HUE047165T2 - Tisztított módosított RNS-t tartalmazó RNS készítmények sejtek átprogramozására - Google Patents

Tisztított módosított RNS-t tartalmazó RNS készítmények sejtek átprogramozására

Info

Publication number
HUE047165T2
HUE047165T2 HUE17195966A HUE17195966A HUE047165T2 HU E047165 T2 HUE047165 T2 HU E047165T2 HU E17195966 A HUE17195966 A HU E17195966A HU E17195966 A HUE17195966 A HU E17195966A HU E047165 T2 HUE047165 T2 HU E047165T2
Authority
HU
Hungary
Prior art keywords
rna
purified modified
preparations
reprogramming cells
modified rna
Prior art date
Application number
HUE17195966A
Other languages
English (en)
Inventor
Gary Dahl
Judith Meis
Anthony Person
Jerome Jendrisak
Katalin Kariko
Drew Weissman
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of HUE047165T2 publication Critical patent/HUE047165T2/hu

Links

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
    • 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
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/1816—Erythropoietin [EPO]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44—Oxidoreductases (1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00—Drugs for dermatological disorders
    • A61P17/14—Drugs for dermatological disorders for baldness or alopecia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/06—Immunosuppressants, e.g. drugs for graft rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712—Cystic fibrosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/505—Erythropoietin [EPO]
    • 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
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • 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
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • C12N5/0606—Pluripotent embryonic cells, e.g. embryonic stem cells [ES]
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0073—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen 1.14.13
    • C12N9/0075—Nitric-oxide synthase (1.14.13.39)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/333—Modified A
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/602—Sox-2
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/603—Oct-3/4
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/604—Klf-4
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/605—Nanog
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/606—Transcription factors c-Myc
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60—Transcription factors
    • C12N2501/608—Lin28
    • 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/02—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from embryonic 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
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with NADH or NADPH as one donor, and incorporation of one atom of oxygen (1.14.13)
    • C12Y114/13039—Nitric-oxide synthase (NADPH dependent) (1.14.13.39)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012—Sphingomyelin phosphodiesterase (3.1.4.12)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Transplantation (AREA)
  • Pulmonology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurology (AREA)
HUE17195966A 2009-12-07 2010-12-07 Tisztított módosított RNS-t tartalmazó RNS készítmények sejtek átprogramozására HUE047165T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US26731209P 2009-12-07 2009-12-07

Publications (1)

Publication Number Publication Date
HUE047165T2 true HUE047165T2 (hu) 2020-04-28

Family

ID=44143383

Family Applications (2)

Application Number Title Priority Date Filing Date
HUE17195966A HUE047165T2 (hu) 2009-12-07 2010-12-07 Tisztított módosított RNS-t tartalmazó RNS készítmények sejtek átprogramozására
HUE16181434A HUE036684T2 (hu) 2009-12-07 2010-12-07 Tisztított, módosított RNS-t tartalmazó RNS készítmények sejtek visszaprogramozásához

Family Applications After (1)

Application Number Title Priority Date Filing Date
HUE16181434A HUE036684T2 (hu) 2009-12-07 2010-12-07 Tisztított, módosított RNS-t tartalmazó RNS készítmények sejtek visszaprogramozásához

Country Status (22)

Country Link
US (6) US8808982B2 (hu)
EP (4) EP3623474A1 (hu)
JP (6) JP2013512690A (hu)
KR (4) KR102505097B1 (hu)
CN (3) CN102947450B (hu)
AU (1) AU2010328310B2 (hu)
BR (1) BR112012013875B8 (hu)
CA (2) CA2783032C (hu)
CY (1) CY1120237T1 (hu)
DK (2) DK3112467T3 (hu)
ES (2) ES2769129T3 (hu)
HK (1) HK1251259B (hu)
HR (2) HRP20180737T1 (hu)
HU (2) HUE047165T2 (hu)
IL (1) IL220219A0 (hu)
LT (2) LT3112467T (hu)
NO (2) NO3112467T3 (hu)
PL (3) PL3287525T3 (hu)
PT (1) PT3112467T (hu)
SG (2) SG10201408129XA (hu)
SI (2) SI3112467T1 (hu)
WO (2) WO2011071936A2 (hu)

