MX2020004587A - Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt. - Google Patents
Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt.Info
- Publication number
- MX2020004587A MX2020004587A MX2020004587A MX2020004587A MX2020004587A MX 2020004587 A MX2020004587 A MX 2020004587A MX 2020004587 A MX2020004587 A MX 2020004587A MX 2020004587 A MX2020004587 A MX 2020004587A MX 2020004587 A MX2020004587 A MX 2020004587A
- Authority
- MX
- Mexico
- Prior art keywords
- arm
- anticodon
- stop codons
- trna
- acceptor
- Prior art date
Links
Classifications
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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
-
- 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
-
- 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
- A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Dispersion Chemistry (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
En ciertas modalidades, la presente invención proporciona un ARN de transferencia (ARNt) modificado que comprende un brazo T, un brazo D, un brazo de anticodón y un brazo aceptor, en donde el brazo T comprende nucleótidos que interaccionan con la proteína del factor de elongación 1 alfa 1, y métodos de uso del mismo. En ciertas modalidades, la presente invención provee un ARN de transferencia (ARNt) modificado que comprende un brazo T, un brazo D, un brazo anticodón y un brazo aceptor, (a) en donde el brazo anticodón comprende un anticodón trinucleotídico, en donde el anticodón es 5'-UCA-3' y reconoce codones de paro TGA, y en donde el brazo aceptor está enlazado operativamente a una arginina, triptófano o glicina; (b) en donde el brazo anticodón comprende un anticodón trinucleotídico, en donde el anticodón es 5'-UUA-3' y reconoce codones de paro TAA, y en donde el brazo aceptor está enlazado operativamente a una glutamina o glutamato; o (c) en donde el brazo anticodón comprende un anticodón trinucleotídico, en donde el anticodón es 5'-CUA-3' y reconoce codones de paro TAG, y en donde el brazo aceptor está enlazado operativamente a un triptófano, glutamato o glutamina.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762580887P | 2017-11-02 | 2017-11-02 | |
| US201862687015P | 2018-06-19 | 2018-06-19 | |
| PCT/US2018/059065 WO2019090154A1 (en) | 2017-11-02 | 2018-11-02 | Methods of rescuing stop codons via genetic reassignment with ace-trna |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2020004587A true MX2020004587A (es) | 2021-04-12 |
Family
ID=66332719
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020004647A MX2020004647A (es) | 2017-11-02 | 2018-11-02 | Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna). |
| MX2020004587A MX2020004587A (es) | 2017-11-02 | 2018-11-02 | Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2020004647A MX2020004647A (es) | 2017-11-02 | 2018-11-02 | Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna). |
Country Status (12)
| Country | Link |
|---|---|
| US (5) | US20230203482A1 (es) |
| EP (2) | EP3703701A4 (es) |
| JP (6) | JP2021502078A (es) |
| KR (2) | KR102930465B1 (es) |
| CN (3) | CN111712248B (es) |
| AU (2) | AU2018360065A1 (es) |
| BR (2) | BR112020008733A2 (es) |
| CA (2) | CA3081737A1 (es) |
| IL (2) | IL274396B2 (es) |
| MX (2) | MX2020004647A (es) |
| RU (2) | RU2020117928A (es) |
| WO (2) | WO2019090154A1 (es) |
Families Citing this family (48)
| 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 |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | 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 | がんワクチン |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| 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 |
| 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) |
