UA125963C2 - Олігонуклеотид для індукції батьківської експресії ube3a - Google Patents

Олігонуклеотид для індукції батьківської експресії ube3a Download PDF

Info

Publication number
UA125963C2
UA125963C2 UAA201806476A UAA201806476A UA125963C2 UA 125963 C2 UA125963 C2 UA 125963C2 UA A201806476 A UAA201806476 A UA A201806476A UA A201806476 A UAA201806476 A UA A201806476A UA 125963 C2 UA125963 C2 UA 125963C2
Authority
UA
Ukraine
Prior art keywords
oligonucleotide
nucleosides
nucleic acid
modified
group
Prior art date
Application number
UAA201806476A
Other languages
English (en)
Ukrainian (uk)
Inventor
Вєроніка Коста
Вероника Коста
Май Хедтьорн
Май Хедтёрн
Маріус Хьонер
Мариус Хёнер
Раві Джагасіа
Рави ДЖАГАСИА
Мадс Аабое Єнсен
Мадс Аабое Енсен
Крістоф Пач
Кристоф ПАЧ
Люккє Педерсен
Люкке ПЕДЕРСЕН
Сьорен Вестергорд Расмуссен
Original Assignee
Ф. Хоффманн-Ля Рош Аг
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=UA125963(C2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ф. Хоффманн-Ля Рош Аг filed Critical Ф. Хоффманн-Ля Рош Аг
Publication of UA125963C2 publication Critical patent/UA125963C2/uk

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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Food Science & Technology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Developmental Biology & Embryology (AREA)
UAA201806476A 2015-11-12 2016-11-11 Олігонуклеотид для індукції батьківської експресії ube3a UA125963C2 (uk)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP15194367 2015-11-12
EP16189502 2016-09-19
PCT/EP2016/077383 WO2017081223A1 (en) 2015-11-12 2016-11-11 Oligonucleotides for inducing paternal ube3a expression

Publications (1)

Publication Number Publication Date
UA125963C2 true UA125963C2 (uk) 2022-07-20

Family

ID=57345910

Family Applications (1)

Application Number Title Priority Date Filing Date
UAA201806476A UA125963C2 (uk) 2015-11-12 2016-11-11 Олігонуклеотид для індукції батьківської експресії ube3a

Country Status (30)

