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
-
- 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
-
- 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—Liposomes
-
- 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
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)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (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 |
|---|---|---|---|
| MX2020004587A MX2020004587A (es) | 2017-11-02 | 2018-11-02 | Métodos de rescate de codones de paro mediante reasignación genética con ace-arnt. |
| 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). |
Family Applications After (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 (4) | US11661600B2 (es) |
| EP (2) | EP3703762A4 (es) |
| JP (4) | JP7474511B2 (es) |
| KR (2) | KR20200083550A (es) |
| CN (2) | CN111712248A (es) |
| AU (2) | AU2018360055A1 (es) |
| BR (2) | BR112020008725A2 (es) |
| CA (2) | CA3081737A1 (es) |
| IL (2) | IL274356A (es) |
| MX (2) | MX2020004587A (es) |
| RU (2) | RU2020117928A (es) |
| WO (2) | WO2019090154A1 (es) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN111712248A (zh) | 2017-11-02 | 2020-09-25 | 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 |
| CA3114108A1 (en) * | 2018-09-26 | 2020-04-02 | Case Western Reserve University | Methods and compositions for treating a premature stop codon-mediated disorder |
| US20230089490A1 (en) * | 2019-10-11 | 2023-03-23 | University Of Massachusetts | Raav-mediated in vivo delivery of suppressor trnas |
| WO2021087401A1 (en) * | 2019-11-01 | 2021-05-06 | Tevard Bio, Inc. | Methods and compositions for treating a premature termination codon-mediated disorder |
| WO2021113218A1 (en) * | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Targeted transfer rnas for treatment of diseases |
| IL297243A (en) * | 2020-04-14 | 2022-12-01 | Flagship Pioneering Innovations Vi Llc | trem compositions and their uses |
| AU2021289600A1 (en) | 2020-06-12 | 2023-01-19 | University Of Rochester | Encoding and expression of ACE-tRNAs |
| JP2024502044A (ja) * | 2020-12-31 | 2024-01-17 | ユニバーシティー オブ アイオワ リサーチ ファンデーション | センス・サプレッサートランスファーｒｎａ組成物ならびに関連する用途及び機能 |
| IL305421A (en) * | 2021-02-25 | 2023-10-01 | Riboz Llc | Engineered expression vectors for increasing synthetic mRNA expression |
| JP2024517809A (ja) * | 2021-05-05 | 2024-04-23 | 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 |
| 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 |
| WO2023122785A1 (en) * | 2021-12-23 | 2023-06-29 | 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 |
| WO2023147348A1 (en) * | 2022-01-25 | 2023-08-03 | Hc Bioscience, Inc. | Universal suppressor trnas and uses thereof |
| WO2023150753A1 (en) | 2022-02-07 | 2023-08-10 | University Of Rochester | Optimized sequences for enhanced trna expression or/and nonsense mutation suppression |
| 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 |
| WO2023205606A1 (en) | 2022-04-18 | 2023-10-26 | Vertex Pharmaceuticals Incorporated | Compositions and methods for enhancing aav therapy and decreasing tropism of aav to the liver |
| WO2023220083A1 (en) * | 2022-05-09 | 2023-11-16 | 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 |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4687737A (en) | 1982-11-12 | 1987-08-18 | Massachusetts Institute Of Technology | Mammalian suppressor genes |
| US5840702A (en) | 1996-03-22 | 1998-11-24 | Uab Research Foundation | Cystic fibrosis treatment |
| EP1045902B1 (en) | 1998-01-14 | 2005-07-06 | Human Gene Therapy Research Institute | HUMAN SUPPRESSOR tRNA OLIGONUCLEOTIDES AND METHODS OF USE FOR SAME |
| IL159311A0 (en) | 2001-06-13 | 2004-06-01 | Remedies for diseases caused by nonsense mutation | |
| US6964859B2 (en) | 2001-10-16 | 2005-11-15 | Massachusetts Institute Of Technology | Suppressor tRNA system |
| EP1968635B1 (en) | 2005-12-14 | 2014-09-17 | Ambrx, Inc. | Compositions containing, methods involving, and uses of non-natural amino acids and polypeptides |
| GB0600948D0 (en) | 2006-01-18 | 2006-02-22 | Univ Dundee | Prevention/Treatment Of Ichthyosis Vulgaris, Atopy And Other Disorders |
| FR2902439A1 (fr) | 2006-06-14 | 2007-12-21 | Univ Rene Descartes Paris V Et | Arn de transfert chimerique et son utilisation pour la production d'arn par une cellule |
| WO2009086497A2 (en) | 2007-12-28 | 2009-07-09 | Ardell David H | Genetic encryption |
