[go: up one dir, main page]

PT3112467T - Preparações de arn compreendendo arn modificado purificado para a reprogramação de células - Google Patents

Preparações de arn compreendendo arn modificado purificado para a reprogramação de células

Info

Publication number
PT3112467T
PT3112467T PT161814348T PT16181434T PT3112467T PT 3112467 T PT3112467 T PT 3112467T PT 161814348 T PT161814348 T PT 161814348T PT 16181434 T PT16181434 T PT 16181434T PT 3112467 T PT3112467 T PT 3112467T
Authority
PT
Portugal
Prior art keywords
rna
composing
cell reprogramming
preparations
modified purified
Prior art date
Application number
PT161814348T
Other languages
English (en)
Inventor
Jendrisak Jerome
Dahl Gary
Meis Judith
Person Anthony
Karikó Katalin
Weissman Drew
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 PT3112467T publication Critical patent/PT3112467T/pt

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
    • 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
    • C12N5/0696Artificially induced pluripotent stem cells, e.g. iPS
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/18Growth factors; Growth regulators
    • A61K38/1816Erythropoietin [EPO]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/50Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/14Drugs for dermatological disorders for baldness or alopecia
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4712Cystic fibrosis
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • C07K14/505Erythropoietin [EPO]
    • 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
    • C12N15/117Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • 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
    • 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
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004Oxidoreductases (1.)
    • C12N9/0071Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0073Oxidoreductases (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
    • C12N9/0075Nitric-oxide synthase (1.14.13.39)
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/78Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y305/00Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004Adenosine deaminase (3.5.4.4)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/17Immunomodulatory nucleic acids
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/333Modified A
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/335Modified T or U
    • 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
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/336Modified G
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/602Sox-2
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/603Oct-3/4
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/604Klf-4
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/605Nanog
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/606Transcription factors c-Myc
    • 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
    • C12N2501/00Active agents used in cell culture processes, e.g. differentation
    • C12N2501/60Transcription factors
    • C12N2501/608Lin28
    • 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
    • C12N2506/00Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/02Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from embryonic 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
    • C12N2510/00Genetically modified cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y114/00Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/13Oxidoreductases 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)
    • C12Y114/13039Nitric-oxide synthase (NADPH dependent) (1.14.13.39)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04012Sphingomyelin 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)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Transplantation (AREA)
  • Cell Biology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Pulmonology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
PT161814348T 2009-12-07 2010-12-07 Preparações de arn compreendendo arn modificado purificado para a reprogramação de células PT3112467T (pt)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
PT3112467T true PT3112467T (pt) 2018-04-16

Family

ID=44143383

Family Applications (1)

Application Number Title Priority Date Filing Date
PT161814348T PT3112467T (pt) 2009-12-07 2010-12-07 Preparações de arn compreendendo arn modificado purificado para a reprogramação de células

Country Status (22)

Country Link
US (6) US8808982B2 (pt)
EP (4) EP3623474A1 (pt)
JP (6) JP2013512690A (pt)
KR (5) KR101878502B1 (pt)
CN (3) CN102947450B (pt)
AU (1) AU2010328310B2 (pt)
BR (1) BR112012013875B8 (pt)
CA (2) CA2783032C (pt)
CY (1) CY1120237T1 (pt)
DK (2) DK3287525T3 (pt)
ES (2) ES2769129T3 (pt)
HK (1) HK1251259B (pt)
HR (2) HRP20180737T1 (pt)
HU (2) HUE047165T2 (pt)
IL (1) IL220219A0 (pt)
LT (2) LT3287525T (pt)
NO (2) NO3112467T3 (pt)
PL (3) PL2510099T3 (pt)
PT (1) PT3112467T (pt)
SG (2) SG181564A1 (pt)
SI (2) SI3287525T1 (pt)
WO (2) WO2011071931A2 (pt)

