JP2005508306A - 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物 - Google Patents

哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物 Download PDF

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JP2005508306A
JP2005508306A JP2003515539A JP2003515539A JP2005508306A JP 2005508306 A JP2005508306 A JP 2005508306A JP 2003515539 A JP2003515539 A JP 2003515539A JP 2003515539 A JP2003515539 A JP 2003515539A JP 2005508306 A JP2005508306 A JP 2005508306A
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rna
nucleic acid
kit
rnai
expression
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JP2005508306A5 (enExample
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マーク カイ
アントン マカフリー
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Leland Stanford Junior University
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Leland Stanford Junior University
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Environmental Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Animal Husbandry (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Diabetes (AREA)
  • Urology & Nephrology (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Rheumatology (AREA)
  • Pathology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
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JP2003515539A 2001-07-23 2002-07-19 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物 Withdrawn JP2005508306A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US30741101P 2001-07-23 2001-07-23
US36066402P 2002-02-27 2002-02-27
PCT/US2002/022869 WO2003010180A1 (en) 2001-07-23 2002-07-19 Methods and compositions for rnai mediated inhibition of gene expression in mammals

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2010249697A Division JP6055164B2 (ja) 2001-07-23 2010-11-08 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物
JP2014187281A Division JP2015044809A (ja) 2001-07-23 2014-09-16 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物

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JP2005508306A true JP2005508306A (ja) 2005-03-31
JP2005508306A5 JP2005508306A5 (enExample) 2006-01-05

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JP2003515539A Withdrawn JP2005508306A (ja) 2001-07-23 2002-07-19 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物
JP2010249697A Expired - Lifetime JP6055164B2 (ja) 2001-07-23 2010-11-08 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物
JP2014187281A Pending JP2015044809A (ja) 2001-07-23 2014-09-16 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物
JP2016159918A Expired - Lifetime JP6247355B2 (ja) 2001-07-23 2016-08-17 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物

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JP2014187281A Pending JP2015044809A (ja) 2001-07-23 2014-09-16 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物
JP2016159918A Expired - Lifetime JP6247355B2 (ja) 2001-07-23 2016-08-17 哺乳類における遺伝子発現のＲＮＡｉによる阻害に関する方法および組成物

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US (3) US20030153519A1 (enExample)
EP (2) EP2280070B1 (enExample)
JP (4) JP2005508306A (enExample)
AT (1) ATE556720T1 (enExample)
CA (2) CA2936534C (enExample)
DK (2) DK1409506T3 (enExample)
ES (2) ES2546829T3 (enExample)
PT (1) PT2280070E (enExample)
WO (1) WO2003010180A1 (enExample)

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