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)
  • 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)
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
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
EP3793567A1 (en) 2021-03-24
IL279391A (en) 2021-01-31
CA3103267A1 (en) 2019-12-19
KR20210040358A (ko) 2021-04-13
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

Similar Documents

Publication Publication Date Title
PH12017550069A1 (en) Tau antisense oligomers and uses thereof
MX2020013667A (es) Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman.
MX2022003116A (es) Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
MX2025014318A (es) Vector de adenovirus de chimpance util para la administracion de neoantigenos
SA519401379B1 (ar) علاج فيروس مرتبط بغدة لمرض هنتنجتون
BR112017004349A2 (pt) terapia com gene de globina para o tratamento de hemoglobinopatias
CA3062698A1 (en) Nucleic acid molecules encoding an engineered antigen receptor and an inhibitory nucleic acid molecule and methods of use thereof
MX2016013832A (es) Composiciones y metodos para tratar hemoglobinopatias.
WO2016037071A3 (en) Methods for treating aging and skin disorders using nucleic acids targeting tyr or mmp1
MX388146B (es) Vectores de virus asociados con adeno (aav) para uso en terapia de genes de coroideremia.
EP4089172A3 (en) Multiple vector system and uses thereof
WO2016065349A8 (en) Short non-coding protein regulatory rnas (sprrnas) and methods of use
MY191210A (en) Novel promoter and uses thereof
MX2013014544A (es) Vectorales de terapia genica para adrenoleucodistrofia y adrenomieloneuropatia.
BR112015024605A2 (pt) sistemas e métodos para a produção visada de proteína terapêutica dentro de célula alvo
EP3800255A3 (en) Rna-guided transcriptional regulation and methods of using the same for the treatment of back pain
EP4678754A3 (en) A modified mglur6 promoter and methods of use
MX2015014423A (es) Administración efectiva de genes grandes por vectores aav duales.
SA517380883B1 (ar) Viii مجموعة تعبير متطورة لحزم والتعبير عن عامل متغير لعلاج اضطراب توقف نزيف الدم
PH12020500570A1 (en) Alpha-synuclein antisense oligonucleotides and uses thereof
CA3057899A1 (en) Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
WO2020104649A3 (en) Novel rna compositions and methods for inhibiting angptl8
MX2018005467A (es) Composiciones que comprenden vectores lentivirales que expresan interleucina-12, y metodos de uso de las mismas.
AU2017248848A1 (en) Enhanced gene delivery methods
CA2986376C (en) Poxvirus-derived promoter, and vector comprising same