AU2013337651B2 - Methods and products for expressing proteins in cells - Google Patents

Methods and products for expressing proteins in cells Download PDF

Info

Publication number
AU2013337651B2
AU2013337651B2 AU2013337651A AU2013337651A AU2013337651B2 AU 2013337651 B2 AU2013337651 B2 AU 2013337651B2 AU 2013337651 A AU2013337651 A AU 2013337651A AU 2013337651 A AU2013337651 A AU 2013337651A AU 2013337651 B2 AU2013337651 B2 AU 2013337651B2
Authority
AU
Australia
Prior art keywords
gene
composition
protein
cell
seq
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
AU2013337651A
Other languages
English (en)
Other versions
AU2013337651A1 (en
Inventor
Matthew Angel
Christopher Rohde
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Factor Bioscience Inc
Original Assignee
Factor Bioscience Inc
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 Factor Bioscience Inc filed Critical Factor Bioscience Inc
Publication of AU2013337651A1 publication Critical patent/AU2013337651A1/en
Priority to AU2018264115A priority Critical patent/AU2018264115B2/en
Application granted granted Critical
Publication of AU2013337651B2 publication Critical patent/AU2013337651B2/en
Priority to AU2021250972A priority patent/AU2021250972A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02—Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/04—Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • 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
    • A61P11/00—Drugs for disorders of the respiratory system
    • A61P11/02—Nasal agents, e.g. decongestants
    • 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
    • A61P11/04—Drugs for disorders of the respiratory system for throat disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/08—Drugs for disorders of the urinary system of the prostate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/10—Drugs for disorders of the urinary system of the bladder
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P15/00—Drugs for genital or sexual disorders; Contraceptives
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • 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
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • 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
    • C07K14/195—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from bacteria
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C12N15/1024—In vivo mutagenesis using high mutation rate "mutator" host strains by inserting genetic material, e.g. encoding an error prone polymerase, disrupting a gene for mismatch repair
    • 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
    • C12N15/63—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
    • C12N15/86—Viral vectors
    • 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
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • 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)
    • 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)
    • C12Y301/21—Endodeoxyribonucleases producing 5'-phosphomonoesters (3.1.21)
    • C12Y301/21004—Type II site-specific deoxyribonuclease (3.1.21.4)
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • G01N33/5014—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics for testing toxicity
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Urology & Nephrology (AREA)
  • Neurosurgery (AREA)
  • Hematology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Pulmonology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Endocrinology (AREA)
AU2013337651A 2012-11-01 2013-11-01 Methods and products for expressing proteins in cells Active AU2013337651B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2018264115A AU2018264115B2 (en) 2012-11-01 2018-11-16 Methods and products for expressing proteins in cells
AU2021250972A AU2021250972A1 (en) 2012-11-01 2021-10-15 Methods and products for expressing proteins in cells

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201261721302P 2012-11-01 2012-11-01
US61/721,302 2012-11-01
US201361785404P 2013-03-14 2013-03-14
US61/785,404 2013-03-14
US201361842874P 2013-07-03 2013-07-03
US61/842,874 2013-07-03
PCT/US2013/068118 WO2014071219A1 (en) 2012-11-01 2013-11-01 Methods and products for expressing proteins in cells

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2018264115A Division AU2018264115B2 (en) 2012-11-01 2018-11-16 Methods and products for expressing proteins in cells

Publications (2)

Publication Number Publication Date
AU2013337651A1 AU2013337651A1 (en) 2015-06-11
AU2013337651B2 true AU2013337651B2 (en) 2018-12-13

Family

ID=50628111

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2013337651A Active AU2013337651B2 (en) 2012-11-01 2013-11-01 Methods and products for expressing proteins in cells
AU2018264115A Active AU2018264115B2 (en) 2012-11-01 2018-11-16 Methods and products for expressing proteins in cells
AU2021250972A Abandoned AU2021250972A1 (en) 2012-11-01 2021-10-15 Methods and products for expressing proteins in cells

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2018264115A Active AU2018264115B2 (en) 2012-11-01 2018-11-16 Methods and products for expressing proteins in cells
AU2021250972A Abandoned AU2021250972A1 (en) 2012-11-01 2021-10-15 Methods and products for expressing proteins in cells

Country Status (11)

Country Link
US (19) US9447395B2 (OSRAM)
EP (2) EP3786298A1 (OSRAM)
JP (5) JP6510416B2 (OSRAM)
KR (4) KR20230154283A (OSRAM)
CN (1) CN104769112A (OSRAM)
AU (3) AU2013337651B2 (OSRAM)
BR (2) BR122019025681B1 (OSRAM)
CA (2) CA2890110C (OSRAM)
MX (2) MX363017B (OSRAM)
RU (2) RU2711249C2 (OSRAM)
WO (1) WO2014071219A1 (OSRAM)

