ES2967788T3 - Expresión especifica de célula de Arnmond - Google Patents

Expresión especifica de célula de Arnmond Download PDF

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Publication number
ES2967788T3
ES2967788T3 ES17851710T ES17851710T ES2967788T3 ES 2967788 T3 ES2967788 T3 ES 2967788T3 ES 17851710 T ES17851710 T ES 17851710T ES 17851710 T ES17851710 T ES 17851710T ES 2967788 T3 ES2967788 T3 ES 2967788T3
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ES
Spain
Prior art keywords
modrna
protein
mir
cell
gene
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Legal status (The legal status 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 status listed.)
Active
Application number
ES17851710T
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English (en)
Spanish (es)
Inventor
Lior Zangi
Ajit Magadum
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Icahn School of Medicine at Mount Sinai
Original Assignee
Icahn School of Medicine at Mount Sinai
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Publication of ES2967788T3 publication Critical patent/ES2967788T3/es
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Classifications

    • 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
    • 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
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/67—General methods for enhancing the expression
    • 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
    • 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

Landscapes

  • 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)
ES17851710T 2016-09-16 2017-09-18 Expresión especifica de célula de Arnmond Active ES2967788T3 (es)

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
ES2967788T3 true ES2967788T3 (es) 2024-05-03

Family

ID=61619271

Family Applications (1)

Application Number Title Priority Date Filing Date
ES17851710T Active ES2967788T3 (es) 2016-09-16 2017-09-18 Expresión especifica de célula de Arnmond

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
BR112019005144A2 (pt) 2019-06-04
AU2017326535A1 (en) 2019-04-18
US11299749B2 (en) 2022-04-12
US20250154523A1 (en) 2025-05-15
WO2018053414A1 (en) 2018-03-22
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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