JP2018533362A - 薬物候補の有効性プロファイルを決定するための方法 - Google Patents
薬物候補の有効性プロファイルを決定するための方法 Download PDFInfo
- Publication number
- JP2018533362A JP2018533362A JP2018521350A JP2018521350A JP2018533362A JP 2018533362 A JP2018533362 A JP 2018533362A JP 2018521350 A JP2018521350 A JP 2018521350A JP 2018521350 A JP2018521350 A JP 2018521350A JP 2018533362 A JP2018533362 A JP 2018533362A
- Authority
- JP
- Japan
- Prior art keywords
- cells
- days
- differentiated
- drug candidate
- cell
- 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.)
- Pending
Links
Images
Classifications
-
- 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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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
- A61P25/08—Antiepileptics; Anticonvulsants
-
- 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
- A61P25/20—Hypnotics; Sedatives
-
- 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
-
- 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
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—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
- C12N15/1137—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 against enzymes
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/0018—Culture media for cell or tissue culture
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0619—Neurons
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0618—Cells of the nervous system
- C12N5/0623—Stem cells
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
- C12N5/06—Animal cells or tissues; Human cells or tissues
- C12N5/0602—Vertebrate cells
- C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
-
- 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
- G01N33/5044—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 involving specific cell types
- G01N33/5058—Neurological 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
-
- 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
- C12N2500/00—Specific components of cell culture medium
- C12N2500/30—Organic components
- C12N2500/38—Vitamins
-
- 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
- C12N2500/00—Specific components of cell culture medium
- C12N2500/30—Organic components
- C12N2500/44—Thiols, e.g. mercaptoethanol
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/01—Modulators of cAMP or cGMP, e.g. non-hydrolysable analogs, phosphodiesterase inhibitors, cholera toxin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/10—Growth factors
- C12N2501/11—Epidermal growth factor [EGF]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/10—Growth factors
- C12N2501/115—Basic fibroblast growth factor (bFGF, FGF-2)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/10—Growth factors
- C12N2501/119—Other fibroblast growth factors, e.g. FGF-4, FGF-8, FGF-10
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/10—Growth factors
- C12N2501/13—Nerve growth factor [NGF]; Brain-derived neurotrophic factor [BDNF]; Cilliary neurotrophic factor [CNTF]; Glial-derived neurotrophic factor [GDNF]; Neurotrophins [NT]; Neuregulins
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/40—Regulators of development
- C12N2501/41—Hedgehog proteins; Cyclopamine (inhibitor)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/60—Transcription factors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/70—Enzymes