| US20120077186A1 (en) | 2010-05-07 | 2012-03-29 | Oregon Health & Science University | Use of cysteine-derived suppressor trnas for non-native amino acid incorporation |
| WO2016036916A1 (en) | 2014-09-03 | 2016-03-10 | Bioatla, Llc | Discovering and producing conditionally active biologic proteins in the same eukaryotic cell production hosts |
| HRP20170589T2 (hr) | 2014-12-22 | 2023-05-12 | Sandoz Ag | Varijante sekvenci |
| CN107614689A (zh) | 2015-03-27 | 2018-01-19 | 昆士兰大学 | 用于将非天然氨基酸并入蛋白质中的平台 |
| WO2017049409A1 (en) | 2015-09-25 | 2017-03-30 | The Centre For Drug Research And Development | Compositions for promoting readthrough of premature termination codons, and methods of using the same |
| CN107177592B (zh) | 2016-03-10 | 2021-03-30 | 北京大学 | 抑制性tRNA通读提前终止密码子疾病中的截短蛋白 |
| BR112018073671A2 (pt) | 2016-05-16 | 2019-02-26 | The Board Of Regents Of The University Of Texas System | composições para a entrega de rnat como nanopartículas e métodos de uso |
| WO2018031531A1 (en) | 2016-08-08 | 2018-02-15 | President And Fellows Of Harvard College | Engineered bacteria for non-invasive imaging and therapeutic applications |
| EP3589751A4 (en) | 2017-03-03 | 2021-11-17 | The Regents of The University of California | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA AND DEAMINASES |
| CN111712248A (zh) | 2017-11-02 | 2020-09-25 | 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 |
| WO2019175316A1 (en) | 2018-03-15 | 2019-09-19 | Universität Hamburg | Synthetic transfer rna with extended anticodon loop |
| CA3114108A1 (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 |
| EP3953469A1 (en) | 2019-04-11 | 2022-02-16 | Arcturus Therapeutics, Inc. | 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 |
| WO2021087401A1 (en) | 2019-11-01 | 2021-05-06 | Tevard Bio, Inc. | Methods and compositions for treating a premature termination codon-mediated disorder |
| AU2020379762A1 (en) | 2019-11-04 | 2022-05-26 | Flagship Pioneering, Inc. | TREM compositions for con-rare codons and related uses |
| WO2021113218A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Targeted transfer rnas for treatment of diseases |
-
2018
- 2018-11-02 CN CN201880085141.4A patent/CN111712248A/zh active Pending
- 2018-11-02 JP JP2020524562A patent/JP7474511B2/ja active Active
- 2018-11-02 BR BR112020008725-4A patent/BR112020008725A2/pt unknown
- 2018-11-02 RU RU2020117928A patent/RU2020117928A/ru unknown
- 2018-11-02 KR KR1020207015631A patent/KR20200083550A/ko not_active Application Discontinuation
- 2018-11-02 KR KR1020207015589A patent/KR20200090171A/ko not_active Application Discontinuation
- 2018-11-02 CA CA3081737A patent/CA3081737A1/en active Pending
- 2018-11-02 WO PCT/US2018/059065 patent/WO2019090154A1/en active Application Filing
- 2018-11-02 CA CA3081692A patent/CA3081692A1/en active Pending
- 2018-11-02 US US16/761,205 patent/US11661600B2/en active Active
- 2018-11-02 EP EP18872286.2A patent/EP3703762A4/en active Pending
- 2018-11-02 BR BR112020008733-5A patent/BR112020008733A2/pt not_active Application Discontinuation
- 2018-11-02 AU AU2018360055A patent/AU2018360055A1/en active Pending
- 2018-11-02 AU AU2018360065A patent/AU2018360065A1/en active Pending
- 2018-11-02 CN CN201880079506.2A patent/CN111818945A/zh active Pending
- 2018-11-02 RU RU2020117787A patent/RU2020117787A/ru unknown
- 2018-11-02 MX MX2020004587A patent/MX2020004587A/es unknown
- 2018-11-02 US US16/760,932 patent/US20230203482A1/en not_active Abandoned
- 2018-11-02 MX MX2020004647A patent/MX2020004647A/es unknown
- 2018-11-02 JP JP2020524637A patent/JP2021502078A/ja active Pending
- 2018-11-02 EP EP18872570.9A patent/EP3703701A4/en active Pending
- 2018-11-02 WO PCT/US2018/059085 patent/WO2019090169A1/en unknown
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2020
- 2020-04-30 IL IL274356A patent/IL274356A/en unknown
- 2020-05-03 IL IL274396A patent/IL274396A/en unknown
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2023
- 2023-04-21 US US18/137,931 patent/US20240182891A1/en active Pending
- 2023-04-21 US US18/137,942 patent/US20230407300A1/en active Pending
- 2023-05-24 JP JP2023085389A patent/JP7474532B2/ja active Active
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2024
- 2024-02-16 JP JP2024022138A patent/JP2024054361A/ja active Pending
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