Families Citing this family (126)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10347710B4 (de) 2003-10-14 2006-03-30 Johannes-Gutenberg-Universität Mainz Rekombinante Impfstoffe und deren Verwendung
DE102005046490A1 (de) 2005-09-28 2007-03-29 Johannes-Gutenberg-Universität Mainz Modifikationen von RNA, die zu einer erhöhten Transkriptstabilität und Translationseffizienz führen
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
CA2831613A1 (en) 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
LT3892295T (lt) 2011-05-24 2023-07-10 BioNTech SE Individualizuotos vakcinos nuo vėžio
JP6261500B2 (ja) 2011-07-22 2018-01-17 プレジデント アンド フェローズ オブ ハーバード カレッジ ヌクレアーゼ切断特異性の評価および改善
US9464124B2 (en) 2011-09-12 2016-10-11 Moderna Therapeutics, Inc. Engineered nucleic acids and methods of use thereof
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
WO2013143555A1 (en) 2012-03-26 2013-10-03 Biontech Ag Rna formulation for immunotherapy
US9283287B2 (en) 2012-04-02 2016-03-15 Moderna Therapeutics, Inc. Modified polynucleotides for the production of nuclear proteins
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9572897B2 (en) 2012-04-02 2017-02-21 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9254311B2 (en) 2012-04-02 2016-02-09 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
AU2013315007A1 (en) 2012-09-17 2015-04-09 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
BR122019025681B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
DK2922554T3 (en) 2012-11-26 2022-05-23 Modernatx Inc Terminalt modificeret rna
JP6484558B2 (ja) 2012-11-28 2019-03-13 バイオエヌテック エールエヌアー ファーマシューティカルズ ゲーエムベーハーＢｉｏｎｔｅｃｈ Ｒｎａ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｇｍｂｈ 癌ワクチンの組み合せ物
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014180490A1 (en) 2013-05-10 2014-11-13 Biontech Ag Predicting immunogenicity of t cell epitopes
KR102733409B1 (ko) 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
FI3702466T3 (fi) * 2013-08-27 2023-03-23 Res Inst Nationwide Childrens Hospital Valmisteita ja menetelmiä amyotrofisen lateraaliskleroosin hoitoon
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US10023626B2 (en) 2013-09-30 2018-07-17 Modernatx, Inc. Polynucleotides encoding immune modulating polypeptides
BR112016007255A2 (pt) 2013-10-03 2017-09-12 Moderna Therapeutics Inc polinucleotídeos que codificam receptor de lipoproteína de baixa densidade
JP2016538276A (ja) 2013-11-05 2016-12-08 ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルＴｈｅ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ Ａｔ Ｎａｔｉｏｎｗｉｄｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ 筋萎縮性側索硬化症の処置のためのＮＦ−κＢおよびＳＯＤ−１を阻害する組成物および方法
PT3066201T (pt) 2013-11-07 2018-06-04 Massachusetts Inst Technology Métodos e composições relacionadas com crispr com arng reguladores
US9068179B1 (en) 2013-12-12 2015-06-30 President And Fellows Of Harvard College Methods for correcting presenilin point mutations
WO2015138739A2 (en) * 2014-03-12 2015-09-17 Precision Biosciences, Inc. Dystrophin gene oxon deletion using engineered nucleases
PT3981437T (pt) 2014-04-23 2025-01-15 Modernatx Inc Vacinas de ácidos nucleicos
US10280419B2 (en) 2014-05-09 2019-05-07 UNIVERSITé LAVAL Reduction of amyloid beta peptide production via modification of the APP gene using the CRISPR/Cas system
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
WO2016045732A1 (en) 2014-09-25 2016-03-31 Biontech Rna Pharmaceuticals Gmbh Stable formulations of lipids and liposomes
WO2016049531A1 (en) 2014-09-26 2016-03-31 Purecircle Usa Inc. Single nucleotide polymorphism (snp) markers for stevia
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
MX388527B (es) * 2014-11-26 2025-03-20 Huvepharma Inc Composiciones adyuvantes y metodos relacionados.
US10195261B2 (en) 2014-11-26 2019-02-05 VaxLiant, LLC Adjuvant compositions and related methods
EP4400584A3 (en) 2014-12-03 2024-10-16 Agilent Technologies, Inc. Guide rna with chemical modifications
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
WO2016128060A1 (en) 2015-02-12 2016-08-18 Biontech Ag Predicting t cell epitopes useful for vaccination
US11241505B2 (en) 2015-02-13 2022-02-08 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CA2979556C (en) 2015-03-24 2023-07-18 VaxLiant, LLC Adjuvant compositions and related methods
EP3280803B1 (en) 2015-04-06 2021-05-26 The Board of Trustees of the Leland Stanford Junior University Chemically modified guide rnas for crispr/cas-mediated gene regulation
WO2017015637A1 (en) 2015-07-22 2017-01-26 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
CN108025074A (zh) * 2015-07-25 2018-05-11 哈比卜·弗罗斯特 用于为癌症和其他病理状态提供治疗或治愈的系统、装置和方法
EA201890565A1 (ru) 2015-08-25 2019-04-30 Дьюк Юниверсити Композиции и способы улучшения специфичности в геномной инженерии с применением рнк-направляемых эндонуклеаз
WO2017059902A1 (en) 2015-10-07 2017-04-13 Biontech Rna Pharmaceuticals Gmbh 3' utr sequences for stabilization of rna
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
US11866754B2 (en) 2015-10-16 2024-01-09 Modernatx, Inc. Trinucleotide mRNA cap analogs
EP3362461B1 (en) 2015-10-16 2022-03-16 Modernatx, Inc. Mrna cap analogs with modified phosphate linkage
EP3365356B1 (en) 2015-10-23 2023-06-28 President and Fellows of Harvard College Nucleobase editors and uses thereof
US11459560B2 (en) * 2015-11-23 2022-10-04 The Regents Of The University Of Colorado Methods and compositions for reprogramming cells
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
CN105494263B (zh) * 2015-12-25 2018-11-30 哈尔滨医科大学 一种产生ho-1/app/psen1三转基因阿尔茨海默病小鼠模型的方法