- C12N2501/72—Transferases [EC 2.]
- C12N2501/727—Kinases (EC 2.7.)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2501/00—Active agents used in cell culture processes, e.g. differentation
- C12N2501/999—Small molecules not provided for elsewhere
-
- 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
- C12N2503/00—Use of cells in diagnostics
- C12N2503/02—Drug screening
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
- C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem 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
- C12N2533/00—Supports or coatings for cell culture, characterised by material
- C12N2533/50—Proteins
- C12N2533/52—Fibronectin; Laminin
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Physics & Mathematics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Cell Biology (AREA)
- Immunology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Hematology (AREA)
- Urology & Nephrology (AREA)
- Toxicology (AREA)
- Pathology (AREA)
- Tropical Medicine & Parasitology (AREA)
- Food Science & Technology (AREA)
- Analytical Chemistry (AREA)
- General Physics & Mathematics (AREA)
- Developmental Biology & Embryology (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021113496A JP2021191262A (ja) | 2015-11-12 | 2021-07-08 | 薬物候補の有効性プロファイルを決定するための方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP15194367.7 | 2015-11-12 | ||
| EP15194367 | 2015-11-12 | ||
| EP16189502.4 | 2016-09-19 | ||
| EP16189502 | 2016-09-19 | ||
| PCT/EP2016/077435 WO2017081254A1 (en) | 2015-11-12 | 2016-11-11 | Methods for determining the efficacy profile of a drug candidate |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021113496A Division JP2021191262A (ja) | 2015-11-12 | 2021-07-08 | 薬物候補の有効性プロファイルを決定するための方法 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| JP2018533362A true JP2018533362A (ja) | 2018-11-15 |
Family
ID=57345910
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018521350A Pending JP2018533362A (ja) | 2015-11-12 | 2016-11-11 | 薬物候補の有効性プロファイルを決定するための方法 |
| JP2018524194A Active JP6751142B2 (ja) | 2015-11-12 | 2016-11-11 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2018521433A Active JP7066609B2 (ja) | 2015-11-12 | 2016-11-11 | 霊長類種に由来する標準化された神経細胞アッセイ |
| JP2020136397A Active JP7279000B2 (ja) | 2015-11-12 | 2020-08-12 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2021113496A Pending JP2021191262A (ja) | 2015-11-12 | 2021-07-08 | 薬物候補の有効性プロファイルを決定するための方法 |
| JP2023009553A Pending JP2023052630A (ja) | 2015-11-12 | 2023-01-25 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2024196762A Pending JP2025024060A (ja) | 2015-11-12 | 2024-11-11 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
Family Applications After (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018524194A Active JP6751142B2 (ja) | 2015-11-12 | 2016-11-11 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2018521433A Active JP7066609B2 (ja) | 2015-11-12 | 2016-11-11 | 霊長類種に由来する標準化された神経細胞アッセイ |
| JP2020136397A Active JP7279000B2 (ja) | 2015-11-12 | 2020-08-12 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2021113496A Pending JP2021191262A (ja) | 2015-11-12 | 2021-07-08 | 薬物候補の有効性プロファイルを決定するための方法 |
| JP2023009553A Pending JP2023052630A (ja) | 2015-11-12 | 2023-01-25 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
| JP2024196762A Pending JP2025024060A (ja) | 2015-11-12 | 2024-11-11 | 父方ube3aの発現を誘導するオリゴヌクレオチド |
