BR112019005144A2 - sistema regulador de expressão, composição, métodos para expressar uma proteína, para induzir/reativar a proliferação de cardiomiócitos neonatais ou adultos e para expressar um gene de interesse, vetor, kit regulador de expressão, e, uso. - Google Patents
sistema regulador de expressão, composição, métodos para expressar uma proteína, para induzir/reativar a proliferação de cardiomiócitos neonatais ou adultos e para expressar um gene de interesse, vetor, kit regulador de expressão, e, uso.Info
- Publication number
- BR112019005144A2 BR112019005144A2 BR112019005144A BR112019005144A BR112019005144A2 BR 112019005144 A2 BR112019005144 A2 BR 112019005144A2 BR 112019005144 A BR112019005144 A BR 112019005144A BR 112019005144 A BR112019005144 A BR 112019005144A BR 112019005144 A2 BR112019005144 A2 BR 112019005144A2
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- Brazil
- Prior art keywords
- protein
- interest
- expression regulatory
- expressing
- cell
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- C—CHEMISTRY; METALLURGY
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- 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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- 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
- A61K31/7084—Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/67—General methods for enhancing the expression
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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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0652—Cells of skeletal and connective tissues; Mesenchyme
- C12N5/0657—Cardiomyocytes; Heart 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
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0676—Pancreatic cells
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Cardiology (AREA)
- Cell Biology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Heart & Thoracic Surgery (AREA)
- General Chemical & Material Sciences (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Rheumatology (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)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
é descrito um sistema regulador de expressão para a transcrição (expressão) específica de célula de uma proteína de interesse, por exemplo um indutor de ciclo celular que reativa a proliferação em cardiomiócitos adultos ou neonatais ou células beta que produzem insulina. o sistema regulador de expressão compreende um primeiro ácido nucleico que codifica um elemento de reconhecimento de microrna que especificamente liga um mir de célula alvo, e uma proteína supressora de tradução; e um segundo ácido nucleico que compreende um motivo de interação de proteína supressora que liga a proteína supressora de tradução, e um gene que codifica uma proteína de interesse. quando uma célula de interesse é cotransfectada com os primeiro e segundo ácidos nucleicos do sistema, a proteína de interesse é expressa em uma maneira específica de célula.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662395701P | 2016-09-16 | 2016-09-16 | |
PCT/US2017/052035 WO2018053414A1 (en) | 2016-09-16 | 2017-09-18 | Cell-specific expression of modrna |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112019005144A2 true BR112019005144A2 (pt) | 2019-06-04 |
Family
ID=61619271
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112019005144A BR112019005144A2 (pt) | 2016-09-16 | 2017-09-18 | sistema regulador de expressão, composição, métodos para expressar uma proteína, para induzir/reativar a proliferação de cardiomiócitos neonatais ou adultos e para expressar um gene de interesse, vetor, kit regulador de expressão, e, uso. |
Country Status (9)
Country | Link |
---|---|
US (2) | US11299749B2 (pt) |
EP (2) | EP4316587A3 (pt) |
JP (2) | JP7432362B2 (pt) |
CN (1) | CN110177578B (pt) |
AU (2) | AU2017326535B2 (pt) |
BR (1) | BR112019005144A2 (pt) |
CA (1) | CA3036710A1 (pt) |
ES (1) | ES2967788T3 (pt) |
WO (1) | WO2018053414A1 (pt) |
Families Citing this family (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ES2967788T3 (es) * | 2016-09-16 | 2024-05-03 | Icahn School Med Mount Sinai | Expresión especifica de célula de Arnmond |
WO2019189545A1 (ja) * | 2018-03-30 | 2019-10-03 | 国立大学法人京都大学 | 細胞の製造方法 |
JP2022543668A (ja) | 2019-08-09 | 2022-10-13 | ナットクラッカー セラピューティクス, インコーポレイテッド | マイクロ流体装置およびその使用方法 |
CN114945672A (zh) * | 2019-11-11 | 2022-08-26 | 西奈山伊坎医学院 | 用于治疗心力衰竭的基因递送系统 |
