RU2426544C2 - Лечение нарушений цнс - Google Patents

Лечение нарушений цнс Download PDF

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Publication number
RU2426544C2
RU2426544C2 RU2008141162/15A RU2008141162A RU2426544C2 RU 2426544 C2 RU2426544 C2 RU 2426544C2 RU 2008141162/15 A RU2008141162/15 A RU 2008141162/15A RU 2008141162 A RU2008141162 A RU 2008141162A RU 2426544 C2 RU2426544 C2 RU 2426544C2
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RU
Russia
Prior art keywords
sirna
gene
sina
use according
nucleotides
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RU2008141162/15A
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English (en)
Russian (ru)
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RU2008141162A (ru
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АНТОН Ана Изабель ХИМЕНЕС (ES)
АНТОН Ана Изабель ХИМЕНЕС
ЯГЕ Анхела СЕСТО (ES)
ЯГЕ Анхела СЕСТО
ГОМЕС Мария Консепсьон ХИМЕНЕС (ES)
ГОМЕС Мария Консепсьон ХИМЕНЕС
ГУЛЬОН Эдуардо ГОМЕС-АСЕБО (ES)
ГУЛЬОН Эдуардо ГОМЕС-АСЕБО
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Силентис С.А.
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Publication of RU2008141162A publication Critical patent/RU2008141162A/ru
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Publication of RU2426544C2 publication Critical patent/RU2426544C2/ru

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    • 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

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  • 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)
RU2008141162/15A 2006-03-17 2007-03-16 Лечение нарушений цнс RU2426544C2 (ru)

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
GB0605337.5 2006-03-17

Publications (2)

Publication Number Publication Date
RU2008141162A RU2008141162A (ru) 2010-04-27
RU2426544C2 true RU2426544C2 (ru) 2011-08-20

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
RU2008141162/15A RU2426544C2 (ru) 2006-03-17 2007-03-16 Лечение нарушений цнс

Country Status (13)

Country Link
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=)

Families Citing this family (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005004794A2 (en) * 2003-06-09 2005-01-20 Alnylam Pharmaceuticals Inc. Method of treating neurodegenerative disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Регистр Лекарственных Средств России.: Издательство OOO «РЛС-2003», выпуск 10, 2003 г., с.749. Аравин А.А и др. Роль двухцепочечной РНК в подавлении экспрессии генов эукариот. - Молекулярная Биология, 2002, том 36, №2, с.240-251. *

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
IL194114A (en) 2012-06-28
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
US8871729B2 (en) 2014-10-28

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