| 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 |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| EP3856253A4 (en) * | 2018-09-26 | 2022-07-06 | Case Western Reserve University | METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES MEDIATED BY PREMATURE STOPCODON |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| 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 |
| JP7712270B2 (ja) * | 2019-11-01 | 2025-07-23 | テバード バイオサイエンシズ インコーポレイテッド | 未成熟終止コドン媒介障害を処置するための方法および組成物 |
| CA3163514A1 (en) * | 2019-12-02 | 2021-06-10 | David HUSS | Targeted transfer rnas for treatment of diseases |
| AU2021255583A1 (en) | 2020-04-14 | 2022-11-10 | Flagship Pioneering Innovations Vi, Llc | Trem compositions and uses thereof |
| EP4165183A1 (en) * | 2020-06-12 | 2023-04-19 | University of Rochester | Encoding and expression of ace-trnas |
| IL303886A (en) * | 2020-12-23 | 2023-08-01 | Flagship Pioneering Inc | Compositions of modified trems and uses thereof |
| KR20230127269A (ko) * | 2020-12-31 | 2023-08-31 | 유니버시티 오브 아이오와 리써치 파운데이션 | 센스, 억제자 트랜스퍼 rna 조성물 및 관련된 용도와기능 |
| AU2022227003A1 (en) * | 2021-02-25 | 2023-09-28 | Riboz, Llc | Engineered expression constructs to increase protein expression from synthetic ribonucleic acid (rna) |
| CN117693586A (zh) * | 2021-05-05 | 2024-03-12 | 特瓦德生物科学股份有限公司 | 用于治疗提前终止密码子介导的病症的方法和组合物 |
| CN113308463A (zh) * | 2021-05-12 | 2021-08-27 | 中国农业科学院哈尔滨兽医研究所（中国动物卫生与流行病学中心哈尔滨分中心） | 经过反密码子编辑的tRNA作为分子开关在精准控制蛋白表达及病毒复制中的应用 |
| JPWO2023048291A1 (es) * | 2021-09-24 | 2023-03-30 | ||
| US20250059538A1 (en) * | 2021-12-23 | 2025-02-20 | Shape Therapeutics Inc. | COMPOSITIONS AND METHODS OF tRNA SILENCING |
| WO2023141261A1 (en) | 2022-01-21 | 2023-07-27 | Hc Bioscience, Inc. | Modified transfer rna with improved activity |
| EP4469573A1 (en) * | 2022-01-25 | 2024-12-04 | HC Bioscience, Inc. | Universal suppressor trnas and uses thereof |
| CA3243439A1 (en) | 2022-02-07 | 2023-08-10 | University Of Rochester | SEQUENCES OPTIMIZED FOR ENHANCED DNA EXPRESSION AND/OR NONSENSE MUTATION DELETION |
| CN114717204B (zh) * | 2022-03-08 | 2023-12-26 | 中国农业科学院哈尔滨兽医研究所（中国动物卫生与流行病学中心哈尔滨分中心） | 复制缺陷型伪狂犬病病毒及其构建方法和应用 |
| 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 |
| US20250295687A1 (en) | 2022-05-09 | 2025-09-25 | Flagship Pioneering Innovations Vi, Llc | Trem compositions and methods of use for treating proliferative disorders |
| WO2023220342A2 (en) * | 2022-05-13 | 2023-11-16 | Shape Therapeutics Inc. | Engineered tranfer rnas |
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| CN120769910A (zh) | 2023-02-23 | 2025-10-10 | 罗切斯特大学 | 用于制备封闭端dna线分子的药剂和方法 |
| WO2024197081A1 (en) | 2023-03-20 | 2024-09-26 | Hc Bioscience, Inc. | Minimally-sized dna threads for trna therapy |
| EP4442826A1 (en) | 2023-04-06 | 2024-10-09 | Universität Hamburg | Synthetic dna construct encoding transfer rna |
| EP4458967A1 (en) | 2023-05-02 | 2024-11-06 | Universität Hamburg | Pharmaceutical composition comprising multiple suppressor transfer rnas |
| WO2024243073A1 (en) * | 2023-05-19 | 2024-11-28 | University Of Iowa Research Foundation | Anti-codon engineered suppressor transfer rnas |
| WO2025080866A1 (en) * | 2023-10-10 | 2025-04-17 | Hc Bioscience, Inc. | Trna therapeutics for hemophilia |
| WO2025101685A1 (en) | 2023-11-09 | 2025-05-15 | University Of Rochester | Suppression of nonsense mutations using anticodon engineered (ace)-trnas |
| CN120519519A (zh) * | 2024-02-21 | 2025-08-22 | 珠海科动生物技术有限公司 | 工程化tRNA表达盒 |
| CN118389493B (zh) * | 2024-05-10 | 2025-04-11 | 北京大学 | 一种定点修饰的工程化tRNA及其应用 |
| CN118147231B (zh) * | 2024-05-11 | 2024-07-12 | 北京大学 | 包含工程化转运核糖核酸分子的核酸构建体及其应用 |
| WO2026006151A2 (en) | 2024-06-24 | 2026-01-02 | University Of Rochester | Functionalization of ace-trna encoding synthetic linear picovectors |
| WO2026030209A1 (en) | 2024-07-29 | 2026-02-05 | University Of Rochester | Use of decoding trnas to boost protein expression or function |
