、及びそれを用いる方法 |
| WO2005094370A2 (en) | 2004-03-29 | 2005-10-13 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| WO2005111211A2 (en) | 2004-05-14 | 2005-11-24 | Rosetta Genomics Ltd. | Micronas and uses thereof |
| US20100184833A1 (en) | 2006-08-11 | 2010-07-22 | Prosenta Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| IL284530B (en) | 2011-11-18 | 2022-07-01 | Alnylam Pharmaceuticals Inc | rnai factors, preparations and methods of using them for the treatment of transthyretin-related diseases |
| HUE033431T2 (en) | 2012-04-23 | 2017-11-28 | Biomarin Tech Bv | RNA modulating oligonucleotides with improved characteristics in the treatment of neuromuscular disorders |
| DK2852668T3 (en) | 2012-07-12 | 2016-05-30 | Proqr Therapeutics Ii Bv | Oligonucleotides TO COMPLETE A CHANGE IN SEQUENCE OF A target RNA molecule THERE IS IN A LIVING CELL |
| US9650627B1 (en) | 2012-07-19 | 2017-05-16 | University Of Puerto Rico | Site-directed RNA editing |
| IL239317B (en) * | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| US20140186919A1 (en) | 2012-12-12 | 2014-07-03 | Feng Zhang | Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation |
| CN105793425B (zh) * | 2013-06-17 | 2021-10-26 | 布罗德研究所有限公司 | 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用 |
| WO2015120197A1 (en) * | 2014-02-05 | 2015-08-13 | Regents Of The University Of California | Rna editing biomarkers for diagnosis, pharmacological screening and prognostication in cancer |
| CN107532162A (zh) | 2014-12-12 | 2018-01-02 | 托德·M·伍尔夫 | 用于利用寡核苷酸编辑细胞中核酸的组合物和方法 |
| WO2016097212A1 (en) | 2014-12-17 | 2016-06-23 | Proqr Therapeutics Ii B.V. | Targeted rna editing |
| WO2016176191A1 (en) * | 2015-04-27 | 2016-11-03 | The Trustees Of The University Of Pennsylvania | Dual aav vector system for crispr/cas9 mediated correction of human disease |
| JP6624743B2 (ja) * | 2015-07-14 | 2019-12-25 | 学校法人福岡大学 | 部位特異的ｒｎａ変異導入方法およびそれに使用する標的編集ガイドｒｎａならびに標的ｒｎａ−標的編集ガイドｒｎａ複合体 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| DE102015012522B3 (de) | 2015-09-26 | 2016-06-02 | Eberhard Karls Universität Tübingen | Verfahren und Substanzen zur gerichteten RNA-Editierung |
| CN114533900A (zh) | 2015-10-09 | 2022-05-27 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| EP3458074B1 (en) | 2016-05-16 | 2024-04-17 | Board of Regents of the University of Texas System | COMPOSITIONS FOR THE DELIVERY OF tRNA AS NANOPARTICLES AND METHODS OF USE THEREWITH |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| GB2574769A (en) * | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| CA3063739A1 (en) | 2017-05-18 | 2018-11-22 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| WO2018237372A1 (en) | 2017-06-23 | 2018-12-27 | Cornell University | Rna molecules, methods of producing circular rna, and treatment methods |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019071048A1 (en) | 2017-10-04 | 2019-04-11 | The Broad Institute, Inc. | SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION |
| AU2018345919B2 (en) | 2017-10-06 | 2025-08-21 | Oregon Health & Science University | Compositions and methods for editing RNA |
| CA3100034A1 (en) | 2018-05-11 | 2019-11-14 | Beam Therapeutics Inc. | Methods of editing single nucleotide polymorphism using programmable base editor systems |
| WO2020001793A1 (en) | 2018-06-29 | 2020-01-02 | Eberhard-Karls-Universität Tübingen | Artificial nucleic acids for rna editing |
| BR112021001343A2 (pt) | 2018-07-24 | 2021-05-04 | Flagship Pioneering Innovations Vi, Llc | composições compreendendo poliribonucleotídeos circulares e seus usos |
| WO2020051555A1 (en) * | 2018-09-06 | 2020-03-12 | The Regents Of The University Of California | Rna and dna base editing via engneered adar recruitment |