Families Citing this family (151)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
ES2937245T3 (es) 2005-08-23 2023-03-27 Univ Pennsylvania ARN que contiene nucleósidos modificados y métodos de uso del mismo
US9249423B2 (en) * 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
US10155038B2 (en) 2007-02-02 2018-12-18 Yale University Cells prepared by transient transfection and methods of use thereof
US8859229B2 (en) * 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
RS58405B1 (sr) 2009-12-01 2019-04-30 Translate Bio Inc Stereoidni derivati za isporuku irnk u humanim genetskim oboljenjima
KR101878502B1 (ko) 2009-12-07 2018-07-13 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 세포 리프로그래밍을 위한 정제된 변형 rna를 포함하는 rna 제제
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
ES2586580T3 (es) 2010-07-06 2016-10-17 Glaxosmithkline Biologicals Sa Inmunización de mamíferos grandes con dosis bajas de ARN
HUE026646T2 (en) 2010-07-06 2016-07-28 Glaxosmithkline Biologicals Sa Preferred liposomes containing lipids of PKA value for delivery of RNA
ES2646669T3 (es) 2010-07-06 2017-12-14 Glaxosmithkline Biologicals Sa Procedimientos de aumento de una respuesta inmunitaria mediante el suministro de ARN
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
DK4226941T3 (da) 2010-08-31 2024-12-02 Glaxosmithkline Biologicals Sa Pegylerede liposomer til levering af immunogen-kodende rna
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR102266691B1 (ko) 2010-10-11 2021-06-23 노파르티스 아게 항원 전달 플랫폼
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
JP6184945B2 (ja) 2011-06-08 2017-08-23 シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド mRNA送達のための脂質ナノ粒子組成物および方法
US11896636B2 (en) 2011-07-06 2024-02-13 Glaxosmithkline Biologicals Sa Immunogenic combination compositions and uses thereof
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
HRP20220250T1 (hr) * 2011-10-03 2022-04-29 Modernatx, Inc. Modificirani nukleozidi, nukleotidi i nukleinske kiseline, te njihove uporabe
MX354995B (es) 2011-12-05 2018-03-27 Factor Bioscience Inc Metodos y productos para transfeccion de celulas.
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 ACUTE TREATMENT USES THEREOF
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
RU2014129863A (ru) * 2011-12-21 2016-02-10 Модерна Терапьютикс, Инк. Способы повышения жизнеспособности или увеличения продолжительности жизни органа или экспланта органа
AU2012362113B2 (en) * 2011-12-30 2017-08-03 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
HK1206612A1 (en) * 2012-04-02 2016-01-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of secreted proteins
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
US10501512B2 (en) 2012-04-02 2019-12-10 Modernatx, Inc. Modified polynucleotides
AU2013243952A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CN104520424B (zh) * 2012-05-13 2019-06-28 美国绿阳生物技术及医药公司 使用合成的信使rna无饲养细胞地衍生人类诱导性多能干细胞
BR112014030677A2 (pt) 2012-06-08 2022-07-19 Shire Human Genetic Therapies distribuição pulmonar de mrna para células-alvo não-pulmonares
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
US9512456B2 (en) 2012-08-14 2016-12-06 Modernatx, Inc. Enzymes and polymerases for the synthesis of RNA
MX2015002917A (es) 2012-09-07 2015-06-03 Genentech Inc Metodos y composiciones para producir hepatocitos inducidos.
MX2015004853A (es) 2012-10-16 2015-12-09 Massachusetts Inst Technology Produccion de acidos ribonucleicos no poliadenilados estables.
KR102315098B1 (ko) 2012-11-01 2021-10-21 팩터 바이오사이언스 인크. 세포에서 단백질을 발현시키는 방법들과 생성물들
SMT202200337T1 (it) 2012-11-26 2022-09-14 Modernatx Inc Rna modificato al livello del terminale
EP3628335B1 (en) * 2012-12-07 2023-11-08 Translate Bio, Inc. Lipidic nanoparticles for mrna delivery in the lungs
DK2959005T3 (da) 2013-02-22 2022-01-03 Univ Leland Stanford Junior Medicinsk anvendelse relateret til telomer forlængelse
EP2968391A1 (en) 2013-03-13 2016-01-20 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
WO2014160243A1 (en) 2013-03-14 2014-10-02 The Trustees Of The University Of Pennsylvania Purification and purity assessment of rna molecules synthesized with modified nucleosides
EP2970955B1 (en) 2013-03-14 2018-11-14 Translate Bio, Inc. Methods for purification of messenger rna
WO2014153052A2 (en) 2013-03-14 2014-09-25 Shire Human Genetic Therapies, Inc. Cftr mrna compositions and related methods and uses