| CN120420340A (zh) * | 2025-05-09 | 2025-08-05 | 北京大学 | 用于治疗抑癌基因无义突变的抑制性转运核糖核酸 |
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| GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| 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 |
| EP3765609A1 (en) | 2018-03-15 | 2021-01-20 | Universität Hamburg | Synthetic transfer rna with extended anticodon loop |
| US10905778B2 (en) | 2018-09-26 | 2021-02-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| EP3856253A4 (en) | 2018-09-26 | 2022-07-06 | Case Western Reserve University | METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISEASES MEDIATED BY PREMATURE STOPCODON |
| WO2020208169A1 (en) | 2019-04-11 | 2020-10-15 | Universität Hamburg | Synthetic transfer rna with extended anticodon loop |
| US20230089490A1 (en) | 2019-10-11 | 2023-03-23 | University Of Massachusetts | Raav-mediated in vivo delivery of suppressor trnas |
| JP7712270B2 (ja) | 2019-11-01 | 2025-07-23 | テバード バイオサイエンシズ インコーポレイテッド | 未成熟終止コドン媒介障害を処置するための方法および組成物 |
| JP2023500116A (ja) | 2019-11-04 | 2023-01-04 | フラッグシップ パイオニアリング， インコーポレイテッド | ｃｏｎ－希少コドンのためのＴＲＥＭ組成物及び関連する使用 |
| CA3163514A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Targeted transfer rnas for treatment of diseases |
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2018
- 2018-11-02 CN CN201880085141.4A patent/CN111712248B/zh active Active
- 2018-11-02 RU RU2020117928A patent/RU2020117928A/ru unknown
- 2018-11-02 CA CA3081737A patent/CA3081737A1/en active Pending
- 2018-11-02 WO PCT/US2018/059065 patent/WO2019090154A1/en not_active Ceased
- 2018-11-02 JP JP2020524637A patent/JP2021502078A/ja active Pending
- 2018-11-02 JP JP2020524562A patent/JP7474511B2/ja active Active
- 2018-11-02 RU RU2020117787A patent/RU2020117787A/ru unknown
- 2018-11-02 IL IL274396A patent/IL274396B2/en unknown
- 2018-11-02 AU AU2018360065A patent/AU2018360065A1/en not_active Abandoned
- 2018-11-02 CN CN202410906067.5A patent/CN119286852A/zh active Pending
- 2018-11-02 US US16/760,932 patent/US20230203482A1/en not_active Abandoned
- 2018-11-02 US US16/761,205 patent/US11661600B2/en active Active
- 2018-11-02 MX MX2020004647A patent/MX2020004647A/es unknown
- 2018-11-02 EP EP18872570.9A patent/EP3703701A4/en active Pending
- 2018-11-02 CN CN201880079506.2A patent/CN111818945A/zh active Pending
- 2018-11-02 BR BR112020008733-5A patent/BR112020008733A2/pt not_active Application Discontinuation
- 2018-11-02 KR KR1020207015589A patent/KR102930465B1/ko active Active
- 2018-11-02 KR KR1020207015631A patent/KR102857378B1/ko active Active
- 2018-11-02 MX MX2020004587A patent/MX2020004587A/es unknown
- 2018-11-02 CA CA3081692A patent/CA3081692A1/en active Pending
- 2018-11-02 EP EP18872286.2A patent/EP3703762A4/en active Pending
- 2018-11-02 WO PCT/US2018/059085 patent/WO2019090169A1/en not_active Ceased
- 2018-11-02 BR BR112020008725-4A patent/BR112020008725A2/pt not_active Application Discontinuation
- 2018-11-02 AU AU2018360055A patent/AU2018360055B2/en active Active
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2020
- 2020-04-30 IL IL274356A patent/IL274356A/en unknown
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2023
- 2023-04-21 US US18/137,931 patent/US12559749B2/en active Active
- 2023-04-21 US US18/137,942 patent/US12565649B2/en active Active
- 2023-05-24 JP JP2023085389A patent/JP7474532B2/ja active Active
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2024
- 2024-02-02 US US18/430,760 patent/US20250115905A1/en active Pending
- 2024-02-16 JP JP2024022138A patent/JP7804358B2/ja active Active
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2025
- 2025-03-05 JP JP2025034647A patent/JP2025111411A/ja active Pending
- 2025-11-07 JP JP2025188664A patent/JP2026021534A/ja active Pending
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