GB2574769A - RNA Targeting of mutations via suppressor tRNAs and deaminases - Google Patents
RNA Targeting of mutations via suppressor tRNAs and deaminases Download PDFInfo
- Publication number
- GB2574769A GB2574769A GB201913819A GB201913819A GB2574769A GB 2574769 A GB2574769 A GB 2574769A GB 201913819 A GB201913819 A GB 201913819A GB 201913819 A GB201913819 A GB 201913819A GB 2574769 A GB2574769 A GB 2574769A
- Authority
- GB
- United Kingdom
- Prior art keywords
- deaminases
- rna targeting
- suppressor trnas
- muscular dystrophy
- mutations via
- 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.)
- Withdrawn
Links
- 230000035772 mutation Effects 0.000 title abstract 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title 1
- 108091060545 Nonsense suppressor Proteins 0.000 title 1
- 230000008685 targeting Effects 0.000 title 1
- 238000013459 approach Methods 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 2
- 201000006938 muscular dystrophy Diseases 0.000 abstract 2
- 108091033409 CRISPR Proteins 0.000 abstract 1
- 238000010354 CRISPR gene editing Methods 0.000 abstract 1
- 108020004705 Codon Proteins 0.000 abstract 1
- 201000003883 Cystic fibrosis Diseases 0.000 abstract 1
- 208000015178 Hurler syndrome Diseases 0.000 abstract 1
- 208000026350 Inborn Genetic disease Diseases 0.000 abstract 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 abstract 1
- 206010028980 Neoplasm Diseases 0.000 abstract 1
- 208000005980 beta thalassemia Diseases 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000035475 disorder Diseases 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 208000016361 genetic disease Diseases 0.000 abstract 1
- 230000009437 off-target effect Effects 0.000 abstract 1
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/53—Ligases (6)
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
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- C12Y—ENZYMES
- C12Y603/00—Ligases forming carbon-nitrogen bonds (6.3)
- C12Y603/05—Carbon-nitrogen ligases with glutamine as amido-N-donor (6.3.5)
- C12Y603/05007—Glutaminyl-tRNA synthase (glutamine-hydrolyzing) (6.3.5.7)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
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- C12N2320/00—Applications; Uses
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- C12N2320/31—Combination therapy
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- C12N2320/34—Allele or polymorphism specific uses
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2795/00—Bacteriophages
- C12N2795/00011—Details
- C12N2795/18011—Details ssRNA Bacteriophages positive-sense
- C12N2795/18111—Leviviridae
- C12N2795/18122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
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- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
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- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762466961P | 2017-03-03 | 2017-03-03 | |
| US201762551732P | 2017-08-29 | 2017-08-29 | |
| PCT/US2018/020762 WO2018161032A1 (en) | 2017-03-03 | 2018-03-02 | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| GB201913819D0 GB201913819D0 (en) | 2019-11-06 |
| GB2574769A true GB2574769A (en) | 2019-12-18 |
Family
ID=63370240
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| GB201913819A Withdrawn GB2574769A (en) | 2017-03-03 | 2018-03-02 | RNA Targeting of mutations via suppressor tRNAs and deaminases |
Country Status (7)
| Country | Link |
|---|---|
| US (8) | US11932856B2 (enExample) |
| EP (1) | EP3589751A4 (enExample) |
| JP (3) | JP2020508685A (enExample) |
| CN (2) | CN118416088A (enExample) |
| GB (1) | GB2574769A (enExample) |
| IL (1) | IL268763A (enExample) |
| WO (1) | WO2018161032A1 (enExample) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| DK3234134T3 (da) * | 2014-12-17 | 2020-07-27 | Proqr Therapeutics Ii Bv | Målrettet rna-redigering |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| JP2020508685A (ja) * | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 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 |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | 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 |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| EP3701042A4 (en) * | 2017-10-23 | 2021-08-11 | The Broad Institute, Inc. | NUCLEIC ACID TARGETED EDITING SYSTEMS, METHODS AND COMPOSITIONS |
