KR101669944B1 - Ｃｎｓ 질환의 치료 - Google Patents

Ｃｎｓ 질환의 치료 Download PDF

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KR101669944B1
KR101669944B1 KR1020087025174A KR20087025174A KR101669944B1 KR 101669944 B1 KR101669944 B1 KR 101669944B1 KR 1020087025174 A KR1020087025174 A KR 1020087025174A KR 20087025174 A KR20087025174 A KR 20087025174A KR 101669944 B1 KR101669944 B1 KR 101669944B1
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homo sapiens
rna
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KR20080111063A (ko
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안톤 아나 이사벨 지메네즈
야구에 안젤라 세스토
고메즈 마 콘셉시온 지메니즈
굴론 에두아르도 고메즈-아세보
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실렌티스 에스.에이.유.
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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)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • 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)
KR1020087025174A 2006-03-17 2007-03-16 Ｃｎｓ 질환의 치료 Active KR101669944B1 (ko)

Applications Claiming Priority (3)

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
PCT/GB2007/050128 WO2007107789A2 (en) 2006-03-17 2007-03-16 Treatment of cns conditions

Related Child Applications (1)

Application Number Title Priority Date Filing Date
KR1020147013792A Division KR101605144B1 (ko) 2006-03-17 2007-03-16 Ｃｎｓ 질환의 치료

Publications (2)

Publication Number Publication Date
KR20080111063A KR20080111063A (ko) 2008-12-22
KR101669944B1 true KR101669944B1 (ko) 2016-10-28

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KR1020087025174A Active KR101669944B1 (ko) 2006-03-17 2007-03-16 Ｃｎｓ 질환의 치료
KR1020147013792A Active KR101605144B1 (ko) 2006-03-17 2007-03-16 Ｃｎｓ 질환의 치료

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Country Link
US (1) US8871729B2 (enExample)
EP (1) EP2004823B1 (enExample)
JP (2) JP5781722B2 (enExample)
KR (2) KR101669944B1 (enExample)
CN (1) CN101448944B (enExample)
AU (1) AU2007228570B2 (enExample)
CA (1) CA2645120C (enExample)
ES (1) ES2582649T3 (enExample)
GB (1) GB0605337D0 (enExample)
IL (1) IL194114A (enExample)
MX (1) MX2008011731A (enExample)
RU (1) RU2426544C2 (enExample)
WO (1) WO2007107789A2 (enExample)

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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
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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
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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
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Also Published As

Publication number Publication date
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
IL194114A (en) 2012-06-28
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

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