MX2021005886A - Compuestos y metodos para reducir la expresion de priones. - Google Patents
Compuestos y metodos para reducir la expresion de priones.Info
- Publication number
- MX2021005886A MX2021005886A MX2021005886A MX2021005886A MX2021005886A MX 2021005886 A MX2021005886 A MX 2021005886A MX 2021005886 A MX2021005886 A MX 2021005886A MX 2021005886 A MX2021005886 A MX 2021005886A MX 2021005886 A MX2021005886 A MX 2021005886A
- Authority
- MX
- Mexico
- Prior art keywords
- disease
- compounds
- methods
- creutzfeldt
- reducing
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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
- C12N15/11—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
- 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
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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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/35—Special therapeutic applications based on a specific dosage / administration regimen
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)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Neurosurgery (AREA)
- Epidemiology (AREA)
- Neurology (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o actividad de ARN de PRNP en una célula o animal y, en determinados casos, para reducir la cantidad de proteína PrP en una célula o animal. Dichos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica distintiva de una enfermedad neurodegenerativa. Dichos síntomas y características distintivas son cambios espongiformes en el cerebro, desarrollo de agregados proteicos anormales, pérdida neuronal, marcadores de pérdida neuronal, demencia que progresa rápidamente y muerte. Estas enfermedades neurodegenerativas incluyen enfermedades priónicas, enfermedad de Creutzfeldt-Jakob (CJD), variante de la enfermedad de Creutzfeldt-Jakob (vCJD), enfermedad de Creutzfeldt- Jakob familiar (fCJD), síndrome de Gerstmann-Straussler-Scheinker, insomnio familiar fatal, kuru, enfermedad de Alzheimer o enfermedad de Parkinson.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862770386P | 2018-11-21 | 2018-11-21 | |
PCT/US2019/062681 WO2020106996A1 (en) | 2018-11-21 | 2019-11-21 | Compounds and methods for reducing prion expression |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2021005886A true MX2021005886A (es) | 2021-06-23 |
Family
ID=70774613
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2021005886A MX2021005886A (es) | 2018-11-21 | 2019-11-21 | Compuestos y metodos para reducir la expresion de priones. |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220025366A1 (es) |
EP (1) | EP3884053A4 (es) |
JP (2) | JP7455831B2 (es) |
KR (1) | KR20210093970A (es) |
CN (1) | CN113286886A (es) |
AU (1) | AU2019384181A1 (es) |
BR (1) | BR112021007476A2 (es) |
CA (1) | CA3117694A1 (es) |
IL (1) | IL283332A (es) |
MX (1) | MX2021005886A (es) |
SG (1) | SG11202103794QA (es) |
WO (1) | WO2020106996A1 (es) |
Families Citing this family (5)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3088112A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
EP4110913A4 (en) * | 2020-02-28 | 2024-07-24 | Univ Massachusetts | OLIGONUCLEOTIDES FOR PRNP MODULATION |
EP4298220A1 (en) * | 2021-02-25 | 2024-01-03 | Alnylam Pharmaceuticals, Inc. | Prion protein (prnp) irna compositions and methods of use thereof |
WO2023060092A1 (en) * | 2021-10-04 | 2023-04-13 | Atalanta Therapeutics, Inc | Compositions and methods for treatment of prion diseases |
WO2023164696A2 (en) * | 2022-02-28 | 2023-08-31 | Ionis Pharmaceuticals, Inc. | Rnai agents of prion expression |
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JOP20190104A1 (ar) * | 2016-11-10 | 2019-05-07 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن atxn3 |
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2019
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CN113286886A (zh) | 2021-08-20 |
TW202039841A (zh) | 2020-11-01 |
US20220025366A1 (en) | 2022-01-27 |
JP2022509625A (ja) | 2022-01-21 |
WO2020106996A1 (en) | 2020-05-28 |
EP3884053A1 (en) | 2021-09-29 |
EP3884053A4 (en) | 2023-02-01 |
SG11202103794QA (en) | 2021-05-28 |
BR112021007476A2 (pt) | 2021-11-03 |
IL283332A (en) | 2021-07-29 |
JP7455831B2 (ja) | 2024-03-26 |
CA3117694A1 (en) | 2020-05-28 |
AU2019384181A1 (en) | 2021-05-20 |
JP2024079716A (ja) | 2024-06-11 |
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