| IL274396B2 (en) * | 2017-11-02 | 2025-10-01 | Wistar Inst | Methods for rescuing stop codons through genetic rearrangement 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 |
| US20230193242A1 (en) * | 2017-12-22 | 2023-06-22 | The Broad Institute, Inc. | Cas12b systems, methods, and compositions for targeted dna base editing |
| US20220073915A1 (en) * | 2018-06-29 | 2022-03-10 | 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 |
| EP3844274A1 (en) | 2018-08-28 | 2021-07-07 | Roche Innovation Center Copenhagen A/S | Neoantigen engineering using splice modulating compounds |
| WO2020069194A1 (en) * | 2018-09-26 | 2020-04-02 | 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 |
| CA3115864A1 (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 |
| WO2020113135A1 (en) * | 2018-11-29 | 2020-06-04 | Flagship Pioneering Innovations V, Inc. | Methods of modulating rna |
| JP2022516919A (ja) * | 2019-01-04 | 2022-03-03 | ザ・ユニバーシティ・オブ・シカゴ | Rnaをモジュレートするためのシステムおよび方法 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| DE112020001306T5 (de) | 2019-03-19 | 2022-01-27 | Massachusetts Institute Of Technology | Verfahren und zusammensetzungen zur editierung von nukleotidsequenzen |
| 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 |
| WO2020211780A1 (en) | 2019-04-15 | 2020-10-22 | Edigene Inc. | Methods and compositions for editing rnas |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP3997229A4 (en) | 2019-07-12 | 2024-07-03 | Peking University | TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| EP4041317A4 (en) * | 2019-10-11 | 2024-05-22 | University of Massachusetts | RAAV-MEDIATED IN VIVO ADMINISTRATION OF ARNT SUPPRESSANTS |
| CA3159944A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Therapeutic editing |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| CA3163514A1 (en) * | 2019-12-02 | 2021-06-10 | David HUSS | Targeted transfer rnas 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 |
| WO2021175289A1 (zh) * | 2020-03-04 | 2021-09-10 | 中国科学院遗传与发育生物学研究所 | 多重基因组编辑方法和系统 |
| 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 |
| CN115516090A (zh) | 2020-04-14 | 2022-12-23 | 旗舰先锋创新Vi有限责任公司 | Trem组合物及其用途 |
| 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) |
| EP4172336A4 (en) * | 2020-06-29 | 2024-07-24 | 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 |
| EP4277990A4 (en) * | 2021-01-12 | 2025-02-12 | Peking University | TARGETED RNA EDITING BY USING ENDOGENOUS ADAR USING MANIPULATED RNAS |
| IL309025A (en) * | 2021-06-15 | 2024-02-01 | Massachusetts Inst Technology | Deaminase-based RNA sensors |
| JP2024533163A (ja) | 2021-09-03 | 2024-09-12 | タシト・セラピューティクス・インコーポレーテッド | スプライソソーム構成要素のリクルートを介したrna編集 |
| 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 |
| CA3249261A1 (en) | 2022-04-18 | 2023-10-26 | Vertex Pharma | COMPOSITIONS AND PROCESSES TO IMPROVE ADENE-ASSOCIATED VIRUS (AAV) THERAPY AND REDUCE AAV TROPISM TO THE LIVER |
| 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) |
| EP4652272A1 (en) * | 2023-01-18 | 2025-11-26 | The Broad Institute Inc. | Prime editing-mediated readthrough of premature termination codons (pert) |
Citations (2)
| 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 |
| US20020156042A1 (en) * | 1998-01-14 | 2002-10-24 | Panchal Rekha G. | Human suppressor tRNA oligonucleotides and methods of use for same |
Family Cites Families (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8314226B2 (en) | 2004-03-29 | 2012-11-20 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| JP5697297B2 (ja) | 2004-05-14 | 2015-04-08 | ロゼッタ ジノミクス リミテッド | マイクロnasおよびその使用 |
| WO2008018795A1 (en) | 2006-08-11 | 2008-02-14 | Prosensa Technologies B.V. | Methods and means for treating dna repeat instability associated genetic disorders |
| MY167390A (en) | 2011-11-18 | 2018-08-16 | Alnylam Pharmaceuticals Inc | Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases |
| BR112014026285B1 (pt) | 2012-04-23 | 2021-08-31 | Biomarin Technologies B.V | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento de distúrbios neuromusculares |
