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.Info
- 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
- Authority
- MX
- Mexico
- Prior art keywords
- specific
- mirna
- vector
- promoter
- oligodendrocyte
- Prior art date
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- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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- 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
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- 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
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- 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
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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
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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
- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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- 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
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- 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
- C12N15/63—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
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/04—Phosphoric diester hydrolases (3.1.4)
- C12Y301/04037—2',3'-Cyclic-nucleotide 3'-phosphodiesterase (3.1.4.37)
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- 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 N.A.
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- 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 N.A.
- C12N2310/141—MicroRNAs, miRNAs
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- 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
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- 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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- 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
- 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
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- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Mycology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (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.
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 (4)
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 |
TW202216996A (zh) | 2020-06-29 | 2022-05-01 | 美商Ionis製藥公司 | 調節plp1之化合物及方法 |
JP2023538284A (ja) * | 2020-08-04 | 2023-09-07 | ダイセルナ ファーマシューティカルズ, インコーポレイテッド | Plp1発現を阻害するための組成物及び方法 |
WO2024149284A1 (en) * | 2023-01-10 | 2024-07-18 | Nanjing Legend Biotech Co., Ltd. | Regulatory sequences and uses thereof |
Family Cites Families (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2008073303A2 (en) * | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
-
2019
- 2019-02-01 TW TW108104341A patent/TW201945009A/zh unknown
- 2019-02-06 KR KR1020207025401A patent/KR20200118122A/ko active Search and Examination
- 2019-02-06 BR BR112020015967-0A patent/BR112020015967A2/pt unknown
- 2019-02-06 CA CA3090230A patent/CA3090230A1/en active Pending
- 2019-02-06 US US16/967,483 patent/US20210087560A1/en not_active Abandoned
- 2019-02-06 MX MX2020008242A patent/MX2020008242A/es unknown
- 2019-02-06 SG SG11202007462YA patent/SG11202007462YA/en unknown
- 2019-02-06 EP EP19751969.7A patent/EP3750996A4/en not_active Withdrawn
- 2019-02-06 CN CN201980011927.6A patent/CN111699259A/zh active Pending
- 2019-02-06 WO PCT/JP2019/004227 patent/WO2019156115A1/ja unknown
- 2019-02-06 AU AU2019219286A patent/AU2019219286A1/en not_active Abandoned
- 2019-02-06 JP JP2019570774A patent/JPWO2019156115A1/ja active Pending
-
2020
- 2020-07-31 IL IL276424A patent/IL276424A/en unknown
- 2020-08-06 PH PH12020551191A patent/PH12020551191A1/en unknown
Also Published As
Publication number | Publication date |
---|---|
KR20200118122A (ko) | 2020-10-14 |
US20210087560A1 (en) | 2021-03-25 |
EP3750996A4 (en) | 2022-02-23 |
TW201945009A (zh) | 2019-12-01 |
WO2019156115A1 (ja) | 2019-08-15 |
BR112020015967A2 (pt) | 2020-12-15 |
AU2019219286A1 (en) | 2020-08-27 |
IL276424A (en) | 2020-09-30 |
SG11202007462YA (en) | 2020-09-29 |
CA3090230A1 (en) | 2019-08-15 |
EP3750996A1 (en) | 2020-12-16 |
JPWO2019156115A1 (ja) | 2021-04-08 |
PH12020551191A1 (en) | 2021-05-17 |
RU2020128850A (ru) | 2022-03-09 |
CN111699259A (zh) | 2020-09-22 |
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