AR124275A1 - Administración selectiva de oligonucleótidos a células gliales - Google Patents
Administración selectiva de oligonucleótidos a células glialesInfo
- Publication number
- AR124275A1 AR124275A1 ARP210102814A ARP210102814A AR124275A1 AR 124275 A1 AR124275 A1 AR 124275A1 AR P210102814 A ARP210102814 A AR P210102814A AR P210102814 A ARP210102814 A AR P210102814A AR 124275 A1 AR124275 A1 AR 124275A1
- Authority
- AR
- Argentina
- Prior art keywords
- target rna
- oligonucleotide
- glial
- level
- cells
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 16
- 210000004498 neuroglial cell Anatomy 0.000 title abstract 14
- 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 Polymers 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 title abstract 2
- 210000002569 neuron Anatomy 0.000 abstract 7
- 230000007423 decrease Effects 0.000 abstract 5
- 238000000034 method Methods 0.000 abstract 4
- 210000000653 nervous system Anatomy 0.000 abstract 3
- 230000003247 decreasing effect Effects 0.000 abstract 2
- 238000012216 screening Methods 0.000 abstract 2
- 230000009699 differential effect Effects 0.000 abstract 1
- 230000002452 interceptive effect Effects 0.000 abstract 1
Classifications
-
- 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
-
- 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
- C12N2310/312—Phosphonates
- C12N2310/3125—Methylphosphonates
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- 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
-
- 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
- C12N2503/00—Use of cells in diagnostics
- C12N2503/02—Drug screening
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)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
Reivindicación 1: Un método para administrar en forma selectiva un oligonucleótido interferente a células gliales, que comprende: poner en contacto una célula glial con un oligonucleótido que comprende una región de complementariedad respecto a un ARN diana expresado en la célula glial, en donde el oligonucleótido puede disminuir la expresión del ARN diana. Reivindicación 43: Un método para disminuir en forma selectiva el nivel de un ARN diana que se expresa en una célula glial, que comprende: poner en contacto una célula glial con un oligonucleótido que comprende una región de complementariedad respecto a un ARN diana expresado en la célula glial, en donde el oligonucleótido puede disminuir la expresión del ARN diana. Reivindicación 85: Un método para el cribado de un oligonucleótido que disminuye en forma selectiva el nivel de un ARN diana expresado en células gliales o células neuronales, que comprende: poner en contacto una célula glial y una célula neuronal con un oligonucleótido posible, en donde el oligonucleótido posible comprende una región de complementariedad respecto a un ARN diana expresado tanto en la célula glial como en la célula neuronal; y medir el nivel de ARN diana en las células gliales y las células neuronales para determinar cualquier efecto diferencial en el nivel de expresión de ARN diana. Reivindicación 96: Un método para el cribado de oligonucleótidos que disminuyan en forma selectiva el nivel de un ARN diana expresado en células gliales o células neuronales, que comprende: administrar un primer oligonucleótido posible al sistema nervioso de un primer sujeto, en donde el primer oligonucleótido posible comprende una región de complementariedad respecto a un primer ARN diana expresado en células gliales y en donde el primer oligonucleótido puede disminuir el nivel de expresión del primer ARN diana; administrar un segundo oligonucleótido posible al sistema nervioso de un segundo sujeto, en donde el segundo oligonucleótido posible comprende una región de complementariedad respecto a un segundo ARN diana expresado en células neuronales y en donde el segundo oligonucleótido puede disminuir el nivel de expresión del segundo ARN diana; y medir el nivel del primer ARN diana en células gliales y el segundo ARN diana en células neuronales en el sistema nervioso del primer sujeto y el segundo sujeto, en donde el primer sujeto y el segundo sujeto son de la misma especie.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063089406P | 2020-10-08 | 2020-10-08 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124275A1 true AR124275A1 (es) | 2023-03-15 |
