IL194114A - Use of RNA compound interferes with the preparation of drugs for the treatment of central nervous system disorder, medicinal preparations for intraperitoneal administration containing one or more SINAs directed to the gene expressed in the central nervous system. - Google Patents
Use of RNA compound interferes with the preparation of drugs for the treatment of central nervous system disorder, medicinal preparations for intraperitoneal administration containing one or more SINAs directed to the gene expressed in the central nervous system.Info
- Publication number
- IL194114A IL194114A IL194114A IL19411408A IL194114A IL 194114 A IL194114 A IL 194114A IL 194114 A IL194114 A IL 194114A IL 19411408 A IL19411408 A IL 19411408A IL 194114 A IL194114 A IL 194114A
- Authority
- IL
- Israel
- Prior art keywords
- cns
- sina
- target gene
- gene
- compound
- Prior art date
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- XXYIANZGUOSQHY-XLPZGREQSA-N thymidine 3'-monophosphate Chemical compound O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](OP(O)(O)=O)C1 XXYIANZGUOSQHY-XLPZGREQSA-N 0.000 description 1
- 230000001988 toxicity Effects 0.000 description 1
- 231100000419 toxicity Toxicity 0.000 description 1
- 210000003437 trachea Anatomy 0.000 description 1
- 230000010474 transient expression Effects 0.000 description 1
- 230000007704 transition Effects 0.000 description 1
- 238000013519 translation Methods 0.000 description 1
- QORWJWZARLRLPR-UHFFFAOYSA-H tricalcium bis(phosphate) Chemical compound [Ca+2].[Ca+2].[Ca+2].[O-]P([O-])([O-])=O.[O-]P([O-])([O-])=O QORWJWZARLRLPR-UHFFFAOYSA-H 0.000 description 1
- IHIXIJGXTJIKRB-UHFFFAOYSA-N trisodium vanadate Chemical compound [Na+].[Na+].[Na+].[O-][V]([O-])([O-])=O IHIXIJGXTJIKRB-UHFFFAOYSA-N 0.000 description 1
- 238000009827 uniform distribution Methods 0.000 description 1
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- 230000009385 viral infection Effects 0.000 description 1
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Classifications
-
- A—HUMAN NECESSITIES
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- A—HUMAN NECESSITIES
- 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
-
- A—HUMAN NECESSITIES
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- 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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K2039/54—Medicinal preparations containing antigens or antibodies characterised by the route of administration
- A61K2039/541—Mucosal route
- A61K2039/543—Mucosal route intranasal
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB0605337.5A GB0605337D0 (en) | 2006-03-17 | 2006-03-17 | Treatment of CNS conditions |
| PCT/GB2007/050128 WO2007107789A2 (en) | 2006-03-17 | 2007-03-16 | Treatment of cns conditions |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| IL194114A0 IL194114A0 (en) | 2011-08-01 |
| IL194114A true IL194114A (en) | 2012-06-28 |
Family
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Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
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| IL194114A IL194114A (en) | 2006-03-17 | 2008-09-15 | Use of RNA compound interferes with the preparation of drugs for the treatment of central nervous system disorder, medicinal preparations for intraperitoneal administration containing one or more SINAs directed to the gene expressed in the central nervous system. |
Country Status (13)
| Country | Link |
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| US (1) | US8871729B2 (https=) |
| EP (1) | EP2004823B1 (https=) |
| JP (2) | JP5781722B2 (https=) |
| KR (2) | KR101669944B1 (https=) |
| CN (1) | CN101448944B (https=) |
| AU (1) | AU2007228570B2 (https=) |
| CA (1) | CA2645120C (https=) |
| ES (1) | ES2582649T3 (https=) |
| GB (1) | GB0605337D0 (https=) |
| IL (1) | IL194114A (https=) |
| MX (1) | MX2008011731A (https=) |
| RU (1) | RU2426544C2 (https=) |
| WO (1) | WO2007107789A2 (https=) |
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| EP2145014B1 (en) * | 2007-04-05 | 2012-12-12 | The J. David Gladstone Institutes | Agents that reduce neuronal overexcitation |
| WO2010080452A2 (en) | 2008-12-18 | 2010-07-15 | Quark Pharmaceuticals, Inc. | siRNA COMPOUNDS AND METHODS OF USE THEREOF |
| NZ714887A (en) * | 2009-09-11 | 2019-07-26 | Ionis Pharmaceuticals Inc | Modulation of huntingtin expression |