EP3411078A1 (en) * 2016-02-02 2018-12-12 Crispr Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017141109A1 (en) * 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017147278A1 (en) 2016-02-25 2017-08-31 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by crispr/cas9 technology
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
AU2017242794B2 (en) * 2016-03-31 2022-12-15 Ethris Gmbh Novel minimal UTR sequences
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
US12065667B2 (en) 2016-04-16 2024-08-20 Ohio State Innovation Foundation Modified Cpf1 MRNA, modified guide RNA, and uses thereof
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
MA45670A (fr) 2016-07-13 2019-05-22 Vertex Pharma Procédés, compositions et kits pour augmenter l'efficacité d'édition du génome
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JP7231935B2 (ja) 2016-08-03 2023-03-08 プレジデント アンド フェローズ オブ ハーバード カレッジ アデノシン核酸塩基編集因子およびそれらの使用
EP3497214B1 (en) 2016-08-09 2023-06-28 President and Fellows of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
US10576167B2 (en) * 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
CN106370765A (zh) * 2016-08-23 2017-02-01 国家烟草质量监督检验中心 一种基于反相色谱飞行时间质谱的喉癌尿液差异代谢物的测定筛选方法
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
CN106381310B (zh) * 2016-08-31 2020-02-04 武汉华美生物工程有限公司 一种用t7噬菌体rna聚合酶和t7启动子系统在哺乳动物细胞表达蛋白的方法
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
CN110214005A (zh) * 2016-11-11 2019-09-06 DNALite治疗学公司 用于基因治疗的结构和方法
KR101908593B1 (ko) * 2016-12-07 2018-10-16 연세대학교 산학협력단 Cftr 유전자 소실 유도 가이드 rna, cftr 변이를 갖는 t84 세포 및 이의 용도
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
EP3585807A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
US11168319B1 (en) * 2017-02-28 2021-11-09 Inari Agriculture Technology, Inc. Plant cell culture
WO2018160592A1 (en) 2017-02-28 2018-09-07 Arcturus Therapeutics, Inc. Translatable molecules and synthesis thereof
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190127797A (ko) 2017-03-10 2019-11-13 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 시토신에서 구아닌으로의 염기 편집제
CN107475255B (zh) * 2017-03-21 2021-03-26 河北医科大学第二医院 一种基因载体介导的基于CRISPR/Cas9基因编辑系统的sgRNA及其用途
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
AU2018261790B2 (en) 2017-05-05 2024-10-03 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2018224166A1 (en) 2017-06-09 2018-12-13 Biontech Rna Pharmaceuticals Gmbh Methods for predicting the usefulness of disease specific amino acid modifications for immunotherapy
US11002729B2 (en) * 2017-06-14 2021-05-11 Wuhan Institute Of Virology, Chinese Academy Of Sciences CD2-associated protein (CD2AP) and its interactive proteins
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019079240A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
US11634722B2 (en) 2018-01-22 2023-04-25 Inari Agriculture Technology, Inc. Plant gene editing systems, methods, and compositions
EP3775202A4 (en) * 2018-03-27 2022-04-06 Factor Bioscience Inc. NUCLEIC ACID BASED THERAPEUTICS
EP3781193A4 (en) 2018-04-18 2022-01-26 Altius Institute For Biomedical Sciences Animal pathogen-derived polypeptides and uses thereof for genetic engineering
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
CN108624592B (zh) * 2018-05-24 2021-08-17 苏州大学张家港工业技术研究院 针对DJ-1基因编辑的sgRNA筛选及其载体与应用
US12264181B2 (en) 2018-06-27 2025-04-01 Altius Institute For Biomedical Sciences Nucleic acid binding domains and methods of use thereof
JP2021529750A (ja) * 2018-06-27 2021-11-04 モデルナティーエックス， インコーポレイテッド 個別化がんワクチンエピトープの選択
CN108949830B (zh) * 2018-08-03 2021-11-26 福州大学 一种在鱼类中实现基因组编辑、精确定点基因敲入的方法
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
CN109628404B (zh) * 2018-12-18 2020-04-28 浙江大学 猪皮下脂肪前体细胞永生化细胞系的构建方法及用途
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
MX2021011426A (es) 2019-03-19 2022-03-11 Broad Inst Inc Metodos y composiciones para editar secuencias de nucleótidos.
EP3956349A1 (en) 2019-04-17 2022-02-23 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110200973A (zh) * 2019-06-19 2019-09-06 南京市儿童医院 Dna依赖性蛋白激酶特异性抑制剂在制备防治肾纤维化药物中的用途
KR20220039719A (ko) 2019-07-03 2022-03-29 팩터 바이오사이언스 인크. 양이온성 지질 및 이의 용도
EP3999103A4 (en) 2019-07-19 2023-11-22 Inari Agriculture Technology, Inc. GENOMIC EDITING FOR ENHANCED HOMOLOGY-DIRECTED REPAIR
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CN110699381A (zh) * 2019-09-17 2020-01-17 合肥瑞灵生物科技有限公司 地中海贫血病基因治疗载体构建方法及其用途
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
WO2021191678A1 (en) 2020-03-23 2021-09-30 Avectas Limited Engineering of dendritic cells for generation of vaccines against sars-cov-2
MX2022014008A (es) 2020-05-08 2023-02-09 Broad Inst Inc Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo.
CN113151181B (zh) * 2021-04-14 2022-11-29 中山大学附属第七医院(深圳) 一种脂肪干细胞重编程为神经元的方法及负载该神经元的细胞适应性水凝胶修复脊髓损伤
CN113373214B (zh) * 2021-06-18 2024-03-29 中国人民解放军军事科学院军事医学研究院 Cd30在诊断脑神经相关疾病中的用途
KR20240055811A (ko) 2021-09-10 2024-04-29 애질런트 테크놀로지스, 인크. 프라임 편집을 위한 화학적 변형을 갖는 가이드 rna
CN114150017B (zh) * 2021-11-30 2023-05-16 长江大学 一种调节猪未成熟睾丸支持细胞乳酸分泌水平的方法及应用
JP2025524469A (ja) 2022-06-24 2025-07-30 チューン セラピューティクス インコーポレイテッド 標的を定めた遺伝子抑制を介して低密度リポタンパク質を低減するための組成物、系、及び方法
WO2024032678A1 (zh) * 2022-08-11 2024-02-15 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途
KR20250057640A (ko) * 2023-10-18 2025-04-29 연세대학교 산학협력단 신경세포 분화 유도용 재조합 벡터 및 이를 포함하는 퇴행성 뇌신경 질환의 예방 또는 치료용 약학적 조성물
CN118667815B (zh) * 2024-07-16 2025-02-14 宁夏大学 一种靶向prdm1改善牛子宫内膜炎的核苷酸序列及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011154393A1 (en) * 2010-06-07 2011-12-15 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Fusion proteins comprising a dna-binding domain of a tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use

