MX2020013667A - Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman. - Google Patents

Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.

Info

Publication number
MX2020013667A
MX2020013667A MX2020013667A MX2020013667A MX2020013667A MX 2020013667 A MX2020013667 A MX 2020013667A MX 2020013667 A MX2020013667 A MX 2020013667A MX 2020013667 A MX2020013667 A MX 2020013667A MX 2020013667 A MX2020013667 A MX 2020013667A
Authority
MX
Mexico
Prior art keywords
mir
treatment
angelman syndrome
expression
angelman
Prior art date
Application number
MX2020013667A
Other languages
English (en)
Spanish (es)
Inventor
Matthew During
Original Assignee
Ovid Therapeutics Inc
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 Ovid Therapeutics Inc filed Critical Ovid Therapeutics Inc
Publication of MX2020013667A publication Critical patent/MX2020013667A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0047Sonopheresis, i.e. ultrasonically-enhanced transdermal delivery, electroporation of a pharmacologically active agent
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/15011Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
MX2020013667A 2018-06-14 2019-06-14 Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman. MX2020013667A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862684774P 2018-06-14 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
MX2020013667A true MX2020013667A (es) 2021-03-02

Family

ID=68839583

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020013667A MX2020013667A (es) 2018-06-14 2019-06-14 Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.

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
AU2018320849A1 (en) * 2017-08-25 2020-03-05 Ovid Therapeutics Inc. Recombinant adeno-associated vectors
KR20210040358A (ko) 2018-06-14 2021-04-13 오비드 테라퓨틱스 인크. 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용
WO2020227604A1 (en) * 2019-05-08 2020-11-12 Nova Southeastern University Regulation of nucleotide excision repair (ner) by microrna for treatment of breast cancer
IL303239A (en) * 2020-12-01 2023-07-01 Univ Pennsylvania Compositions and their uses for the treatment of Engelmann syndrome

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
US20150267193A1 (en) * 2011-10-14 2015-09-24 Ramanuj Dasgupta MicroRNAs and Methods of Using Same
EA201492116A1 (ru) * 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
WO2014004572A2 (en) 2012-06-25 2014-01-03 Isis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
EP3291843B1 (en) 2015-05-07 2023-03-22 University of South Florida Modified ube3a gene for a gene therapy approach for angelman syndrome
US20190298843A1 (en) * 2015-05-11 2019-10-03 Purdue Research Foundation Ligand ionophore conjugates
EP4541420A3 (en) * 2015-07-17 2025-06-25 Ovid Therapeutics Inc. Methods of treating developmental disorders with gaboxadol
KR20210040358A (ko) 2018-06-14 2021-04-13 오비드 테라퓨틱스 인크. 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용

Also Published As

Publication number Publication date
WO2019241624A1 (en) 2019-12-19
JP2021527087A (ja) 2021-10-11
EP3793567A1 (en) 2021-03-24
US11130952B2 (en) 2021-09-28
CA3103267A1 (en) 2019-12-19
KR20210040358A (ko) 2021-04-13
EP3793567A4 (en) 2021-08-11
US20200332294A1 (en) 2020-10-22
CN112739353A (zh) 2021-04-30
IL279391A (en) 2021-01-31
US20190382761A1 (en) 2019-12-19
US10704048B2 (en) 2020-07-07
AU2019285298A1 (en) 2021-01-07

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