JP2020508685A5 - - Google Patents
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- 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.)
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- 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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3589751A4 (OSRAM) |
| JP (3) | JP2020508685A (OSRAM) |
| CN (2) | CN118416088A (OSRAM) |
| GB (1) | GB2574769A (OSRAM) |
| IL (1) | IL268763A (OSRAM) |
| WO (1) | WO2018161032A1 (OSRAM) |
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 |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| US11932856B2 (en) * | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC 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 |
| CN111712248B (zh) | 2017-11-02 | 2024-07-26 | 衣阿华大学研究基金会 | 使用ACE-tRNA经由遗传重分配拯救终止密码子的方法 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3728575A4 (en) * | 2017-12-22 | 2021-11-24 | The Broad Institute, Inc. | CAS12B SYSTEMS, METHODS AND COMPOSITIONS FOR SPECIFIC EDITING OF DNA BASES |
| CN112752844A (zh) * | 2018-06-29 | 2021-05-04 | 蒂宾根大学 | 用于rna编辑的人工核酸 |
| WO2020033601A1 (en) * | 2018-08-07 | 2020-02-13 | The Broad Institute, Inc. | Novel cas12b enzymes and systems |
| WO2020043750A1 (en) | 2018-08-28 | 2020-03-05 | Roche Innovation Center Copenhagen A/S | Neoantigen engineering using splice modulating compounds |
| AU2019350869A1 (en) * | 2018-09-26 | 2021-05-20 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| US10905778B2 (en) | 2018-09-26 | 2021-02-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| WO2020074001A1 (en) * | 2018-10-12 | 2020-04-16 | 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 |
| EP3887516A1 (en) * | 2018-11-29 | 2021-10-06 | Flagship Pioneering Innovations V, Inc. | Methods of modulating rna |
| US20220048962A1 (en) * | 2019-01-04 | 2022-02-17 | The University Of Chicago | Systems and methods for modulating rna |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| 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) |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | 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 |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| CN115397983A (zh) * | 2019-12-02 | 2022-11-25 | 西普治疗公司 | 用于治疗疾病的靶向转移rna |
| 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 |
| US20240247258A1 (en) * | 2020-03-12 | 2024-07-25 | Frest Inc. | Oligonucleotide and target rna site-specific editing method |
| BR112022020780A2 (pt) | 2020-04-14 | 2022-11-29 | Flagship Pioneering Innovations Vi Llc | Composições de trem e usos das mesmas |
| WO2021231680A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2) |
| WO2022003684A1 (en) * | 2020-06-29 | 2022-01-06 | 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 |
| JP2024523877A (ja) * | 2021-06-15 | 2024-07-02 | マサチューセッツ インスティテュート オブ テクノロジー | デアミナーゼベースのｒｎａセンサー |
| MX2024002647A (es) | 2021-09-03 | 2024-05-10 | Tacit Therapeutics Inc | Edición de arn a través del reclutamiento de componentes de espliceosomas. |
| IL316014A (en) * | 2022-03-30 | 2024-11-01 | Univ Pittsburgh Commonwealth Sys Higher Education | Adeno-associated virus vectors for delivering nucleic acids to retinal cells |
| 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 |
| EP4652271A1 (en) * | 2023-01-18 | 2025-11-26 | 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 |
| AU2225199A (en) * | 1998-01-14 | 1999-08-02 | Human Gene Therapy Research Institute | Human suppressor trna oligonucleotides and methods of use for same |
| US8314226B2 (en) | 2004-03-29 | 2012-11-20 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| CA2566519C (en) | 2004-05-14 | 2020-04-21 | Rosetta Genomics Ltd. | Micrornas and uses thereof |
| ES2373246T3 (es) | 2006-08-11 | 2012-02-01 | Prosensa Technologies B.V. | Oligonucleótidos monocatenarios complementarios de elementos repetitivos para el tratamiento de trastornos genéticos asociados a la inestabilidad de repeticiones de adn. |
| PE20142362A1 (es) | 2011-11-18 | 2015-01-30 | Alnylam Pharmaceuticals Inc | Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr) |
| HRP20171254T1 (hr) | 2012-04-23 | 2018-01-12 | Biomarin Technologies B.V. | Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja |
| NZ703824A (en) | 2012-07-12 | 2018-06-29 | Proqr Therapeutics Ii Bv | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
| US9650627B1 (en) | 2012-07-19 | 2017-05-16 | University Of Puerto Rico | Site-directed RNA editing |