Country Link
US (9) US10494633B2 (enExample)
EP (5) EP4220360B9 (enExample)
JP (7) JP2018533362A (enExample)
KR (4) KR102482440B1 (enExample)
CN (5) CN116064540A (enExample)
AU (4) AU2016352836B2 (enExample)
CA (1) CA3004799C (enExample)
CL (3) CL2018001189A1 (enExample)
CO (1) CO2018004550A2 (enExample)
CR (3) CR20180264A (enExample)
DK (2) DK3374509T3 (enExample)
ES (2) ES2991800T3 (enExample)
FI (1) FI4220360T3 (enExample)
HK (2) HK1258309A1 (enExample)
HR (2) HRP20241138T1 (enExample)
HU (2) HUE053197T2 (enExample)
IL (3) IL300119A (enExample)
LT (2) LT4220360T (enExample)
MA (1) MA67580B1 (enExample)
MX (4) MX2018004978A (enExample)
PE (2) PE20210451A1 (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
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
EP3626823A1 (en) 2009-09-11 2020-03-25 Ionis Pharmaceuticals, Inc. Modulation of huntingtin expression
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
BR112014001244A2 (pt) 2011-07-19 2017-02-21 Wave Life Sciences Pte Ltd métodos para a síntese de ácidos nucléicos funcionalizados
EP2864479B1 (en) 2012-06-25 2018-08-15 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
ES2862073T3 (es) 2012-07-13 2021-10-06 Wave Life Sciences Ltd Grupo auxiliar asimétrico
CN112007045A (zh) 2012-07-13 2020-12-01 波涛生命科学有限公司 手性控制
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
US10400243B2 (en) 2014-11-25 2019-09-03 Ionis Pharmaceuticals, Inc. Modulation of UBE3A-ATS expression
MA67580B1 (fr) 2015-11-12 2024-09-30 F. Hoffmann-La Roche Ag Oligonucléotides pour induire l'expression de paternal ube3a
AU2018375807B2 (en) * 2017-12-01 2025-04-10 The Texas A&M University System Angelman syndrome antisense treatment
AU2019218987B2 (en) 2018-02-12 2025-04-24 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
AU2019344006B2 (en) 2018-09-21 2025-11-27 University Of Connecticut Compositions and methods to restore paternal UBE3A gene expression in human Angelman Syndrome
CN113316722A (zh) * 2019-01-17 2021-08-27 豪夫迈·罗氏有限公司 E3泛素连接酶(ube3a)蛋白质靶标
CA3130431A1 (en) * 2019-02-20 2020-08-27 Roche Innovation Center Copenhagen A/S Phosphonoacetate gapmer oligonucleotides
CR20210538A (es) 2019-03-29 2021-12-06 Ionis Pharmaceuticals Inc Compuestos y métodos para modular ube3a-ats
EA202193214A1 (ru) 2019-05-22 2022-03-14 Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл Гены ube3a и кассеты экспрессии и их использование
WO2020239714A1 (en) 2019-05-28 2020-12-03 F. Hoffmann-La Roche Ag Method to generate monocytic progenitor cells
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
JP2023529457A (ja) * 2020-06-09 2023-07-10 ロシュ イノベーション センター コペンハーゲン エーエス 治療用ポリヌクレオチドに使用するためのグアノシン類似体
WO2022158487A1 (ja) 2021-01-21 2022-07-28 藤森工業株式会社 積層フィルムおよび包装袋
WO2023217890A1 (en) 2022-05-10 2023-11-16 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting cfp-elk1 intergene region
WO2024227765A2 (en) 2023-05-04 2024-11-07 F. Hoffmann-La Roche Ag Oligonucleotides capable of upregulating glucocerebrosidase expression
WO2025100980A1 (ko) * 2023-11-08 2025-05-15 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
AU2916292A (en) 1991-10-24 1993-05-21 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
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
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6238921B1 (en) 1998-03-26 2001-05-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of human mdm2 expression
AU1853600A (en) * 1999-01-06 2000-07-24 Choong-Chin Liew Method for the detection of gene transcripts in blood and uses thereof
RU2233844C2 (ru) 1999-02-12 2004-08-10 Санкио Компани Лимитед Новые нуклеозидные и олигонуклеотидные аналоги
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US20020098511A1 (en) 1999-12-02 2002-07-25 Karen Heichman Protein-protein interactions
US6300132B1 (en) 1999-12-17 2001-10-09 Isis Pharmaceuticals, Inc. Antisense inhibition of telomeric repeat binding factor 2 expression
WO2001092582A1 (en) * 2000-06-01 2001-12-06 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
EP2270024B1 (en) 2001-06-21 2018-10-24 Ionis 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
EP1578927A2 (en) 2002-08-14 2005-09-28 Pharmacia Corporation Antisense modulation of nav1.3 expression
JP2006500066A (ja) 2002-09-25 2006-01-05 ファルマシア・コーポレーション ミクロソームのプロスタグランジンｅ２シンターゼ発現のアンチセンス調節
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As 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
JP5713377B2 (ja) 2005-12-28 2015-05-07 ザ スクリプス リサーチ インスティテュート 薬物標的としての天然アンチセンスおよび非コードｒｎａ転写物
EP1984381B1 (en) 2006-01-27 2010-09-29 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
WO2007131237A2 (en) 2006-05-05 2007-11-15 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of ptp1b
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'
WO2008113832A2 (en) 2007-03-22 2008-09-25 Santaris Pharma A/S SHORT RNA 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
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US8546556B2 (en) 2007-11-21 2013-10-01 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
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
WO2010036698A1 (en) 2008-09-24 2010-04-01 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
JP5843775B2 (ja) * 2009-10-13 2016-01-13 ステムセル テクノロジーズ インコーポレーティッド 幹細胞を分化させるための重量オスモル濃度の操作法
NO2516648T3 (enExample) 2009-12-23 2018-04-07
US8846637B2 (en) 2010-06-08 2014-09-30 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
AU2011282217B2 (en) 2010-07-19 2015-12-03 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (DMPK) expression
KR101788903B1 (ko) * 2010-08-19 2017-10-23 에프. 호프만-라 로슈 아게 체세포의 유도된 재프로그래밍된 신경 줄기 세포 (ｉｒｎｓｃｓ) 로의 전환
WO2012064806A2 (en) 2010-11-11 2012-05-18 The University Of North Carolina At Chapel Hill Methods and compositions for unsilencing imprinted genes
EP3521451A1 (en) 2010-11-17 2019-08-07 Ionis 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
AU2012244816B2 (en) 2011-04-20 2015-12-10 Roche Glycart Ag Method and constructs for the pH dependent passage of the blood-brain-barrier
EP2751270B1 (en) 2011-08-29 2018-08-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
CN104136451A (zh) 2011-09-07 2014-11-05 玛瑞纳生物技术有限公司 具有构象限制的单体的核酸化合物的合成和用途
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP2864479B1 (en) * 2012-06-25 2018-08-15 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
CA2889044A1 (en) 2012-11-15 2014-05-22 Roche Innovation Center Copenhagen A/S Anti apob antisense conjugate compounds
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
EP3119881B1 (en) * 2014-03-21 2023-03-01 FUJIFILM Cellular Dynamics, Inc. Production of midbrain dopaminergic neurons and methods for the use thereof
MA67580B1 (fr) * 2015-11-12 2024-09-30 F. Hoffmann-La Roche Ag Oligonucléotides pour induire l'expression de paternal ube3a
JP2020528739A (ja) 2017-06-28 2020-10-01 ユニヴァーシティ オブ サウス フロリダ アンジェルマン症候群の遺伝子治療法のための改変ｕｂｅ３ａ遺伝子
AU2018375807B2 (en) 2017-12-01 2025-04-10 The Texas A&M University System Angelman syndrome antisense treatment
KR20200116088A (ko) 2018-01-26 2020-10-08 에프. 호프만-라 로슈 아게 방사성 표지된 올리고뉴클레오티드 및 이의 제조 방법
CR20210538A (es) 2019-03-29 2021-12-06 Ionis Pharmaceuticals Inc Compuestos y métodos para modular ube3a-ats