| AU2010324658A1 (en) | 2009-11-26 | 2012-05-03 | Quark Pharmaceuticals, Inc. | siRNA compounds comprising terminal substitutions |
| US20110135613A1 (en) * | 2009-12-03 | 2011-06-09 | The J. David Gladstone Institutes | Methods for treating apolipoprotein e4-associated disorders |
| EP2862929B1 (en) | 2009-12-09 | 2017-09-06 | Quark Pharmaceuticals, Inc. | Compositions and methods for treating diseases, disorders or injury of the CNS |
| WO2011106104A2 (en) * | 2010-02-26 | 2011-09-01 | Memorial Sloan-Kettering Cancer Center | Methods and compositions for the detection and treatment of cancer involving mirnas and mirna inhibitors and targets |
| EP2380595A1 (en) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| CA2796722C (en) | 2010-04-19 | 2021-06-01 | Nlife Therapeutics, S.L. | Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types |
| WO2012058210A1 (en) * | 2010-10-29 | 2012-05-03 | Merck Sharp & Dohme Corp. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA) |
| AU2011338682B2 (en) | 2010-12-06 | 2017-04-27 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising threose modifications |
| WO2012123819A1 (en) * | 2011-03-15 | 2012-09-20 | Optinose As Et Al | Nasal delivery |
| US9486540B2 (en) * | 2012-03-09 | 2016-11-08 | Northeastern University | Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders |
| EP2831232A4 (en) * | 2012-03-30 | 2015-11-04 | Univ Washington | METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME |
| US20140302510A1 (en) * | 2012-04-06 | 2014-10-09 | Centre National De La Recherche Scientifique | Method of diagnosis, prognostic or treatment of neurodegenerative diseases |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| EP2911695B1 (en) | 2012-10-26 | 2023-11-29 | Palomo Limited | Compositions and methods for the treatment of parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| CN105264091B (zh) * | 2013-03-14 | 2020-02-28 | Ionis制药公司 | 用于调节tau表达的组合物和方法 |
| US9951330B2 (en) | 2013-07-11 | 2018-04-24 | The Trustees Of Columbia University In The City Of New York | Micrornas that silence tau expression |
| 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 |
| EP3027223A1 (en) | 2013-07-31 | 2016-06-08 | QBI Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| WO2015089375A1 (en) * | 2013-12-13 | 2015-06-18 | The General Hospital Corporation | Soluble high molecular weight (hmw) tau species and applications thereof |
| US10513701B2 (en) | 2015-03-25 | 2019-12-24 | Università Degli Studi Di Trento | RNA interference mediated therapy for neurodegenerative diseases |
| PH12018501207B1 (en) | 2015-12-21 | 2024-02-23 | Novartis Ag | Compositions and methods for decreasing tau expression |
| HUE065170T2 (hu) | 2016-09-29 | 2024-05-28 | Biogen Ma Inc | Vegyületek és módszerek a TAU expresszió csökkentésére |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| MX2020009532A (es) * | 2018-03-13 | 2020-10-05 | Janssen Pharmaceutica Nv | Oligonucleotidos modificados para uso en el tratamiento de tauopatias. |
| US20210363523A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for mapt modulation |
| AU2021246024A1 (en) * | 2020-03-30 | 2022-10-27 | Alnylam Pharmaceuticals, Inc. | Microtubule associated protein Tau (MAPT) iRNA agent compositions and methods of use thereof |
| CN113817728A (zh) * | 2020-06-19 | 2021-12-21 | 四川大学华西医院 | 一种有效干扰Tau蛋白表达的重组慢病毒及其应用 |
| TW202328449A (zh) * | 2021-09-24 | 2023-07-16 | 美商艾拉倫製藥公司 | 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法 |
| CN119173631A (zh) * | 2022-05-12 | 2024-12-20 | 迪克纳制药公司 | 用于抑制mapt表达的组合物和方法 |
| KR20250129743A (ko) * | 2022-12-29 | 2025-08-29 | 보이저 테라퓨틱스, 인크. | Mapt를 조절하기 위한 조성물 및 방법 |
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| CA2116460A1 (en) | 1993-03-02 | 1994-09-03 | Mitsubishi Chemical Corporation | Preventive or therapeutic agents for alzheimer's disease, a screening method of alzheimer's disease and tau-protein kinase i originated from human being |
| US6410046B1 (en) * | 1996-11-19 | 2002-06-25 | Intrabrain International Nv | Administering pharmaceuticals to the mammalian central nervous system |
| WO2002086105A1 (en) | 2001-04-20 | 2002-10-31 | Chiron Corporation | Delivery of polynucleotide agents to the central nervous sysstem |
| US20050159376A1 (en) * | 2002-02-20 | 2005-07-21 | Slrna Therapeutics, Inc. | RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA) |