| CN105209621B (zh) | 2012-12-12 | 2021-05-25 | 布罗德研究所有限公司 | 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化 |
| EP4299741A3 (en) * | 2012-12-12 | 2024-02-28 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| KR20160056869A (ko) * | 2013-06-17 | 2016-05-20 | 더 브로드 인스티튜트, 인코퍼레이티드 | 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용 |
| US20170191057A1 (en) * | 2014-02-05 | 2017-07-06 | 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 |
| JP6851319B2 (ja) * | 2015-04-27 | 2021-03-31 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | ヒト疾患のＣＲＩＳＰＲ／Ｃａｓ９媒介性の修正のためのデュアルＡＡＶベクター系 |
| US11390865B2 (en) | 2015-07-14 | 2022-07-19 | Fukuoka University | Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex |
| 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 |
| JP7511326B2 (ja) | 2015-10-09 | 2024-07-05 | ウェイブ ライフ サイエンシズ リミテッド | オリゴヌクレオチド組成物およびその方法 |
| PL3458074T3 (pl) | 2016-05-16 | 2024-11-12 | Board Of Regents Of The University Of Texas System | KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| NZ751483A (en) | 2016-09-01 | 2022-07-01 | Proqr Therapeutics Ii Bv | Chemically modified single-stranded rna-editing oligonucleotides |
| WO2018134301A1 (en) | 2017-01-19 | 2018-07-26 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in rna editing |
| US11932856B2 (en) * | 2017-03-03 | 2024-03-19 | The Regents Of The University Of California | RNA targeting of mutations via suppressor tRNAs and deaminases |
| US11866697B2 (en) | 2017-05-18 | 2024-01-09 | The Broad Institute, Inc. | Systems, methods, and compositions for targeted nucleic acid editing |
| EP3642342A4 (en) | 2017-06-23 | 2021-03-17 | Cornell University | RNA MOLECULES, CIRCULAR RNA PRODUCTION METHODS, AND PROCESSING METHODS |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| KR20250068785A (ko) | 2017-10-04 | 2025-05-16 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물 |
| EP3691747A4 (en) | 2017-10-06 | 2021-10-06 | Oregon Health & Science University | RNA COMPOSITIONS AND EDITING PROCEDURES |
| CN119587720A (zh) | 2018-05-11 | 2025-03-11 | 比姆医疗股份有限公司 | 利用可编程碱基编辑器系统编辑单核苷酸多态性的方法 |
| CN112752844A (zh) | 2018-06-29 | 2021-05-04 | 蒂宾根大学 | 用于rna编辑的人工核酸 |
| AU2019312269A1 (en) | 2018-07-24 | 2021-03-04 | Flagship Pioneering Innovations Vi, Llc | Compositions comprising circular polyribonucleotides and uses thereof |
| CA3110998A1 (en) * | 2018-09-06 | 2020-03-12 | The Regents Of The University Of California | Rna and dna base editing via engneered adar recruitment |
| WO2020074001A1 (en) | 2018-10-12 | 2020-04-16 | Peking University | Methods and Compositions for Editing RNAs |
| AU2020210645B2 (en) | 2019-01-22 | 2025-04-24 | Korro Bio, Inc. | RNA-editing oligonucleotides and uses thereof |
| TW202043469A (zh) | 2019-01-22 | 2020-12-01 | 美商科羅生物公司 | Ｒｎａ編輯之寡核苷酸及其用途 |
| JP2022518731A (ja) | 2019-01-22 | 2022-03-16 | コロ バイオ， インコーポレイテッド | Ｒｎａ編集オリゴヌクレオチド及びその使用 |
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2018
- 2018-03-02 US US16/490,494 patent/US11932856B2/en active Active
- 2018-03-02 WO PCT/US2018/020762 patent/WO2018161032A1/en not_active Ceased
- 2018-03-02 EP EP18760638.9A patent/EP3589751A4/en active Pending
- 2018-03-02 CN CN202410435171.0A patent/CN118416088A/zh active Pending
- 2018-03-02 CN CN201880029395.4A patent/CN110612353A/zh active Pending
- 2018-03-02 GB GB201913819A patent/GB2574769A/en not_active Withdrawn
- 2018-03-02 JP JP2019547704A patent/JP2020508685A/ja active Pending
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2019
- 2019-08-18 IL IL26876319A patent/IL268763A/en unknown
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2020
- 2020-05-01 US US16/864,911 patent/US11479775B2/en active Active
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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
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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
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2023
- 2023-02-17 JP JP2023023308A patent/JP2023075118A/ja active Pending
- 2023-11-13 US US18/507,935 patent/US20240352465A1/en active Pending
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2024
- 2024-07-26 US US18/786,394 patent/US20240392303A1/en active Pending
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2025
- 2025-01-21 JP JP2025008232A patent/JP2025066771A/ja active Pending
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