ES2651147T3 - Oligonucleótidos para modular la expresión génica y usos de los mismos - Google Patents
Oligonucleótidos para modular la expresión génica y usos de los mismos Download PDFInfo
- Publication number
- ES2651147T3 ES2651147T3 ES13716415.8T ES13716415T ES2651147T3 ES 2651147 T3 ES2651147 T3 ES 2651147T3 ES 13716415 T ES13716415 T ES 13716415T ES 2651147 T3 ES2651147 T3 ES 2651147T3
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- oligonucleotides
- gene expression
- modulate gene
- nucleotides
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- 108091034117 Oligonucleotide Proteins 0.000 title abstract 2
- 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 1
- 230000014509 gene expression Effects 0.000 title 1
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 108020004491 Antisense DNA Proteins 0.000 abstract 1
- 102000053602 DNA Human genes 0.000 abstract 1
- 239000000427 antigen Substances 0.000 abstract 1
- 108091007433 antigens Proteins 0.000 abstract 1
- 102000036639 antigens Human genes 0.000 abstract 1
- 239000003816 antisense DNA Substances 0.000 abstract 1
- 230000000295 complement effect Effects 0.000 abstract 1
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical class O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
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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
- C12N15/1135—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 oncogenes or tumor suppressor genes
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- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/47—Quinolines; Isoquinolines
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- 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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- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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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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- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
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- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
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- Oncology (AREA)
- Neurology (AREA)
- Physics & Mathematics (AREA)
- Inorganic Chemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| IT000275A ITMI20120275A1 (it) | 2012-02-24 | 2012-02-24 | Oligonucleotidi per la modulazione dell'espressione genica e loro usi |
| ITMI20120275 | 2012-02-24 | ||
| PCT/IB2013/051410 WO2013124807A2 (en) | 2012-02-24 | 2013-02-21 | Oligonucleotides for modulating gene expression and uses thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2651147T3 true ES2651147T3 (es) | 2018-01-24 |
Family
ID=45999941
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES13716415.8T Active ES2651147T3 (es) | 2012-02-24 | 2013-02-21 | Oligonucleótidos para modular la expresión génica y usos de los mismos |
Country Status (28)
| Country | Link |
|---|---|
| US (2) | US10023867B2 (enExample) |
| EP (1) | EP2817407B1 (enExample) |
| JP (2) | JP6333739B2 (enExample) |
| KR (1) | KR101992925B1 (enExample) |
| CN (2) | CN104169421A (enExample) |
| AR (1) | AR090129A1 (enExample) |
| AU (2) | AU2013223694B2 (enExample) |
| BR (1) | BR112014020885B1 (enExample) |
| CA (1) | CA2864060C (enExample) |
| CY (1) | CY1120084T1 (enExample) |
| DK (1) | DK2817407T3 (enExample) |
| ES (1) | ES2651147T3 (enExample) |
| HK (1) | HK1201878A1 (enExample) |
| HU (1) | HUE034846T2 (enExample) |
| IN (1) | IN2014MN01493A (enExample) |
| IT (1) | ITMI20120275A1 (enExample) |
| LT (1) | LT2817407T (enExample) |
| MA (1) | MA35935B1 (enExample) |
| NO (1) | NO2817407T3 (enExample) |
| PL (1) | PL2817407T3 (enExample) |
| PT (1) | PT2817407T (enExample) |
| RS (1) | RS56631B1 (enExample) |
| RU (1) | RU2648140C2 (enExample) |
| SG (2) | SG11201404852WA (enExample) |
