KR20200119782A - 발작 장애 치료 시 mir101 또는 mir128의 용도 - Google Patents

발작 장애 치료 시 mir101 또는 mir128의 용도 Download PDF

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Publication number
KR20200119782A
KR20200119782A KR1020207019430A KR20207019430A KR20200119782A KR 20200119782 A KR20200119782 A KR 20200119782A KR 1020207019430 A KR1020207019430 A KR 1020207019430A KR 20207019430 A KR20207019430 A KR 20207019430A KR 20200119782 A KR20200119782 A KR 20200119782A
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South Korea
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vector
epilepsy
seizure
patient
promoter
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KR1020207019430A
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English (en)
Korean (ko)
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매튜 듀링
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오비드 테라퓨틱스 인크.
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Publication of KR20200119782A publication Critical patent/KR20200119782A/ko
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00—Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0047—Sonopheresis, i.e. ultrasonically-enhanced transdermal delivery, electroporation of a pharmacologically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Mycology (AREA)
  • Dispersion Chemistry (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020207019430A 2017-12-06 2018-12-06 발작 장애 치료 시 mir101 또는 mir128의 용도 Withdrawn KR20200119782A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762595255P 2017-12-06 2017-12-06
US62/595,255 2017-12-06
PCT/US2018/064158 WO2019113266A1 (en) 2017-12-06 2018-12-06 Use of mir101 or mir128 in the treatment of seizure disorders

Publications (1)

Publication Number Publication Date
KR20200119782A true KR20200119782A (ko) 2020-10-20

Family

ID=66658874

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207019430A Withdrawn KR20200119782A (ko) 2017-12-06 2018-12-06 발작 장애 치료 시 mir101 또는 mir128의 용도

Country Status (10)

Country Link
US (2) US10870855B2 (https=)
EP (1) EP3703820A4 (https=)
JP (1) JP2021505619A (https=)
KR (1) KR20200119782A (https=)
CN (1) CN111417397A (https=)
AU (1) AU2018378622A1 (https=)
CA (1) CA3084985A1 (https=)
IL (1) IL274838A (https=)
MX (2) MX388998B (https=)
WO (1) WO2019113266A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3084985A1 (en) 2017-12-06 2019-06-13 Ovid Therapeutics Inc. Use of mir101 or mir128 in the treatment of seizure disorders
BR112022002794A2 (pt) * 2019-08-15 2022-08-09 Childrens Hospital Philadelphia Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
WO2025164317A1 (ja) * 2024-01-31 2025-08-07 国立大学法人群馬大学 改良型ミクログリア標的ａａｖベクター

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US4315514A (en) 1980-05-08 1982-02-16 William Drewes Method and apparatus for selective cell destruction
US4923437A (en) 1986-07-18 1990-05-08 Gordon Robert T Process for applying a localized magnetic or electric field
US5059415A (en) 1989-02-21 1991-10-22 The State Of Oregon Acting By And Through The Oregon State Board Of Higher Education On Behalf Of Oregon Health Method for diagnostically imaging lesions in the brain inside a blood-brain barrier
US5112596A (en) 1990-04-23 1992-05-12 Alkermes, Inc. Method for increasing blood-brain barrier permeability by administering a bradykinin agonist of blood-brain barrier permeability
US5438989A (en) 1990-08-10 1995-08-08 Hochman; Darryl Solid tumor, cortical function, and nerve tissue imaging methods and device
US5465718A (en) 1990-08-10 1995-11-14 Hochman; Daryl Solid tumor, cortical function, and nerve tissue imaging methods and device
DE4227800C2 (de) 1991-08-21 1996-12-19 Toshiba Kawasaki Kk Thrombuslösende Behandlungsvorrichtung
US5291890A (en) 1991-08-29 1994-03-08 General Electric Company Magnetic resonance surgery using heat waves produced with focussed ultrasound
US5524620A (en) 1991-11-12 1996-06-11 November Technologies Ltd. Ablation of blood thrombi by means of acoustic energy
JP3325300B2 (ja) 1992-02-28 2002-09-17 株式会社東芝 超音波治療装置
US5368032A (en) 1993-11-09 1994-11-29 General Electric Company Manually positioned focussed energy system guided by medical imaging
US5526814A (en) 1993-11-09 1996-06-18 General Electric Company Automatically positioned focussed energy system guided by medical imaging
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US5752515A (en) 1996-08-21 1998-05-19 Brigham & Women's Hospital Methods and apparatus for image-guided ultrasound delivery of compounds through the blood-brain barrier
EP1937151A4 (en) 2005-09-19 2011-07-06 Univ Columbia Systems and methods for opening of the blood-brain barrier of a subject using ultrasound
EP2068925A4 (en) * 2007-05-07 2011-08-31 Medimmune Llc ANTI-ICOS ANTIBODIES AND THEIR USE FOR THE TREATMENT OF CANCER, TRANSPLANTATIONS AND AUTOIMMUNE DISEASES
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WO2017024111A1 (en) * 2015-08-04 2017-02-09 The University Of Chicago Inhibitors of cacna1a/alpha1a subunit internal ribosomal entry site (ires) and methods of treating spinocerebellar ataxia type 6
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CA3056410A1 (en) * 2017-03-15 2018-09-20 Ovid Therapeutics, Inc. Use of designer receptors exclusively activated by designer drugs in the treatment of seizure disorders
CA3084985A1 (en) 2017-12-06 2019-06-13 Ovid Therapeutics Inc. Use of mir101 or mir128 in the treatment of seizure disorders

Also Published As

Publication number Publication date
WO2019113266A1 (en) 2019-06-13
CA3084985A1 (en) 2019-06-13
MX2020005952A (es) 2022-01-03
EP3703820A1 (en) 2020-09-09
AU2018378622A1 (en) 2020-05-28
JP2021505619A (ja) 2021-02-18
US20210095292A1 (en) 2021-04-01
US20190169619A1 (en) 2019-06-06
US10870855B2 (en) 2020-12-22
EP3703820A4 (en) 2020-12-09
MX2021011598A (es) 2021-10-13
MX388998B (es) 2025-03-11
IL274838A (en) 2020-07-30
CN111417397A (zh) 2020-07-14
US11761006B2 (en) 2023-09-19

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