Families Citing this family (124)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2735531T3 (es) 2005-08-23 2019-12-19 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
WO2008097926A2 (en) * 2007-02-02 2008-08-14 Yale University Transient transfection with rna
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
TR201901311T4 (tr) 2009-12-01 2019-02-21 Translate Bio Inc İnsan genetik hastalıklarında mRNA'nın teslimi için steroid türevi.
KR102505097B1 (ko) 2009-12-07 2023-03-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 ｒｎａ를 포함하는 ｒｎａ 제제
WO2011130624A2 (en) 2010-04-16 2011-10-20 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
PL3243526T3 (pl) 2010-07-06 2020-05-18 Glaxosmithkline Biologicals S.A. Dostarczanie rna w celu wyzwolenia wielu szlaków immunologicznych
MX2013000164A (es) 2010-07-06 2013-03-05 Novartis Ag Liposomas con lipidos que tienen valor de pka ventajoso para suministro de arn.
US10487332B2 (en) 2010-07-06 2019-11-26 Glaxosmithkline Biologicals Sa Immunisation of large mammals with low doses of RNA
WO2012019168A2 (en) 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DK3981427T3 (da) 2010-08-31 2022-07-11 Glaxosmithkline Biologicals Sa Pegylerede liposomer til afgivelse af immunogen-kodende RNA
DE19177059T1 (de) 2010-10-01 2021-10-07 Modernatx, Inc. N1-methyl-pseudouracile enthältendes ribonucleinsäuren sowie ihre verwendungen
EP4098325A1 (en) 2010-10-11 2022-12-07 GlaxoSmithKline Biologicals S.A. Antigen delivery platforms
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
DE12722942T1 (de) 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
CN103906527B (zh) 2011-06-08 2020-07-10 川斯勒佰尔公司 Mrna递送的脂质纳米颗粒组合物和方法
ES2656050T3 (es) 2011-07-06 2018-02-22 Glaxosmithkline Biologicals Sa Composiciones de combinación inmunogénica y usos de las mismas
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
JP6113737B2 (ja) 2011-10-03 2017-04-12 モデルナティエックス インコーポレイテッドＭｏｄｅｒｎａＴＸ，Ｉｎｃ． 修飾型のヌクレオシド、ヌクレオチドおよび核酸、ならびにそれらの使用方法
RU2624139C2 (ru) 2011-12-05 2017-06-30 Фэктор Байосайенс Инк. Способы и препараты для трансфекции клеток
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
EP2791159A4 (en) * 2011-12-14 2015-10-14 Moderna Therapeutics Inc MODIFIED NUCLEIC ACIDS, AND SHORT-TERM CARE USES THEREOF
MX2014007233A (es) 2011-12-16 2015-02-04 Moderna Therapeutics Inc Composiciones de nucleosidos, nucleotidos y acidos nucleicos modificados.
CN104968354A (zh) * 2011-12-21 2015-10-07 现代治疗公司 增加器官或器官外植体的活力或寿命的方法
EP4372081A2 (en) * 2011-12-30 2024-05-22 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect
US10501513B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides for the production of oncology-related proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
EP2834259A4 (en) 2012-04-02 2016-08-24 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
CA2868391A1 (en) * 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
DK2850179T3 (da) * 2012-05-13 2019-06-17 Allele Biotechnology & Pharmaceuticals Inc Feeder-cellefri afledning af human-inducerede pluripotente stamceller med syntetisk messenger-rna
