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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JP2010530239A5 (enExample
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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)
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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)

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JP2010530239A JP2010530239A (ja) 2010-09-09
JP2010530239A5 JP2010530239A5 (enExample) 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)

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US (1) US8217018B2 (enExample)
EP (1) EP2014769B1 (enExample)
JP (1) JP5401451B2 (enExample)
AT (1) ATE462787T1 (enExample)
CA (1) CA2690730C (enExample)
DE (1) DE602007005629D1 (enExample)
WO (1) WO2009007855A2 (enExample)

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* Cited by examiner, † Cited by third party
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EP2297341A4 (en) * 2008-05-09 2013-01-09 Univ British Columbia METHOD AND COMPOSITIONS FOR TREATING MORBUS HUNTINGTON
US8679750B2 (en) 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
ES2616051T3 (es) 2008-12-02 2017-06-09 Wave Life Sciences Japan, Inc. Método para la síntesis de ácidos nucleicos modificados en el átomo de fósforo
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011097643A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP2591356A1 (en) * 2010-07-09 2013-05-15 Ecole Polytechnique Fédérale de Lausanne (EPFL) Method for in-vitro monitoring of neuronal disorders and use thereof
EP2620428B1 (en) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
JP6128529B2 (ja) 2011-07-19 2017-05-17 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. 官能化核酸の合成のための方法
ES2651514T3 (es) 2011-08-11 2018-01-26 Ionis Pharmaceuticals, Inc. Compuestos oligoméricos gapados que comprenden deoxirribonucleósidos modificados en 5 ' -en la brecha y usos de ellos
MX374399B (es) 2012-02-29 2025-03-06 Sangamo Biosciences Inc Composiciones y sus usos para tratar y prevenir la enfermedad de huntington.
US9598458B2 (en) 2012-07-13 2017-03-21 Wave Life Sciences Japan, Inc. Asymmetric auxiliary group
MX356830B (es) 2012-07-13 2018-06-15 Shin Nippon Biomedical Laboratories Ltd Adyuvante de acido nucleico quiral.
US9982257B2 (en) 2012-07-13 2018-05-29 Wave Life Sciences Ltd. Chiral control
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
KR20150070278A (ko) * 2012-10-15 2015-06-24 아이시스 파마수티컬즈 인코포레이티드 C9orf72 발현 조절용 조성물
JP2016516804A (ja) 2013-04-17 2016-06-09 ファイザー・インク 心血管疾患を治療するためのn−ピペリジン−3−イルベンズアミド誘導体
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
US10369201B2 (en) * 2013-11-11 2019-08-06 Sangamo Therapeutics, Inc. Methods and compositions for treating Huntington's disease
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
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
WO2015108046A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
DK3094728T3 (da) 2014-01-16 2022-05-16 Wave Life Sciences Ltd Kiralt design
CA2947035A1 (en) * 2014-05-08 2015-11-12 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
SMT202000165T1 (it) 2015-04-16 2020-05-08 Ionis Pharmaceuticals Inc Composizioni per modulare l'espressione di c9orf72
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés

Also Published As

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

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