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
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- A—HUMAN NECESSITIES
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- 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
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- 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
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- 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
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- 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)
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- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
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- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Chemical Kinetics & Catalysis (AREA)
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- Biophysics (AREA)
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- Plant Pathology (AREA)
- Psychology (AREA)
- Epidemiology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (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)
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| 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 |
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| IL194114A0 IL194114A0 (en) | 2011-08-01 |
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| EP (1) | EP2004823B1 (enExample) |
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| KR (2) | KR101669944B1 (enExample) |
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| MX (1) | MX2008011731A (enExample) |
| RU (1) | RU2426544C2 (enExample) |
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| EP2145014B1 (en) * | 2007-04-05 | 2012-12-12 | The J. David Gladstone Institutes | Agents that reduce neuronal overexcitation |
| US20110288155A1 (en) | 2008-12-18 | 2011-11-24 | Elena Feinstein | Sirna compounds and methods of use thereof |
| RU2562861C2 (ru) * | 2009-09-11 | 2015-09-10 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии гентингтина |
| 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 |
| US8778904B2 (en) | 2009-12-09 | 2014-07-15 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the CNS |
| AU2011219029B2 (en) * | 2010-02-26 | 2017-02-02 | Columbia University | 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 |
| DK2560687T3 (en) | 2010-04-19 | 2017-09-18 | Nlife Therapeutics S L | Preparations and Methods for Selective Delivery of Oligonucleotide Molecules to Specific Neuron Types |
| US9260471B2 (en) | 2010-10-29 | 2016-02-16 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA) |
| EP2649181B1 (en) | 2010-12-06 | 2016-04-27 | Quark Pharmaceuticals, Inc. | Double stranded oligonucleotide compounds comprising positional modifications |
| US9949923B2 (en) * | 2011-03-15 | 2018-04-24 | Optinose As | Nasal delivery |
| WO2013053954A1 (en) * | 2011-10-14 | 2013-04-18 | Centre National De La Recherche Scientifique | Method of diagnosis, prognostic or treatment of neurodegenerative diseases |
| 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 |
| WO2013148260A1 (en) | 2012-03-30 | 2013-10-03 | Washington University | Methods for modulating tau expression for reducing seizure and modifying a neurodegenerative syndrome |
| US9611473B2 (en) | 2012-09-12 | 2017-04-04 | Quark Pharmaceuticals, Inc. | Double-stranded nucleic acid compounds |
| AU2013336581A1 (en) | 2012-10-26 | 2015-06-11 | Nlife Therapeutics, S.L. | Compositions and methods for the treatment of Parkinson disease by the selective delivery of oligonucleotide molecules to specific neuron types |
| ES2807379T3 (es) * | 2013-03-14 | 2021-02-22 | Ionis Pharmaceuticals Inc | Composiciones y métodos para regular la expresión de Tau |
| JP2016523980A (ja) * | 2013-07-11 | 2016-08-12 | ザ トラスティーズ オブ コロンビア ユニバーシティ イン ザ シティ オブ ニューヨーク | タウ発現を抑制するマイクロｒｎａ |
| 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 |