| WO2014011053A1 (en) | 2012-07-12 | 2014-01-16 | Proqr Therapeutics B.V. | 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 |
| ES2576126T3 (es) | 2012-12-12 | 2016-07-05 | The Broad Institute, Inc. | Modificación por tecnología genética y optimización de sistemas, métodos y composiciones enzimáticas mejorados para la manipulación de secuencias |
| KR20150105956A (ko) * | 2012-12-12 | 2015-09-18 | 더 브로드 인스티튜트, 인코퍼레이티드 | 서열 조작 및 치료적 적용을 위한 시스템, 방법 및 조성물의 전달, 유전자 조작 및 최적화 |
| CA2915795C (en) * | 2013-06-17 | 2021-07-13 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
| 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 |
| DK3234134T3 (da) | 2014-12-17 | 2020-07-27 | Proqr Therapeutics Ii Bv | Målrettet rna-redigering |
| 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 |
| EP3323890A4 (en) | 2015-07-14 | 2019-01-30 | Fukuoka University | METHOD OF INDUSTRYING SITE-SPECIFIC RNA MUTATIONS, TARGETED 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 |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| CN109414451B (zh) | 2016-05-16 | 2022-08-12 | 德克萨斯大学系统董事会 | 用于作为纳米粒递送tRNA的组合物及其使用方法 |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| JP2020508685A (ja) * | 2017-03-03 | 2020-03-26 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 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 |
| CN111417727A (zh) | 2017-05-18 | 2020-07-14 | 博德研究所 | 用于靶向核酸编辑的系统、方法和组合物 |
| EP3642342A4 (en) | 2017-06-23 | 2021-03-17 | Cornell University | RNA MOLECULES, CIRCULAR RNA PRODUCTION METHODS, AND PROCESSING METHODS |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| KR20200074104A (ko) | 2017-10-04 | 2020-06-24 | 더 브로드 인스티튜트, 인코퍼레이티드 | 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물 |
| IL273817B2 (en) | 2017-10-06 | 2025-03-01 | Univ Oregon Health & Science | RNA editing agents and methods |
| KR20250133482A (ko) | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| US20220073915A1 (en) | 2018-06-29 | 2022-03-10 | Eberhard-Karls-Universität-Tübingen | Artificial nucleic acids for rna editing |
| AU2019312269A1 (en) | 2018-07-24 | 2021-03-04 | Flagship Pioneering Innovations Vi, Llc | Compositions comprising circular polyribonucleotides and uses thereof |
| US20220010333A1 (en) * | 2018-09-06 | 2022-01-13 | The Regents Of The University Of California | Rna and dna base editing via engineered adar recruitment |
| CA3115864A1 (en) | 2018-10-12 | 2020-04-16 | Peking University | Methods and compositions for editing rnas |
| EP3914259A4 (en) | 2019-01-22 | 2023-05-17 | Korro Bio, Inc. | RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE |
| CA3127243A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| WO2020154343A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
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- 2025-01-21 JP JP2025008232A patent/JP2025066771A/ja active Pending
Patent Citations (2)
| 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 |
| US20020156042A1 (en) * | 1998-01-14 | 2002-10-24 | Panchal Rekha G. | Human suppressor tRNA oligonucleotides and methods of use for same |
Non-Patent Citations (3)
| Title |
|---|
| BIDDLE, W et al., "Modification of Orthogonal tRNAs: Unexpected Consequences for Sense Codon Reassignment", Nucleic Acids Research, (20161023), vol. 44, no. 21, pages 10042 - 10050, abtract; page 10013, column 1, paragraph 2; page 10044, column 2, paragraph 2; * |
| GATTI, "SMRT Compounds Correct Nonsense Mutations in Primary Immunodeficiency and Other Genetic Models", Annals of the New York Academy of Sciences, (20120200), vol. 1250, pages 1 - 13, Figure 1; page 4, paragraph 3 * |
| KATREKAR, D et al., "In vivo RNA Targeting of Point Mutations via Suppressor tRNAs and Adenosine Deaminases", BioRxiv, (20171027), pages 1 - 25; whole document; * |
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| CN118416088A (zh) | 2024-08-02 |
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| US20230139474A1 (en) | 2023-05-04 |
| US20200263180A1 (en) | 2020-08-20 |
| US11608503B2 (en) | 2023-03-21 |
| JP2025066771A (ja) | 2025-04-23 |
| US20240352465A1 (en) | 2024-10-24 |
| JP2023075118A (ja) | 2023-05-30 |
| US11479775B2 (en) | 2022-10-25 |
| IL268763A (en) | 2019-10-31 |
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