IL268763A - Targeted RNA mutations using repressor tRNAs and deaminases - Google Patents
Targeted RNA mutations using repressor tRNAs and deaminasesInfo
- Publication number
- IL268763A IL268763A IL26876319A IL26876319A IL268763A IL 268763 A IL268763 A IL 268763A IL 26876319 A IL26876319 A IL 26876319A IL 26876319 A IL26876319 A IL 26876319A IL 268763 A IL268763 A IL 268763A
- Authority
- IL
- Israel
- Prior art keywords
- deaminases
- rna targeting
- suppressor trnas
- mutations via
- via suppressor
- Prior art date
Links
- 108091032973 (ribonucleotides)n+m Proteins 0.000 title 1
- 108020004566 Transfer RNA Proteins 0.000 title 1
- 230000035772 mutation Effects 0.000 title 1
- 230000008685 targeting Effects 0.000 title 1
Classifications
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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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/115—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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/53—Ligases (6)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- 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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (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)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
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 (1)
| Publication Number | Publication Date |
|---|---|
| IL268763A true IL268763A (en) | 2019-10-31 |
Family
ID=63370240
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL26876319A IL268763A (en) | 2017-03-03 | 2019-08-18 | Targeted RNA mutations using repressor 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 (56)
| 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 |
| ES2810375T3 (es) * | 2014-12-17 | 2021-03-08 | Proqr Therapeutics Ii Bv | Edición direccionada de ARN |
| SG10202104041PA (en) | 2015-10-23 | 2021-06-29 | Harvard College | Nucleobase editors and uses thereof |
| 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 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| EP3592381A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Cancer vaccine |
| WO2018165629A1 (en) | 2017-03-10 | 2018-09-13 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| 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. | SYSTEMS, PROCEDURES AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITING |
| WO2019090169A1 (en) | 2017-11-02 | 2019-05-09 | The Wistar Institute Of Anatomy And Biology | Methods of rescuing stop codons via genetic reassignment with ace-trna |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes 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 |
| JP7347830B2 (ja) * | 2018-06-29 | 2023-09-20 | エバーハルト カール ウニヴェルジテート テュービンゲン | Rna編集のための人工核酸 |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| WO2020033601A1 (en) * | 2018-08-07 | 2020-02-13 | 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 |
| 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 |
| US10905778B2 (en) | 2018-09-26 | 2021-02-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| EP4527931A1 (en) * | 2018-10-12 | 2025-03-26 | Peking University | Methods and compositions for editing rnas |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| JP2022513159A (ja) * | 2018-11-29 | 2022-02-07 | フラッグシップ パイオニアリング イノベーションズ ブイ, インコーポレイテッド | Rnaを調節する方法 |
| 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 |
| EP3942043A2 (en) | 2019-03-19 | 2022-01-26 | The Broad Institute, 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 |
| EP3953469A1 (en) * | 2019-04-11 | 2022-02-16 | Arcturus Therapeutics, Inc. | Synthetic transfer rna with extended anticodon loop |
| LU101182B1 (en) * | 2019-04-11 | 2020-10-12 | Univ Hamburg | Synthetic transfer RNA with extended anticodon loop |
| PH12021552613A1 (en) | 2019-04-15 | 2022-06-27 | Edigene Therapeutics Beijing Inc | METHODS AND COMPOSITIONS FOR EDITING RNAs |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN114269919B (zh) | 2019-07-12 | 2023-08-18 | 北京大学 | 采用工程化rna利用内源adar进行靶向的rna编辑 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US20230089490A1 (en) * | 2019-10-11 | 2023-03-23 | University Of Massachusetts | Raav-mediated in vivo delivery of suppressor trnas |
| CA3163514A1 (en) * | 2019-12-02 | 2021-06-10 | David HUSS | Targeted transfer rnas for treatment of diseases |
| WO2021113390A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Compositions for treatment of diseases |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| 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 |
| US20230053353A1 (en) * | 2020-01-09 | 2023-02-23 | The Regents Of The University Of California | Targeting transfer rna for the suppression of nonsense mutations in messenger rna |
| US20240117368A1 (en) * | 2020-03-04 | 2024-04-11 | Suzhou Qi Biodesign Biotechnology Company Limited | Multiplex genome editing method and system |
| 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) |
| US20230242906A1 (en) * | 2020-06-29 | 2023-08-03 | Bar-Ilan University | Composition and methods for identifying antisense guide rna for rna editing |
| US20230313231A1 (en) * | 2020-09-08 | 2023-10-05 | The Regents Of The University Of California | Rna and dna base editing via engineered adar |
| 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 |
| WO2022150974A1 (en) * | 2021-01-12 | 2022-07-21 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| AU2022291759A1 (en) * | 2021-06-15 | 2023-12-21 | Massachusetts Institute Of Technology | Deaminase-based rna sensors |