EP2858679B1 (en) 2012-06-08 2021-02-24 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2014028429A2 (en) 2012-08-14 2014-02-20 Moderna Therapeutics, Inc. Enzymes and polymerases for the synthesis of rna
BR112015005011A2 (pt) 2012-09-07 2018-04-17 Genentech Inc métodos e composições para produzir hepatócitos induzidos.
EP2909321A4 (en) * 2012-10-16 2016-06-22 Massachusetts Inst Technology STABLE NON-POLYADENYLNA RNA PRODUCTION
RU2019143431A (ru) 2012-11-01 2020-04-28 Фэктор Байосайенс Инк. Способы и продукты для экспрессии белков в клетках
CA2892529C (en) 2012-11-26 2023-04-25 Moderna Therapeutics, Inc. Terminally modified rna
EP2959005B1 (en) 2013-02-22 2021-10-06 The Board of Trustees of the Leland Stanford Junior University Medical use relating to telomere extension
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US20160032316A1 (en) * 2013-03-14 2016-02-04 The Trustees Of The University Of Pennsylvania Purification and Purity Assessment of RNA Molecules Synthesized with Modified Nucleosides
BR112015022660A2 (pt) 2013-03-14 2017-10-31 Shire Human Genetic Therapies métodos para a purificação de rna mensageiro
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
BR112015022868B1 (pt) 2013-03-14 2023-05-16 Ethris Gmbh Composições de mrna de cftr e usos e métodos relacionados
EP2983804A4 (en) 2013-03-15 2017-03-01 Moderna Therapeutics, Inc. Ion exchange purification of mrna
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
EP3578663A1 (en) 2013-03-15 2019-12-11 ModernaTX, Inc. Manufacturing methods for production of rna transcripts
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
US8980864B2 (en) * 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014186334A1 (en) 2013-05-15 2014-11-20 Robert Kruse Intracellular translation of circular rna
JP7019233B2 (ja) 2013-07-11 2022-02-15 モデルナティエックス インコーポレイテッド ＣＲＩＳＰＲ関連タンパク質をコードする合成ポリヌクレオチドおよび合成ｓｇＲＮＡを含む組成物ならびに使用方法
KR102250027B1 (ko) * 2013-07-26 2021-05-07 교토후고리츠다이가쿠호진 골아 세포 및 그 조제 방법
KR20230074639A (ko) * 2013-08-28 2023-05-30 벡톤 디킨슨 앤드 컴퍼니 대량의 동시 단일 세포 분석
EP3041934A1 (en) 2013-09-03 2016-07-13 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015048744A2 (en) 2013-09-30 2015-04-02 Moderna Therapeutics, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
CA2926218A1 (en) 2013-10-03 2015-04-09 Moderna Therapeutics, Inc. Polynucleotides encoding low density lipoprotein receptor
WO2015061491A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
CA2928188A1 (en) 2013-10-22 2015-04-30 Shire Human Genetic Therapies, Inc. Mrna therapy for argininosuccinate synthetase deficiency
EP3542802A1 (en) 2013-11-01 2019-09-25 CureVac AG Modified rna with decreased immunostimulatory properties
CA2925021A1 (en) 2013-11-01 2015-05-07 Curevac Ag Modified rna with decreased immunostimulatory properties
KR101665165B1 (ko) * 2013-12-04 2016-10-12 단국대학교 산학협력단 신규한 rna 앱타머 및 그의 용도
MX2016009771A (es) 2014-01-31 2016-11-14 Factor Bioscience Inc Metodos y productos para la produccion y administracion de acido nucleico.