Family Cites Families (122)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3539465A (en) 1968-10-08 1970-11-10 Ncr Co Encapsulation of hydrophilic liquid-in-oil emulsions
US5843780A (en) 1995-01-20 1998-12-01 Wisconsin Alumni Research Foundation Primate embryonic stem cells
US6743771B2 (en) * 1995-12-29 2004-06-01 Novactyl, Inc. Methods and compositions for controlling protein assembly or aggregation
JP2001508302A (ja) 1997-01-10 2001-06-26 ライフ テクノロジーズ，インコーポレイテッド 胚性幹細胞血清置換
AU9319398A (en) 1997-09-19 1999-04-05 Sequitur, Inc. Sense mrna therapy
US7621606B2 (en) 2001-08-27 2009-11-24 Advanced Cell Technology, Inc. Trans-differentiation and re-differentiation of somatic cells and production of cells for cell therapies
US20110171185A1 (en) 1999-06-30 2011-07-14 Klimanskaya Irina V Genetically intact induced pluripotent cells or transdifferentiated cells and methods for the production thereof
EP1322656B1 (en) 2000-09-26 2008-01-16 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory activity of immunostimulatory oligonucleotide analogs by positional chemical changes
AU2002330465B2 (en) 2001-09-21 2006-07-27 Kyoto University Screening method for a reprogramming agent, reprogramming agent screened by the method, method for using the reprogramming agent, method for differentiating an undifferentiated fusion cell, and production method of cells, tissues and organs.
CA2463914A1 (en) 2001-10-18 2003-04-24 Ixion Biotechnology, Inc. Conversion of liver stem and progenitor cells to pancreatic functional cells
US7276489B2 (en) 2002-10-24 2007-10-02 Idera Pharmaceuticals, Inc. Modulation of immunostimulatory properties of oligonucleotide-based compounds by optimal presentation of 5′ ends
GB0202149D0 (en) 2002-01-30 2002-03-20 Univ Edinburgh Pluripotency determining factors and uses thereof
CA2524495A1 (en) 2003-06-03 2005-01-13 Eli Lilly And Company Modulation of survivin expression
US20070134796A1 (en) 2005-07-26 2007-06-14 Sangamo Biosciences, Inc. Targeted integration and expression of exogenous nucleic acid sequences
US7682828B2 (en) 2003-11-26 2010-03-23 Whitehead Institute For Biomedical Research Methods for reprogramming somatic cells
ES2383813T3 (es) 2004-09-08 2012-06-26 Wisconsin Alumni Research Foundation Método de cultivo y cultivo de células madre embrionarias
DK3196296T3 (en) 2004-09-08 2019-02-04 Wisconsin Alumini Res Foundation Cultivation of human embryonic stem cells
JP5735194B2 (ja) * 2005-01-25 2015-06-17 セル セラピューティクス インコーポレーテッド 改善された生体内半減期を有する生物学的に活性なタンパク質
EP1904528B1 (en) 2005-07-13 2012-10-31 Novo Nordisk Health Care AG Host cell protein knock-out cells for production of therapeutic proteins
US8323666B2 (en) 2005-08-01 2012-12-04 Allergan, Inc. Botulinum toxin compositions
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
US9012219B2 (en) 2005-08-23 2015-04-21 The Trustees Of The University Of Pennsylvania RNA preparations comprising purified modified RNA for reprogramming cells
US8476070B2 (en) 2005-08-29 2013-07-02 Technion Research & Development Foundation Limited Media for culturing stem cells
US8129187B2 (en) 2005-12-13 2012-03-06 Kyoto University Somatic cell reprogramming by retroviral vectors encoding Oct3/4. Klf4, c-Myc and Sox2
EP4223769A3 (en) 2005-12-13 2023-11-01 Kyoto University Nuclear reprogramming factor
US8278104B2 (en) 2005-12-13 2012-10-02 Kyoto University Induced pluripotent stem cells produced with Oct3/4, Klf4 and Sox2
CA2634329A1 (en) 2005-12-22 2007-07-19 Csl Behring Gmbh Octanoate-reduced human albumin
DE102006051516A1 (de) * 2006-10-31 2008-05-08 Curevac Gmbh (Basen-)modifizierte RNA zur Expressionssteigerung eines Proteins
GB0623635D0 (en) 2006-11-27 2007-01-03 Stem Cell Sciences Uk Ltd Pluripotent cell growth media
