CN102625809B - 亨廷顿表达的调节 - Google Patents
亨廷顿表达的调节 Download PDFInfo
- Publication number
- CN102625809B CN102625809B CN201080051393.9A CN201080051393A CN102625809B CN 102625809 B CN102625809 B CN 102625809B CN 201080051393 A CN201080051393 A CN 201080051393A CN 102625809 B CN102625809 B CN 102625809B
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- A61K31/00—Medicinal preparations containing organic active ingredients
- 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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- 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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- 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
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
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- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biomedical Technology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Genetics & Genomics (AREA)
- Neurology (AREA)
- Molecular Biology (AREA)
- Neurosurgery (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Diabetes (AREA)
- Physical Education & Sports Medicine (AREA)
- Psychiatry (AREA)
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- Cardiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
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- Microbiology (AREA)
- Hospice & Palliative Care (AREA)
- Rheumatology (AREA)
- Endocrinology (AREA)
- Pain & Pain Management (AREA)
- Heart & Thoracic Surgery (AREA)
- Hematology (AREA)
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202010532625.8A CN111705058A (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
| CN201510262406.1A CN104894129B (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US24185309P | 2009-09-11 | 2009-09-11 | |
| US61/241,853 | 2009-09-11 | ||
| PCT/US2010/048532 WO2011032045A1 (en) | 2009-09-11 | 2010-09-10 | Modulation of huntingtin expression |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202010532625.8A Division CN111705058A (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
| CN201510262406.1A Division CN104894129B (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN102625809A CN102625809A (zh) | 2012-08-01 |
| CN102625809B true CN102625809B (zh) | 2015-06-24 |
Family
ID=43732828
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201080051393.9A Active CN102625809B (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
| CN201510262406.1A Active CN104894129B (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
| CN202010532625.8A Pending CN111705058A (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201510262406.1A Active CN104894129B (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
| CN202010532625.8A Pending CN111705058A (zh) | 2009-09-11 | 2010-09-10 | 亨廷顿表达的调节 |
Country Status (19)
| Country | Link |
|---|---|
| US (8) | US8906873B2 (enExample) |
| EP (3) | EP3178933B1 (enExample) |
| JP (5) | JP5809146B2 (enExample) |
| KR (5) | KR101774526B1 (enExample) |
| CN (3) | CN102625809B (enExample) |
| AU (4) | AU2010292003B2 (enExample) |
| BR (1) | BR112012005448B1 (enExample) |
| CA (2) | CA2931725C (enExample) |
| DK (1) | DK2475675T3 (enExample) |
| ES (2) | ES2615534T3 (enExample) |
| HU (1) | HUE033113T2 (enExample) |
| IL (4) | IL218482A (enExample) |
| MX (3) | MX340397B (enExample) |
| NZ (3) | NZ714887A (enExample) |
| PL (1) | PL2475675T3 (enExample) |
| PT (1) | PT2475675T (enExample) |
| RU (2) | RU2562861C2 (enExample) |
| SI (1) | SI2475675T1 (enExample) |
| WO (1) | WO2011032045A1 (enExample) |
Families Citing this family (55)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2203173E (pt) | 2007-10-26 | 2016-03-15 | Academisch Ziekenhuis Leiden | Resumo |
| 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 |
| CA2732343C (en) * | 2008-07-29 | 2017-05-09 | The Board Of Regents Of The University Of Texas System | Selective inhibition of polyglutamine protein expression |
| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| RU2562861C2 (ru) | 2009-09-11 | 2015-09-10 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии гентингтина |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| 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 |
| WO2012144906A1 (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 |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| EP2839006B1 (en) * | 2012-04-20 | 2018-01-03 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| WO2014059341A2 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Antisense compounds and uses thereof |
| TW202246503A (zh) * | 2013-07-19 | 2022-12-01 | 美商百健Ma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 |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| 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 |
| 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 |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| KR20180030461A (ko) | 2015-05-29 | 2018-03-23 | 더 보드 오브 트러스티스 오브 더 리랜드 스탠포드 쥬니어 유니버시티 | 연장된 뉴클레오타이드 반복을 포함하는 유전자의 유해 영향 감소를 위한 뉴클레오사이드 제제 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| CN108697709A (zh) | 2015-12-10 | 2018-10-23 | Ptc医疗公司 | 用于治疗亨廷顿病的方法 |
| EP3881857B1 (en) | 2016-02-18 | 2025-04-16 | 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 |
| AU2017368050A1 (en) | 2016-11-29 | 2019-06-20 | Puretech Lyt, Inc. | Exosomes for delivery of therapeutic agents |
| AU2018282154B2 (en) | 2017-06-05 | 2022-04-07 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| CN111182898B (zh) | 2017-06-28 | 2024-04-16 | Ptc医疗公司 | 用于治疗亨廷顿氏病的方法 |
| WO2019005993A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | METHODS OF TREATING HUNTINGTON'S DISEASE |
| BR112020019373A2 (pt) | 2018-03-27 | 2020-12-29 | Ptc Therapeutics, Inc. | Compostos para o tratamento da doença de hutington |
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