EP3991751A1 (en) * 2014-02-10 2022-05-04 The Board of Trustees of the Leland Stanford Junior University Activation of innate immunity for enhanced nuclear reprogramming of somatic cells with mrna
RU2746406C2 (ru) 2014-04-23 2021-04-13 МОДЕРНАТиЭкс, ИНК. Вакцины на основе нуклеиновых кислот
BR112016024632A2 (pt) 2014-04-25 2018-01-30 Shire Human Genetic Therapies métodos de purificação de rna mensageiro
US10286086B2 (en) 2014-06-19 2019-05-14 Modernatx, Inc. Alternative nucleic acid molecules and uses thereof
US10407683B2 (en) 2014-07-16 2019-09-10 Modernatx, Inc. Circular polynucleotides
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3041948B1 (en) 2014-11-10 2019-01-09 Modernatx, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
JP7199809B2 (ja) 2015-02-13 2023-01-06 ファクター バイオサイエンス インコーポレイテッド 核酸製品及びその投与方法
AU2016286364B2 (en) 2015-06-30 2022-07-28 Ethris Gmbh ATP-binding cassette family coding polyribonucleotides and formulations thereof
US10662409B2 (en) * 2015-09-09 2020-05-26 Trustees Of Tufts College Methods of generating neural stem cells
US11434486B2 (en) 2015-09-17 2022-09-06 Modernatx, Inc. Polynucleotides containing a morpholino linker
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
US11446398B2 (en) 2016-04-11 2022-09-20 Obsidian Therapeutics, Inc. Regulated biocircuit systems
EP3445850B1 (en) 2016-04-22 2021-10-27 BioNTech SE Methods for providing single-stranded rna
BR112018073683A2 (pt) 2016-05-18 2019-02-26 Modernatx, Inc. polinucleotídeos codificadores de relaxina
SG11201810827VA (en) * 2016-06-03 2018-12-28 Stemgenics Inc Direct reprogramming of a human somatic cell to a selected (predetermined) differentiated cell with functionalized nanoparticles
CN106226524B (zh) * 2016-07-07 2018-10-09 福建省农业科学院食用菌研究所 一种食用菌dsRNA病毒的检测方法
IL264439B1 (en) 2016-08-17 2024-04-01 Factor Bioscience Inc A suitable viral preparation containing a synthetic messenger RNA encoding a gene editing protein for use in cancer treatment and a synthetic messenger RNA encoding a gene editing protein for use in therapy
MX2019002904A (es) 2016-09-14 2019-09-26 Modernatx Inc Composiciones de arn de alta pureza y métodos para su preparación.
CA3036710A1 (en) * 2016-09-16 2018-03-22 Icahn School Of Medicine At Mount Sinai Cell-specific expression of modrna
CA3054062A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Novel codon-optimized cftr mrna
MA47680A (fr) 2017-02-28 2020-01-08 Sanofi Sa Arn thérapeutique
WO2018188730A1 (en) * 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
AU2018268859A1 (en) 2017-05-16 2019-12-12 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding CFTR
WO2018232357A1 (en) 2017-06-15 2018-12-20 Modernatx, Inc. Rna formulations
US10034951B1 (en) 2017-06-21 2018-07-31 New England Biolabs, Inc. Use of thermostable RNA polymerases to produce RNAs having reduced immunogenicity
EP3668977A4 (en) 2017-08-18 2021-04-21 Modernatx, Inc. HPLC ANALYTICAL PROCESSES
WO2019036685A1 (en) * 2017-08-18 2019-02-21 Modernatx, Inc. METHODS FOR HPLC ANALYSIS
