CL2021002585A1 - Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central - Google Patents
Composiciones y métodos para inhibir la expresión génica en el sistema nervioso centralInfo
- Publication number
- CL2021002585A1 CL2021002585A1 CL2021002585A CL2021002585A CL2021002585A1 CL 2021002585 A1 CL2021002585 A1 CL 2021002585A1 CL 2021002585 A CL2021002585 A CL 2021002585A CL 2021002585 A CL2021002585 A CL 2021002585A CL 2021002585 A1 CL2021002585 A1 CL 2021002585A1
- Authority
- CL
- Chile
- Prior art keywords
- nervous system
- central nervous
- methods
- compositions
- gene expression
- Prior art date
Links
- 210000003169 central nervous system Anatomy 0.000 title abstract 3
- 230000014509 gene expression Effects 0.000 title abstract 2
- 239000000203 mixture Substances 0.000 title abstract 2
- 230000002401 inhibitory effect Effects 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 102000009645 Mitochondrial Aldehyde Dehydrogenase Human genes 0.000 abstract 1
- 108010009513 Mitochondrial Aldehyde Dehydrogenase Proteins 0.000 abstract 1
- 208000012902 Nervous system disease Diseases 0.000 abstract 1
- 208000025966 Neurological disease 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 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 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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- 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
- C12N15/1137—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 enzymes
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- C12Y102/01—Oxidoreductases acting on the aldehyde or oxo group of donors (1.2) with NAD+ or NADP+ as acceptor (1.2.1)
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Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962829595P | 2019-04-04 | 2019-04-04 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2021002585A1 true CL2021002585A1 (es) | 2022-05-20 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2021002585A CL2021002585A1 (es) | 2019-04-04 | 2021-10-04 | Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20220170025A1 (https=) |
| EP (1) | EP3947683A1 (https=) |
| JP (1) | JP2022526419A (https=) |
| KR (1) | KR20210148264A (https=) |
| CN (1) | CN113748208A (https=) |
| AU (1) | AU2020252560A1 (https=) |
| BR (1) | BR112021019793A2 (https=) |
| CA (1) | CA3135402A1 (https=) |
| CL (1) | CL2021002585A1 (https=) |
| IL (1) | IL286868A (https=) |
| MX (1) | MX2021012126A (https=) |
| SG (1) | SG11202110896WA (https=) |
| WO (1) | WO2020206350A1 (https=) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019143621A1 (en) | 2018-01-16 | 2019-07-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting aldh2 expression |
| CN119499394A (zh) | 2019-11-01 | 2025-02-25 | 阿尔尼拉姆医药品有限公司 | 亨廷顿（HTT）iRNA药剂组合物及其使用方法 |
| EP4073251A1 (en) | 2019-12-13 | 2022-10-19 | Alnylam Pharmaceuticals, Inc. | Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof |
| EP4244356A1 (en) * | 2020-11-13 | 2023-09-20 | Dicerna Pharmaceuticals, Inc. | Chemical modifications for inhibiting expression of aldh2 |
| AR124713A1 (es) * | 2021-01-28 | 2023-04-26 | Dicerna Pharmaceuticals Inc | Composiciones y métodos para inhibir la expresión de genes en el sistema nervioso central |
| WO2022174000A2 (en) | 2021-02-12 | 2022-08-18 | Alnylam Pharmaceuticals, Inc. | Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases |
| CN117222739A (zh) | 2021-02-25 | 2023-12-12 | 阿尔尼拉姆医药品有限公司 | 朊病毒蛋白(prnp)irna组合物和其使用方法 |
| WO2022192519A1 (en) | 2021-03-12 | 2022-09-15 | Alnylam Pharmaceuticals, Inc. | Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof |
| WO2022212231A2 (en) | 2021-03-29 | 2022-10-06 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| PE20250074A1 (es) * | 2021-05-28 | 2025-01-13 | Novo Nordisk As | Composiciones y metodos para inhibir la expresion del componente 1 de reduccion de amidoxima mitocondrial (marc1) |
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| PT1309726E (pt) | 2000-03-30 | 2010-03-08 | Whitehead Biomedical Inst | Mediadores de interferência por rna específicos de sequência de rna |
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| US20050019915A1 (en) * | 2001-06-21 | 2005-01-27 | Bennett C. Frank | Antisense modulation of superoxide dismutase 1, soluble expression |
| JP2005508196A (ja) | 2001-11-07 | 2005-03-31 | アプレラ コーポレイション | 核酸分析の汎用ヌクレオチド |
| US20070265220A1 (en) | 2004-03-15 | 2007-11-15 | City Of Hope | Methods and compositions for the specific inhibition of gene expression by double-stranded RNA |
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| JP2012504389A (ja) | 2008-09-22 | 2012-02-23 | 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 | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
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| JP2017522046A (ja) | 2014-06-06 | 2017-08-10 | ソルスティス バイオロジクス，リミティッド | 生物可逆的および生物不可逆的基を有するポリヌクレオチド構築物 |
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| EP4029942A1 (en) * | 2017-04-05 | 2022-07-20 | Silence Therapeutics GmbH | Products and compositions |
| CA3118327A1 (en) * | 2017-11-13 | 2019-05-16 | Silence Therapeutics Gmbh | Nucleic acids for inhibiting expression of aldh2 in a cell |
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| WO2019143621A1 (en) * | 2018-01-16 | 2019-07-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting aldh2 expression |
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| SG11202110896WA (en) | 2021-10-28 |
| CN113748208A (zh) | 2021-12-03 |
| BR112021019793A2 (pt) | 2021-12-07 |
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| US20220170025A1 (en) | 2022-06-02 |
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