EP2099496A2 (en) 2006-12-08 2009-09-16 Massachusetts Institute of Technology Delivery of nanoparticles and/or agents to cells
US10829733B2 (en) 2007-01-04 2020-11-10 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
JP2010515464A (ja) 2007-01-11 2010-05-13 イエール・ユニバーシテイ Ｈｉｖ細胞表面受容体の標的化不活性化のための組成物および方法
US8859229B2 (en) 2007-02-02 2014-10-14 Yale University Transient transfection with RNA
US9249423B2 (en) 2007-02-02 2016-02-02 Yale University Method of de-differentiating and re-differentiating somatic cells using RNA
CN101743306A (zh) 2007-03-23 2010-06-16 威斯康星校友研究基金会 体细胞重编程
EP3878949B1 (en) 2007-04-07 2025-06-04 Whitehead Institute for Biomedical Research Reprogramming of somatic cells
AU2008286249B2 (en) 2007-12-10 2013-10-10 Kyoto University Efficient method for nuclear reprogramming
EP2072618A1 (en) 2007-12-14 2009-06-24 Johannes Gutenberg-Universität Mainz Use of RNA for reprogramming somatic cells
US9534205B2 (en) 2008-03-17 2017-01-03 The Scripps Research Institute Combined chemical and genetic approaches for generation of induced pluripotent stem cells
US20110045001A1 (en) 2008-03-28 2011-02-24 Biontex Laboratories Gmbh Transfection results of non-viral gene delivery systems by influencing of the innate immune system
AU2009238629C1 (en) * 2008-04-14 2015-04-30 Sangamo Therapeutics, Inc. Linear donor constructs for targeted integration
WO2009127230A1 (en) 2008-04-16 2009-10-22 Curevac Gmbh MODIFIED (m)RNA FOR SUPPRESSING OR AVOIDING AN IMMUNOSTIMULATORY RESPONSE AND IMMUNOSUPPRESSIVE COMPOSITION
KR101871192B1 (ko) 2008-06-04 2018-06-27 후지필름 셀룰러 다이내믹스, 인코포레이티드 비-바이러스 접근법을 사용한 ｉＰＳ 세포의 생산 방법
GB2460552B (en) 2008-06-05 2011-09-07 Iti Scotland Ltd Stem cell culture media and methods
US8669048B2 (en) 2008-06-24 2014-03-11 Parkinson's Institute Pluripotent cell lines and methods of use thereof
US20100184033A1 (en) 2008-07-16 2010-07-22 West Michael D Methods to accelerate the isolation of novel cell strains from pluripotent stem cells and cells obtained thereby
US20110263015A1 (en) 2008-08-20 2011-10-27 Virxsys Corporation Compositions and methods for generation of pluripotent stem cells
DK3450545T5 (da) 2008-10-24 2024-09-09 Wisconsin Alumni Res Found Pluripotente stamceller opnået ved ikke-viral omprogrammering
EP2192174B1 (en) * 2008-11-21 2015-11-11 Fraunhofer-Gesellschaft zur Förderung der angewandten Forschung e.V. Reprogramming cells toward a pluripotent state
US20110023141A1 (en) * 2008-12-04 2011-01-27 Sigma-Aldrich Co. Genomic editing of genes involved with parkinson's disease
WO2010079340A2 (en) 2009-01-08 2010-07-15 Eisai R & D Management Co., Ltd. Assay
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
EP2213990B1 (fr) 2009-01-22 2016-08-17 Johnson Controls Technology Company Procédé de calibration et/ou de correction d'un dispositif d'affichage ayant une aiguille, l'aiguille étant mobile en rotation autour d'un axe de rotation
WO2010093655A2 (en) 2009-02-10 2010-08-19 University Of Dayton Enhanced method for producing stem-like cells from somatic cells
US10894944B2 (en) 2009-04-10 2021-01-19 Monash University Cell culture media
DK2421957T3 (da) 2009-04-22 2021-01-25 Viacyte Inc Cellesammensætninger afledt af dedifferentierede omprogrammerede celler
WO2010123501A1 (en) 2009-04-22 2010-10-28 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
US10837020B2 (en) 2009-04-22 2020-11-17 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long RNA molecules
US8496941B2 (en) 2009-06-03 2013-07-30 National Institute Of Advanced Industrial Science And Technology Vectors for generating pluripotent stem cells and methods of producing pluripotent stem cells using the same
ES2587963T3 (es) 2009-07-31 2016-10-27 Ethris Gmbh ARN con una combinación de nucleótidos no modificados y modificados para la expresión de proteínas
