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.

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Publication number
BR112019005144A2
BR112019005144A2 BR112019005144-9A BR112019005144A BR112019005144A2 BR 112019005144 A2 BR112019005144 A2 BR 112019005144A2 BR 112019005144 A BR112019005144 A BR 112019005144A BR 112019005144 A2 BR112019005144 A2 BR 112019005144A2
Authority
BR
Brazil
Prior art keywords
protein
interest
expression regulatory
expressing
cell
Prior art date
Application number
BR112019005144-9A
Other languages
English (en)
Portuguese (pt)
Inventor
Zangi Lior
Magadum Ajit
Original Assignee
Icahn School Of Medicine At Mount Sinai
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Icahn School Of Medicine At Mount Sinai filed Critical Icahn School Of Medicine At Mount Sinai
Publication of BR112019005144A2 publication Critical patent/BR112019005144A2/pt

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7084Compounds having two nucleosides or nucleotides, e.g. nicotinamide-adenine dinucleotide, flavine-adenine dinucleotide
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67General methods for enhancing the expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0652Cells of skeletal and connective tissues; Mesenchyme
    • C12N5/0657Cardiomyocytes; Heart cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0676Pancreatic 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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Cardiology (AREA)
  • Cell Biology (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Vascular Medicine (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
BR112019005144-9A 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. BR112019005144A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662395701P 2016-09-16 2016-09-16
US62/395,701 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
BR112019005144-9A 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 (3) US11299749B2 (https=)
EP (2) EP3512567B1 (https=)
JP (2) JP7432362B2 (https=)
CN (1) CN110177578B (https=)
AU (2) AU2017326535B2 (https=)
BR (1) BR112019005144A2 (https=)
CA (1) CA3036710A1 (https=)
ES (1) ES2967788T3 (https=)
WO (1) WO2018053414A1 (https=)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna
JP7440868B2 (ja) * 2018-03-30 2024-02-29 国立大学法人京都大学 細胞の製造方法
BR112022002365A2 (pt) 2019-08-09 2022-04-26 Nutcracker Therapeutics Inc Métodos e aparelhos para fabricar e para remover material de uma composição terapêutica
WO2021092440A1 (en) 2019-11-07 2021-05-14 Icahn School Of Medicine At Mount Sinai Synthetic modified rna and uses thereof
EP4058584A4 (en) * 2019-11-11 2023-12-20 Icahn School of Medicine at Mount Sinai GENE DELIVERY SYSTEMS FOR THE TREATMENT OF HEART FAILURE
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
EP4514965A1 (en) * 2022-04-27 2025-03-05 The Penn State Research Foundation Modrna-based cas endonuclease and base editor and uses thereof
US20230364267A1 (en) * 2022-05-13 2023-11-16 The Uab Research Foundation Pre-clinical modified rna approaches used in large animals for muscle and vascular regeneration
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
US20260035707A1 (en) * 2022-07-26 2026-02-05 Modernatx, Inc. Engineered polynucleotides for temporal control of expression
CN116785501A (zh) * 2022-08-04 2023-09-22 上海交通大学医学院附属上海儿童医学中心 促进心脏组织修复的modRNA转染改造的细胞及其应用
WO2024151975A1 (en) 2023-01-13 2024-07-18 Igor Lednev Detecting degradation of a polynucleotide by raman spectroscopy
CN119120571B (zh) * 2024-09-11 2026-03-17 武汉科技大学 一种治疗hiv感染的靶向外泌体嵌合抗原受体分子的构建及其应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH08506008A (ja) 1992-11-18 1996-07-02 アーチ ディベロプメント コーポレイション 心筋及び血管平滑筋へのアデノウィルス介在遺伝子移入
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
KR20240136456A (ko) * 2009-12-07 2024-09-13 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 rna를 포함하는 rna 제제
US9458509B2 (en) * 2010-07-22 2016-10-04 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
EP2694660B1 (en) 2011-04-03 2018-08-08 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
CA2915627A1 (en) * 2012-06-15 2013-12-19 The General Hospital Corporation Inhibitors of micrornas that regulate production of atrial natriuretic peptide (anp) as therapeutics and uses thereof
WO2014093574A1 (en) * 2012-12-13 2014-06-19 Moderna Therapeutics, Inc. Modified polynucleotides for altering cell phenotype
US10538740B2 (en) 2014-03-20 2020-01-21 Kyoto University Method for sorting cardiomyocytes
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules
EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna

Also Published As

Publication number Publication date
JP2023123520A (ja) 2023-09-05
EP3512567B1 (en) 2023-11-01
JP2019528736A (ja) 2019-10-17
CA3036710A1 (en) 2018-03-22
CN110177578B (zh) 2024-02-13
US12215336B2 (en) 2025-02-04
US20190203226A1 (en) 2019-07-04
EP3512567A4 (en) 2020-03-18
US20220220501A1 (en) 2022-07-14
AU2017326535B2 (en) 2023-08-24
EP4316587A3 (en) 2024-04-24
CN110177578A (zh) 2019-08-27
AU2017326535A1 (en) 2019-04-18
US11299749B2 (en) 2022-04-12
US20250154523A1 (en) 2025-05-15
WO2018053414A1 (en) 2018-03-22
ES2967788T3 (es) 2024-05-03
EP4316587A2 (en) 2024-02-07
EP3512567A1 (en) 2019-07-24
JP7432362B2 (ja) 2024-02-16
EP3512567C0 (en) 2023-11-01
AU2023270313A1 (en) 2024-01-04

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