JP2010500025A - 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 - Google Patents

霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 Download PDF

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JP2010500025A
JP2010500025A JP2009523833A JP2009523833A JP2010500025A JP 2010500025 A JP2010500025 A JP 2010500025A JP 2009523833 A JP2009523833 A JP 2009523833A JP 2009523833 A JP2009523833 A JP 2009523833A JP 2010500025 A JP2010500025 A JP 2010500025A
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nucleic acid
strand
seq
expression
vector
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JP2010500025A5 (enExample
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ケンメラー，ウィリアム・エフ
ケイトー，マイケル・ディー
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Medtronic Inc
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Medtronic Inc
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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
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2009523833A 2006-08-09 2007-08-08 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列 Pending JP2010500025A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US11/501,634 US7902352B2 (en) 2005-05-06 2006-08-09 Isolated nucleic acid duplex for reducing huntington gene expression
PCT/US2007/017638 WO2008021136A2 (en) 2006-08-09 2007-08-08 Methods and sequences to suppress primate huntington gene expression in vivo

Publications (2)

Publication Number Publication Date
JP2010500025A true JP2010500025A (ja) 2010-01-07
JP2010500025A5 JP2010500025A5 (enExample) 2011-10-20

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JP2009523833A Pending JP2010500025A (ja) 2006-08-09 2007-08-08 霊長類ハンチントン遺伝子発現をｉｎｖｉｖｏで抑制する方法および配列

Country Status (5)

Country Link
US (1) US7902352B2 (enExample)
EP (1) EP2056881A4 (enExample)
JP (1) JP2010500025A (enExample)
CN (1) CN101557831A (enExample)
WO (1) WO2008021136A2 (enExample)

Cited By (2)

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JP2018506304A (ja) * 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
US11603529B2 (en) 2017-09-22 2023-03-14 Genzyme Corporation Variant RNAi

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US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
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RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
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CN104661664B (zh) 2012-07-13 2020-07-03 波涛生命科学有限公司 手性控制
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
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JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
AU2015264263B2 (en) 2014-05-20 2021-08-05 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
BR112023019981A2 (pt) 2021-03-29 2023-12-12 Alnylam Pharmaceuticals Inc Composições do agente irna de huntingtina (htt) e métodos de uso das mesmas

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