WO2011036640A2 (en) * 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
CA2779039A1 (en) * 2009-10-29 2011-05-05 Mcmaster University Generating induced pluripotent stem cells and progenitor cells from fibroblasts
JP5898086B2 (ja) 2009-11-04 2016-04-06 セルラー ダイナミクス インターナショナル， インコーポレイテッド 化学物質を用いるエピソームリプログラミング
CA2780726A1 (en) 2009-11-11 2011-05-19 Tariq M. Rana Method for generation and regulation of ips cells and compositions thereof
LT3112467T (lt) 2009-12-07 2018-06-25 The Trustees Of The University Of Pennsylvania Išgrynintą modifikuotą rnr apimantys rnr preparatai, skirti ląstelių perprogramavimui
US20130189741A1 (en) 2009-12-07 2013-07-25 Cellscript, Inc. Compositions and methods for reprogramming mammalian cells
ES2696825T3 (es) * 2009-12-10 2019-01-18 Univ Minnesota Modificación del ADN inducida por el efector TAL
US8557972B2 (en) 2009-12-21 2013-10-15 University Of Washington Through Its Center For Commercialization Compositions and methods for transfection of RNA and controlled stabilization of transfected RNA
US8962331B2 (en) 2010-02-01 2015-02-24 The Board Of Trustees Of The Leland Stanford Junior University Method of making induced pluripotent stem cell from adipose stem cells using minicircle DNA vectors
WO2011110886A1 (en) 2010-03-09 2011-09-15 Biolamina Ab Composition and method for enabling proliferation of pluripotent human stem cells
CA2796464C (en) 2010-04-16 2021-08-03 Immune Disease Institute, Inc. Sustained polypeptide expression from synthetic, modified rnas and uses thereof
WO2011140397A2 (en) 2010-05-05 2011-11-10 The Regents Of The University Of California Office Of The President Stem cell defined media for xeno-free and feeder free conditions and uses thereof
US8048675B1 (en) 2010-05-12 2011-11-01 Ipierian, Inc. Integration-free human induced pluripotent stem cells from blood
US20130145487A1 (en) * 2010-05-12 2013-06-06 Cellectis Meganuclease variants cleaving a dna target sequence from the dystrophin gene and uses thereof
EP2571512B1 (en) * 2010-05-17 2017-08-23 Sangamo BioSciences, Inc. Novel dna-binding proteins and uses thereof
US20110201118A1 (en) * 2010-06-14 2011-08-18 Iowa State University Research Foundation, Inc. Nuclease activity of tal effector and foki fusion protein
CN103003416B (zh) * 2010-06-15 2016-11-16 细胞动力学国际有限公司 从小体积的外周血产生诱导性多潜能干细胞
WO2012019122A2 (en) 2010-08-05 2012-02-09 Wisconsin Alumni Research Foundation Simplified basic media for human pluripotent cell culture
EP3578205A1 (en) 2010-08-06 2019-12-11 ModernaTX, Inc. A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof
WO2012021632A2 (en) * 2010-08-10 2012-02-16 The Johns Hopkins University Generation and use of pluripotent stem cells
GB201014169D0 (en) * 2010-08-25 2010-10-06 Cambridge Entpr Ltd In vitro hepatic differentiation
JP5794588B2 (ja) 2010-09-14 2015-10-14 国立大学法人京都大学 効率的な人工多能性幹細胞の樹立方法
EP3511420B1 (en) * 2010-09-27 2021-04-07 Sangamo Therapeutics, Inc. Compositions for inhibiting viral entry into cells
CN103429606A (zh) 2010-10-01 2013-12-04 现代治疗公司 设计核酸及其使用方法
EP2625278A1 (en) * 2010-10-08 2013-08-14 Regents of the University of Minnesota A method to increase gene targeting frequency
WO2012060473A1 (en) 2010-11-04 2012-05-10 Kyoto University Method of efficiently establishing induced pluripotent stem cells
US9267123B2 (en) 2011-01-05 2016-02-23 Sangamo Biosciences, Inc. Methods and compositions for gene correction
US9499592B2 (en) * 2011-01-26 2016-11-22 President And Fellows Of Harvard College Transcription activator-like effectors
KR20120096395A (ko) * 2011-02-22 2012-08-30 주식회사 툴젠 뉴클레아제에 의해 유전자 변형된 세포를 농축시키는 방법
EP2678434B1 (en) * 2011-02-25 2018-11-07 Recombinetics, Inc. Genetically modified animals and methods for making the same
CA2832807A1 (en) * 2011-03-07 2012-09-13 Massachusetts Institute Of Technology Methods for transfecting cells with nucleic acids
