MX340397B - Modulacion de expresion de huntingtina. - Google Patents
Modulacion de expresion de huntingtina.Info
- Publication number
- MX340397B MX340397B MX2012003006A MX2012003006A MX340397B MX 340397 B MX340397 B MX 340397B MX 2012003006 A MX2012003006 A MX 2012003006A MX 2012003006 A MX2012003006 A MX 2012003006A MX 340397 B MX340397 B MX 340397B
- Authority
- MX
- Mexico
- Prior art keywords
- modulation
- huntingtin expression
- compositions
- compounds
- methods
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 abstract 2
- 239000000203 mixture Substances 0.000 abstract 2
- 208000023105 Huntington disease Diseases 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 208000024891 symptom Diseases 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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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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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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- C12N2310/10—Type of nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- 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
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- 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 ===---===
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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Abstract
La presente invención se refiere a métodos, compuestos y composiciones para reducir la expresión de ARNm y proteína de huntingtina en un animal. Dichos métodos, compuestos, y composiciones son útiles para tratar, prevenir, retrasar, o mejorar la enfermedad de Huntington, o un síntoma de la misma. Un oligonucleótido de cadena individual dirigido a un ácido nucleico que codifica para huntingtina y que puede inhibir la expresión de huntingtina, en el cual el oligonucleótido modificado comprende: un segmento de espacio que consiste de diez desoxinucleósidos ligados; un segmento de ala 5´que consiste de cinco nucleósidos ligados; y un segmento de la 3´que consiste de cinco nucleósidos ligados; en el cual el segmento de espacio está posicionado entre el segmento de ala 5´y el segmento de la 3´, en el cual cada nucleósido de cada segmento de ala comprende un azúcar 2´-O-metoxietilado; en el cual los enlazamientos internucleósido dentro del segmento de espacio, los enlazamientos que conectan el segmento de espacio al segmento de ala 5´o 3´, y los enlazamientos para los nucleósidos más hacia el extremo 5´y más hacia el extremo 3´de cada segmento de ala son todos enlazamientos de fosforotioato, los enlazamientos internucleósido que conectan el resto de los nucleósidos de los segmentos de ala tanto 5´como 3´son enlazamientos de fosfoldiéster; y en el cual todas la scitosinas son 5-metilcitosinas; y en donde la secuencia de nucleobase del iligonucleótido consiste de una secuencia recitada en SEQ ID NO: 22 ó 32.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US24185309P | 2009-09-11 | 2009-09-11 | |
| PCT/US2010/048532 WO2011032045A1 (en) | 2009-09-11 | 2010-09-10 | Modulation of huntingtin expression |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| MX2012003006A MX2012003006A (es) | 2012-06-28 |
| MX340397B true MX340397B (es) | 2016-07-07 |
Family
ID=43732828
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017016415A MX380175B (es) | 2009-09-11 | 2010-09-10 | Modulacion de expresion de huntingtina. |
| MX2012003006A MX340397B (es) | 2009-09-11 | 2010-09-10 | Modulacion de expresion de huntingtina. |
| MX2016004031A MX353152B (es) | 2009-09-11 | 2012-03-09 | Modulación de expresión de huntingtina. |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017016415A MX380175B (es) | 2009-09-11 | 2010-09-10 | Modulacion de expresion de huntingtina. |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2016004031A MX353152B (es) | 2009-09-11 | 2012-03-09 | Modulación de expresión de huntingtina. |
Country Status (19)
| Country | Link |
|---|---|
| US (8) | US8906873B2 (es) |
| EP (3) | EP3178933B1 (es) |
| JP (5) | JP5809146B2 (es) |
| KR (5) | KR102279458B1 (es) |
| CN (3) | CN111705058A (es) |
| AU (4) | AU2010292003B2 (es) |
| BR (1) | BR112012005448B1 (es) |
| CA (2) | CA2773886C (es) |
| DK (1) | DK2475675T3 (es) |
| ES (2) | ES2772825T3 (es) |
| HU (1) | HUE033113T2 (es) |
| IL (4) | IL218482A (es) |
| MX (3) | MX380175B (es) |
| NZ (3) | NZ624783A (es) |
| PL (1) | PL2475675T3 (es) |
| PT (1) | PT2475675T (es) |
| RU (2) | RU2751847C9 (es) |
| SI (1) | SI2475675T1 (es) |
| WO (1) | WO2011032045A1 (es) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2203173T3 (pl) | 2007-10-26 | 2016-06-30 | Academisch Ziekenhuis Leiden | Środki i sposoby przeciwdziałania zaburzeniom mięśni |
| EP2119783A1 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| AU2009276763B2 (en) * | 2008-07-29 | 2015-07-16 | The Board Of Regents Of The University Of Texas Sytem | Selective inhibition of polyglutamine protein expression |
| CA2744987C (en) | 2008-12-02 | 2018-01-16 | Chiralgen, Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
| CA2767253A1 (en) | 2009-07-06 | 2011-01-13 | Ontorii, Inc. | Novel nucleic acid prodrugs and methods of use thereof |
| KR102279458B1 (ko) | 2009-09-11 | 2021-07-21 | 아이오니스 파마수티컬즈, 인코포레이티드 | 헌팅틴 발현의 조절 |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| US8569254B2 (en) | 2010-12-10 | 2013-10-29 | National Yang Ming University | Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same |
| CA2833223A1 (en) * | 2011-04-22 | 2012-10-26 | Prosensa Technologies B.V. | New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1) |
| CA2839437A1 (en) * | 2011-06-16 | 2012-12-20 | Isis Pharmaceuticals, Inc. | Antisense modulation of fibroblast growth factor receptor 4 expression |
| WO2013012758A1 (en) | 2011-07-19 | 2013-01-24 | Ontorii, Inc. | Methods for the synthesis of functionalized nucleic acids |
| JP2015509922A (ja) | 2012-01-27 | 2015-04-02 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するrna調節オリゴヌクレオチド |
| EP3336189A1 (en) * | 2012-04-20 | 2018-06-20 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| JP6268157B2 (ja) | 2012-07-13 | 2018-01-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| KR102712879B1 (ko) | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| CN104684923B (zh) | 2012-07-13 | 2018-09-28 | 株式会社新日本科学 | 手性核酸佐剂 |
| EP4144845B1 (en) | 2012-10-12 | 2024-04-24 | Ionis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
| TWI702046B (zh) | 2013-07-19 | 2020-08-21 | 美商Ionis製藥公司 | 用於調節τ蛋白表現之組合物 |
| CN103601798B (zh) * | 2013-11-11 | 2015-11-18 | 杭州璞题生物科技有限公司 | 亨廷顿蛋白的酰基化修饰方法 |
| US10144933B2 (en) | 2014-01-15 | 2018-12-04 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| EP4137572A1 (en) | 2014-01-16 | 2023-02-22 | Wave Life Sciences Ltd. | Chiral design |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric sirna |
| CA2978521A1 (en) | 2015-05-29 | 2016-12-08 | National Yang-Ming University | Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| CA2995110A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| MX382671B (es) | 2015-12-10 | 2025-03-13 | Ptc Therapeutics Inc | Compuestos para usarse en el tratamiento o mejoramiento de la enfermedad de huntington |
| EP3416663B1 (en) | 2016-02-18 | 2021-04-14 | The Penn State Research Foundation | Generating gabaergic neurons in brains |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US10457940B2 (en) * | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3548005A4 (en) | 2016-11-29 | 2020-06-17 | Puretech Health LLC | Exosomes for delivery of therapeutic agents |
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| CA3064590A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | Two-tailed self-delivering sirna and related methods |
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