Also Published As

Publication number Publication date
AU2023200410B2 (en) 2024-10-31
US11852627B2 (en) 2023-12-26
KR20250121139A (ko) 2025-08-11
RU2018119315A3 (enExample) 2020-03-26
DK3374509T3 (da) 2021-02-15
US20170191064A1 (en) 2017-07-06
NZ741585A (en) 2024-01-26
PE20181157A1 (es) 2018-07-19
ES2852998T3 (es) 2021-09-14
CN116064539A (zh) 2023-05-05
CA3004799C (en) 2024-05-14
RU2018119315A (ru) 2019-12-13
IL281211B2 (en) 2024-01-01
US20200348286A1 (en) 2020-11-05
ZA201802494B (en) 2024-01-31
AU2023200410A1 (en) 2023-02-23
US20220146496A1 (en) 2022-05-12
HK1253325A1 (zh) 2019-06-14
EP3374499A1 (en) 2018-09-19
CL2021002159A1 (es) 2022-03-11
RS61529B9 (sr) 2024-04-30
JP7279000B2 (ja) 2023-05-22
PH12018501005A1 (en) 2019-01-28
RU2742007C2 (ru) 2021-02-01
US11320421B2 (en) 2022-05-03
CR20220077A (es) 2023-04-11
KR20180070611A (ko) 2018-06-26
JP2021191262A (ja) 2021-12-16
HRP20210227T1 (hr) 2021-03-19
IL300119A (en) 2023-03-01
FI4220360T3 (fi) 2024-08-27
CR20180264A (es) 2018-08-06
MX2022016377A (es) 2023-01-30
IL258488A (en) 2018-05-31
JP7066609B2 (ja) 2022-05-13
IL258488B (en) 2021-03-25
EP3798307A1 (en) 2021-03-31
KR20200137027A (ko) 2020-12-08
IL281211B1 (en) 2023-09-01
MX2022016376A (es) 2023-01-30
CN108350431A (zh) 2018-07-31
MA67580B1 (fr) 2024-09-30
US20250116656A2 (en) 2025-04-10
IL281211A (en) 2021-04-29
US20200057052A1 (en) 2020-02-20
CO2018004550A2 (es) 2018-11-22
ES2852998T4 (es) 2024-04-25
US20190154664A1 (en) 2019-05-23
HUE053197T2 (hu) 2021-06-28
EP4220360B9 (en) 2024-10-16
PL3374509T3 (pl) 2021-05-04
MX2022016381A (es) 2023-01-30
AU2022211910B2 (en) 2024-06-13
AU2022211910A1 (en) 2022-11-10
HRP20241138T1 (hr) 2024-11-22
US10718753B2 (en) 2020-07-21
JP2019500851A (ja) 2019-01-17
US20240085402A1 (en) 2024-03-14
JP2018533362A (ja) 2018-11-15
RS66036B1 (sr) 2024-11-29
KR20230008233A (ko) 2023-01-13
CR20200118A (es) 2021-04-21
WO2017081250A1 (en) 2017-05-18
KR102482440B1 (ko) 2022-12-29
SG11201803951QA (en) 2018-06-28
US10739332B2 (en) 2020-08-11
RS61529B1 (sr) 2021-04-29
JP2018533954A (ja) 2018-11-22
AU2016352836B2 (en) 2020-06-25
CL2018001189A1 (es) 2018-08-31
US10494633B2 (en) 2019-12-03
SI3374509T1 (sl) 2021-04-30
US20190310244A1 (en) 2019-10-10
DK4220360T3 (da) 2024-09-16
LT4220360T (lt) 2024-09-10
US20230296587A1 (en) 2023-09-21
EP4220360A2 (en) 2023-08-02
HUE067905T2 (hu) 2024-11-28
HK1258434A1 (zh) 2019-11-08
KR102185465B1 (ko) 2020-12-03
KR102840652B1 (ko) 2025-08-04
MX2018004978A (es) 2018-07-06
JP2021003113A (ja) 2021-01-14
CN108350432A (zh) 2018-07-31
ES2991800T3 (es) 2024-12-04
AU2016352836A1 (en) 2018-04-26
CN108291226B (zh) 2022-11-04
CN116064540A (zh) 2023-05-05
US12259380B2 (en) 2025-03-25
EP4220360A3 (en) 2023-08-23
EP3374509A1 (en) 2018-09-19
PL4220360T3 (pl) 2024-12-16
JP2023052630A (ja) 2023-04-11
CN108291226A (zh) 2018-07-17
LT3374509T (lt) 2021-03-10
EP3374509B1 (en) 2020-12-16
JP2025024060A (ja) 2025-02-19
AU2020203573A1 (en) 2020-06-18
HK1258309A1 (zh) 2019-11-08
CL2020000889A1 (es) 2020-08-28
WO2017081254A1 (en) 2017-05-18
EP3374498A1 (en) 2018-09-19
AU2020203573B2 (en) 2022-07-07
BR112018007633A2 (pt) 2018-11-06
PE20210451A1 (es) 2021-03-08
JP6751142B2 (ja) 2020-09-02
SI4220360T1 (sl) 2024-10-30
EP4220360B1 (en) 2024-06-26
US20190144824A1 (en) 2019-05-16
WO2017081223A1 (en) 2017-05-18
CA3004799A1 (en) 2017-05-18