CN107563149B (zh) * 2017-08-21 2020-10-23 上海派森诺生物科技股份有限公司 全长转录本的结构注释和比对结果评估方法
EP3675817A1 (en) 2017-08-31 2020-07-08 Modernatx, Inc. Methods of making lipid nanoparticles
JP7424968B2 (ja) * 2017-09-13 2024-01-30 バイオエヌテック エスエー 細胞におけるｒｎａ発現を増強する方法
US20210010034A1 (en) * 2018-03-13 2021-01-14 The Bosrd of Trustees of the Leland Stanford Junior university Transient cellular reprogramming for reversal of cell aging
US20220403001A1 (en) 2018-06-12 2022-12-22 Obsidian Therapeutics, Inc. Pde5 derived regulatory constructs and methods of use in immunotherapy
CN108676855B (zh) * 2018-07-24 2021-09-28 内蒙古赛科星家畜种业与繁育生物技术研究院有限公司 标准曲线法荧光定量pcr鉴定牛转基因拷贝数的方法及引物
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
EP3861108A1 (en) 2018-10-04 2021-08-11 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed rna
US20210386788A1 (en) 2018-10-24 2021-12-16 Obsidian Therapeutics, Inc. Er tunable protein regulation
WO2020091137A1 (en) * 2018-10-31 2020-05-07 National Cancer Center Composition comprising material for regulating oct4 modification to repress stemness
WO2020168466A1 (en) * 2019-02-19 2020-08-27 Stemirna Therapeutics Co., Ltd. Modified nucleoside and synthetic methods thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN112390838A (zh) * 2019-08-14 2021-02-23 斯微(上海)生物科技有限公司 一种改性核苷及其合成方法
CN110894493A (zh) * 2019-10-28 2020-03-20 吉林大学 一种重编程间充质干细胞及其制备方法
US20230055044A1 (en) * 2020-01-24 2023-02-23 I Peace, Inc. Method for manufacturing induced pluripotent stem cells
US11576966B2 (en) 2020-02-04 2023-02-14 CureVac SE Coronavirus vaccine
KR20210116324A (ko) 2020-03-12 2021-09-27 기초과학연구원 게놈 서열 변이를 가지는 세포 사멸 유도용 조성물 및 상기 조성물을 이용한 세포 사멸 유도 방법
AU2021252164A1 (en) 2020-04-09 2022-12-15 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
IL302830A (en) * 2020-11-13 2023-07-01 Egenesis Inc Cells, tissues, organs and animals with one or more genes that have been modified for improved survival and tolerance of a different-sex transplant
MX2023007574A (es) 2020-12-22 2023-09-29 CureVac SE "vacuna de arn contra variantes de sars-cov-2.
US20230042860A1 (en) * 2021-07-15 2023-02-09 Turn Biotechnologies, Inc. Polycistronic expression vectors
WO2023288287A2 (en) 2021-07-15 2023-01-19 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs and methods of use for cell rejuvenation and for treatment
CN113408945B (zh) * 2021-07-15 2023-03-24 广西中烟工业有限责任公司 一种烤烟纯度的检测方法、装置、电子设备及存储介质
US20230046606A1 (en) 2021-07-15 2023-02-16 Turn Biotechnologies, Inc. Synthetic, persistent rna constructs with on/off mechanism for controlled expression and methods of use
CN114196676B (zh) * 2021-11-29 2023-07-18 中国农业科学院北京畜牧兽医研究所 Itga2基因在调控猪米色脂肪形成中的应用
CN114561381A (zh) * 2022-03-14 2022-05-31 桂林医学院 免疫mRNA及其制备方法和应用
CN114540387B (zh) * 2022-03-28 2024-04-26 仁景(苏州)生物科技有限公司 一种ires序列介导的非帽依赖型的基因表达载体及其应用
AU2023274159A1 (en) 2022-09-07 2024-03-21 Eyegene Inc. COMPOSITION FOR IN-VIVO DELIVERING mRNA CONTAINING MODIFIED NUCLEOTIDE
CN117511947B (zh) * 2024-01-08 2024-03-29 艾斯拓康医药科技(北京)有限公司 一种优化的5`utr序列及其应用

