UY39297A - Compuestos y métodos para reducir la expresión de kcnt1 - Google Patents
Compuestos y métodos para reducir la expresión de kcnt1Info
- Publication number
- UY39297A UY39297A UY0001039297A UY39297A UY39297A UY 39297 A UY39297 A UY 39297A UY 0001039297 A UY0001039297 A UY 0001039297A UY 39297 A UY39297 A UY 39297A UY 39297 A UY39297 A UY 39297A
- Authority
- UY
- Uruguay
- Prior art keywords
- compounds
- methods
- kcnt1
- pharmaceutical compositions
- expression
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 title abstract 4
- 101000944018 Homo sapiens Potassium channel subfamily T member 1 Proteins 0.000 title abstract 3
- 102100033508 Potassium channel subfamily T member 1 Human genes 0.000 title abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 abstract 3
- 206010010904 Convulsion Diseases 0.000 abstract 2
- 230000000926 neurological effect Effects 0.000 abstract 2
- 208000024891 symptom Diseases 0.000 abstract 2
- 208000014644 Brain disease Diseases 0.000 abstract 1
- 208000032274 Encephalopathy Diseases 0.000 abstract 1
- 230000005856 abnormality Effects 0.000 abstract 1
- 230000003542 behavioural effect Effects 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 206010015037 epilepsy Diseases 0.000 abstract 1
- 230000001617 migratory effect Effects 0.000 abstract 1
Classifications
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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
- C12N15/1138—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 against receptors or cell surface proteins
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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
- C12N2310/11—Antisense
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/334—Modified C
- C12N2310/3341—5-Methylcytosine
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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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
Landscapes
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Abstract
Se proporcionan compuestos, métodos y composiciones farmacéuticas para reducir la cantidad o actividad del ARN de KCNT1 en una célula o sujeto, y en determinadas circunstancias reducir la cantidad de proteína de KCNT1 en una célula o un sujeto. Estos compuestos, métodos y composiciones farmacéuticas son útiles para mejorar al menos un síntoma o característica de una afección neurológica. Dichos síntomas y características incluyen convulsiones, encefalopatía y anomalías del comportamiento. Los ejemplos no taxativos de afecciones neurológicas que se benefician de estos compuestos, métodos y composiciones farmacéuticas son la epilepsia de la infancia con crisis focales migratorias (EIMFS).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202063044345P | 2020-06-25 | 2020-06-25 | |
US202063079438P | 2020-09-16 | 2020-09-16 |
Publications (1)
Publication Number | Publication Date |
---|---|
UY39297A true UY39297A (es) | 2021-12-31 |
Family
ID=79281885
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
UY0001039297A UY39297A (es) | 2020-06-25 | 2021-06-25 | Compuestos y métodos para reducir la expresión de kcnt1 |
Country Status (6)
Country | Link |
---|---|
US (1) | US20240229042A1 (es) |
EP (1) | EP4171576A2 (es) |
JP (1) | JP2023533153A (es) |
CN (1) | CN115701998A (es) |
UY (1) | UY39297A (es) |
WO (1) | WO2021263082A2 (es) |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20020048787A1 (en) * | 2000-06-14 | 2002-04-25 | Ming-Hui Wei | Isolated human transporter proteins, nucleic acid molecules encoding human transporter proteins, and uses thereof |
US20070059710A1 (en) * | 2004-02-27 | 2007-03-15 | Applera Corporation | Genetic polymorphisms associated with stroke, methods of detection and uses thereof |
WO2018227247A1 (en) * | 2017-06-13 | 2018-12-20 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating conditions associated with gain-of-function mutations in kcnt1 |
-
2021
- 2021-06-25 US US18/001,853 patent/US20240229042A1/en active Pending
- 2021-06-25 CN CN202180045166.3A patent/CN115701998A/zh active Pending
- 2021-06-25 UY UY0001039297A patent/UY39297A/es unknown
- 2021-06-25 JP JP2022574424A patent/JP2023533153A/ja active Pending
- 2021-06-25 EP EP21828398.4A patent/EP4171576A2/en active Pending
- 2021-06-25 WO PCT/US2021/039049 patent/WO2021263082A2/en unknown
Also Published As
Publication number | Publication date |
---|---|
WO2021263082A3 (en) | 2022-02-10 |
JP2023533153A (ja) | 2023-08-02 |
CN115701998A (zh) | 2023-02-14 |
WO2021263082A2 (en) | 2021-12-30 |
EP4171576A2 (en) | 2023-05-03 |
US20240229042A1 (en) | 2024-07-11 |
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