CL2021002159A1 - Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud no. 201801189) - Google Patents

Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud no. 201801189)

Info

Publication number
CL2021002159A1
CL2021002159A1 CL2021002159A CL2021002159A CL2021002159A1 CL 2021002159 A1 CL2021002159 A1 CL 2021002159A1 CL 2021002159 A CL2021002159 A CL 2021002159A CL 2021002159 A CL2021002159 A CL 2021002159A CL 2021002159 A1 CL2021002159 A1 CL 2021002159A1
Authority
CL
Chile
Prior art keywords
ube3a
oligonucleotides
induce
divisional application
expression
Prior art date
Application number
CL2021002159A
Other languages
English (en)
Inventor
Veronica Costa
Maj Hedtjärn
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
Application filed by Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of CL2021002159A1 publication Critical patent/CL2021002159A1/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 [CRISPRs]
    • 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)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Hematology (AREA)
  • Urology & Nephrology (AREA)
  • Toxicology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Food Science & Technology (AREA)
  • Analytical Chemistry (AREA)
  • Developmental Biology & Embryology (AREA)

Abstract

La presente invención se refiere a oligonucleótidos que son capaces de inducir la expresión de la ubiquitín-proteína ligasa E3A (UBE3A) a partir del alelo parterno en neuronas animales o humanas. Los oligonucleótidos con diana en el supresor del alelo paterno de UBE3A mediante hibridación con el ARN no codificante largo de SNHG14 cadena abajo de SNORD109B. Asimismo, la presente invención se refiere a composiciones y métodos farmacéuticos para el tratamiento del síndrome de Angelman.
CL2021002159A 2015-11-12 2021-08-13 Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud no. 201801189) CL2021002159A1 (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP15194367 2015-11-12
EP16189502 2016-09-19
CL2018001189A CL2018001189A1 (es) 2015-11-12 2018-05-03 Oligonucleótidos para inducir la expresión paterna de ube3a
CL2020000889A CL2020000889A1 (es) 2015-11-12 2020-04-02 Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud 201801189)

Publications (1)

Publication Number Publication Date
CL2021002159A1 true CL2021002159A1 (es) 2022-03-11

Family

ID=57345910

Family Applications (3)

Application Number Title Priority Date Filing Date
CL2018001189A CL2018001189A1 (es) 2015-11-12 2018-05-03 Oligonucleótidos para inducir la expresión paterna de ube3a
CL2020000889A CL2020000889A1 (es) 2015-11-12 2020-04-02 Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud 201801189)
CL2021002159A CL2021002159A1 (es) 2015-11-12 2021-08-13 Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud no. 201801189)

Family Applications Before (2)

Application Number Title Priority Date Filing Date
CL2018001189A CL2018001189A1 (es) 2015-11-12 2018-05-03 Oligonucleótidos para inducir la expresión paterna de ube3a
CL2020000889A CL2020000889A1 (es) 2015-11-12 2020-04-02 Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud 201801189)

Country Status (29)

Country Link
US (9) US10494633B2 (es)
EP (5) EP3374509B1 (es)
JP (6) JP6751142B2 (es)
KR (3) KR102482440B1 (es)
CN (5) CN108350431A (es)
AU (4) AU2016352836B2 (es)
BR (1) BR112018007633A2 (es)
CA (1) CA3004799C (es)
CL (3) CL2018001189A1 (es)
CO (1) CO2018004550A2 (es)
CR (3) CR20220077A (es)
DK (1) DK3374509T3 (es)
ES (1) ES2852998T4 (es)
HK (3) HK1253325A1 (es)
HR (1) HRP20210227T1 (es)
HU (1) HUE053197T2 (es)
IL (3) IL281211B2 (es)
LT (1) LT3374509T (es)
MX (4) MX2018004978A (es)
PE (2) PE20181157A1 (es)
PH (1) PH12018501005A1 (es)
PL (1) PL3374509T3 (es)
RS (1) RS61529B9 (es)
RU (1) RU2742007C2 (es)
SG (1) SG11201803951QA (es)
SI (1) SI3374509T1 (es)
UA (1) UA125963C2 (es)
WO (3) WO2017081223A1 (es)
ZA (1) ZA201802494B (es)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11852627B2 (en) 2015-11-12 2023-12-26 Hoffmann-La Roche Inc. Oligonucleotides for inducing paternal UBE3A expression

