JP5781722B2 - Ｃｎｓ状態の治療 - Google Patents

Ｃｎｓ状態の治療 Download PDF

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Publication number
JP5781722B2
JP5781722B2 JP2008558913A JP2008558913A JP5781722B2 JP 5781722 B2 JP5781722 B2 JP 5781722B2 JP 2008558913 A JP2008558913 A JP 2008558913A JP 2008558913 A JP2008558913 A JP 2008558913A JP 5781722 B2 JP5781722 B2 JP 5781722B2
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JP
Japan
Prior art keywords
sirna
sina
gene
strand
nucleotide
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JP2008558913A
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English (en)
Japanese (ja)
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JP2009530257A5 (enExample
JP2009530257A (ja
Inventor
アナ・イサベル・ヒメネス・アントン
アンジェラ・セスト・ジャゲ
マ・コンセプシオン・ヒメネス・ゴメス
エドゥアルド・ゴメス−アセボ・グジョン
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シレンティス・エセ・ア・ウ
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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

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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)
JP2008558913A 2006-03-17 2007-03-16 Ｃｎｓ状態の治療 Active JP5781722B2 (ja)

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
JP2013161765A Division JP5923466B2 (ja) 2006-03-17 2013-08-02 Ｃｎｓ状態の治療

Publications (3)

Publication Number Publication Date
JP2009530257A JP2009530257A (ja) 2009-08-27
JP2009530257A5 JP2009530257A5 (enExample) 2010-03-11
JP5781722B2 true JP5781722B2 (ja) 2015-09-24

Family

ID=36292900

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2008558913A Active JP5781722B2 (ja) 2006-03-17 2007-03-16 Ｃｎｓ状態の治療
JP2013161765A Active JP5923466B2 (ja) 2006-03-17 2013-08-02 Ｃｎｓ状態の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2013161765A Active JP5923466B2 (ja) 2006-03-17 2013-08-02 Ｃｎｓ状態の治療

Country Status (13)

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
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
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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
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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