| IL143379A (en) | 2001-05-24 | 2013-11-28 | Yissum Res Dev Co | Oligonucleotide against human ache isoform r and its uses |
| EP1432724A4 (en) | 2002-02-20 | 2006-02-01 | Sirna Therapeutics Inc | RNA inhibition mediated inhibition of MAP KINASE GENES |
| GB2406568B (en) | 2002-02-20 | 2005-09-28 | Sirna Therapeutics Inc | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
| EP1495120B1 (en) | 2002-04-18 | 2012-10-10 | Acuity Pharmaceuticals, Inc | Means and methods for the specific modulation of target genes in the eye |
| US20030225031A1 (en) | 2002-05-21 | 2003-12-04 | Quay Steven C. | Administration of acetylcholinesterase inhibitors to the cerebral spinal fluid |
| US20040254146A1 (en) | 2002-05-21 | 2004-12-16 | Nastech Pharmaceutical Company Inc. | Carboxylate salts of galantamine and their pharmaceutical use |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US20040241854A1 (en) | 2002-08-05 | 2004-12-02 | Davidson Beverly L. | siRNA-mediated gene silencing |
| US20050106731A1 (en) | 2002-08-05 | 2005-05-19 | Davidson Beverly L. | siRNA-mediated gene silencing with viral vectors |
| CA2881743A1 (en) | 2002-09-25 | 2004-04-08 | University Of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
| US20050008617A1 (en) | 2002-09-28 | 2005-01-13 | Massachusetts Institute Of Technology | Compositions and methods for delivery of short interfering RNA and short hairpin RNA |
| AU2003290586B2 (en) * | 2002-11-04 | 2009-07-02 | University Of Massachusetts | Allele-specific RNA interference |
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2005004794A2 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| EP1636342A4 (en) | 2003-06-20 | 2008-10-08 | Isis Pharmaceuticals Inc | OLIGOMER COMPOUNDS FOR USE IN THE MODULATION OF GENES |
| CA2528963A1 (en) | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina) |
| US20070185042A1 (en) | 2003-08-08 | 2007-08-09 | The Brigham And Women's Hospital, Inc. President And Fellows Of Harvard College | Sirna based methods for treating alzheimer's disease |
| WO2005045037A2 (en) | 2003-10-23 | 2005-05-19 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| WO2005105995A2 (en) | 2004-04-14 | 2005-11-10 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20050265927A1 (en) | 2004-05-17 | 2005-12-01 | Yale University | Intranasal delivery of nucleic acid molecules |
| WO2006041922A2 (en) | 2004-10-08 | 2006-04-20 | Dara Biosciences, Inc. | Agents and methods for administration to the central nervous system |
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2006
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2007
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- 2007-03-16 CN CN200780017978.7A patent/CN101448944B/zh active Active
- 2007-03-16 EP EP07733553.7A patent/EP2004823B1/en active Active
- 2007-03-16 CA CA2645120A patent/CA2645120C/en active Active
- 2007-03-16 US US12/293,230 patent/US8871729B2/en active Active
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- 2007-03-16 ES ES07733553.7T patent/ES2582649T3/es active Active
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- 2007-03-16 KR KR1020087025174A patent/KR101669944B1/ko active Active
- 2007-03-16 RU RU2008141162/15A patent/RU2426544C2/ru active
- 2007-03-16 KR KR1020147013792A patent/KR101605144B1/ko active Active
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Also Published As
| Publication number | Publication date |
|---|---|
| GB0605337D0 (en) | 2006-04-26 |
| WO2007107789A2 (en) | 2007-09-27 |
| JP5923466B2 (ja) | 2016-05-24 |
| EP2004823A2 (en) | 2008-12-24 |
| JP5781722B2 (ja) | 2015-09-24 |
| ES2582649T3 (es) | 2016-09-14 |
| RU2008141162A (ru) | 2010-04-27 |
| KR20080111063A (ko) | 2008-12-22 |
| CA2645120A1 (en) | 2007-09-27 |
| MX2008011731A (es) | 2009-03-05 |
| AU2007228570A1 (en) | 2007-09-27 |
| KR20140076634A (ko) | 2014-06-20 |
| US20090176728A1 (en) | 2009-07-09 |
| CA2645120C (en) | 2015-02-24 |
| HK1126812A1 (zh) | 2009-09-11 |
| IL194114A0 (en) | 2011-08-01 |
| WO2007107789A3 (en) | 2008-05-08 |
| CN101448944B (zh) | 2014-02-12 |
| JP2013234197A (ja) | 2013-11-21 |
| JP2009530257A (ja) | 2009-08-27 |
| AU2007228570B2 (en) | 2013-09-19 |
| KR101605144B1 (ko) | 2016-03-21 |
| KR101669944B1 (ko) | 2016-10-28 |
| EP2004823B1 (en) | 2016-05-25 |
| AU2007228570A8 (en) | 2008-10-23 |
| CN101448944A (zh) | 2009-06-03 |
| RU2426544C2 (ru) | 2011-08-20 |
| US8871729B2 (en) | 2014-10-28 |
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