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
AU2010270714B2 (en) 2009-07-06 2015-08-13 Wave Life Sciences Ltd. Novel nucleic acid prodrugs and methods use thereof
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
CA2879023C (en) 2012-07-13 2017-03-28 Wave Life Sciences Japan Asymmetric auxiliary group
CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
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
KR20230152178A (ko) 2014-01-16 2023-11-02 웨이브 라이프 사이언시스 리미티드 키랄 디자인
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
SG11202003596WA (en) * 2017-12-01 2020-05-28 Texas A & M Univ Sys Angelman syndrome antisense treatment
JP7317029B2 (ja) 2018-02-12 2023-07-28 アイオーニス ファーマシューティカルズ， インコーポレーテッド 修飾化合物及びその使用
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
MX2021003303A (es) * 2018-09-21 2021-06-23 Univ Connecticut Composiciones y metodos para restaurar la expresion del gen ube3a paterno en sindrome de angelman humano.
US20220091137A1 (en) 2019-01-17 2022-03-24 Hoffmann-La Roche Inc. E3 ubiquitin ligase (ube3a) protein targets
JP2022527105A (ja) * 2019-03-29 2022-05-30 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｕｂｅ３ａ－ａｔｓを調節するための化合物及び方法
CA3139678A1 (en) * 2019-05-22 2020-11-26 The University Of North Carolina At Chapel Hill Ube3a genes and expression cassettes and their use
CN113874494A (zh) 2019-05-28 2021-12-31 豪夫迈·罗氏有限公司 产生单核细胞祖细胞的方法
WO2021249993A1 (en) * 2020-06-09 2021-12-16 Roche Innovation Center Copenhagen A/S Guanosine analogues for use in therapeutic polynucleotides
EP4282651A1 (en) 2021-01-21 2023-11-29 Fujimori Kogyo Co., Ltd. Layered film and packaging bag
WO2023217890A1 (en) 2022-05-10 2023-11-16 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting cfp-elk1 intergene region

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
ATE239484T1 (de) 1991-10-24 2003-05-15 Isis Pharmaceuticals Inc Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen
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
EP2253639A1 (en) 1997-09-12 2010-11-24 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6238921B1 (en) 1998-03-26 2001-05-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of human mdm2 expression
CA2359816C (en) * 1999-01-06 2010-08-03 Genenews Inc. Method for the detection of gene transcripts in blood and uses thereof
ID30093A (id) 1999-02-12 2001-11-01 Sankyo Co Analog-analog nukleosida dan oligonukleotida baru
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
CA2396460A1 (en) 1999-12-02 2001-06-07 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
WO2003000707A2 (en) * 2001-06-21 2003-01-03 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
BR0313455A (pt) 2002-08-14 2007-07-31 Pharmacia Corp modulação "antisense" da expressão de nav1.3
AU2003270900A1 (en) * 2002-09-25 2004-04-19 Pharmacia Corporation Antisense modulation of microsomal prostaglandin e2 synthase expression
EP2752488B1 (en) 2002-11-18 2020-02-12 Roche Innovation Center Copenhagen A/S Antisense design
GB2421029B (en) 2003-08-29 2008-04-09 Wisconsin Alumni Res Found Method of in vitro differentiation of neural stem cells, motor neurons and dopamine neurons from primate embryonic stem cells
WO2007031091A2 (en) 2005-09-15 2007-03-22 Santaris Pharma A/S Rna antagonist compounds for the modulation of p21 ras expression
WO2007087113A2 (en) 2005-12-28 2007-08-02 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
AU2007211080B9 (en) 2006-01-27 2012-05-03 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
ES2386578T3 (es) 2006-05-05 2012-08-23 Isis Pharmaceuticals, Inc. Compuestos y procedimientos para modular la expresión de PCSK9
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
ES2389737T3 (es) 2006-05-11 2012-10-31 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en 5'
DK2149605T3 (da) 2007-03-22 2013-09-30 Santaris Pharma As Korte RNA antagonist forbindelser til modulering af det ønskede 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
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
DK2176280T4 (en) 2007-07-05 2015-07-20 Isis Pharmaceuticals Inc 6-Disubstituerede bicykliske nukleinsyreanaloge
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
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
US8642334B2 (en) * 2009-02-17 2014-02-04 Memorial Sloan Kettering Cancer Center Methods of neural conversion of human embryonic stem cells
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
AU2010306377B2 (en) 2009-10-13 2015-01-22 Stemcell Technologies Canada Inc. Manipulation of osmolality for differentiating stem cells
CN102869776B (zh) 2009-12-23 2017-06-23 库尔纳公司 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病
EP2580228B1 (en) 2010-06-08 2016-03-23 Ionis 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
CN106434648A (zh) 2010-07-19 2017-02-22 F·C·贝内特 肌强直性营养障碍蛋白激酶(dmpk)表达的调节
EP2606126B1 (en) * 2010-08-19 2016-05-18 F.Hoffmann-La Roche Ag Conversion of somatic cells to induced reprogrammed neural stem cells (irnscs)
WO2012064806A2 (en) 2010-11-11 2012-05-18 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
DK2694644T3 (en) 2011-03-30 2018-04-16 Cellular Dynamics Int Inc Priming of pluripotent stem cells for neural differentiation
BR112013026423A2 (pt) 2011-04-20 2016-11-29 Roche Glycart Ag método e construtos para a passagem de pendente do ph da barreira sangue-cérebro
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
SG11201401314PA (en) 2011-09-07 2014-09-26 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
SI3461895T1 (sl) 2012-06-25 2020-10-30 Ionis Pharmaceuticals, Inc. Modulacija ekspresije UBE3A-ATS
CN104884618A (zh) 2012-11-15 2015-09-02 罗氏创新中心哥本哈根有限公司 寡核苷酸缀合物
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
US9694036B2 (en) 2014-03-21 2017-07-04 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
BR112019027692A2 (pt) 2017-06-28 2020-11-24 University Of South Florida gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman
SG11202003596WA (en) 2017-12-01 2020-05-28 Texas A & M Univ Sys Angelman syndrome antisense treatment
KR20200116088A (ko) 2018-01-26 2020-10-08 에프. 호프만-라 로슈 아게 방사성 표지된 올리고뉴클레오티드 및 이의 제조 방법
JP2022527105A (ja) 2019-03-29 2022-05-30 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｕｂｅ３ａ－ａｔｓを調節するための化合物及び方法