US10077439B2 (en) 2013-03-15 2018-09-18 Modernatx, Inc. Removal of DNA fragments in mRNA production process
WO2014144767A1 (en) 2013-03-15 2014-09-18 Moderna Therapeutics, Inc. Ion exchange purification of mrna
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
WO2014152031A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Ribonucleic acid purification
WO2014152027A1 (en) 2013-03-15 2014-09-25 Moderna Therapeutics, Inc. Manufacturing methods for production of rna transcripts
JP6625521B2 (ja) 2013-05-15 2020-01-08 リボカイン,エルエルシー 環状rnaの細胞内翻訳
HUE056760T2 (hu) 2013-07-11 2022-03-28 Modernatx Inc A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik
US10557120B2 (en) 2013-07-26 2020-02-11 Kyoto Prefectural Public University Corporation Osteoblast and method for preparing same
GB2546833B (en) * 2013-08-28 2018-04-18 Cellular Res Inc Microwell for single cell analysis comprising single cell and single bead oligonucleotide capture labels
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
EP3052511A4 (en) 2013-10-02 2017-05-31 Moderna Therapeutics, Inc. Polynucleotide molecules and uses thereof
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
MX2016005239A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Tratamiento con acido ribonucleico mensajero para la fenilcetonuria.
CA2925021C (en) 2013-11-01 2025-05-06 Curevac Ag MODIFIED MESSENGER RIBONUCLE ACID (MRNA) WITH REDUCED IMMUNOSTIMULATING PROPERTIES
EP3062798B1 (en) 2013-11-01 2020-05-06 CureVac AG Modified rna with decreased immunostimulatory properties
KR101665165B1 (ko) * 2013-12-04 2016-10-12 단국대학교 산학협력단 신규한 rna 앱타머 및 그의 용도
RU2714404C2 (ru) 2014-01-31 2020-02-14 Фэктор Байосайенс Инк. Способы и продукты для получения и доставки нуклеиновых кислот
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
SG10201912038TA (en) 2014-04-23 2020-02-27 Modernatx Inc Nucleic acid vaccines
SG11201608725YA (en) 2014-04-25 2016-11-29 Shire Human Genetic Therapies Methods for purification of messenger rna
WO2015196128A2 (en) 2014-06-19 2015-12-23 Moderna Therapeutics, Inc. Alternative nucleic acid molecules and uses thereof
WO2016011222A2 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Circular polynucleotides
WO2016011226A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3461904A1 (en) 2014-11-10 2019-04-03 ModernaTX, Inc. Alternative nucleic acid molecules containing reduced uracil content and uses thereof
CA2976376A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CA2990883A1 (en) 2015-06-30 2017-01-05 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
WO2017049286A1 (en) 2015-09-17 2017-03-23 Moderna Therapeutics, Inc. Polynucleotides containing a morpholino linker
PL3350333T3 (pl) 2015-09-17 2022-03-07 Modernatx, Inc. Polinukleotydy zawierające region stabilizujący ogon
EP3353313B1 (en) * 2015-09-23 2024-08-07 Pfizer Inc. Cells and method of cell culture
AU2016336344A1 (en) 2015-10-05 2018-04-19 Modernatx, Inc. Methods for therapeutic administration of messenger ribonucleic acid drugs
WO2017180587A2 (en) 2016-04-11 2017-10-19 Obsidian Therapeutics, Inc. Regulated biocircuit systems
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SMT202100681T1 (it) 2016-04-22 2022-01-10 BioNTech SE Procedimenti per fornire rna a singolo filamento
AU2017268394A1 (en) 2016-05-18 2019-01-03 Modernatx, Inc. Polynucleotides encoding relaxin
KR20190028665A (ko) * 2016-06-03 2019-03-19 스템제닉스 인코포레이티드 기능화된 나노입자(functionalized nanoparticles)를 사용하여 선택된(예정된) 분화된 세포에 대한 사람 체세포의 직접적인 재프로그래밍
US12385034B2 (en) 2016-06-24 2025-08-12 Modernatx, Inc. Methods and apparatus for filtration
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
US10576167B2 (en) 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN109937253B (zh) 2016-09-14 2023-06-30 摩登纳特斯有限公司 高纯度rna组合物及其制备方法
ES2967788T3 (es) * 2016-09-16 2024-05-03 Icahn School Med Mount Sinai Expresión especifica de célula de Arnmond
EP3538146A4 (en) 2016-11-11 2020-07-15 ModernaTX, Inc. INFLUENZA VACCINE
AU2018224326B2 (en) 2017-02-27 2024-01-04 Translate Bio, Inc. Novel codon-optimized CFTR mRNA
CN110337305A (zh) 2017-02-28 2019-10-15 赛诺菲 治疗性rna
WO2018188730A1 (en) 2017-04-11 2018-10-18 Biontech Rna Pharmaceuticals Gmbh Rna for treatment of autoimmune diseases
CN111031790A (zh) 2017-04-20 2020-04-17 E开创生物技术股份有限公司 产生基因修改的动物的方法