| US20210355494A1 (en) | 2018-10-12 | 2021-11-18 | Peking University | Methods and compositions for editing rnas |
| US11453878B2 (en) | 2019-01-22 | 2022-09-27 | Korro Bio, Inc. | RNA-editing oligonucleotides and uses thereof |
| KR20210132022A (ko) | 2019-01-22 | 2021-11-03 | 코로 바이오, 인크. | Rna-편집 올리고뉴클레오타이드 및 그의 용도 |
| KR20210129645A (ko) | 2019-01-22 | 2021-10-28 | 코로 바이오, 인크. | Rna-편집 올리고뉴클레오타이드 및 그의 용도 |
-
2018
- 2018-03-02 GB GB201913819A patent/GB2574769A/en not_active Withdrawn
- 2018-03-02 EP EP18760638.9A patent/EP3589751A4/en active Pending
- 2018-03-02 WO PCT/US2018/020762 patent/WO2018161032A1/en not_active Ceased
- 2018-03-02 CN CN202410435171.0A patent/CN118416088A/zh active Pending
- 2018-03-02 JP JP2019547704A patent/JP2020508685A/ja active Pending
- 2018-03-02 US US16/490,494 patent/US11932856B2/en active Active
- 2018-03-02 CN CN201880029395.4A patent/CN110612353A/zh active Pending
-
2019
- 2019-08-18 IL IL26876319A patent/IL268763A/en unknown
-
2020
- 2020-05-01 US US16/864,911 patent/US11479775B2/en active Active
-
2021
- 2021-02-08 US US17/170,693 patent/US20210163948A1/en not_active Abandoned
- 2021-02-08 US US17/170,671 patent/US11608503B2/en active Active
-
2022
- 2022-03-04 US US17/687,411 patent/US20220186226A1/en not_active Abandoned
- 2022-08-25 US US17/822,338 patent/US20230139474A1/en not_active Abandoned
-
2023
- 2023-02-17 JP JP2023023308A patent/JP2023075118A/ja active Pending
- 2023-11-13 US US18/507,935 patent/US20240352465A1/en active Pending
-
2024
- 2024-07-26 US US18/786,394 patent/US20240392303A1/en active Pending
-
2025
- 2025-01-21 JP JP2025008232A patent/JP2025066771A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020508685A5 (cg-RX-API-DMAC7.html) | ||
| US20230365962A1 (en) | Targeted rna editing by leveraging endogenous adar using engineered rnas | |
| US20230139474A1 (en) | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES | |
| JP7381494B2 (ja) | 転写調節要素及びその使用 | |
| CN112040988A (zh) | 修饰的aav衣壳多肽用于治疗肌肉疾病 | |
| BR112021006844A2 (pt) | métodos para editar um rna alvo e para tratar ou prevenir uma doença ou condição, rna editado ou uma célula hospedeira tendo um rna editado, rna que recruta desaminase, construção, biblioteca, composição, célula hospedeira, e, kit para editar um rna alvo . | |
| JP2022115976A (ja) | ユートロフィン遺伝子を標的とした筋ジストロフィーの治療方法 | |
| EA036064B1 (ru) | Гибридный белок альбумин-связывающей молекулы и аргининдезиминазы для лечения рака и аргинин-зависимых болезней | |
| US20240254464A1 (en) | Cleavage-inactive cas12f1, cleavage-inactive cas12f1-based fusion protein, crispr gene-editing system comprising same, and preparation method and use thereof | |
| CN116949012B (zh) | 一种融合蛋白及其应用 | |
| KR20220008863A (ko) | 유전자 치료를 위한 변형된 아데노-연관 바이러스(aav) 입자들 | |
| WO2022232442A2 (en) | Multiplex crispr/cas9-mediated target gene activation system | |
| US20240376466A1 (en) | Genome editing compositions and methods for treatment of fanconi anemia | |
| Svahn et al. | Adding functional entities to plasmids | |
| EP4502153A1 (en) | Gene editing system for treating usher syndrome | |
| JP2022551931A (ja) | 改変エンドヌクレアーゼおよび関連方法 | |
| KR20240141024A (ko) | E1347A DddAtox 변이체-TnpB fusion 단백질을 이용한 유전자 가위 시스템 | |
| US20250223587A1 (en) | Compositions and methods for treating a congenital eye disease | |
| AU2019258830B2 (en) | Modified AAV capsid polypeptides for treatment of muscular diseases | |
| US20230220007A1 (en) | Setd7 epigenetic modulators | |
| WO2025153509A1 (en) | Improved prime editing polypeptide | |
| KR20240027748A (ko) | Rbm20 돌연변이의 게놈 편집 | |
| WO2024229240A2 (en) | Compositions and methods for treating stargardt disease | |
| WO2023196220A2 (en) | Method for genome-wide functional perturbation of human microsatellites using engineered zinc fingers |