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11852627B2 (en) 2015-11-12 2023-12-26 Hoffmann-La Roche Inc. Oligonucleotides for inducing paternal UBE3A expression

Also Published As

Publication number Publication date
US10718753B2 (en) 2020-07-21
US11320421B2 (en) 2022-05-03
JP2021191262A (ja) 2021-12-16
RU2018119315A (ru) 2019-12-13
AU2020203573B2 (en) 2022-07-07
PL3374509T3 (pl) 2021-05-04
JP6751142B2 (ja) 2020-09-02
JP2023052630A (ja) 2023-04-11
JP2019500851A (ja) 2019-01-17
ES2852998T3 (es) 2021-09-14
KR20230008233A (ko) 2023-01-13
CN108291226B (zh) 2022-11-04
US10739332B2 (en) 2020-08-11
LT3374509T (lt) 2021-03-10
HRP20210227T1 (hr) 2021-03-19
US20190144824A1 (en) 2019-05-16
DK3374509T3 (da) 2021-02-15
IL281211B2 (en) 2024-01-01
UA125963C2 (uk) 2022-07-20
JP2018533954A (ja) 2018-11-22
CR20220077A (es) 2023-04-11
AU2023200410A1 (en) 2023-02-23
US10494633B2 (en) 2019-12-03
EP3374509A1 (en) 2018-09-19
PH12018501005A1 (en) 2019-01-28
WO2017081254A1 (en) 2017-05-18
CN116064539A (zh) 2023-05-05
EP4220360A3 (en) 2023-08-23
CR20180264A (es) 2018-08-06
IL258488A (en) 2018-05-31
WO2017081223A1 (en) 2017-05-18
CN108350432A (zh) 2018-07-31
KR20180070611A (ko) 2018-06-26
HK1253325A1 (zh) 2019-06-14
CO2018004550A2 (es) 2018-11-22
HUE053197T2 (hu) 2021-06-28
US20200057052A1 (en) 2020-02-20
EP3374498A1 (en) 2018-09-19
CL2020000889A1 (es) 2020-08-28
US20240085402A1 (en) 2024-03-14
MX2022016377A (es) 2023-01-30
US20230296587A1 (en) 2023-09-21
JP7279000B2 (ja) 2023-05-22
SI3374509T1 (sl) 2021-04-30
US20170191064A1 (en) 2017-07-06
EP4220360B1 (en) 2024-06-26
RS61529B9 (sr) 2024-04-30
IL300119A (en) 2023-03-01
JP7066609B2 (ja) 2022-05-13
CN116064540A (zh) 2023-05-05
HK1258434A1 (zh) 2019-11-08
AU2022211910A1 (en) 2022-11-10
US20200348286A1 (en) 2020-11-05
PE20210451A1 (es) 2021-03-08
JP2018533362A (ja) 2018-11-15
US20190154664A1 (en) 2019-05-23
AU2016352836A1 (en) 2018-04-26
AU2016352836B2 (en) 2020-06-25
NZ741585A (en) 2024-01-26
PE20181157A1 (es) 2018-07-19
IL258488B (en) 2021-03-25
IL281211B1 (en) 2023-09-01
ES2852998T4 (es) 2024-04-25
EP3374509B1 (en) 2020-12-16
CA3004799A1 (en) 2017-05-18
AU2020203573A1 (en) 2020-06-18
WO2017081250A1 (en) 2017-05-18
RU2742007C2 (ru) 2021-02-01
ZA201802494B (en) 2024-01-31
US11852627B2 (en) 2023-12-26
CL2018001189A1 (es) 2018-08-31
KR102482440B1 (ko) 2022-12-29
