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
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
DE602007005629T
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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
Original Assignee
Commissariat a lEnergie Atomique CEA
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Application filed by Commissariat a lEnergie Atomique CEA filed Critical Commissariat a lEnergie Atomique CEA
Publication of DE602007005629D1 publication Critical patent/DE602007005629D1/de
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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 N.A.
    • 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
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 (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
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 (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
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
KR101881596B1 (ko) 2008-12-02 2018-07-24 웨이브 라이프 사이언시스 재팬 인코포레이티드 인 원자 변형된 핵산의 합성 방법
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
US8957040B2 (en) 2010-02-08 2015-02-17 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
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
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
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
EP2734208B1 (de) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Verfahren zur synthese von funktionalisierten nukleinsäuren
US10202599B2 (en) 2011-08-11 2019-02-12 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
CA2865011C (en) 2012-02-29 2021-06-15 Sangamo Biosciences, Inc. Methods and compositions for treating huntington's disease
KR102213609B1 (ko) 2012-07-13 2021-02-08 웨이브 라이프 사이언시스 리미티드 키랄 제어
EP2872485B1 (de) 2012-07-13 2020-12-16 Wave Life Sciences Ltd. Asymmetrische hilfsgruppe
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
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
CA2888486A1 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP2986599A1 (de) 2013-04-17 2016-02-24 Pfizer Inc. N-piperidin-3-ylbenzamid-derivate zur behandlung von herz-kreislauf-erkrankungen
SG11201602597YA (en) 2013-10-11 2016-05-30 Ionis Pharmaceuticals Inc Compositions for modulating c9orf72 expression
BR112016010175A2 (pt) * 2013-11-11 2017-10-03 Sangamo Biosciences Inc Repressor genético, seus usos, e composição farmacêutica
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
WO2015108047A1 (ja) 2014-01-15 2015-07-23 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
AU2015207773B2 (en) 2014-01-16 2021-06-17 Wave Life Sciences Ltd. Chiral design
BR112016025849A2 (pt) * 2014-05-08 2017-10-17 Chdi Foundation Inc métodos e composições para o tratamento da doença de huntington
PL3283080T3 (pl) 2015-04-16 2020-07-27 Ionis Pharmaceuticals, Inc. Kompozycja do modulowania ekspresji c9orf72
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
US11260073B2 (en) 2015-11-02 2022-03-01 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
US8217018B2 (en) 2012-07-10
WO2009007855A3 (en) 2009-04-02
US20100299768A1 (en) 2010-11-25
ATE462787T1 (de) 2010-04-15
EP2014769A1 (de) 2009-01-14
CA2690730A1 (en) 2009-01-15
EP2014769B1 (de) 2010-03-31
WO2009007855A2 (en) 2009-01-15
CA2690730C (en) 2018-07-31
JP5401451B2 (ja) 2014-01-29
JP2010530239A (ja) 2010-09-09

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

8364 No opposition during term of opposition