KR20210040358A - 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 - Google Patents

안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 Download PDF

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Publication number
KR20210040358A
KR20210040358A KR1020217001109A KR20217001109A KR20210040358A KR 20210040358 A KR20210040358 A KR 20210040358A KR 1020217001109 A KR1020217001109 A KR 1020217001109A KR 20217001109 A KR20217001109 A KR 20217001109A KR 20210040358 A KR20210040358 A KR 20210040358A
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KR
South Korea
Prior art keywords
vector
microrna
promoter
patient
mir145
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
KR1020217001109A
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English (en)
Korean (ko)
Inventor
매튜 듀링
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오비드 테라퓨틱스 인크.
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Publication of KR20210040358A publication Critical patent/KR20210040358A/ko
Withdrawn legal-status Critical Current

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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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020217001109A 2018-06-14 2019-06-14 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용 Withdrawn KR20210040358A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862684774P 2018-06-14 2018-06-14
US62/684,774 2018-06-14
PCT/US2019/037174 WO2019241624A1 (en) 2018-06-14 2019-06-14 Use of mir-92a or mir-145 in the treatment of angelman syndrome

Publications (1)

Publication Number Publication Date
KR20210040358A true KR20210040358A (ko) 2021-04-13

Family

ID=68839583

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217001109A Withdrawn KR20210040358A (ko) 2018-06-14 2019-06-14 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용

Country Status (10)

Country Link
US (2) US10704048B2 (https=)
EP (1) EP3793567A4 (https=)
JP (1) JP2021527087A (https=)
KR (1) KR20210040358A (https=)
CN (1) CN112739353A (https=)
AU (1) AU2019285298A1 (https=)
CA (1) CA3103267A1 (https=)
IL (1) IL279391A (https=)
MX (1) MX2020013667A (https=)
WO (1) WO2019241624A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020002148A (es) * 2017-08-25 2020-07-20 Ovid Therapeutics Inc Vectores adenoasociados recombinantes.
MX2020013667A (es) 2018-06-14 2021-03-02 Ovid Therapeutics Inc Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.
WO2020227604A1 (en) * 2019-05-08 2020-11-12 Nova Southeastern University Regulation of nucleotide excision repair (ner) by microrna for treatment of breast cancer
KR20230128001A (ko) * 2020-12-01 2023-09-01 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 엔젤만 증후군의 치료를 위한 조성물 및 이의 용도

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
WO2006128245A1 (en) 2005-06-03 2006-12-07 Southern Adelaide Health Service-Flinders Medical Centre Targeting cells with altered microrna expression
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
CN102676516B (zh) * 2011-03-17 2014-06-04 中国医学科学院肿瘤研究所 microRNA 145的新用途
WO2013056216A1 (en) * 2011-10-14 2013-04-18 New York University Micrornas and methods of using same
AU2013262709A1 (en) * 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
ES2809199T3 (es) 2012-06-25 2021-03-03 Ionis Pharmaceuticals Inc Modulación de la expresión de UBE3A-ATS
HUE062186T2 (hu) * 2015-05-07 2023-09-28 Univ South Florida Módosított UBE3A gén az Angelman-szindróma génterápiás megközelítésére
US20190298843A1 (en) * 2015-05-11 2019-10-03 Purdue Research Foundation Ligand ionophore conjugates
IL256912B2 (en) * 2015-07-17 2024-01-01 Ovid Therapeutics Inc Methods of treating developmental disorders with gaboxadol
MX2020013667A (es) 2018-06-14 2021-03-02 Ovid Therapeutics Inc Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.

Also Published As

Publication number Publication date
US11130952B2 (en) 2021-09-28
WO2019241624A1 (en) 2019-12-19
US20200332294A1 (en) 2020-10-22
US20190382761A1 (en) 2019-12-19
MX2020013667A (es) 2021-03-02
EP3793567A1 (en) 2021-03-24
IL279391A (en) 2021-01-31
CA3103267A1 (en) 2019-12-19
US10704048B2 (en) 2020-07-07
CN112739353A (zh) 2021-04-30
JP2021527087A (ja) 2021-10-11
AU2019285298A1 (en) 2021-01-07
EP3793567A4 (en) 2021-08-11

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Patent event date: 20210113

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