JP4316373B2 - ヒトのアセチルコリンエステラーゼ(ache)に対するアンチセンスオリゴヌクレオチド及びその使用 - Google Patents

ヒトのアセチルコリンエステラーゼ(ache)に対するアンチセンスオリゴヌクレオチド及びその使用 Download PDF

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JP4316373B2
JP4316373B2 JP2003509100A JP2003509100A JP4316373B2 JP 4316373 B2 JP4316373 B2 JP 4316373B2 JP 2003509100 A JP2003509100 A JP 2003509100A JP 2003509100 A JP2003509100 A JP 2003509100A JP 4316373 B2 JP4316373 B2 JP 4316373B2
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ache
antisense
muscle
mrna
pharmaceutical composition
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JP2004536103A (ja
JP2004536103A5 (enExample
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ソレク・ヘルモナ
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イスム リサーチ ディベロップメント カンパニー オブ ザ ヘブライ ユニバーシティ オブ エルサレム
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    • 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
    • C12N15/1137—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 against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/01—Carboxylic ester hydrolases (3.1.1)
    • C12Y301/01007—Acetylcholinesterase (3.1.1.7)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
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  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
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  • Pain & Pain Management (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2003509100A 2001-05-24 2002-05-23 ヒトのアセチルコリンエステラーゼ(ache)に対するアンチセンスオリゴヌクレオチド及びその使用 Expired - Fee Related JP4316373B2 (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IL143379A IL143379A (en) 2001-05-24 2001-05-24 Oligonucleotide against human ache isoform r and its uses
PCT/IL2002/000411 WO2003002739A1 (en) 2001-05-24 2002-05-23 Antisense oligonucleotide against human acetylcholinesterase (ache) and uses thereof

Publications (3)

Publication Number Publication Date
JP2004536103A JP2004536103A (ja) 2004-12-02
JP2004536103A5 JP2004536103A5 (enExample) 2006-01-05
JP4316373B2 true JP4316373B2 (ja) 2009-08-19

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US (3) US7074915B2 (enExample)
EP (1) EP1390493B1 (enExample)
JP (1) JP4316373B2 (enExample)
AU (1) AU2002256873B2 (enExample)
CA (1) CA2458806C (enExample)
ES (1) ES2397302T3 (enExample)
IL (1) IL143379A (enExample)
NZ (1) NZ529549A (enExample)
WO (1) WO2003002739A1 (enExample)

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JP6047270B2 (ja) 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー.フェー. Dnaリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
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US20060178333A1 (en) 2006-08-10
IL143379A0 (en) 2002-04-21
WO2003002739A1 (en) 2003-01-09
ES2397302T3 (es) 2013-03-06
US20090281165A1 (en) 2009-11-12
US7074915B2 (en) 2006-07-11
EP1390493A1 (en) 2004-02-25
US7456154B2 (en) 2008-11-25
CA2458806A1 (en) 2003-01-09
IL143379A (en) 2013-11-28
CA2458806C (en) 2012-10-09
US20030216344A1 (en) 2003-11-20
NZ529549A (en) 2008-07-31
AU2002256873B2 (en) 2007-07-05
EP1390493B1 (en) 2012-11-07

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