IL270631B2 (en) 2017-05-16 2024-03-01 Translate Bio Inc Treatment of cystic fibrosis through the administration of mRNA with an optimal codon encoding ctfr
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
EP3668979A4 (en) * 2017-08-18 2021-06-02 Modernatx, Inc. METHOD OF 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
US11744801B2 (en) 2017-08-31 2023-09-05 Modernatx, Inc. Methods of making lipid nanoparticles
WO2019053003A1 (en) * 2017-09-13 2019-03-21 Biontech Rna Pharmaceuticals Gmbh METHODS FOR ENHANCING RNA EXPRESSION IN CELL
MX2020009491A (es) * 2018-03-13 2020-10-28 Univ Leland Stanford Junior Reprogramacion celular transitoria para revertir el envejecimiento celular.
EP3806888B1 (en) 2018-06-12 2024-01-31 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
CN112930396B (zh) 2018-08-24 2024-05-24 川斯勒佰尔公司 用于纯化信使rna的方法
WO2020061457A1 (en) 2018-09-20 2020-03-26 Modernatx, Inc. Preparation of lipid nanoparticles and methods of administration thereof
US11072808B2 (en) 2018-10-04 2021-07-27 New England Biolabs, Inc. Methods and compositions for increasing capping efficiency of transcribed RNA
AU2019355177A1 (en) 2018-10-04 2021-05-06 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
US20200157157A1 (en) 2018-11-21 2020-05-21 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
WO2020168466A1 (en) * 2019-02-19 2020-08-27 Stemirna Therapeutics Co., Ltd. Modified nucleoside and synthetic methods thereof
MX2021011031A (es) 2019-03-11 2021-12-10 Modernatx Inc Proceso de transcripción in vitro en lotes alimentados.
CN114450265B (zh) 2019-07-03 2024-12-24 菲克特生物科学股份有限公司 阳离子脂质及其用途
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
AU2020329226A1 (en) * 2019-08-14 2022-02-24 Modernatx, Inc. Processes for purifying downstream products of in vitro transcription
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
JP2023505387A (ja) * 2019-11-11 2023-02-08 厚雄 越智 体細胞をリプログラムするための方法およびキット
WO2021149824A1 (ja) * 2020-01-24 2021-07-29 アイ ピース, インコーポレイテッド 人工多能性幹細胞の作製方法
US12194089B2 (en) 2020-02-04 2025-01-14 CureVac SE Coronavirus vaccine
EP4119166A4 (en) 2020-03-12 2024-06-05 Institute for Basic Science COMPOSITION FOR INDUCING APOPTOSIS OF CELLS HAVING GENOMIC SEQUENCE VARIATION AND METHOD FOR INDUCING APOPTOSIS OF CELLS USING THE COMPOSITION
WO2021205077A1 (en) 2020-04-09 2021-10-14 Finncure Oy Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses
US12194157B2 (en) 2020-04-09 2025-01-14 Finncure Oy Carrier for targeted delivery to a host
GB2635873B (en) * 2020-11-13 2025-11-26 Egenesis Inc Cells, tissues, organs, and animals having one or more modified genes for enhanced xenograft survival and tolerance
US11918643B2 (en) 2020-12-22 2024-03-05 CureVac SE RNA vaccine against SARS-CoV-2 variants
JP2022104813A (ja) 2020-12-29 2022-07-11 アイ ピース,インコーポレイテッド リプログラミング因子を導入された細胞の培養方法
US12329811B2 (en) 2021-01-11 2025-06-17 Modernatx, Inc. Seasonal RNA influenza virus vaccines
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
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
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
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
KR102785258B1 (ko) * 2022-09-07 2025-03-26 아이진 주식회사 변형핵산 함유 mRNA의 체내 전달용 조성물
DE202023106198U1 (de) 2022-10-28 2024-03-21 CureVac SE Impfstoff auf Nukleinsäurebasis
CN115992146B (zh) * 2022-11-11 2025-04-18 上海交通大学医学院附属第九人民医院 经修饰的mRNA分子及相关产品和用途
WO2024129826A2 (en) * 2022-12-14 2024-06-20 Grann Pharmaceuticals Inc. Compositions and methods for modulating hsp70 activity
WO2024249954A1 (en) 2023-05-31 2024-12-05 Capstan Therapeutics, Inc. Lipid nanoparticle formulations and compositions
WO2025024442A1 (en) * 2023-07-26 2025-01-30 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating conditions associated with bsep deficiency
CN116898988A (zh) * 2023-08-15 2023-10-20 臻赫医药(杭州)有限公司 皮肤重编程因子制剂、生物材料及用途
WO2025038982A2 (en) * 2023-08-17 2025-02-20 Duke University Regulators of t cell exhaustion
WO2025144938A1 (en) 2023-12-26 2025-07-03 Emmune, Inc. Systems for nucleic acid transfer
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
KR20250110708A (ko) 2024-01-11 2025-07-21 서울대학교산학협력단 역분화 유전자의 간 특이적 전달을 통한 간 재생 방법
CN118516381B (zh) * 2024-07-22 2024-11-01 北京剂泰医药科技有限公司 编码荧光素酶的mRNA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
US6218531B1 (en) * 1997-06-25 2001-04-17 Promega Corporation Method of isolating RNA