CA2831613A1 (en) * 2011-03-31 2012-10-04 Moderna Therapeutics, Inc. Delivery and formulation of engineered nucleic acids
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
JP5996630B2 (ja) * 2011-04-05 2016-09-21 セレクティスＣｅｌｌｅｃｔｉｓ コンパクトｔａｌｅ−ヌクレアーゼを作製する方法及びその使用
US9862926B2 (en) 2011-06-27 2018-01-09 Cellscript, Llc. Inhibition of innate immune response
US20130040302A1 (en) 2011-07-11 2013-02-14 Thomas J. Burke Methods for cell reprogramming and genome engineering
KR102061557B1 (ko) * 2011-09-21 2020-01-03 상가모 테라퓨틱스, 인코포레이티드 이식 유전자 발현의 조절을 위한 방법 및 조성물
KR20190099538A (ko) 2011-10-03 2019-08-27 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도
BR112014013664A2 (pt) * 2011-12-05 2020-11-03 Factor Bioscience Inc. métodos e produtos para transfectar células
US8497124B2 (en) 2011-12-05 2013-07-30 Factor Bioscience Inc. Methods and products for reprogramming cells to a less differentiated state
EP4144378A1 (en) 2011-12-16 2023-03-08 ModernaTX, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
CA2859691A1 (en) 2011-12-21 2013-06-27 Moderna Therapeutics, Inc. Methods of increasing the viability or longevity of an organ or organ explant
LT3144389T (lt) 2011-12-30 2018-08-10 Cellscript, Llc In vitro susintetintos viengrandės rnr gavimas ir panaudojimas įvedimui į žinduolio ląsteles, siekiant sužadinti biologinį arba biocheminį poveikį
AU2013243954A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US20130274129A1 (en) * 2012-04-04 2013-10-17 Geneart Ag Tal-effector assembly platform, customized services, kits and assays
JP2015513919A (ja) 2012-04-10 2015-05-18 ロッシ，ジョン，ジェイ 膵癌細胞への治療的送達および診断的送達のためのｒｎａアプタマー
KR20150045935A (ko) 2012-04-24 2015-04-29 더 브리검 앤드 우먼즈 하스피털, 인크. 다능성 세포를 다시 생성하는 방법
US9738879B2 (en) * 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
US10155929B2 (en) 2012-05-13 2018-12-18 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free derivation of human-induced pluripotent stem cells with synthetic messenger RNA
US10119150B2 (en) 2012-05-13 2018-11-06 Allele Biotechnology & Pharmaceuticals, Inc. Feeder-free Derivation of human-induced pluripotent stem cells with synthetic messenger RNA
JP6448531B2 (ja) 2012-05-13 2019-01-09 アリール バイオテクノロジー アンド ファーマシューティカルズ， インコーポレイテッド 合成メッセンジャーｒｎａを使用したヒト人工多能性幹細胞のフィーダーフリー誘導
EP3816281A1 (en) * 2012-07-11 2021-05-05 Sangamo Therapeutics, Inc. Methods and compositions for the treatment of lysosomal storage diseases
EP2684892A1 (en) * 2012-07-13 2014-01-15 Association Française contre les Myopathies Compositions and methods for duchenne muscular dystrophy gene therapy
ES2824024T3 (es) * 2012-10-10 2021-05-11 Sangamo Therapeutics Inc Compuestos modificadores de células T y usos de los mismos
BR122019025681B1 (pt) 2012-11-01 2023-04-18 Factor Bioscience Inc Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula
US20140140969A1 (en) * 2012-11-20 2014-05-22 Sangamo Biosciences, Inc. Methods and compositions for muscular dystrophies
AU2013355327A1 (en) * 2012-12-05 2015-06-11 Sangamo Therapeutics, Inc. Methods and compositions for regulation of metabolic disorders
PT2959005T (pt) 2013-02-22 2021-12-30 Univ Leland Stanford Junior Utilização médica relacionada com extensão de telómero
KR102733409B1 (ko) * 2013-06-05 2024-11-21 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
CN104878020B (zh) 2015-04-29 2017-09-15 华中农业大学 一种控制水稻叶片直立性发育的基因及其应用
US10576167B2 (en) * 2016-08-17 2020-03-03 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011154393A1 (en) * 2010-06-07 2011-12-15 Helmholtz Zentrum München Deutsches Forschungszentrum Für Gesundheit Und Umwelt (Gmbh) Fusion proteins comprising a dna-binding domain of a tal effector protein and a non-specific cleavage domain of a restriction nuclease and their use