| IL311101A (en) | 2021-09-03 | 2024-04-01 | Tacit Therapeutics Inc | RNA editing through recruitment of SPLICEOSOME components |
| US20250327092A1 (en) * | 2022-03-30 | 2025-10-23 | University Of Pittsburgh - Of The Commonwealth System Of Higher Education | Adeno-associated virus vectors for nucleic acid delivery to retinal cells |
| CA3249261A1 (en) | 2022-04-18 | 2023-10-26 | Vertex Pharmaceuticals Incorporated | 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 |
| WO2024137990A2 (en) | 2022-12-21 | 2024-06-27 | Trustees Of Boston University | Compositions and methods for controlled mrna translation and stability |
| EP4652272A1 (en) * | 2023-01-18 | 2025-11-26 | The Broad Institute Inc. | Prime editing-mediated readthrough of premature termination codons (pert) |
| EP4652271A1 (en) * | 2023-01-18 | 2025-11-26 | The Broad Institute, Inc. | Base editing-mediated readthrough of premature termination codons (bert) |
| CN121380197B (zh) * | 2025-12-22 | 2026-04-24 | 北京大学 | 基于假尿苷修饰的rna单碱基编辑系统及方法 |
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 |
| EP1045902B1 (en) * | 1998-01-14 | 2005-07-06 | Human Gene Therapy Research Institute | HUMAN SUPPRESSOR tRNA OLIGONUCLEOTIDES AND METHODS OF USE FOR SAME |
| 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 |
| JP6047270B2 (ja) | 2006-08-11 | 2016-12-21 | バイオマリン テクノロジーズ ベー.フェー. | Dnaリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段 |
| 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 |
| EP3604536B1 (en) | 2012-04-23 | 2024-05-29 | Vico Therapeutics B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders |
| US9605255B2 (en) | 2012-07-12 | 2017-03-28 | Proqr Therapeutics Ii 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 |
| IL239317B (en) * | 2012-12-12 | 2022-07-01 | Broad Inst Inc | Providing, engineering and optimizing systems, methods and compositions for sequence manipulation and therapeutic applications |
| 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 |
| US12359197B2 (en) | 2014-12-12 | 2025-07-15 | Etagen Pharma, Inc. | Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides |
| ES2810375T3 (es) | 2014-12-17 | 2021-03-08 | Proqr Therapeutics Ii Bv | Edición direccionada de ARN |
| 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 |
| 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 |
| WO2017015575A1 (en) | 2015-07-22 | 2017-01-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| DE102015012522B3 (de) | 2015-09-26 | 2016-06-02 | Eberhard Karls Universität Tübingen | Verfahren und Substanzen zur gerichteten RNA-Editierung |
| EP3359523A4 (en) | 2015-10-09 | 2019-07-24 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR |
| US20170354672A1 (en) | 2016-05-16 | 2017-12-14 | The 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 |
| CA3035293A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| 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 |
| WO2018213708A1 (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 FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT |
| 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 |
| CN111727247A (zh) | 2017-10-04 | 2020-09-29 | 博德研究所 | 用于靶向核酸编辑的系统、方法和组合物 |
| US20200291383A1 (en) | 2017-10-06 | 2020-09-17 | Oregon Health & Science University | Compositions and methods for editing rna |
| KR20250133482A (ko) | 2018-05-11 | 2025-09-05 | 빔 테라퓨틱스, 인크. | 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법 |
| JP7347830B2 (ja) | 2018-06-29 | 2023-09-20 | エバーハルト カール ウニヴェルジテート テュービンゲン | Rna編集のための人工核酸 |
| CN112567038A (zh) | 2018-07-24 | 2021-03-26 | 旗舰创业创新第六有限责任公司 | 包含环状多核糖核苷酸的组合物及其用途 |
| US20220010333A1 (en) * | 2018-09-06 | 2022-01-13 | The Regents Of The University Of California | Rna and dna base editing via engineered adar recruitment |
| EP4527931A1 (en) | 2018-10-12 | 2025-03-26 | Peking University | Methods and compositions for editing rnas |
| CA3126947A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| JP2022519184A (ja) | 2019-01-22 | 2022-03-22 | 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 |
| CA3127243A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
-
2018
- 2018-03-02 CN CN202410435171.0A patent/CN118416088A/zh active Pending
- 2018-03-02 GB GB201913819A patent/GB2574769A/en not_active Withdrawn
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- 2018-03-02 EP EP18760638.9A patent/EP3589751A4/en active Pending
- 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
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2019
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2020
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2021
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2022
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| GB201913819D0 (en) | 2019-11-06 |
| JP2020508685A (ja) | 2020-03-26 |
| US11608503B2 (en) | 2023-03-21 |
| JP2023075118A (ja) | 2023-05-30 |
| WO2018161032A1 (en) | 2018-09-07 |
| US11479775B2 (en) | 2022-10-25 |
| US20220186226A1 (en) | 2022-06-16 |
| US20230139474A1 (en) | 2023-05-04 |
| EP3589751A4 (en) | 2021-11-17 |
| CN118416088A (zh) | 2024-08-02 |
| EP3589751A1 (en) | 2020-01-08 |
| US20210198673A1 (en) | 2021-07-01 |
| US20200263180A1 (en) | 2020-08-20 |
| US20240352465A1 (en) | 2024-10-24 |
| GB2574769A (en) | 2019-12-18 |
| US20200277607A1 (en) | 2020-09-03 |
| US11932856B2 (en) | 2024-03-19 |
| CN110612353A (zh) | 2019-12-24 |
| US20240392303A1 (en) | 2024-11-28 |
| US20210163948A1 (en) | 2021-06-03 |
| JP2025066771A (ja) | 2025-04-23 |
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