EP4351623A1 (en) | 2021-06-09 | 2024-04-17 | Icahn School of Medicine at Mount Sinai | Gene combination as a broad spectrum antiviral |
EP4355882A2 (en) * | 2021-06-15 | 2024-04-24 | Modernatx, Inc. | Engineered polynucleotides for cell-type or microenvironment-specific expression |
CN114085834A (zh) * | 2021-11-16 | 2022-02-25 | 珠海中科先进技术研究院有限公司 | 一种癌细胞导向电路组及应用 |
WO2023212649A1 (en) * | 2022-04-27 | 2023-11-02 | The Penn State Research Foundation | Modrna-based cas endonuclease and base editor and uses thereof |
EP4282963A1 (en) * | 2022-05-23 | 2023-11-29 | Eberhard Karls Universität Tübingen, Medizinische Fakultät | Nucleic acid modified biological cell with expansion-dependent gene expression |
WO2024015729A1 (en) * | 2022-07-11 | 2024-01-18 | Icahn School Of Medicine At Mount Sinai | Regulatory system for expression of a gene of interest in a target cell and method of use thereof |
WO2024026254A1 (en) * | 2022-07-26 | 2024-02-01 | Modernatx, Inc. | Engineered polynucleotides for temporal control of expression |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
ATE307212T1 (de) * | 1992-11-18 | 2005-11-15 | Arch Dev Corp | Adenovirus-gelenkter gen-transfer zum herz-und glatten vaskulären muskel |
US20090005336A1 (en) * | 2007-05-08 | 2009-01-01 | Zhiguo Wang | Use of the microRNA miR-1 for the treatment, prevention, and diagnosis of cardiac conditions |
JP2013512690A (ja) * | 2009-12-07 | 2013-04-18 | ザ トラスティース オブ ザ ユニバーシティ オブ ペンシルベニア | 細胞を再プログラム化するための精製された修飾rnaを含むrna調製物 |
US9458509B2 (en) * | 2010-07-22 | 2016-10-04 | President And Fellows Of Harvard College | Multiple input biologic classifier circuits for cells |
US10086043B2 (en) | 2011-04-03 | 2018-10-02 | The General Hospital Corporation | Efficient protein expression in vivo using modified RNA (MOD-RNA) |
EP2584040A1 (en) * | 2011-10-17 | 2013-04-24 | Pharmahungary 2000 Kft. | Compounds for treatment of ischemic injury |
US9388412B2 (en) * | 2012-06-15 | 2016-07-12 | The General Hospital Corporation | Inhibitors of microRNAs that regulate production of atrial natriuretic peptide (ANP) as therapeutics and uses thereof |
EP2931914A4 (en) * | 2012-12-13 | 2016-08-17 | Moderna Therapeutics Inc | MODIFIED POLYNUCLEOTIDES FOR CHANGING THE CELL PHENOTYPE |
WO2015141827A1 (ja) * | 2014-03-20 | 2015-09-24 | 国立大学法人京都大学 | 心筋細胞の選別方法 |
WO2016040395A1 (en) * | 2014-09-08 | 2016-03-17 | Massachusetts Institute Of Technology | Rna-based logic circuits with rna binding proteins, aptamers and small molecules |
ES2967788T3 (es) * | 2016-09-16 | 2024-05-03 | Icahn School Med Mount Sinai | Expresión especifica de célula de Arnmond |
-
2017
- 2017-09-18 ES ES17851710T patent/ES2967788T3/es active Active
- 2017-09-18 BR BR112019005144A patent/BR112019005144A2/pt unknown
- 2017-09-18 EP EP23205982.4A patent/EP4316587A3/en active Pending
- 2017-09-18 WO PCT/US2017/052035 patent/WO2018053414A1/en active Application Filing
- 2017-09-18 JP JP2019514770A patent/JP7432362B2/ja active Active
- 2017-09-18 EP EP17851710.8A patent/EP3512567B1/en active Active
- 2017-09-18 CA CA3036710A patent/CA3036710A1/en active Pending
- 2017-09-18 AU AU2017326535A patent/AU2017326535B2/en active Active
- 2017-09-18 CN CN201780070205.9A patent/CN110177578B/zh active Active
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2019
- 2019-03-15 US US16/354,814 patent/US11299749B2/en active Active
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2022
- 2022-03-28 US US17/705,609 patent/US20220220501A1/en active Pending
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2023
- 2023-06-07 JP JP2023093720A patent/JP2023123520A/ja active Pending
- 2023-11-23 AU AU2023270313A patent/AU2023270313A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
EP3512567A1 (en) | 2019-07-24 |
JP2019528736A (ja) | 2019-10-17 |
EP3512567B1 (en) | 2023-11-01 |
AU2017326535A1 (en) | 2019-04-18 |
ES2967788T3 (es) | 2024-05-03 |
EP4316587A2 (en) | 2024-02-07 |
EP3512567A4 (en) | 2020-03-18 |
CN110177578A (zh) | 2019-08-27 |
AU2017326535B2 (en) | 2023-08-24 |
CA3036710A1 (en) | 2018-03-22 |
US11299749B2 (en) | 2022-04-12 |
JP7432362B2 (ja) | 2024-02-16 |
US20220220501A1 (en) | 2022-07-14 |
AU2023270313A1 (en) | 2024-01-04 |
WO2018053414A1 (en) | 2018-03-22 |
US20190203226A1 (en) | 2019-07-04 |
EP4316587A3 (en) | 2024-04-24 |
JP2023123520A (ja) | 2023-09-05 |
EP3512567C0 (en) | 2023-11-01 |
CN110177578B (zh) | 2024-02-13 |
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