Also Published As

Publication number Publication date
BR122019025681B1 (pt) 2023-04-18
KR102121086B1 (ko) 2020-06-09
US11339409B2 (en) 2022-05-24
US9487768B2 (en) 2016-11-08
US20200056207A1 (en) 2020-02-20
JP6510416B2 (ja) 2019-05-08
EP2914728A1 (en) 2015-09-09
JP7436406B2 (ja) 2024-02-21
US11332758B2 (en) 2022-05-17
US11332759B2 (en) 2022-05-17
US20200032295A1 (en) 2020-01-30
JP2018115207A (ja) 2018-07-26
US10724053B2 (en) 2020-07-28
KR20150080573A (ko) 2015-07-09
JP2024056815A (ja) 2024-04-23
MX2019002498A (es) 2021-11-16
RU2711249C2 (ru) 2020-01-15
US9376669B2 (en) 2016-06-28
US10415060B2 (en) 2019-09-17
US20160251649A1 (en) 2016-09-01
AU2018264115A1 (en) 2018-12-06
US20170362612A1 (en) 2017-12-21
US10752919B2 (en) 2020-08-25
US20200040364A1 (en) 2020-02-06
KR102596302B1 (ko) 2023-11-01
KR20210127818A (ko) 2021-10-22
RU2015120524A (ru) 2016-12-20
AU2013337651A1 (en) 2015-06-11
US12006508B2 (en) 2024-06-11
US9447395B2 (en) 2016-09-20
EP2914728A4 (en) 2016-05-25
JP2021090435A (ja) 2021-06-17
RU2019143431A (ru) 2020-04-28
EP3786298A1 (en) 2021-03-03
BR122019025678B1 (pt) 2023-04-18
JP6793146B2 (ja) 2020-12-02
CA3150985A1 (en) 2014-05-08
US20200325496A1 (en) 2020-10-15
US10590437B2 (en) 2020-03-17
MX363017B (es) 2019-03-04
US10767195B2 (en) 2020-09-08
CA2890110A1 (en) 2014-05-08
US20190345519A1 (en) 2019-11-14
US10752918B2 (en) 2020-08-25
JP6890565B2 (ja) 2021-06-18
JP2015534817A (ja) 2015-12-07
US20160251639A1 (en) 2016-09-01
EP2914728B1 (en) 2020-07-08
KR102315098B1 (ko) 2021-10-21
KR20230154283A (ko) 2023-11-07
AU2018264115B2 (en) 2021-08-12
BR112015009804A2 (pt) 2017-08-22
US20150267189A1 (en) 2015-09-24
US20170015983A1 (en) 2017-01-19
US20200032298A1 (en) 2020-01-30
CA2890110C (en) 2023-05-02
US20220243228A1 (en) 2022-08-04
HK1214304A1 (en) 2016-07-22
US20200032297A1 (en) 2020-01-30
US11339410B2 (en) 2022-05-24
JP2018113985A (ja) 2018-07-26
MX2015005346A (es) 2015-07-14
CN104769112A (zh) 2015-07-08
WO2014071219A1 (en) 2014-05-08
US20150275193A1 (en) 2015-10-01
KR20200067921A (ko) 2020-06-12
US20170218400A1 (en) 2017-08-03
US9758797B2 (en) 2017-09-12
US9464285B2 (en) 2016-10-11
US9657282B2 (en) 2017-05-23
US20200032296A1 (en) 2020-01-30
US20200032299A1 (en) 2020-01-30
AU2021250972A1 (en) 2021-11-11
US10752917B2 (en) 2020-08-25
US20200040363A1 (en) 2020-02-06
US20200040365A1 (en) 2020-02-06

Similar Documents

Publication Publication Date Title
US20220243228A1 (en) Methods and products for expressing proteins in cells
HK40048692A (en) Methods and products for expressing proteins in cells
HK1214304B (en) Methods and products for expressing proteins in cells

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)