Similar Documents

Publication Publication Date Title
UA125963C2 (uk) Олігонуклеотид для індукції батьківської експресії ube3a
JP7002603B2 (ja) Ｐｄ－ｌ１発現低減用のオリゴヌクレオチド
JP7379387B2 (ja) Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
CN111433361B (zh) 天使综合征反义治疗
US11485975B2 (en) Oligonucleotides for modulating TMEM106B expression
CN113785060A (zh) 用于调节atxn2表达的寡核苷酸
WO2018130584A1 (en) Antisense oligonucleotides for modulating nfkb2 expression
US20210214727A1 (en) Enhanced oligonucleotides for inhibiting scn9a expression
US20200216845A1 (en) Antisense oligonucleotides for modulating rela expression
US20190367920A1 (en) Antisense oligonucleotides for modulating nfkb1 expression
HK40090602A (zh) 用於诱导父本ube3a表达的寡核苷酸
HK40090600A (zh) 用於诱导父本ube3a表达的寡核苷酸
HK40060810A (en) Oligonucleotides for modulating atxn2 expression
US20190338286A1 (en) Antisense oligonucleotides for modulating rel expression
HK40043583A (en) Oligonucleotides for modulating atxn2 expression
EA046118B1 (ru) Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения
HK1253325B (en) Oligonucleotides for inducing paternal ube3a expression
HK1262526A1 (en) Antisense oligonucleotides for modulating htra1 expression
BR112018007633B1 (pt) Oligonucleotídeos para indução de expressão de ube3a paterna
WO2019038228A1 (en) OLIGONUCLEOTIDES FOR MODULATION OF TOM1 EXPRESSION
BR122021008010B1 (pt) Oligonucleotídeos para indução de expressão de ube3a paterna,conjugado, composição farmacêutica, uso dos mesmos e método para induçãode expressão de ube3a em uma célula alvo onde expressão de ube3a paterna ésuprimida