Country Status (30)
| Country | Link |
|---|---|
| US (9) | US10494633B2 (enExample) |
| EP (5) | EP3374498A1 (enExample) |
| JP (7) | JP2018533362A (enExample) |
| KR (4) | KR20250121139A (enExample) |
| CN (5) | CN108350431A (enExample) |
| AU (4) | AU2016352836B2 (enExample) |
| CA (1) | CA3004799C (enExample) |
| CL (3) | CL2018001189A1 (enExample) |
| CO (1) | CO2018004550A2 (enExample) |
| CR (3) | CR20200118A (enExample) |
| DK (2) | DK3374509T3 (enExample) |
| ES (2) | ES2991800T3 (enExample) |
| FI (1) | FI4220360T3 (enExample) |
| HK (2) | HK1258309A1 (enExample) |
| HR (2) | HRP20210227T1 (enExample) |
| HU (2) | HUE053197T2 (enExample) |
| IL (3) | IL281211B2 (enExample) |
| LT (2) | LT3374509T (enExample) |
| MA (1) | MA67580B1 (enExample) |
| MX (4) | MX2018004978A (enExample) |
| PE (2) | PE20181157A1 (enExample) |
| PH (1) | PH12018501005A1 (enExample) |
| PL (2) | PL4220360T3 (enExample) |
| RS (2) | RS61529B9 (enExample) |
| RU (1) | RU2742007C2 (enExample) |
| SG (1) | SG11201803951QA (enExample) |
| SI (2) | SI4220360T1 (enExample) |
| UA (1) | UA125963C2 (enExample) |
| WO (3) | WO2017081254A1 (enExample) |
| ZA (1) | ZA201802494B (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022517803A (ja) * | 2019-01-17 | 2022-03-10 | エフ.ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
Families Citing this family (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2009323766B2 (en) | 2008-12-02 | 2016-10-06 | Wave Life Sciences Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| SG177564A1 (en) | 2009-07-06 | 2012-02-28 | Ontorii Inc | Novel nucleic acid prodrugs and methods of use thereof |
| KR102173836B1 (ko) | 2009-09-11 | 2020-11-05 | 아이오니스 파마수티컬즈, 인코포레이티드 | 헌팅틴 발현의 조절 |
| EP2620428B1 (en) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| PL3461895T3 (pl) | 2012-06-25 | 2021-01-11 | Ionis Pharmaceuticals, Inc. | Modulacja ekspresji ube3a-ats |
| SG10201912895PA (en) | 2012-07-13 | 2020-02-27 | Wave Life Sciences Ltd | Chiral control |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| PT3094728T (pt) | 2014-01-16 | 2022-05-19 | Wave Life Sciences Ltd | Desenho quiral |
| WO2016086104A1 (en) | 2014-11-25 | 2016-06-02 | Ionis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| CN108350431A (zh) | 2015-11-12 | 2018-07-31 | 豪夫迈·罗氏有限公司 | 用于确定候选药物的功效特征的方法 |
| RU2020121752A (ru) * | 2017-12-01 | 2021-12-30 | ТЕХАССКАЯ УНИВЕРСИТЕТСКАЯ СИСТЕМА А энд М | Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты |
| WO2019157531A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| CN108795935B (zh) * | 2018-05-23 | 2022-06-21 | 中国农业科学院上海兽医研究所(中国动物卫生与流行病学中心上海分中心) | 日本血吸虫SjELAV-like 1基因的siRNA及其应用 |
| US12221609B2 (en) | 2018-09-21 | 2025-02-11 | University Of Connecticut | Compositions and methods to restore paternal UBE3A gene expression in human Angelman syndrome |
| MX2021009950A (es) * | 2019-02-20 | 2021-09-21 | Roche Innovation Ct Copenhagen As | Oligonucleotidos gapmeros de fosfonoacetato. |
| UA128437C2 (uk) * | 2019-03-29 | 2024-07-10 | Айоніс Фармасьютикалз, Інк. | Сполуки і способи модулювання ube3a-ats |
| MX2021014248A (es) | 2019-05-22 | 2022-01-06 | Univ North Carolina Chapel Hill | Genes ube3a y casetes de expresion y su uso. |
| JP7701879B2 (ja) | 2019-05-28 | 2025-07-02 | エフ. ホフマン-ラ ロシュ アーゲー | 単球前駆細胞を生成するための方法 |
| TW202204607A (zh) * | 2020-04-16 | 2022-02-01 | 英商羅斯林科技有限公司 | 誘導性多功能幹細胞(iPSC)之誘導 |
| CN115702243A (zh) * | 2020-06-09 | 2023-02-14 | 罗氏创新中心哥本哈根有限公司 | 用于治疗性多核苷酸的鸟苷类似物 |
| JPWO2022158487A1 (enExample) | 2021-01-21 | 2022-07-28 | ||
| JP2025516554A (ja) | 2022-05-10 | 2025-05-30 | エフ. ホフマン-ラ ロシュ アーゲー | Cfp-elk1遺伝子間領域を標的とするアンチセンスオリゴヌクレオチド |
| WO2024227765A2 (en) | 2023-05-04 | 2024-11-07 | F. Hoffmann-La Roche Ag | Oligonucleotides capable of upregulating glucocerebrosidase expression |
| KR20250068540A (ko) * | 2023-11-08 | 2025-05-16 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025125542A1 (en) | 2023-12-15 | 2025-06-19 | F. Hoffmann-La Roche Ag | Intrathecal dosage method |
| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015143342A1 (en) * | 2014-03-21 | 2015-09-24 | Cellular Dynamics International, Inc. | Production of midbrain dopaminergic neurons and methods for the use thereof |
Family Cites Families (58)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4373084A (en) | 1981-07-07 | 1983-02-08 | E. I. Du Pont De Nemours And Company | Sulfur-modified polypropylene ether glycols, a method for preparing them, and polyurethanes prepared therefrom |
| JP2823959B2 (ja) | 1991-10-24 | 1998-11-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6617162B2 (en) | 2001-12-18 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Antisense modulation of estrogen receptor alpha expression |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ/エス | オリゴヌクレオチド類似体 |
| US6238921B1 (en) | 1998-03-26 | 2001-05-29 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of human mdm2 expression |
| CA2702148C (en) * | 1999-01-06 | 2014-03-04 | Genenews Inc. | Method of profiling gene expression in a human subject having an infectious disease |
| ATE287897T2 (de) | 1999-02-12 | 2005-02-15 | Sankyo Co | Analoga von nukleosiden und oligonukleotiden |
| CN102180924A (zh) | 1999-05-04 | 2011-09-14 | 桑塔里斯制药公司 | L-核糖-lna类似物 |
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| AU1936201A (en) | 1999-12-02 | 2001-06-12 | Myriad Genetics, Inc. | Protein-protein interactions |
| US6300132B1 (en) | 1999-12-17 | 2001-10-09 | Isis Pharmaceuticals, Inc. | Antisense inhibition of telomeric repeat binding factor 2 expression |
| AU2001268163A1 (en) * | 2000-06-01 | 2001-12-11 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the ube3a gene |
| US20030087855A1 (en) | 2001-09-13 | 2003-05-08 | Isis Pharmaceuticals Inc. | Antisense modulation of protein kinase R expression |
| CA2451643C (en) * | 2001-06-21 | 2012-11-13 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| US20050124572A1 (en) * | 2002-06-17 | 2005-06-09 | Freier Susan M. | Compositions and their uses directed to signal tranducers |
| CA2495398A1 (en) | 2002-08-14 | 2004-02-26 | Pharmacia Corporation | Antisense modulation of nav1.3 expression |
| EP1575498A2 (en) * | 2002-09-25 | 2005-09-21 | Pharmacia Corporation | Antisense modulation of microsomal prostaglandin e2 synthase expression |
| ATE442152T1 (de) | 2002-11-18 | 2009-09-15 | Santaris Pharma As | Antisense-entwurf |
| EP1670901A4 (en) | 2003-08-29 | 2007-09-05 | Wisconsin Alumni Res Found | METHOD FOR IN VITRO DIFFERENTIATION OF NEURAL STEM CELLS, MOTOR NEURONES AND DOPAMINE NEURONES FROM EMBRYONAL PRIMATIVE STEM CELLS |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| CN103301475B (zh) | 2005-12-28 | 2016-08-03 | 斯克里普斯研究所 | 药物组合物和表达载体以及调节基因表达的方法和核酸分子的应用 |
| CN102908630B (zh) | 2006-01-27 | 2014-11-19 | Isis制药公司 | 6-修饰的双环核酸类似物 |
| EP2015758B1 (en) | 2006-05-05 | 2014-04-02 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating expression apob |
| EP2066684B1 (en) | 2006-05-11 | 2012-07-18 | Isis Pharmaceuticals, Inc. | 5'-modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| US8580756B2 (en) | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| PE20090064A1 (es) | 2007-03-26 | 2009-03-02 | Novartis Ag | Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende |
| ES2388590T3 (es) | 2007-05-30 | 2012-10-16 | Isis Pharmaceuticals, Inc. | Análogos de ácidos nucleicos bicíclicos con puente aminometileno N-sustituido. |
| WO2008154401A2 (en) | 2007-06-08 | 2008-12-18 | Isis Pharmaceuticals, Inc. | Carbocyclic bicyclic nucleic acid analogs |
| WO2009006478A2 (en) | 2007-07-05 | 2009-01-08 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| WO2009090182A1 (en) | 2008-01-14 | 2009-07-23 | Santaris Pharma A/S | C4'-substituted - dna nucleotide gapmer oligonucleotides |
| DK2285819T3 (da) | 2008-04-04 | 2013-12-02 | Isis Pharmaceuticals Inc | Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider |
| US8501805B2 (en) | 2008-09-24 | 2013-08-06 | Isis Pharmaceuticals, Inc. | Substituted alpha-L-bicyclic nucleosides |
| WO2010096496A2 (en) * | 2009-02-17 | 2010-08-26 | Memorial Sloan-Kettering Cancer Center | Methods of neural conversion of human embryonic stem cells |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| WO2011044684A1 (en) | 2009-10-13 | 2011-04-21 | Stemcell Technologies Inc. | Manipulation of osmolality for differentiating stem cells |
| KR101891352B1 (ko) | 2009-12-23 | 2018-08-24 | 큐알엔에이, 인크. | 간세포 성장 인자(hgf)에 대한 천연 안티센스 전사체의 억제에 의한 hgf 관련 질환의 치료 |
| WO2011156202A1 (en) | 2010-06-08 | 2011-12-15 | Isis Pharmaceuticals, Inc. | Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| JP5998131B2 (ja) | 2010-07-14 | 2016-09-28 | カッパーアールエヌエー,インコーポレイテッド | Discslargehomolog(dlg)dlg1への天然アンチセンス転写物の阻害によるdlg関連疾患の治療 |
| KR101900770B1 (ko) | 2010-07-19 | 2018-09-20 | 아이오니스 파마수티컬즈, 인코포레이티드 | 근육긴장성 이영양증-단백질 키나제(dmpk) 발현의 조절 방법 |
| BR112013003031A2 (pt) * | 2010-08-19 | 2016-06-14 | Hoffmann La Roche | método de produção de células hastes neurais (chn), células hastes neurais e seu uso, composição terapêutica e banco biológico de nscs |
| WO2012064806A2 (en) | 2010-11-11 | 2012-05-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| US9487752B2 (en) | 2011-03-30 | 2016-11-08 | Cellular Dynamics International, Inc. | Priming of pluripotent stem cells for neural differentiation |
| CN103502273A (zh) | 2011-04-20 | 2014-01-08 | 罗氏格黎卡特股份公司 | 用于pH依赖性通过血脑屏障的方法和构建体 |
| EP3640332A1 (en) | 2011-08-29 | 2020-04-22 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| WO2013036868A1 (en) | 2011-09-07 | 2013-03-14 | Marina Biotech Inc. | Synthesis and uses of nucleic acid compounds with conformationally restricted monomers |
| US9221864B2 (en) | 2012-04-09 | 2015-12-29 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| PL3461895T3 (pl) * | 2012-06-25 | 2021-01-11 | Ionis Pharmaceuticals, Inc. | Modulacja ekspresji ube3a-ats |
| ES2724853T3 (es) | 2012-11-15 | 2019-09-16 | Roche Innovation Ct Copenhagen As | Conjugados de oligonucleótidos |
| WO2014077693A1 (en) | 2012-11-16 | 2014-05-22 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Means and methods for reducing an effect of aging in a mammalian cell |
| CN108350431A (zh) | 2015-11-12 | 2018-07-31 | 豪夫迈·罗氏有限公司 | 用于确定候选药物的功效特征的方法 |
| WO2019006107A1 (en) | 2017-06-28 | 2019-01-03 | University Of South Florida | MODIFIED UBE3A GENE FOR GENE THERAPY APPROACH TO ANGELMAN'S SYNDROME |
| RU2020121752A (ru) | 2017-12-01 | 2021-12-30 | ТЕХАССКАЯ УНИВЕРСИТЕТСКАЯ СИСТЕМА А энд М | Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты |
| BR112020010436A2 (pt) | 2018-01-26 | 2020-11-24 | F. Hoffmann-La Roche Ag | oligonucleotídeo radiomarcado, processos para a preparação de um oligonucleotídeo radiomarcado, uso do oligonucleotídeo radiomarcado, método para determinar a biodistribuição e farmacocinética e oligonucleotídeo |
| UA128437C2 (uk) | 2019-03-29 | 2024-07-10 | Айоніс Фармасьютикалз, Інк. | Сполуки і способи модулювання ube3a-ats |
-
2016
- 2016-11-11 CN CN201680065987.2A patent/CN108350431A/zh active Pending
- 2016-11-11 CR CR20200118A patent/CR20200118A/es unknown
- 2016-11-11 SI SI201631843T patent/SI4220360T1/sl unknown
- 2016-11-11 CN CN201680066009.XA patent/CN108350432A/zh active Pending
- 2016-11-11 JP JP2018521350A patent/JP2018533362A/ja active Pending
- 2016-11-11 WO PCT/EP2016/077435 patent/WO2017081254A1/en not_active Ceased
- 2016-11-11 HR HRP20210227TT patent/HRP20210227T1/hr unknown
- 2016-11-11 AU AU2016352836A patent/AU2016352836B2/en active Active
- 2016-11-11 PL PL23152543.7T patent/PL4220360T3/pl unknown
- 2016-11-11 PE PE2018000752A patent/PE20181157A1/es unknown
- 2016-11-11 ES ES23152543T patent/ES2991800T3/es active Active
- 2016-11-11 IL IL281211A patent/IL281211B2/en unknown
- 2016-11-11 DK DK16798440.0T patent/DK3374509T3/da active
- 2016-11-11 DK DK23152543.7T patent/DK4220360T3/da active
- 2016-11-11 RS RS20210241A patent/RS61529B9/sr unknown
- 2016-11-11 WO PCT/EP2016/077383 patent/WO2017081223A1/en not_active Ceased
- 2016-11-11 WO PCT/EP2016/077429 patent/WO2017081250A1/en not_active Ceased
- 2016-11-11 EP EP16797824.6A patent/EP3374498A1/en not_active Withdrawn
- 2016-11-11 EP EP16798672.8A patent/EP3374499A1/en not_active Withdrawn
- 2016-11-11 MA MA67580A patent/MA67580B1/fr unknown
- 2016-11-11 CA CA3004799A patent/CA3004799C/en active Active
- 2016-11-11 CR CR20180264A patent/CR20180264A/es unknown
- 2016-11-11 JP JP2018524194A patent/JP6751142B2/ja active Active
- 2016-11-11 MX MX2018004978A patent/MX2018004978A/es unknown
- 2016-11-11 KR KR1020257024980A patent/KR20250121139A/ko active Pending
- 2016-11-11 SI SI201631093T patent/SI3374509T1/sl unknown
- 2016-11-11 RS RS20241003A patent/RS66036B1/sr unknown
- 2016-11-11 PL PL16798440T patent/PL3374509T3/pl unknown
- 2016-11-11 PE PE2020000810A patent/PE20210451A1/es unknown
- 2016-11-11 CN CN202211275978.XA patent/CN116064540A/zh active Pending
- 2016-11-11 KR KR1020207033999A patent/KR102482440B1/ko active Active
- 2016-11-11 HR HRP20241138TT patent/HRP20241138T1/hr unknown
- 2016-11-11 EP EP16798440.0A patent/EP3374509B1/en active Active
- 2016-11-11 UA UAA201806476A patent/UA125963C2/uk unknown
- 2016-11-11 HK HK19100676.0A patent/HK1258309A1/zh unknown
- 2016-11-11 RU RU2018119315A patent/RU2742007C2/ru active
- 2016-11-11 HK HK19100670.6A patent/HK1258434A1/zh unknown
- 2016-11-11 CN CN202211275954.4A patent/CN116064539A/zh active Pending
- 2016-11-11 SG SG11201803951QA patent/SG11201803951QA/en unknown
- 2016-11-11 EP EP20204481.4A patent/EP3798307A1/en active Pending
- 2016-11-11 LT LTEP16798440.0T patent/LT3374509T/lt unknown
- 2016-11-11 CR CR20220077A patent/CR20220077A/es unknown
- 2016-11-11 LT LTEP23152543.7T patent/LT4220360T/lt unknown
- 2016-11-11 EP EP23152543.7A patent/EP4220360B9/en active Active
- 2016-11-11 KR KR1020187013292A patent/KR102185465B1/ko active Active
- 2016-11-11 KR KR1020227045154A patent/KR102840652B1/ko active Active
- 2016-11-11 CN CN201680066253.6A patent/CN108291226B/zh active Active
- 2016-11-11 IL IL300119A patent/IL300119A/en unknown
- 2016-11-11 FI FIEP23152543.7T patent/FI4220360T3/fi active
- 2016-11-11 HU HUE16798440A patent/HUE053197T2/hu unknown
- 2016-11-11 JP JP2018521433A patent/JP7066609B2/ja active Active
- 2016-11-11 HU HUE23152543A patent/HUE067905T2/hu unknown
- 2016-11-11 ES ES16798440T patent/ES2852998T4/es active Active
- 2016-11-14 US US15/351,113 patent/US10494633B2/en active Active
-
2018
- 2018-04-03 IL IL258488A patent/IL258488B/en active IP Right Grant
- 2018-04-16 ZA ZA2018/02494A patent/ZA201802494B/en unknown
- 2018-04-23 MX MX2022016381A patent/MX2022016381A/es unknown
- 2018-04-23 MX MX2022016377A patent/MX2022016377A/es unknown
- 2018-04-23 MX MX2022016376A patent/MX2022016376A/es unknown
- 2018-04-26 CO CONC2018/0004550A patent/CO2018004550A2/es unknown
- 2018-05-03 CL CL2018001189A patent/CL2018001189A1/es unknown
- 2018-05-08 PH PH12018501005A patent/PH12018501005A1/en unknown
- 2018-05-11 US US15/977,970 patent/US20190154664A1/en not_active Abandoned
- 2018-05-11 US US15/977,895 patent/US20190144824A1/en not_active Abandoned
-
2019
- 2019-04-18 US US16/388,714 patent/US10739332B2/en active Active
- 2019-10-24 US US16/663,024 patent/US10718753B2/en active Active
-
2020
- 2020-04-02 CL CL2020000889A patent/CL2020000889A1/es unknown
- 2020-05-29 AU AU2020203573A patent/AU2020203573B2/en active Active
- 2020-07-20 US US16/933,445 patent/US11320421B2/en active Active
- 2020-08-12 JP JP2020136397A patent/JP7279000B2/ja active Active
-
2021
- 2021-07-08 JP JP2021113496A patent/JP2021191262A/ja active Pending
- 2021-08-13 CL CL2021002159A patent/CL2021002159A1/es unknown
-
2022
- 2022-01-21 US US17/581,089 patent/US11852627B2/en active Active
- 2022-08-05 AU AU2022211910A patent/AU2022211910B2/en active Active
-
2023
- 2023-01-25 AU AU2023200410A patent/AU2023200410B2/en active Active
- 2023-01-25 JP JP2023009553A patent/JP2023052630A/ja active Pending
- 2023-02-22 US US18/172,707 patent/US12259380B2/en active Active
- 2023-10-20 US US18/491,513 patent/US20250116656A2/en active Pending
-
2024
- 2024-11-11 JP JP2024196762A patent/JP2025024060A/ja active Pending
Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015143342A1 (en) * | 2014-03-21 | 2015-09-24 | Cellular Dynamics International, Inc. | Production of midbrain dopaminergic neurons and methods for the use thereof |
Non-Patent Citations (3)
| Title |
|---|
| CARTER R. L. ET AL.: "Reversal of cellular phenotypes in neural cells derived from Huntington's disease monkey-induced plu", STEM CELL REPORTS, vol. 3, no. 4, JPN6020025859, 2014, pages 585 - 593, XP055345080, ISSN: 0004462212, DOI: 10.1016/j.stemcr.2014.07.011 * |
| SIRENKO O. ET AL.: "High-content high-throughput assays for characterizing the viability and morphology of human iPSC-de", ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES, vol. vol. 12, no9/10, JPN6020025861, 2014, pages 536 - 547, ISSN: 0004307410 * |
| SUNDBERG M. ET AL.: "Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safe", STEM CELLS, vol. 31, no. 8, JPN6020025860, 2013, pages 1548 - 1562, XP055189032, ISSN: 0004462213, DOI: 10.1002/stem.1415 * |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022517803A (ja) * | 2019-01-17 | 2022-03-10 | エフ.ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
| JP7241185B2 (ja) | 2019-01-17 | 2023-03-16 | エフ. ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP7066609B2 (ja) | 霊長類種に由来する標準化された神経細胞アッセイ | |
| US9005966B2 (en) | Generation of pluripotent cells from fibroblasts | |
| EP3000877B1 (en) | Neural stem cells | |
| US20180298405A1 (en) | Methods and compositions to increase human somatic cell nuclear transfer (scnt) efficiency by removing histone h3-lysine trimethylation, and derivation of human nt-esc | |
| EP2558571A2 (en) | Sustained polypeptide expression from synthetic, modified rnas and uses thereof | |
| HK1212384A1 (en) | Methods and compositions for producing induced hepatocytes | |
| CN112996906A (zh) | 脑类器官的制造方法 | |
| Parr et al. | An update on stem cell biology and engineering for brain development | |
| CN107002031B (zh) | 选择分化细胞的方法 | |
| CN118475685A (zh) | 神经嵴细胞的培养方法及制造方法 | |
| WO2014114023A1 (zh) | 一种能够大规模并快速高纯度地诱导间充质干细胞转决定成造血干细胞的方法 | |
| WO2013011093A1 (en) | Novel method for generation of neural progenitor cells | |
| KR101133559B1 (ko) | 인간 중배엽줄기세포 유래의 신규 miRNA | |
| Qu et al. | Differentiation of reprogrammed human adipose mesenchymal stem cells toward neural cells with defined transcription factors | |
| WO2015088162A1 (ko) | 줄기세포로부터 연골세포로의 분화 촉진용 조성물 | |
| WO2022138787A1 (ja) | 医薬組成物 | |
| Yao et al. | Generation of induced pluripotent stem cells with high efficiency from human embryonic renal cortical cells | |
| Zheng | Uncover The Genetic Mechanism Governing Pluripotency Maintenance in Stem Cells | |
| JP2023090959A (ja) | 幹細胞の製造方法、及び癌細胞化のリスク低減方法 | |
| Akter | Modeling Movement Disorder DYT1 Dystonia Using Patient-Specific Neurons | |
| Reé | Establishment and characterization of a DGCR8 monoallelic human pluripotent stem cell line | |
| CN120787257A (zh) | 始发态多能干细胞的制造方法 | |
| Zenobi | CHARACTERIZATION OF MOLECULAR MECHANISMS DRIVING EPIGENETIC CONVERSION AND PHENOTYPE SWITCH OF FIBROBLASTS INTO INSULIN SECRETING CELLS | |
| Roessler et al. | GENETIC AND EPIGENETIC FEATURES OF INDUCED PLURIPOTENT STEM CELL DERIVED DOPAMINERGIC NEURONS | |
| TW200951227A (en) | Determination of the biological function of a target gene in a cell |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A621 | Written request for application examination |
Free format text: JAPANESE INTERMEDIATE CODE: A621 Effective date: 20191024 |
|
| A977 | Report on retrieval |
Free format text: JAPANESE INTERMEDIATE CODE: A971007 Effective date: 20200626 |
|
| A131 | Notification of reasons for refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A131 Effective date: 20200716 |
|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20201013 |
|
| RD04 | Notification of resignation of power of attorney |
Free format text: JAPANESE INTERMEDIATE CODE: A7424 Effective date: 20210309 |
|
| A02 | Decision of refusal |
Free format text: JAPANESE INTERMEDIATE CODE: A02 Effective date: 20210310 |