Family
ID=78536682
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP210102814A AR124275A1 (es) | 2020-10-08 | 2021-10-12 | Administración selectiva de oligonucleótidos a células gliales |
Country Status (11)
| Country | Link |
|---|---|
| EP (1) | EP4225919A1 (es) |
| JP (1) | JP2023548658A (es) |
| KR (1) | KR20230104880A (es) |
| CN (1) | CN116761886A (es) |
| AR (1) | AR124275A1 (es) |
| AU (1) | AU2021356639A1 (es) |
| CA (1) | CA3194697A1 (es) |
| IL (1) | IL301940A (es) |
| MX (1) | MX2023004109A (es) |
| TW (1) | TW202228729A (es) |
| WO (1) | WO2022077024A1 (es) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230374522A1 (en) * | 2022-04-15 | 2023-11-23 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for modulating scap activity |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003040395A2 (en) | 2001-11-07 | 2003-05-15 | Applera Corporation | Universal nucleotides for nucleic acid analysis |
| DE10317389A1 (de) * | 2003-04-15 | 2004-11-04 | Dr. Kübler GmbH | Synergistisch wirkende Kombination von Phenylbutyrat und Aminobenzoesäure zur Krebstherapie |
| WO2007030167A1 (en) | 2005-09-02 | 2007-03-15 | Nastech Pharmaceutical Company Inc. | Modification of double-stranded ribonucleic acid molecules |
| US20100098664A1 (en) * | 2007-11-28 | 2010-04-22 | Mathieu Jean-Francois Desclaux | Lentiviral vectors allowing RNAi mediated inhibition of GFAP and vimentin expression |
| EP3067359A1 (en) | 2008-09-23 | 2016-09-14 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference |
| WO2011005860A2 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | 5' phosphate mimics |
| US9725479B2 (en) | 2010-04-22 | 2017-08-08 | Ionis Pharmaceuticals, Inc. | 5′-end derivatives |
| WO2013063313A1 (en) | 2011-10-25 | 2013-05-02 | Isis Pharmaceuticals, Inc. | Antisense modulation of gccr expression |
| AU2013356383B2 (en) | 2012-12-06 | 2017-08-31 | Merck Sharp & Dohme Corp. | Disulfide masked prodrug compositions and methods |
| US20170114341A1 (en) | 2014-06-06 | 2017-04-27 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
| EP3865576A1 (en) | 2014-12-15 | 2021-08-18 | Dicerna Pharmaceuticals, Inc. | Ligand-modified double-stranded nucleic acids |
| JP7249080B2 (ja) | 2016-08-23 | 2023-03-30 | ディセルナ ファーマシューティカルズ インコーポレイテッド | 可逆的に修飾されたオリゴヌクレオチドを含む組成物及びその使用 |
| CN110072530A (zh) | 2016-09-02 | 2019-07-30 | 迪克纳制药公司 | 4′-磷酸酯类似物和包含其的寡核苷酸 |
| CA3098623A1 (en) * | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
-
2021
- 2021-10-08 IL IL301940A patent/IL301940A/en unknown
- 2021-10-08 KR KR1020237014927A patent/KR20230104880A/ko unknown
- 2021-10-08 WO PCT/US2021/071785 patent/WO2022077024A1/en active Application Filing
- 2021-10-08 CA CA3194697A patent/CA3194697A1/en active Pending
- 2021-10-08 CN CN202180082834.XA patent/CN116761886A/zh active Pending
- 2021-10-08 AU AU2021356639A patent/AU2021356639A1/en active Pending
- 2021-10-08 EP EP21805812.1A patent/EP4225919A1/en active Pending
- 2021-10-08 JP JP2023521711A patent/JP2023548658A/ja active Pending
- 2021-10-08 TW TW110137436A patent/TW202228729A/zh unknown
- 2021-10-08 MX MX2023004109A patent/MX2023004109A/es unknown
- 2021-10-12 AR ARP210102814A patent/AR124275A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| KR20230104880A (ko) | 2023-07-11 |
| TW202228729A (zh) | 2022-08-01 |
| EP4225919A1 (en) | 2023-08-16 |
| WO2022077024A1 (en) | 2022-04-14 |
| IL301940A (en) | 2023-06-01 |
| MX2023004109A (es) | 2023-06-28 |
| CA3194697A1 (en) | 2022-04-14 |
| CN116761886A (zh) | 2023-09-15 |
| JP2023548658A (ja) | 2023-11-20 |
| AU2021356639A1 (en) | 2023-05-18 |
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