JP2002508299A (ja) * 1997-09-19 2002-03-19 セクイター, インク. センスmRNA治療
JP2004527236A (ja) 2001-02-14 2004-09-09 ベイラー カレッジ オブ メディスン Rna増幅の方法及び組成物
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
AU2003297557B2 (en) 2002-11-21 2009-02-26 Cellscript, Inc. Methods for using primers that encode one strand of a double-stranded promoter
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
JP2008504827A (ja) * 2004-07-02 2008-02-21 プロチバ バイオセラピューティクス インコーポレイティッド 免疫賦活性siRNA分子およびその使用方法
ES2937245T3 (es) 2005-08-23 2023-03-27 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
WO2007120863A2 (en) 2006-04-14 2007-10-25 Epicentre Technologies 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
JP2010528622A (ja) 2007-05-30 2010-08-26 ザ ジェネラル ホスピタル コーポレイション 体細胞から多能性細胞を生成する方法
US8039214B2 (en) 2007-06-29 2011-10-18 Cellscript, Inc. Synthesis of tagged nucleic acids
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
US20100330677A1 (en) * 2008-02-11 2010-12-30 Cambridge Enterprise Limited Improved Reprogramming of Mammalian Cells, and 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
WO2010008486A2 (en) * 2008-06-24 2010-01-21 Parkinsons 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
EP2421563B1 (en) 2009-04-22 2017-04-12 Massachusetts Institute of Technology Innate immune suppression enables repeated delivery of long rna molecules
EP3165234B1 (de) * 2009-07-31 2019-04-03 ethris GmbH Rna mit einer kombination aus unmodifizierten und modifizierten nucleotiden zur proteinexpression
KR101878502B1 (ko) 2009-12-07 2018-07-13 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 세포 리프로그래밍을 위한 정제된 변형 rna를 포함하는 rna 제제
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
US8822663B2 (en) 2010-08-06 2014-09-02 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
CA2821992A1 (en) 2010-10-01 2012-04-05 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
US8765414B2 (en) 2011-03-15 2014-07-01 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
AU2012362113B2 (en) 2011-12-30 2017-08-03 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
HUE047165T2 (hu) 2020-04-28
CN102947450A (zh) 2013-02-27
JP2024041917A (ja) 2024-03-27
US20180265848A1 (en) 2018-09-20
CN114317612B (zh) 2024-10-29
HRP20180737T1 (hr) 2018-06-29
KR102505097B1 (ko) 2023-03-02
JP2019162125A (ja) 2019-09-26
CA2783032A1 (en) 2011-06-16
BR112012013875B1 (pt) 2020-04-14
SG181564A1 (en) 2012-07-30
EP3112467A1 (en) 2017-01-04
US9371544B2 (en) 2016-06-21
JP7475406B2 (ja) 2024-04-26
SI3287525T1 (sl) 2020-03-31
JP6842495B2 (ja) 2021-03-17
KR20130009944A (ko) 2013-01-24
SG10201408129XA (en) 2015-02-27
US11739300B2 (en) 2023-08-29
CA3170391A1 (en) 2011-06-16
LT3287525T (lt) 2020-02-10
PL3287525T3 (pl) 2020-05-18
EP3623474A1 (en) 2020-03-18
WO2011071936A3 (en) 2011-10-13
CN107090436B (zh) 2021-11-30
DK3287525T3 (da) 2020-01-20
EP3287525B1 (en) 2019-10-23
NO3112467T3 (pt) 2018-07-14
KR20230035422A (ko) 2023-03-13
AU2010328310B2 (en) 2015-05-21
US20160369243A1 (en) 2016-12-22
NO2510099T3 (pt) 2018-01-06
JP2022185047A (ja) 2022-12-13
CN114317612A (zh) 2022-04-12
AU2010328310A1 (en) 2012-06-28
SI3112467T1 (en) 2018-06-29
ES2665681T3 (es) 2018-04-26
JP7763277B2 (ja) 2025-10-31
CY1120237T1 (el) 2019-07-10
US8808982B2 (en) 2014-08-19
KR102703156B1 (ko) 2024-09-06
KR20180081836A (ko) 2018-07-17
DK3112467T3 (en) 2018-04-23
ES2769129T3 (es) 2020-06-24
JP2013512690A (ja) 2013-04-18
EP2510099A2 (en) 2012-10-17
JP2016171804A (ja) 2016-09-29
KR102171849B1 (ko) 2020-10-30
KR101878502B1 (ko) 2018-07-13
PL3112467T3 (pl) 2018-07-31
JP2021078516A (ja) 2021-05-27
BR112012013875A2 (pt) 2015-11-03
US20210024895A1 (en) 2021-01-28
CA2783032C (en) 2022-08-30
KR20200124331A (ko) 2020-11-02
HUE036684T2 (hu) 2018-07-30
WO2011071931A2 (en) 2011-06-16
US20140315988A1 (en) 2014-10-23
WO2011071931A3 (en) 2011-11-17
EP3287525A1 (en) 2018-02-28
US10006007B2 (en) 2018-06-26
US20110143436A1 (en) 2011-06-16
EP3112467B1 (en) 2018-02-14
HRP20200246T1 (hr) 2020-05-29
EP2510099A4 (en) 2014-06-11
WO2011071936A2 (en) 2011-06-16
PL2510099T3 (pl) 2018-01-31
US11028370B2 (en) 2021-06-08
KR20240136456A (ko) 2024-09-13
US20160251629A1 (en) 2016-09-01
IL220219A0 (en) 2012-07-31
EP2510099B1 (en) 2017-08-09
HK1251259B (en) 2020-07-03
JP7152541B2 (ja) 2022-10-12
CN102947450B (zh) 2016-11-23
CN107090436A (zh) 2017-08-25
LT3112467T (lt) 2018-06-25
BR112012013875B8 (pt) 2020-06-23

