JP5401451B2 - 変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 - Google Patents

変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 Download PDF

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JP5401451B2
JP5401451B2 JP2010512808A JP2010512808A JP5401451B2 JP 5401451 B2 JP5401451 B2 JP 5401451B2 JP 2010512808 A JP2010512808 A JP 2010512808A JP 2010512808 A JP2010512808 A JP 2010512808A JP 5401451 B2 JP5401451 B2 JP 5401451B2
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nucleic acid
htt
acid molecule
expression
interfering nucleic
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JP2010530239A (ja
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ペリン,ヴァレリー
デグロン,ニコル
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Commissariat a lEnergie Atomique et aux Energies Alternatives CEA
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
JP2010512808A 2007-06-18 2008-06-18 変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用 Expired - Fee Related JP5401451B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP07290751A EP2014769B1 (en) 2007-06-18 2007-06-18 Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
EP07290751.2 2007-06-18
PCT/IB2008/002603 WO2009007855A2 (en) 2007-06-18 2008-06-18 Reversible sirna-based silencing of mutant and endogenous wild-type huntingtin gene and its application for the treatment of huntington's disease

Publications (3)

Publication Number Publication Date
JP2010530239A JP2010530239A (ja) 2010-09-09
JP2010530239A5 JP2010530239A5 (https=) 2011-06-16
JP5401451B2 true JP5401451B2 (ja) 2014-01-29

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JP2010512808A Expired - Fee Related JP5401451B2 (ja) 2007-06-18 2008-06-18 変異および内因性野生型ハンチンチン遺伝子のsiRNAによる可逆的サイレンシングおよびハンチントン病の治療のためのその応用

Country Status (7)

Country Link
US (1) US8217018B2 (https=)
EP (1) EP2014769B1 (https=)
JP (1) JP5401451B2 (https=)
AT (1) ATE462787T1 (https=)
CA (1) CA2690730C (https=)
DE (1) DE602007005629D1 (https=)
WO (1) WO2009007855A2 (https=)

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* Cited by examiner, † Cited by third party
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CA2726866A1 (en) * 2008-05-09 2009-11-12 The University Of British Columbia Methods and compositions for the treatment of huntington's disease
US8679750B2 (en) 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
RU2612521C2 (ru) 2009-07-06 2017-03-09 Онтории, Инк. Новые пролекарства нуклеиновых кислот и способы их применения
CA2789038A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
US8957040B2 (en) 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2012004778A1 (en) * 2010-07-09 2012-01-12 Ecole Polytechnique Federale De Lausanne (Epfl) Method for in-vitro monitoring of neuronal disorders and use thereof
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Wave Life Sciences Japan 不斉補助基
CN103796657B (zh) 2011-07-19 2017-07-11 波涛生命科学有限公司 合成官能化核酸的方法
US10202599B2 (en) 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2013130824A1 (en) 2012-02-29 2013-09-06 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
CA2879066C (en) 2012-07-13 2019-08-13 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant
PL2872485T3 (pl) 2012-07-13 2021-05-31 Wave Life Sciences Ltd. Asymetryczna grupa pomocnicza
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
CN104968783B (zh) 2012-10-15 2019-12-10 Ionis制药公司 用于调节c9orf72表达的组合物
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
EP2986599A1 (en) 2013-04-17 2016-02-24 Pfizer Inc. N-piperidin-3-ylbenzamide derivatives for treating cardiovascular diseases
US10221414B2 (en) 2013-10-11 2019-03-05 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
WO2015070212A1 (en) * 2013-11-11 2015-05-14 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
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
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
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
BR112016016400A2 (pt) 2014-01-16 2017-10-03 Wave Life Sciences Ltd Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos
WO2015171932A1 (en) * 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
RS60230B1 (sr) 2015-04-16 2020-06-30 Ionis Pharmaceuticals Inc Kompozicije za moduliranje ekspresije c9orf72
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS

Also Published As

Publication number Publication date
EP2014769A1 (en) 2009-01-14
WO2009007855A3 (en) 2009-04-02
CA2690730C (en) 2018-07-31
ATE462787T1 (de) 2010-04-15
EP2014769B1 (en) 2010-03-31
US8217018B2 (en) 2012-07-10
CA2690730A1 (en) 2009-01-15
WO2009007855A2 (en) 2009-01-15
DE602007005629D1 (de) 2010-05-12
JP2010530239A (ja) 2010-09-09
US20100299768A1 (en) 2010-11-25

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