PE20211238A1 - Tratamiento antisentido del sindrome de angelman - Google Patents
Tratamiento antisentido del sindrome de angelmanInfo
- Publication number
- PE20211238A1 PE20211238A1 PE2020000649A PE2020000649A PE20211238A1 PE 20211238 A1 PE20211238 A1 PE 20211238A1 PE 2020000649 A PE2020000649 A PE 2020000649A PE 2020000649 A PE2020000649 A PE 2020000649A PE 20211238 A1 PE20211238 A1 PE 20211238A1
- Authority
- PE
- Peru
- Prior art keywords
- seq
- ube3a
- oligonucleotides
- angelman syndrome
- treatment
- Prior art date
Links
- 208000009575 Angelman syndrome Diseases 0.000 title abstract 2
- 230000000692 anti-sense effect Effects 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 3
- 108700028369 Alleles Proteins 0.000 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 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 230000008775 paternal effect Effects 0.000 abstract 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 abstract 1
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 1
- 108091046869 Telomeric non-coding RNA Proteins 0.000 abstract 1
- 102000006275 Ubiquitin-Protein Ligases Human genes 0.000 abstract 1
- 108010083111 Ubiquitin-Protein Ligases Proteins 0.000 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 238000009396 hybridization Methods 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 150000007523 nucleic acids Chemical group 0.000 abstract 1
- 102000004169 proteins and genes Human genes 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
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- 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
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
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- C12N2310/341—Gapmers, i.e. of the type ===---===
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- Health & Medical Sciences (AREA)
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Abstract
En la presente invencion se refiere a oligonucleotidos antisentido que son capaces de inducir la expresion de la proteina ubiquitina ligasa E3A (UBE3A), dicho oligonucleotido comprende una secuencia de nucleotidos contigua de 10 a 30 nucleotidos de longitud con al menos 98 % de complementariedad con una porcion contigua de la secuencia de acido nucleico SEQ ID NO:1, SEQ ID NO:2, SEQ ID NO:3, SEQ ID NO:4 o SEQ ID NO:5 o SEQ ID NO: 6, 7, 8, 9, 10 u 11. Los oligonucleotidos se dirigen al supresor del alelo paterno de UBE3A por hibridacion al ARN no codificante largo de SNHG14 en el extremo 5' de UBE3A-AS lo cual hace que el alelo paterno se inactive y no codifique proteinas, dichos oligonucleotidos son utiles para el tratamiento del sindrome de Angelman.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593431P | 2017-12-01 | 2017-12-01 | |
| US201862676034P | 2018-05-24 | 2018-05-24 | |
| PCT/US2018/063416 WO2019109001A1 (en) | 2017-12-01 | 2018-11-30 | Angelman syndrome antisense treatment |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20211238A1 true PE20211238A1 (es) | 2021-07-09 |
Family
ID=66664231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2020000649A PE20211238A1 (es) | 2017-12-01 | 2018-11-30 | Tratamiento antisentido del sindrome de angelman |
Country Status (16)
| Country | Link |
|---|---|
| US (5) | US11198869B2 (es) |
| EP (2) | EP3717646A4 (es) |
| JP (2) | JP7297320B2 (es) |
| KR (2) | KR102723989B1 (es) |
| CN (2) | CN111433361B (es) |
| AU (1) | AU2018375807A1 (es) |
| BR (1) | BR112020009431A2 (es) |
| CA (1) | CA3079755A1 (es) |
| CL (1) | CL2020001344A1 (es) |
| CO (1) | CO2020006361A2 (es) |
| IL (1) | IL274146B2 (es) |
| MX (1) | MX2020005680A (es) |
| PE (1) | PE20211238A1 (es) |
| RU (1) | RU2020121752A (es) |
| SG (1) | SG11202003596WA (es) |
| WO (1) | WO2019109001A1 (es) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2016352836B2 (en) | 2015-11-12 | 2020-06-25 | F. Hoffmann-La Roche Ag | Oligonucleotides for inducing paternal UBE3A expression |
| CA3091912A1 (en) * | 2018-02-27 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| CA3135180A1 (en) | 2019-03-29 | 2020-10-08 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating ube3a-ats |
| US11732263B2 (en) * | 2020-06-29 | 2023-08-22 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating PLP1 |
| US20230416851A1 (en) * | 2020-10-06 | 2023-12-28 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| AU2023231550A1 (en) * | 2022-03-07 | 2024-09-26 | Ovid Therapeutics Inc. | shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME |
Family Cites Families (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2823959B2 (ja) | 1991-10-24 | 1998-11-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド |
| KR100872437B1 (ko) * | 2000-12-01 | 2008-12-05 | 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. | Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자 |
| EP1799859B1 (en) | 2004-09-17 | 2014-07-02 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| EP1676834A1 (en) | 2004-12-30 | 2006-07-05 | Sanofi-Aventis Deutschland GmbH | Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| WO2008109357A1 (en) * | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting apob gene expression and uses thereof |
| US8580756B2 (en) | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| DK2285819T3 (da) | 2008-04-04 | 2013-12-02 | Isis Pharmaceuticals Inc | Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider |
| EP2370451B1 (en) | 2008-12-02 | 2016-11-16 | Wave Life Sciences Japan, Inc. | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2011017521A2 (en) | 2009-08-06 | 2011-02-10 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| RU2013150331A (ru) | 2011-04-20 | 2015-05-27 | Рош Гликарт Аг | СПОСОБ И УСТРОЙСТВА ДЛЯ рН-ЗАВИСИМОГО ПРОХОЖДЕНИЯ ГЕМАТОЭНЦЕФАЛИЧЕСКОГО БАРЬЕРА |
| DK2751270T3 (en) | 2011-08-29 | 2018-10-29 | Ionis Pharmaceuticals Inc | OLIGOMER-CONJUGATE COMPLEXES AND THEIR USE |
| US9221864B2 (en) | 2012-04-09 | 2015-12-29 | Isis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| BR112014028634A2 (pt) * | 2012-05-16 | 2017-06-27 | Rana Therapeutics Inc | composições e métodos para modulação da expressão de utrn |
| US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
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| KR20240164968A (ko) | 2014-01-16 | 2024-11-21 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
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