EP3374499A1 (en) 2018-09-19
EP3798307A1 (en) 2021-03-31
EP4220360A2 (en) 2023-08-02
RS61529B1 (sr) 2021-04-29
MX2022016376A (es) 2023-01-30
CA3004799C (en) 2024-05-14
RU2018119315A3 (es) 2020-03-26
CN108291226A (zh) 2018-07-17
MX2018004978A (es) 2018-07-06
HK1258309A1 (zh) 2019-11-08
IL281211A (en) 2021-04-29
KR102185465B1 (ko) 2020-12-03
CN108350431A (zh) 2018-07-31
US20190310244A1 (en) 2019-10-10
BR112018007633A2 (pt) 2018-11-06
CR20200118A (es) 2021-04-21
KR20200137027A (ko) 2020-12-08
JP2021003113A (ja) 2021-01-14
US20220146496A1 (en) 2022-05-12
AU2022211910B2 (en) 2024-06-13
MX2022016381A (es) 2023-01-30
SG11201803951QA (en) 2018-06-28

Similar Documents

Publication Publication Date Title
CL2021002159A1 (es) Oligonucleótidos para inducir la expresión paterna de ube3a (divisional solicitud no. 201801189)
CY1122329T1 (el) Anti-siglec-8 αντισωματα και μeθοδοι χρhσεως αυτων
NI201600071A (es) Compuestos de inhibidor de autotaxina
BR112017011923A2 (pt) bactéria modificada para tratar doenças associadas com hiperamonemia
CL2020001344A1 (es) Tratamiento antisentido del síndrome de angelman.
MX2018015410A (es) Esteres de oxaborol y sus usos.
PH12017501370A1 (en) Bivalent swine influenza virus vaccine
CL2018000451A1 (es) Composiciones de micropartículas que comprenden saflufenacil
MX370628B (es) Composiciones y metodos para tratar las espinillas.
PH12017500304A1 (en) Vaccine compositions against dengue virus diseases
NI201600070A (es) Inhibidores tetracíclicos de autotaxina
CO2017007076A2 (es) Compuestos de indenilo, composiciones farmacéuticas y usos médicos del mismo
CR20170157A (es) Derivados de nucleósidos sustituidos con 4´-vinilo como inhibidores de la replicación del arn del virus respiratorio sincitial
BR112017028224A2 (pt) composição, e, método para proteger um leitão contra uma doença associada a pestivírus.
BR112015023260A2 (pt) método de tratamento de grão com ozônio
BR112018001286A2 (pt) ceifeira autopropulsada com acesso dianteiro ao aparelho de processamento de cultura
BR112018073300A2 (pt) métodos para tratamento de doenças associadas com as células ilc3
TH1701003135A (th) องค์ประกอบและวิธีการที่ประกอบรวมด้วยไตรกลีเซอร์ไรด์โซ่ปานกลางสำหรับการบำบัดรักษาโรคลมชัก
TH178097A (es)
TH164752A (th) กระบวนการสำหรับการเตรียมของเอสเทอร์จากกรดซัคซินิค
TH164180A (th) กระบวนการสำหรับการเตรียมของเอสเทอร์จากกรดซัคซินิค