| SI (1) | SI2817407T1 (enExample) |
| SM (1) | SMT201800042T1 (enExample) |
| TN (1) | TN2014000327A1 (enExample) |
| WO (1) | WO2013124807A2 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ITMI20120275A1 (it) * | 2012-02-24 | 2013-08-25 | Biogenera Societa A Responsabilita Limitata | Oligonucleotidi per la modulazione dell'espressione genica e loro usi |
| US10174315B2 (en) | 2012-05-16 | 2019-01-08 | The General Hospital Corporation | Compositions and methods for modulating hemoglobin gene family expression |
| CN104540946A (zh) | 2012-05-16 | 2015-04-22 | Rana医疗有限公司 | 用于调节utrn表达的组合物和方法 |
| CN104583402A (zh) | 2012-05-16 | 2015-04-29 | Rana医疗有限公司 | 用于调节mecp2表达的组合物和方法 |
| JP2015523854A (ja) | 2012-05-16 | 2015-08-20 | ラナ セラピューティクス インコーポレイテッド | Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法 |
| EA201492120A1 (ru) | 2012-05-16 | 2015-10-30 | Рана Терапьютикс, Инк. | Композиции и способы для модулирования экспрессии atp2a2 |
| US10837014B2 (en) | 2012-05-16 | 2020-11-17 | Translate Bio Ma, Inc. | Compositions and methods for modulating SMN gene family expression |
| JP6811338B2 (ja) * | 2016-12-13 | 2021-01-13 | エーエム サイエンシーズ インコーポレイテッド | Ｂ型肝炎の予防または治療用医薬組成物 |
| EP4497432A4 (en) * | 2022-03-22 | 2025-07-30 | Bion Inc | PROTEIN DELIVERY COMPOSITION CONTAINING BILE SALT AND CATIONIC PEPTIDE AND USE THEREOF |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2300938A1 (en) * | 1997-08-20 | 1999-02-25 | Somagenics, Inc. | Antisense and antigene therapeutics with improved binding properties and methods for their use |
| WO2001061030A2 (en) * | 2000-02-14 | 2001-08-23 | Bollon Arthur P | LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION |
| JP2004524371A (ja) * | 2001-04-13 | 2004-08-12 | ザ・ポピュレイション・カウンシル，インコーポレイテッド | 核レセプターを介するペプチド核酸の細胞核内導入 |
| US20030087861A1 (en) * | 2001-05-17 | 2003-05-08 | Iversen Patrick L | Combined approach to treatment of cancer using a c-myc antisense oligomer |
| EP1448590A4 (en) * | 2002-02-20 | 2004-12-15 | Sirna Therapeutics Inc | Inhibition of the myc and myb genes mediated by RNA interference mediated or genes of the corresponding synthetic pathways |
| ITMI20030860A1 (it) * | 2003-04-29 | 2004-10-30 | Univ Bologna | Metodo per l'inibizione selettiva del gene n-myc |
| WO2005045032A2 (en) | 2003-10-20 | 2005-05-19 | Sima Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF EARLY GROWTH RESPONSE GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20050287667A1 (en) * | 2004-06-01 | 2005-12-29 | Pronai Therapeutics, Inc. | Methods and compositions for the inhibition of gene expression |
| GB0415263D0 (en) * | 2004-07-07 | 2004-08-11 | Norwegian Radium Hospital Res | Method |
| US20090202565A1 (en) * | 2004-10-25 | 2009-08-13 | Mark Aron Labow | Torc polynucleotides and polypeptides and method of use |
| US20090324587A1 (en) * | 2005-12-01 | 2009-12-31 | Neal Clifford Goodwin | Cancer Therapies and Pharmaceutical Compositions Used Therein |
| US7625874B2 (en) * | 2006-04-11 | 2009-12-01 | The Regents Of The University Of California | Compositions and methods for treating diseases associated with T-box and N-Myc |
| US20080038783A1 (en) * | 2006-06-29 | 2008-02-14 | Applera Corporation | Compositions and Methods Pertaining to Guanylation of PNA Oligomers |
| US20090047295A1 (en) | 2007-07-12 | 2009-02-19 | Berry David A | Methods and Compositions for Reducing Stemness in Oncogenesis |
| JP2011511051A (ja) * | 2008-02-05 | 2011-04-07 | アンティソマ リサーチ リミテッド | 生物学的物質およびそれらの使用 |
| KR20110091796A (ko) * | 2008-12-04 | 2011-08-12 | 오피케이오 큐알엔에이, 엘엘씨 | 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료 |
| CN102058535B (zh) * | 2010-12-21 | 2013-05-01 | 中国人民解放军总医院 | 叶酸受体靶向的新型脂质体 |
| ITMI20120275A1 (it) * | 2012-02-24 | 2013-08-25 | Biogenera Societa A Responsabilita Limitata | Oligonucleotidi per la modulazione dell'espressione genica e loro usi |
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