MX2020008242A - Promotor especifico de oligodendrocitos, mirna especifico para el gen de plp1, vector que incluye el promotor y/o mirna, y composicion farmaceutica que incluye el vector. - Google Patents

Promotor especifico de oligodendrocitos, mirna especifico para el gen de plp1, vector que incluye el promotor y/o mirna, y composicion farmaceutica que incluye el vector.

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Publication number
MX2020008242A
MX2020008242A MX2020008242A MX2020008242A MX2020008242A MX 2020008242 A MX2020008242 A MX 2020008242A MX 2020008242 A MX2020008242 A MX 2020008242A MX 2020008242 A MX2020008242 A MX 2020008242A MX 2020008242 A MX2020008242 A MX 2020008242A
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Mexico
Prior art keywords
specific
mirna
vector
promoter
oligodendrocyte
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MX2020008242A
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English (en)
Inventor
Takashi Okada
Heng Li
Ken Inoue
Yu OHKI
Makoto Koizumi
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Nat Center Neurology & Psychiatry
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Publication date
Application filed by Nat Center Neurology & Psychiatry filed Critical Nat Center Neurology & Psychiatry
Publication of MX2020008242A publication Critical patent/MX2020008242A/es

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    • C12N15/09—Recombinant DNA-technology
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A—HUMAN NECESSITIES
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/04—Phosphoric diester hydrolases (3.1.4)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • 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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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Physical Education & Sports Medicine (AREA)
  • Mycology (AREA)
  • Psychology (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)

Abstract

Un objeto de la presente invención es proveer un vector con capacidad de supresión, específica de oligodendrocitos, de la expresión del gen de PLP1, para tratar la PMD ocasionada por la anormal idad del gen de PLP1, y un promotor y miRNA para el mismo, y una composición farmacéutica que comprende el vector. El promotor específico de oligodendrocitos de la presente invención comprende un ácido nucleico que tiene una identidad de secuencias de por lo menos 90% con una secuencia nucleotídica establecida en la SEQ ID NO: 1. El miRNA de la presente invención, específico para el gen de PLP1, comprende un par de secuencias nucleotídicas que consisten en una secuencia antisentido y una secuencia sentido específicas.
MX2020008242A 2018-02-07 2019-02-06 Promotor especifico de oligodendrocitos, mirna especifico para el gen de plp1, vector que incluye el promotor y/o mirna, y composicion farmaceutica que incluye el vector. MX2020008242A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
JP2018019950 2018-02-07
PCT/JP2019/004227 WO2019156115A1 (ja) 2018-02-07 2019-02-06 オリゴデンドロサイト特異的プロモータ、ＰＬＰ１遺伝子に特異的なｍｉＲＮＡ、該プロモータ及び／又は該ｍｉＲＮＡを含むベクター並びに該ベクターを含む医薬組成物

Publications (1)

Publication Number Publication Date
MX2020008242A true MX2020008242A (es) 2020-09-25

Family

ID=67549420

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020008242A MX2020008242A (es) 2018-02-07 2019-02-06 Promotor especifico de oligodendrocitos, mirna especifico para el gen de plp1, vector que incluye el promotor y/o mirna, y composicion farmaceutica que incluye el vector.

Country Status (14)

Country Link
US (1) US20210087560A1 (es)
EP (1) EP3750996A4 (es)
JP (1) JPWO2019156115A1 (es)
KR (1) KR20200118122A (es)
CN (1) CN111699259A (es)
AU (1) AU2019219286A1 (es)
BR (1) BR112020015967A2 (es)
CA (1) CA3090230A1 (es)
IL (1) IL276424A (es)
MX (1) MX2020008242A (es)
PH (1) PH12020551191A1 (es)
SG (1) SG11202007462YA (es)
TW (1) TW201945009A (es)
WO (1) WO2019156115A1 (es)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2023130528A (ja) * 2020-06-24 2023-09-21 田辺三菱製薬株式会社 Ｐｌｐ１発現を調節するための化合物、方法及び医薬組成物
EP4172338A2 (en) 2020-06-29 2023-05-03 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating plp1
MX2023001450A (es) * 2020-08-04 2023-04-14 Dicerna Pharmaceuticals Inc Composiciones y metodos para la inhibicion de la expresion de plp1.

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2097538A4 (en) * 2006-12-07 2011-11-30 Switchgear Genomics TRANSCRIPTION REAGULATION ELEMENTS OF BIOLOGICAL PATHS, TOOLS AND METHODS

Also Published As

Publication number Publication date
WO2019156115A1 (ja) 2019-08-15
EP3750996A4 (en) 2022-02-23
BR112020015967A2 (pt) 2020-12-15
TW201945009A (zh) 2019-12-01
JPWO2019156115A1 (ja) 2021-04-08
EP3750996A1 (en) 2020-12-16
RU2020128850A (ru) 2022-03-09
IL276424A (en) 2020-09-30
US20210087560A1 (en) 2021-03-25
SG11202007462YA (en) 2020-09-29
AU2019219286A1 (en) 2020-08-27
KR20200118122A (ko) 2020-10-14
CN111699259A (zh) 2020-09-22
PH12020551191A1 (en) 2021-05-17
CA3090230A1 (en) 2019-08-15

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