CN110177578B - modRNA的细胞特异性表达 - Google Patents

modRNA的细胞特异性表达 Download PDF

Info

Publication number
CN110177578B
CN110177578B CN201780070205.9A CN201780070205A CN110177578B CN 110177578 B CN110177578 B CN 110177578B CN 201780070205 A CN201780070205 A CN 201780070205A CN 110177578 B CN110177578 B CN 110177578B
Authority
CN
China
Prior art keywords
nucleic acid
protein
mir
modrna
seq
Prior art date
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
CN201780070205.9A
Other languages
English (en)
Chinese (zh)
Other versions
CN110177578A (zh
Inventor
L·赞吉
A·马加杜姆
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
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 CN110177578A publication Critical patent/CN110177578A/zh
Application granted granted Critical
Publication of CN110177578B publication Critical patent/CN110177578B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

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)
CN201780070205.9A 2016-09-16 2017-09-18 modRNA的细胞特异性表达 Active CN110177578B (zh)

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 (2)

Publication Number Publication Date
CN110177578A CN110177578A (zh) 2019-08-27
CN110177578B true CN110177578B (zh) 2024-02-13

Family

ID=61619271

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201780070205.9A Active CN110177578B (zh) 2016-09-16 2017-09-18 modRNA的细胞特异性表达

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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012012739A2 (en) * 2010-07-22 2012-01-26 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
CN102947450A (zh) * 2009-12-07 2013-02-27 宾夕法尼亚州大学信托人 用于重编程细胞的包含纯化的经修饰的rna的rna制剂
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

Family Cites Families (8)

* 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
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
EP3512567B1 (en) * 2016-09-16 2023-11-01 Icahn School of Medicine at Mount Sinai Cell-specific expression of modrna

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102947450A (zh) * 2009-12-07 2013-02-27 宾夕法尼亚州大学信托人 用于重编程细胞的包含纯化的经修饰的rna的rna制剂
WO2012012739A2 (en) * 2010-07-22 2012-01-26 President And Fellows Of Harvard College Multiple input biologic classifier circuits for cells
WO2016040395A1 (en) * 2014-09-08 2016-03-17 Massachusetts Institute Of Technology Rna-based logic circuits with rna binding proteins, aptamers and small molecules

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction;Lior等;《Nat Biotechnol》;20140615;898-907页 *

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
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
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

Similar Documents

Publication Publication Date Title
CN110177578B (zh) modRNA的细胞特异性表达
US12410445B2 (en) Vector system for expressing regulatory RNA
KR102121086B1 (ko) 세포에서 단백질을 발현시키는 방법들과 생성물들
CN120435557A (zh) 通过靶向基因阻遏调节乙型肝炎病毒的组合物、系统和方法
Mao et al. MicroRNA-23a is involved in tumor necrosis factor-α induced apoptosis in mesenchymal stem cells and myocardial infarction
JP7620938B2 (ja) Klf誘導心筋再生
JP2011507554A (ja) 遺伝子発現を増加させるための方法および組成物
CN104548134A (zh) miR-144及其抑制剂的应用
CN121868329A (zh) 减轻缺血再灌注损伤的合成物和方法
Dong et al. Non‐coding RNAs in cardiomyocyte proliferation and cardiac regeneration: Dissecting their therapeutic values
EP2545941A1 (en) Method for proliferating cardiomyocytes using micro-rna
JP2020037599A (ja) 新規な治療用抗癌薬の製造及び使用
TW201735932A (zh) 使用微核醣核酸前驅物作爲誘導cd34陽性成體幹細胞增殖之藥物
US20220186228A1 (en) Synthetic microrna mimics
Xue et al. Binding of LncDACH1 to dystrophin impairs the membrane trafficking of Nav1. 5 protein and increases ventricular arrhythmia susceptibility
US20250163420A1 (en) Promotion of Cardiomyocyte Proliferation and Regenerative Treatment of the Heart by Inhibition of microRNA-128
Pan et al. Binding of LncRNA-DACH1 to dystrophin impairs the membrane trafficking of Nav1. 5 protein and increases ventricular arrhythmia susceptibility
Joshi LIN28/let-7 Axis as a Regulator of Myocardial Ischemic Injury
HK40006800A (en) Isolation of extracellular vesicles from red blood cells for gene therapy

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant