PE20181157A1 - Oligonucleotidos para inducir la expresion paterna de ube3a - Google Patents

Oligonucleotidos para inducir la expresion paterna de ube3a

Info

Publication number
PE20181157A1
PE20181157A1 PE2018000752A PE2018000752A PE20181157A1 PE 20181157 A1 PE20181157 A1 PE 20181157A1 PE 2018000752 A PE2018000752 A PE 2018000752A PE 2018000752 A PE2018000752 A PE 2018000752A PE 20181157 A1 PE20181157 A1 PE 20181157A1
Authority
PE
Peru
Prior art keywords
ube3a
oligonucleotides
induce
expression
relates
Prior art date
Application number
PE2018000752A
Other languages
English (en)
Spanish (es)
Inventor
Veronica Costa
Maj Hedtjarn
Marius Hoener
Ravi Jagasia
Mads Aaboe Jensen
Christoph Patsch
Lykke Pedersen
Søren Vestergaard Rasmussen
Original Assignee
Hoffmann La Roche
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=57345910&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PE20181157(A1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of PE20181157A1 publication Critical patent/PE20181157A1/es

Links

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)
PE2018000752A 2015-11-12 2016-11-11 Oligonucleotidos para inducir la expresion paterna de ube3a PE20181157A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP15194367 2015-11-12
EP16189502 2016-09-19

Publications (1)

Publication Number Publication Date
PE20181157A1 true PE20181157A1 (es) 2018-07-19

Family

ID=57345910

Family Applications (2)

Application Number Title Priority Date Filing Date
PE2018000752A PE20181157A1 (es) 2015-11-12 2016-11-11 Oligonucleotidos para inducir la expresion paterna de ube3a
PE2020000810A PE20210451A1 (es) 2015-11-12 2016-11-11 Oligonucleotidos para inducir la expresion paterna de ube3a

Family Applications After (1)

Application Number Title Priority Date Filing Date
PE2020000810A PE20210451A1 (es) 2015-11-12 2016-11-11 Oligonucleotidos para inducir la expresion paterna de ube3a

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)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
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
EP3911957A1 (en) 2019-01-17 2021-11-24 F. Hoffmann-La Roche AG E3 ubiquitin ligase (ube3a) protein targets
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

Family Cites Families (59)

* Cited by examiner, † Cited by third party
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
WO2015143342A1 (en) 2014-03-21 2015-09-24 Cellular Dynamics International, Inc. Production of midbrain dopaminergic neurons and methods for the use thereof
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

Also Published As

Publication number Publication date
AU2023200410A1 (en) 2023-02-23
CL2020000889A1 (es) 2020-08-28
EP4220360A3 (en) 2023-08-23
JP7066609B2 (ja) 2022-05-13
CA3004799A1 (en) 2017-05-18
US20190310244A1 (en) 2019-10-10
US20250116656A2 (en) 2025-04-10
UA125963C2 (uk) 2022-07-20
US10718753B2 (en) 2020-07-21
KR102840652B1 (ko) 2025-08-04
IL281211B2 (en) 2024-01-01
US20230296587A1 (en) 2023-09-21
IL258488A (en) 2018-05-31
NZ741585A (en) 2024-01-26
CN116064539A (zh) 2023-05-05
HRP20210227T1 (hr) 2021-03-19
SI3374509T1 (sl) 2021-04-30
SG11201803951QA (en) 2018-06-28
AU2022211910A1 (en) 2022-11-10
US20200057052A1 (en) 2020-02-20
LT3374509T (lt) 2021-03-10
JP2018533954A (ja) 2018-11-22
ES2852998T4 (es) 2024-04-25
IL300119A (en) 2023-03-01
US20190144824A1 (en) 2019-05-16
AU2022211910B2 (en) 2024-06-13
EP3798307A1 (en) 2021-03-31
IL281211A (en) 2021-04-29
CL2018001189A1 (es) 2018-08-31
BR112018007633A2 (pt) 2018-11-06
HK1258309A1 (zh) 2019-11-08
US11852627B2 (en) 2023-12-26
DK3374509T3 (da) 2021-02-15
JP2018533362A (ja) 2018-11-15
WO2017081254A1 (en) 2017-05-18
ES2852998T3 (es) 2021-09-14
PE20210451A1 (es) 2021-03-08
KR102185465B1 (ko) 2020-12-03
KR20180070611A (ko) 2018-06-26
AU2016352836B2 (en) 2020-06-25
MX2022016381A (es) 2023-01-30
JP2025024060A (ja) 2025-02-19
PL3374509T3 (pl) 2021-05-04
HRP20241138T1 (hr) 2024-11-22
EP4220360A2 (en) 2023-08-02
WO2017081223A1 (en) 2017-05-18
CR20180264A (es) 2018-08-06
EP3374509A1 (en) 2018-09-19
RU2018119315A (ru) 2019-12-13
RS61529B1 (sr) 2021-04-29
CN108291226B (zh) 2022-11-04
CA3004799C (en) 2024-05-14
MX2018004978A (es) 2018-07-06
RU2742007C2 (ru) 2021-02-01
US20240085402A1 (en) 2024-03-14
CN116064540A (zh) 2023-05-05
JP2021003113A (ja) 2021-01-14
PL4220360T3 (pl) 2024-12-16
AU2020203573B2 (en) 2022-07-07
FI4220360T3 (fi) 2024-08-27
ZA201802494B (en) 2024-01-31
CN108350431A (zh) 2018-07-31
EP3374498A1 (en) 2018-09-19
KR20230008233A (ko) 2023-01-13
EP3374509B1 (en) 2020-12-16
RS66036B1 (sr) 2024-11-29
EP4220360B9 (en) 2024-10-16
CO2018004550A2 (es) 2018-11-22
CN108291226A (zh) 2018-07-17
RU2018119315A3 (enExample) 2020-03-26
ES2991800T3 (es) 2024-12-04
JP7279000B2 (ja) 2023-05-22
JP2021191262A (ja) 2021-12-16
DK4220360T3 (da) 2024-09-16
EP4220360B1 (en) 2024-06-26
IL281211B1 (en) 2023-09-01
US20190154664A1 (en) 2019-05-23
SI4220360T1 (sl) 2024-10-30
LT4220360T (lt) 2024-09-10
US11320421B2 (en) 2022-05-03
WO2017081250A1 (en) 2017-05-18
JP2023052630A (ja) 2023-04-11
US10494633B2 (en) 2019-12-03
RS61529B9 (sr) 2024-04-30
JP6751142B2 (ja) 2020-09-02
AU2023200410B2 (en) 2024-10-31
US20200348286A1 (en) 2020-11-05
US20170191064A1 (en) 2017-07-06
PH12018501005A1 (en) 2019-01-28
HK1253325A1 (zh) 2019-06-14
AU2016352836A1 (en) 2018-04-26
KR20200137027A (ko) 2020-12-08
MA67580B1 (fr) 2024-09-30
US10739332B2 (en) 2020-08-11
HUE067905T2 (hu) 2024-11-28
CN108350432A (zh) 2018-07-31
HK1258434A1 (zh) 2019-11-08
IL258488B (en) 2021-03-25
US20220146496A1 (en) 2022-05-12
EP3374499A1 (en) 2018-09-19
JP2019500851A (ja) 2019-01-17
AU2020203573A1 (en) 2020-06-18
CL2021002159A1 (es) 2022-03-11
US12259380B2 (en) 2025-03-25
HUE053197T2 (hu) 2021-06-28
MX2022016377A (es) 2023-01-30
KR102482440B1 (ko) 2022-12-29
MX2022016376A (es) 2023-01-30
KR20250121139A (ko) 2025-08-11
CR20220077A (es) 2023-04-11
CR20200118A (es) 2021-04-21

Similar Documents

Publication Publication Date Title
PE20210451A1 (es) Oligonucleotidos para inducir la expresion paterna de ube3a
CL2020001344A1 (es) Tratamiento antisentido del síndrome de angelman.
BR112017024384A2 (pt) bactérias modificadas para reduzir hiperfenilalaninemia
EA201690881A1 (ru) Соединения-ингибиторы аутотаксина
MX2016001587A (es) Inhibidores de kdm1a para el tratamiento de enfermedades.
CO6571889A2 (es) Antidotos anticoagulantes
CO2018001840A2 (es) Composiciones biofarmacéuticas que comprenden anticuerpos anti-il-5
TN2018000351A1 (en) Oxaborole esters and uses thereof
BR112017004197A2 (pt) composições de vacina contra doenças do vírus da dengue
NI201600070A (es) Inhibidores tetracíclicos de autotaxina
CL2018000451A1 (es) Composiciones de micropartículas que comprenden saflufenacil
BR112018004310A2 (pt) derivados de espinosina e método de preparação
MX2017014206A (es) Introgresion de un rendimieno qtl en plantas de cucumis sativus.
MX2022001772A (es) Formulaciones de isotretinoina y usos y metodos de las mismas.
BR112018001286A2 (pt) ceifeira autopropulsada com acesso dianteiro ao aparelho de processamento de cultura
BR112018069498A2 (pt) composições e métodos para repelir e matar carrapatos e desprender carrapatos em alimentação
TH171892A (th) สารประกอบมีฤทธิ์ทางการรักษาโรคและวิธีการใช้สารเหล่านั้น
TH1701003244A (th) อนุพันธ์พิเพอริดีนในฐานะเป็นตัวยับยั้ง hdac1/2
TH164180A (th) กระบวนการสำหรับการเตรียมของเอสเทอร์จากกรดซัคซินิค
TH1701003135A (th) องค์ประกอบและวิธีการที่ประกอบรวมด้วยไตรกลีเซอร์ไรด์โซ่ปานกลางสำหรับการบำบัดรักษาโรคลมชัก
TH181825A (th) อนุพันธ์ไดอะซาคาร์บาโซลในรูป tau-pet-ลิแกนด์
TH1601003574A (th) ลิปิดซึ่งประกอบรวมด้วยกรดไขมันไม่อิ่มตัวหลายตำแหน่งชนิดสายยาว