JP2010530239A5 - - Google Patents
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- Publication number
- JP2010530239A5 JP2010530239A5 JP2010512808A JP2010512808A JP2010530239A5 JP 2010530239 A5 JP2010530239 A5 JP 2010530239A5 JP 2010512808 A JP2010512808 A JP 2010512808A JP 2010512808 A JP2010512808 A JP 2010512808A JP 2010530239 A5 JP2010530239 A5 JP 2010530239A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- acid molecule
- short interfering
- interfering nucleic
- double
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 22
- 230000002452 interceptive effect Effects 0.000 claims 20
- 108020004707 nucleic acids Proteins 0.000 claims 20
- 102000039446 nucleic acids Human genes 0.000 claims 20
- 239000002773 nucleotide Substances 0.000 claims 8
- 125000003729 nucleotide group Chemical group 0.000 claims 8
- 239000013604 expression vector Substances 0.000 claims 5
- 230000000692 anti-sense effect Effects 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 230000035897 transcription Effects 0.000 claims 3
- 238000013518 transcription Methods 0.000 claims 3
- 208000023105 Huntington disease Diseases 0.000 claims 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- 108091034117 Oligonucleotide Proteins 0.000 claims 2
- 108090000623 proteins and genes Proteins 0.000 claims 2
- 230000005026 transcription initiation Effects 0.000 claims 2
- 239000013598 vector Substances 0.000 claims 2
- 101001030705 Homo sapiens Huntingtin Proteins 0.000 claims 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 claims 1
- 230000002238 attenuated effect Effects 0.000 claims 1
- 230000002950 deficient Effects 0.000 claims 1
- 229960003722 doxycycline Drugs 0.000 claims 1
- XQTWDDCIUJNLTR-CVHRZJFOSA-N doxycycline monohydrate Chemical compound O.O=C1C2=C(O)C=CC=C2[C@H](C)[C@@H]2C1=C(O)[C@]1(O)C(=O)C(C(N)=O)=C(O)[C@@H](N(C)C)[C@@H]1[C@H]2O XQTWDDCIUJNLTR-CVHRZJFOSA-N 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 102000054185 human HTT Human genes 0.000 claims 1
- 230000000977 initiatory effect Effects 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 239000002213 purine nucleotide Substances 0.000 claims 1
- 150000003212 purines Chemical class 0.000 claims 1
- 150000003230 pyrimidines Chemical class 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
- 230000010076 replication Effects 0.000 claims 1
- 238000011808 rodent model Methods 0.000 claims 1
- 230000005030 transcription termination Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP07290751A EP2014769B1 (en) | 2007-06-18 | 2007-06-18 | Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease |
| EP07290751.2 | 2007-06-18 | ||
| PCT/IB2008/002603 WO2009007855A2 (en) | 2007-06-18 | 2008-06-18 | Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2010530239A JP2010530239A (ja) | 2010-09-09 |
| JP2010530239A5 true JP2010530239A5 (OSRAM) | 2011-06-16 |
| JP5401451B2 JP5401451B2 (ja) | 2014-01-29 |
Family
ID=38476952
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2010512808A Expired - Fee Related JP5401451B2 (ja) | 2007-06-18 | 2008-06-18 | 変異および内因性野生型ハンチンチン遺伝子のｓｉＲＮＡによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US8217018B2 (OSRAM) |
| EP (1) | EP2014769B1 (OSRAM) |
| JP (1) | JP5401451B2 (OSRAM) |
| AT (1) | ATE462787T1 (OSRAM) |
| CA (1) | CA2690730C (OSRAM) |
| DE (1) | DE602007005629D1 (OSRAM) |
| WO (1) | WO2009007855A2 (OSRAM) |
Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
| EP2297341A4 (en) * | 2008-05-09 | 2013-01-09 | Univ British Columbia | METHOD AND COMPOSITIONS FOR TREATING MORBUS HUNTINGTON |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| IN2012DN00720A (OSRAM) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
| EP2534262B1 (en) | 2010-02-08 | 2016-12-14 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| DK2534248T3 (en) | 2010-02-08 | 2018-11-19 | Ionis Pharmaceuticals Inc | SELECTIVE REDUCTION OF ALLELVARIANS |
| WO2012004778A1 (en) * | 2010-07-09 | 2012-01-12 | Ecole Polytechnique Federale De Lausanne (Epfl) | Method for in-vitro monitoring of neuronal disorders and use thereof |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| MX347361B (es) | 2011-07-19 | 2017-04-12 | Wave Life Sciences Ltd | Metodos para la sintesis de acidos nucleicos funcionalizados. |
| ES2651514T3 (es) | 2011-08-11 | 2018-01-26 | Ionis Pharmaceuticals, Inc. | Compuestos oligoméricos gapados que comprenden deoxirribonucleósidos modificados en 5 ' -en la brecha y usos de ellos |
| HK1203566A1 (zh) | 2012-02-29 | 2015-10-30 | 桑格摩生物科学股份有限公司 | 治療亨廷頓氏病的方法和組合物 |
| AU2013287630B2 (en) | 2012-07-13 | 2017-05-25 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| WO2014062736A1 (en) | 2012-10-15 | 2014-04-24 | Isis Pharmaceuticals, Inc. | Methods for monitoring c9orf72 expression |
| AU2013331434B2 (en) * | 2012-10-15 | 2019-08-08 | Ionis Pharmaceuticals, Inc. | Compositions for modulating C90RF72 expression |
| JP2016516804A (ja) | 2013-04-17 | 2016-06-09 | ファイザー・インク | 心血管疾患を治療するためのｎ−ピペリジン−３−イルベンズアミド誘導体 |
| RU2748426C2 (ru) | 2013-10-11 | 2021-05-25 | Ионис Фармасьютикалз, Инк. | Композиции для модуляции экспрессии c90rf72 |
| JP2016537341A (ja) * | 2013-11-11 | 2016-12-01 | サンガモ バイオサイエンシーズ， インコーポレイテッド | ハンチントン病を処置するための方法および組成物 |
| 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 |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | 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 |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| JP2017515819A (ja) * | 2014-05-08 | 2017-06-15 | サンガモ セラピューティクス， インコーポレイテッド | ハンチントン病を処置するための方法および組成物 |
| EP3722424A1 (en) | 2015-04-16 | 2020-10-14 | Ionis Pharmaceuticals, Inc. | Compositions for modulating c9orf72 expression |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US11260073B2 (en) | 2015-11-02 | 2022-03-01 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulating C90RF72 |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
-
2007
- 2007-06-18 DE DE602007005629T patent/DE602007005629D1/de active Active
- 2007-06-18 AT AT07290751T patent/ATE462787T1/de not_active IP Right Cessation
- 2007-06-18 EP EP07290751A patent/EP2014769B1/en not_active Not-in-force
-
2008
- 2008-06-18 CA CA2690730A patent/CA2690730C/en not_active Expired - Fee Related
- 2008-06-18 JP JP2010512808A patent/JP5401451B2/ja not_active Expired - Fee Related
- 2008-06-18 US US12/665,137 patent/US8217018B2/en not_active Expired - Fee Related
- 2008-06-18 WO PCT/IB2008/002603 patent/WO2009007855A2/en not_active Ceased
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