AR127537A1 - Compuestos y métodos para reducir la expresión de psd3 - Google Patents
Compuestos y métodos para reducir la expresión de psd3Info
- Publication number
- AR127537A1 AR127537A1 ARP220102985A ARP220102985A AR127537A1 AR 127537 A1 AR127537 A1 AR 127537A1 AR P220102985 A ARP220102985 A AR P220102985A AR P220102985 A ARP220102985 A AR P220102985A AR 127537 A1 AR127537 A1 AR 127537A1
- Authority
- AR
- Argentina
- Prior art keywords
- psd3
- reduce
- liver disease
- methods
- animal
- Prior art date
Links
- 101000736367 Homo sapiens PH and SEC7 domain-containing protein 3 Proteins 0.000 title abstract 3
- 102100036231 PH and SEC7 domain-containing protein 3 Human genes 0.000 title abstract 3
- 150000001875 compounds Chemical class 0.000 title abstract 2
- 208000008338 non-alcoholic fatty liver disease Diseases 0.000 abstract 4
- 208000007082 Alcoholic Fatty Liver Diseases 0.000 abstract 2
- 208000026594 alcoholic fatty liver disease Diseases 0.000 abstract 2
- 208000006454 hepatitis Diseases 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 206010053219 non-alcoholic steatohepatitis Diseases 0.000 abstract 2
- 208000022309 Alcoholic Liver disease Diseases 0.000 abstract 1
- 206010008609 Cholangitis sclerosing Diseases 0.000 abstract 1
- 206010008909 Chronic Hepatitis Diseases 0.000 abstract 1
- 206010019708 Hepatic steatosis Diseases 0.000 abstract 1
- 208000033981 Hereditary haemochromatosis Diseases 0.000 abstract 1
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Chemical class Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 abstract 1
- 239000003795 chemical substances by application Substances 0.000 abstract 1
- 208000019425 cirrhosis of liver Diseases 0.000 abstract 1
- 235000014113 dietary fatty acids Nutrition 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 229930195729 fatty acid Natural products 0.000 abstract 1
- 239000000194 fatty acid Substances 0.000 abstract 1
- 150000004665 fatty acids Chemical class 0.000 abstract 1
- 231100000283 hepatitis Toxicity 0.000 abstract 1
- 206010073071 hepatocellular carcinoma Diseases 0.000 abstract 1
- 231100000844 hepatocellular carcinoma Toxicity 0.000 abstract 1
- 208000019423 liver disease Diseases 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 201000000742 primary sclerosing cholangitis Diseases 0.000 abstract 1
- 208000010157 sclerosing cholangitis Diseases 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
-
- 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
-
- 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.
-
- 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
- C12N2310/321—2'-O-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
- C12N2310/32—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
- C12N2310/32—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
-
- 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
-
- 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 ===---===
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Se proporcionan composiciones en cuestión que incluyen agentes oligoméricos, oligonucleótidos modificados, compuestos oligoméricos y composiciones farmacéuticas, y métodos de uso de estos, para reducir la cantidad o la actividad de ARN de PSD3 en una célula o animal, y en ciertos casos reducir la cantidad de la proteína PSD3 en una célula o animal. Tales composiciones son útiles para tratar la hepatopatía, enfermedad de ácido graso (FLD), enfermedad de hígado graso no alcohólica (NAFLD), esteatosis hepática, esteatohepatitis no alcohólica (NASH), cirrosis hepática, carcinoma hepatocelular, hepatopatía alcohólica, esteatohepatitis alcohólica (ASH), hepatitis por HCV, hepatitis crónica, hemocromatosis hereditaria o colangitis esclerosante primaria.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163274405P | 2021-11-01 | 2021-11-01 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR127537A1 true AR127537A1 (es) | 2024-02-07 |
Family
ID=86157477
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220102985A AR127537A1 (es) | 2021-11-01 | 2022-11-01 | Compuestos y métodos para reducir la expresión de psd3 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20230167446A1 (es) |
| AR (1) | AR127537A1 (es) |
| AU (1) | AU2022377400A1 (es) |
| CA (1) | CA3236950A1 (es) |
| IL (1) | IL312128A (es) |
| TW (1) | TW202333746A (es) |
| WO (1) | WO2023073661A2 (es) |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ITMI20051248A1 (it) * | 2005-07-01 | 2007-01-02 | Vimar Spa | Modem per bus per impianti elettrici civili ed industriali |
| GB2465902B (en) * | 2008-08-25 | 2010-12-08 | Excaliard Pharmaceuticals Inc | Antisense oligonucleotides directed against connective tissue growth factor and uses thereof |
| US20110251091A1 (en) * | 2008-09-12 | 2011-10-13 | Cornell University | Thyroid tumors identified |
| US20130165470A1 (en) * | 2011-12-21 | 2013-06-27 | The Procter & Gamble Company | Methods for Detecting and Treating Rhinovirus Infection |
| WO2020041791A1 (en) * | 2018-08-24 | 2020-02-27 | Locana, Inc. | Fasl immunomodulatory gene therapy compositions and methods for use |
| WO2021226663A1 (en) * | 2020-05-11 | 2021-11-18 | The Florey Institute Of Neuroscience And Mental Health | Compositions and methods for treating disorders associated with loss-of-function mutations in syngap1 |
| TW202227102A (zh) * | 2020-09-22 | 2022-07-16 | 瑞典商阿斯特捷利康公司 | 治療脂肪肝病之方法 |
| EP4301766A1 (en) * | 2021-03-02 | 2024-01-10 | Ionis Pharmaceuticals, Inc. | Purification methods for oligomeric compounds |
| EP4351719A1 (en) * | 2021-05-19 | 2024-04-17 | Empirico Inc. | Modulation of coasy expression |
-
2022
- 2022-10-31 IL IL312128A patent/IL312128A/en unknown
- 2022-10-31 AU AU2022377400A patent/AU2022377400A1/en active Pending
- 2022-10-31 CA CA3236950A patent/CA3236950A1/en active Pending
- 2022-10-31 TW TW111141302A patent/TW202333746A/zh unknown
- 2022-10-31 WO PCT/IB2022/060478 patent/WO2023073661A2/en active Application Filing
- 2022-10-31 US US18/051,089 patent/US20230167446A1/en active Pending
- 2022-11-01 AR ARP220102985A patent/AR127537A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| IL312128A (en) | 2024-06-01 |
| WO2023073661A2 (en) | 2023-05-04 |
| US20230167446A1 (en) | 2023-06-01 |
| TW202333746A (zh) | 2023-09-01 |
| CA3236950A1 (en) | 2023-05-04 |
| AU2022377400A1 (en) | 2024-05-02 |
| WO2023073661A3 (en) | 2023-06-22 |
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