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5824497A (en) * 1995-02-10 1998-10-20 Mcmaster University High efficiency translation of mRNA molecules
JP2002507121A (ja) * 1997-06-25 2002-03-05 プロメガ コーポレイション Ｒｎａの単離方法
US20030083272A1 (en) * 1997-09-19 2003-05-01 Lahive & Cockfield, Llp Sense mrna therapy
US20030186237A1 (en) 2001-02-14 2003-10-02 Baylor College Of Medicine Methods and compositions of amplifying RNA
FR2822164B1 (fr) 2001-03-19 2004-06-18 Centre Nat Rech Scient Polypeptides derives des arn polymerases, et leurs utilisations
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US8137911B2 (en) 2001-05-22 2012-03-20 Cellscript, Inc. Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
WO2004048594A2 (en) 2002-11-21 2004-06-10 Epicentre Technologies Preparation and use of single-stranded transcription substrates for synthesis of transcription products corresponding to target sequences
US9567591B2 (en) * 2003-05-15 2017-02-14 Mello Biotechnology, Inc. Generation of human embryonic stem-like cells using intronic RNA
US20050153333A1 (en) 2003-12-02 2005-07-14 Sooknanan Roy R. Selective terminal tagging of nucleic acids
WO2006002538A1 (en) * 2004-07-02 2006-01-12 Protiva Biotherapeutics, Inc. Immunostimulatory sirna molecules and uses therefor
ES2735531T3 (es) * 2005-08-23 2019-12-19 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US20070087437A1 (en) * 2005-10-14 2007-04-19 Jifan Hu Methods for rejuvenating cells in vitro and in vivo
EP2010659B1 (en) 2006-04-14 2014-06-18 CellScript, Inc. Kits and methods for generating 5' capped RNA
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
KR101107256B1 (ko) 2007-03-27 2012-01-19 삼성전자주식회사 모션 벡터에 기반한 적응적 프레임율 변환 방법 및 장치 및적응적 프레임율 변환 기능을 가지는 디스플레이 장치
CN101802172A (zh) 2007-05-30 2010-08-11 通用医疗公司 由体细胞产生多能细胞的方法
EP2173872B1 (en) 2007-06-29 2014-04-02 CellScript, Inc. Copy dna and sense rna
EP2072618A1 (en) * 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
GB0801215D0 (en) * 2008-01-23 2008-02-27 Univ Sheffield Cell re-programming
EP2250252A2 (en) * 2008-02-11 2010-11-17 Cambridge Enterprise Limited Improved reprogramming of mammalian cells, and the cells obtained
WO2009127230A1 (en) * 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
US8669048B2 (en) * 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
GB0905507D0 (en) 2009-03-31 2009-05-13 Dow Corning Organopol Ysiloxane Compositions Containing An Active Material
US20100273220A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
WO2011012316A2 (de) * 2009-07-31 2011-02-03 Ludwig-Maximilians-Universität Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
KR102505097B1 (ko) 2009-12-07 2023-03-02 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 세포 리프로그래밍을 위한 정제된 변형 ｒｎａ를 포함하는 ｒｎａ 제제
WO2011130624A2 (en) * 2010-04-16 2011-10-20 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
WO2012019168A2 (en) * 2010-08-06 2012-02-09 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DE19177059T1 (de) 2010-10-01 2021-10-07 Modernatx, Inc. N1-methyl-pseudouracile enthältendes ribonucleinsäuren sowie ihre verwendungen
WO2012148586A1 (en) * 2011-03-15 2012-11-01 The Board Of Trustees Of The Leland Stanford Junior University Gpcr fusion protein containing an n-terminal autonomously folding stable domain, and crystals of the same
DE12722942T1 (de) * 2011-03-31 2021-09-30 Modernatx, Inc. Freisetzung und formulierung von manipulierten nukleinsäuren
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
EP4372081A2 (en) 2011-12-30 2024-05-22 Cellscript, Llc Making and using in vitro-synthesized ssrna for introducing into mammalian cells to induce a biological or biochemical effect

