DE602007005629D1 - Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington - Google Patents

Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington

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Publication number
DE602007005629D1
DE602007005629D1 DE602007005629T DE602007005629T DE602007005629D1 DE 602007005629 D1 DE602007005629 D1 DE 602007005629D1 DE 602007005629 T DE602007005629 T DE 602007005629T DE 602007005629 T DE602007005629 T DE 602007005629T DE 602007005629 D1 DE602007005629 D1 DE 602007005629D1
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DE
Germany
Prior art keywords
huntington
disease
treatment
mutated
endogenous
Prior art date
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Active
Application number
DE602007005629T
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German (de)
English (en)
Inventor
Valerie Perrin
Nicole Deglon
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Commissariat a lEnergie Atomique et aux Energies Alternatives CEA
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Commissariat a lEnergie Atomique CEA
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Publication of DE602007005629D1 publication Critical patent/DE602007005629D1/de
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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Classifications

    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Landscapes

  • 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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
DE602007005629T 2007-06-18 2007-06-18 Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington Active DE602007005629D1 (de)

Applications Claiming Priority (1)

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

Publications (1)

Publication Number Publication Date
DE602007005629D1 true DE602007005629D1 (de) 2010-05-12

Family

ID=38476952

Family Applications (1)

Application Number Title Priority Date Filing Date
DE602007005629T Active DE602007005629D1 (de) 2007-06-18 2007-06-18 Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington

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=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
JP5401451B2 (ja) 2014-01-29
EP2014769B1 (en) 2010-03-31
US8217018B2 (en) 2012-07-10
CA2690730A1 (en) 2009-01-15
WO2009007855A2 (en) 2009-01-15
JP2010530239A (ja) 2010-09-09
US20100299768A1 (en) 2010-11-25

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Owner name: COMMISSARIAT A L ENERGIE ATOMIQUE ET AUX ENERG, FR

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