Similar Documents

Publication Publication Date Title
PT3112467T (pt) Preparações de arn compreendendo arn modificado purificado para a reprogramação de células
PT2240572E (pt) Utilização de arn para a reprogramação de células somáticas
EP2647644A4 (en) 2'-O-MODIFIED RNA
EP2638163A4 (en) POLYCOMB-ASSOCIATED NON-CODING RNAS
EP2379922A4 (en) Valve system
PT2406399T (pt) Proteínas mirac
EP2509515A4 (en) WITHERS
BRPI1010035A2 (pt) Células deficientes em fucosilação
BRPI0922912A2 (pt) peptídeos de 4-amino-4-oxobutanoil como inibidores de replicação viral
BRPI1011048A2 (pt) plasmídeo independente de antibiótico
IT1397083B1 (it) Marcatore biochimico serico
BR112012001462A2 (pt) processo para preparar metil mercaptano.
EP2454074A4 (en) MODULAR COLLECTOR SYSTEM
IT1402673B1 (it) Macchina combinata per la realizzazione di libri
BR112013011716A2 (pt) distribuidor reforçado para lança de pós-combustão
BRPI0810088A2 (pt) Regime de vacinação para vacinas de células b
HK40073099B (zh) 用於重编程细胞的包含纯化的经修饰的rna的rna制剂
AU2024266827A1 (en) RNA preparations comprising purified modified RNA for reprogramming cells
IT1393632B1 (it) Gruppo di taglio trasversale
DK2401556T3 (da) Planart elektrodesystem
ES1069648Y (es) Expositor perfeccionado
IT1392579B1 (it) Macchina smistatrice per flat
DE112010003601A5 (de) Stapelsystem
UA18655S (uk) Столик-підставка для газет
UA18175S (uk) Розподільча проставка для виготовлення шпал