Also Published As

Publication number Publication date
SG181564A1 (en) 2012-07-30
IL220219A0 (en) 2012-07-31
US10006007B2 (en) 2018-06-26
LT3287525T (lt) 2020-02-10
KR20180081836A (ko) 2018-07-17
JP2022185047A (ja) 2022-12-13
BR112012013875A2 (pt) 2015-11-03
US20180265848A1 (en) 2018-09-20
US8808982B2 (en) 2014-08-19
SG10201408129XA (en) 2015-02-27
KR20230035422A (ko) 2023-03-13
US11739300B2 (en) 2023-08-29
JP7475406B2 (ja) 2024-04-26
EP3112467A1 (en) 2017-01-04
CN102947450B (zh) 2016-11-23
CN107090436B (zh) 2021-11-30
JP2021078516A (ja) 2021-05-27
US20140315988A1 (en) 2014-10-23
AU2010328310B2 (en) 2015-05-21
KR20200124331A (ko) 2020-11-02
KR20130009944A (ko) 2013-01-24
CY1120237T1 (el) 2019-07-10
EP2510099A2 (en) 2012-10-17
WO2011071931A3 (en) 2011-11-17
DK3112467T3 (en) 2018-04-23
KR102171849B1 (ko) 2020-10-30
JP2024041917A (ja) 2024-03-27
AU2010328310A1 (en) 2012-06-28
BR112012013875B1 (pt) 2020-04-14
DK3287525T3 (da) 2020-01-20
EP3287525B1 (en) 2019-10-23
WO2011071936A2 (en) 2011-06-16
US20160251629A1 (en) 2016-09-01
CA2783032C (en) 2022-08-30
CN107090436A (zh) 2017-08-25
SI3287525T1 (sl) 2020-03-31
PL3287525T3 (pl) 2020-05-18
HRP20180737T1 (hr) 2018-06-29
PT3112467T (pt) 2018-04-16
JP6842495B2 (ja) 2021-03-17
CA2783032A1 (en) 2011-06-16
PL2510099T3 (pl) 2018-01-31
SI3112467T1 (en) 2018-06-29
US20110143436A1 (en) 2011-06-16
US20160369243A1 (en) 2016-12-22
ES2769129T3 (es) 2020-06-24
EP3623474A1 (en) 2020-03-18
KR102505097B1 (ko) 2023-03-02
JP2013512690A (ja) 2013-04-18
CA3170391A1 (en) 2011-06-16
JP2016171804A (ja) 2016-09-29
US11028370B2 (en) 2021-06-08
JP7152541B2 (ja) 2022-10-12
PL3112467T3 (pl) 2018-07-31
HUE036684T2 (hu) 2018-07-30
EP3112467B1 (en) 2018-02-14
EP3287525A1 (en) 2018-02-28
ES2665681T3 (es) 2018-04-26
KR101878502B1 (ko) 2018-07-13
HRP20200246T1 (hr) 2020-05-29
HK1251259B (zh) 2020-07-03
NO2510099T3 (hu) 2018-01-06
US20210024895A1 (en) 2021-01-28
EP2510099B1 (en) 2017-08-09
WO2011071936A3 (en) 2011-10-13
WO2011071931A2 (en) 2011-06-16
US9371544B2 (en) 2016-06-21
NO3112467T3 (hu) 2018-07-14
BR112012013875B8 (pt) 2020-06-23
JP2019162125A (ja) 2019-09-26
LT3112467T (lt) 2018-06-25
EP2510099A4 (en) 2014-06-11
CN114317612A (zh) 2022-04-12
CN102947450A (zh) 2013-02-27

Similar Documents

Publication Publication Date Title
HK1251259B (zh) 用於重編程細胞的包含純化的經修飾的rna的rna製劑
IL272848B (en) Methods for reprogramming cells and their use
HRP20170138T1 (hr) Ponovno dobivanje proteina
HK1214626A1 (zh) 重編程細胞
PL2240572T3 (pl) Wykorzystanie rna dla przeprogramowania komórek somatycznych
EP3460064B8 (en) Efficient protein expression in vivo using modified rna (mod-rna)
EP2432794A4 (en) TRANSACTIVATION RNA PERMUTATIONS
GB0800524D0 (en) Cell culture system
IL223642A (en) Inorganic elements to enhance gene expression
PT2401556E (pt) Sistema de eléctrodo plano
GB0900845D0 (en) Reprogramming cells
AU2009905627A0 (en) Gene expression in plants
AU2009900788A0 (en) Method for administering cells
IL217286A0 (en) Introducing dna into plant cells
AU2009903983A0 (en) Plant gene expression
GB0922428D0 (en) Cell culture
GB0815496D0 (en) Reprogramming cells
GB0813770D0 (en) Reprogramming cells
GB0901897D0 (en) Follicle culture
SG10201500857UA (en) Modified biotin-binding protein