ATE462787T1 - 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
ATE462787T1
ATE462787T1 AT07290751T AT07290751T ATE462787T1 AT E462787 T1 ATE462787 T1 AT E462787T1 AT 07290751 T AT07290751 T AT 07290751T AT 07290751 T AT07290751 T AT 07290751T AT E462787 T1 ATE462787 T1 AT E462787T1
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AT
Austria
Prior art keywords
huntington
disease
treatment
application
type
Prior art date
Application number
AT07290751T
Other languages
English (en)
Inventor
Valerie Perrin
Nicole Deglon
Original Assignee
Commissariat Energie Atomique
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Publication date
Application filed by Commissariat Energie Atomique filed Critical Commissariat Energie Atomique
Application granted granted Critical
Publication of ATE462787T1 publication Critical patent/ATE462787T1/de

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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (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)
AT07290751T 2007-06-18 2007-06-18 Reversibles sirna-silencing eines mutierten und endogenen huntington-wildtypgens und dessen anwendung zur behandlung von morbus huntington ATE462787T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP07290751A EP2014769B1 (de) 2007-06-18 2007-06-18 Reversibles siRNA-Silencing eines mutierten und endogenen Huntington-Wildtypgens und dessen Anwendung zur Behandlung von Morbus Huntington

Publications (1)

Publication Number Publication Date
ATE462787T1 true ATE462787T1 (de) 2010-04-15

Family

ID=38476952

Family Applications (1)

Application Number Title Priority Date Filing Date
AT07290751T ATE462787T1 (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 (de)
EP (1) EP2014769B1 (de)
JP (1) JP5401451B2 (de)
AT (1) ATE462787T1 (de)
CA (1) CA2690730C (de)
DE (1) DE602007005629D1 (de)
WO (1) WO2009007855A2 (de)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8679750B2 (en) * 2008-05-09 2014-03-25 The University Of British Columbia Methods and compositions for the treatment of Huntington'S disease
WO2009135322A1 (en) * 2008-05-09 2009-11-12 The Universtity Of British Columbia Methods and compositions for the treatment of huntington's disease
RU2572826C2 (ru) 2008-12-02 2016-01-20 Чиралджен, Лтд. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
SG10201403841QA (en) 2009-07-06 2014-09-26 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
JP6006120B2 (ja) 2010-02-08 2016-10-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 対立遺伝子多様体の選択的低減
JP6018506B2 (ja) 2010-02-08 2016-11-02 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 対立遺伝子多様体の選択的低減
US20130109048A1 (en) * 2010-07-09 2013-05-02 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
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
EP2742136B1 (de) 2011-08-11 2017-09-27 Ionis Pharmaceuticals, Inc. Gapmerverbindungen mit 5'-modifizierten desoxyribonukleotiden im gap und verwendungen davon
CN104204225A (zh) 2012-02-29 2014-12-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
WO2014010718A1 (ja) 2012-07-13 2014-01-16 株式会社新日本科学 キラル核酸アジュバント
EP2872147B1 (de) 2012-07-13 2022-12-21 Wave Life Sciences Ltd. Verfahren zur herstellung von chiralen oligonukleotiden
RU2693381C2 (ru) 2012-07-13 2019-07-02 Уэйв Лайф Сайенсес Лтд. Асимметричная вспомогательная группа
RU2730677C2 (ru) * 2012-10-15 2020-08-24 Ионис Фармасьютикалз, Инк. Соединение для модуляции экспрессии гена c9orf72 и его применение
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
BR112015026513A2 (pt) 2013-04-17 2017-07-25 Pfizer derivados de n-piperidin-3-ilbenzamida para tratar as doenças cardiovasculares
SG11201602597YA (en) 2013-10-11 2016-05-30 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
EP3095459A4 (de) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chirales nukleinsäureadjuvans mit antitumorwirkung und antitumormittel
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
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 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
CA2936712A1 (en) 2014-01-16 2015-07-23 Meena Chiral design
EP3139966B1 (de) * 2014-05-08 2020-11-18 Sangamo Therapeutics, Inc. Zusammensetzungen zur verwendung bei der behandlung der huntington-krankheit
MX366128B (es) 2015-04-16 2019-06-28 Ionis Pharmaceuticals Inc Composiciones para modular la expresion de c90rf72.
CR20180107A (es) 2015-07-22 2018-04-03 Wave Life Sciences Ltd Composiciones de oligonucleótidos y métodos de los mismos
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
JP2010530239A (ja) 2010-09-09
JP5401451B2 (ja) 2014-01-29
WO2009007855A2 (en) 2009-01-15
US8217018B2 (en) 2012-07-10
CA2690730C (en) 2018-07-31
US20100299768A1 (en) 2010-11-25
CA2690730A1 (en) 2009-01-15
EP2014769A1 (de) 2009-01-14
WO2009007855A3 (en) 2009-04-02
DE602007005629D1 (de) 2010-05-12
EP2014769B1 (de) 2010-03-31

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