| US20160208247A1 (en) | 2013-07-31 | 2016-07-21 | Qbi Enterprises Ltd. | Methods of use of sphingolipid polyalkylamine oligonucleotide compounds |
| JP2017505756A (ja) * | 2013-12-13 | 2017-02-23 | ザ ジェネラル ホスピタル コーポレイション | 可溶性高分子量（ｈｍｗ）タウ種およびその用途 |
| WO2016151523A1 (en) * | 2015-03-25 | 2016-09-29 | Università Degli Studi Di Trento | Rna interference mediated therapy for neurodegenerative diseases |
| JOP20200228A1 (ar) * | 2015-12-21 | 2017-06-16 | Novartis Ag | تركيبات وطرق لخفض تعبير البروتين tau |
| JOP20190065A1 (ar) | 2016-09-29 | 2019-03-28 | Ionis Pharmaceuticals Inc | مركبات وطرق لتقليل التعبير عن tau |
| 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 |
| AU2019233612A1 (en) | 2018-03-13 | 2020-09-10 | Janssen Pharmaceutica Nv | Modified oligonucleotides for use in treatment of tauopathies |
| MX2022011516A (es) * | 2020-03-18 | 2023-01-04 | Univ Massachusetts | Oligonucleotidos para la modulacion de mapt. |
| 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 |
| AU2023269281A1 (en) * | 2022-05-12 | 2024-10-10 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting mapt expression |
| CA3272944A1 (en) * | 2022-12-29 | 2024-07-04 | Voyager Therapeutics Inc | MAPPING COMPOSITIONS AND REGULATION METHODS |
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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 |
| WO2005003350A2 (en) | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
| JP2004532849A (ja) | 2001-04-20 | 2004-10-28 | カイロン コーポレイション | 中枢神経系へのポリヌクレオチド薬剤の送達 |
| 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 |
| 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) |
| AU2003207708A1 (en) | 2002-02-20 | 2003-09-09 | Sirna Therapeutics, Inc. | Rna interference mediated inhibition of map kinase genes |
| CA2482904A1 (en) | 2002-04-18 | 2003-10-23 | Lynkeus Biotech Gmbh | Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification |
| 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 |
| ATE513843T1 (de) | 2002-09-25 | 2011-07-15 | Univ Massachusetts | Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna |
| AU2003279004B2 (en) | 2002-09-28 | 2009-10-08 | Massachusetts Institute Of Technology | Influenza therapeutic |
| ES2334125T3 (es) * | 2002-11-04 | 2010-03-05 | University Of Massachusetts | Interferencia de arn especifico de alelos. |
| US7829694B2 (en) | 2002-11-26 | 2010-11-09 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| EP1635763B1 (en) * | 2003-06-09 | 2012-08-08 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| WO2005044976A2 (en) | 2003-06-20 | 2005-05-19 | Isis Pharmaceuticals, Inc. | Oligomeric compounds for use in gene modulation |
| 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) |
| EP1735443A2 (en) | 2004-04-14 | 2006-12-27 | 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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Also Published As
| Publication number | Publication date |
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| EP2004823A2 (en) | 2008-12-24 |
| EP2004823B1 (en) | 2016-05-25 |
| US20090176728A1 (en) | 2009-07-09 |
| JP5923466B2 (ja) | 2016-05-24 |
| AU2007228570B2 (en) | 2013-09-19 |
| GB0605337D0 (en) | 2006-04-26 |
| CA2645120A1 (en) | 2007-09-27 |
| RU2008141162A (ru) | 2010-04-27 |
| ES2582649T3 (es) | 2016-09-14 |
| IL194114A0 (en) | 2011-08-01 |
| JP2009530257A (ja) | 2009-08-27 |
| AU2007228570A8 (en) | 2008-10-23 |
| JP5781722B2 (ja) | 2015-09-24 |
| MX2008011731A (es) | 2009-03-05 |
| WO2007107789A2 (en) | 2007-09-27 |
| JP2013234197A (ja) | 2013-11-21 |
| CN101448944A (zh) | 2009-06-03 |
| AU2007228570A1 (en) | 2007-09-27 |
| CA2645120C (en) | 2015-02-24 |
| US8871729B2 (en) | 2014-10-28 |
| HK1126812A1 (zh) | 2009-09-11 |
| CN101448944B (zh) | 2014-02-12 |
| RU2426544C2 (ru) | 2011-08-20 |
| KR101605144B1 (ko) | 2016-03-21 |
| WO2007107789A3 (en) | 2008-05-08 |
| KR20140076634A (ko) | 2014-06-20 |
| KR20080111063A (ko) | 2008-12-22 |
| KR101669944B1 (ko) | 2016-10-28 |
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