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
German (de)
English (en)
Inventor
Valerie Perrin
Nicole Deglon
Original Assignee
Commissariat Energie Atomique
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Application granted granted Critical
Publication of ATE462787T1 publication Critical patent/ATE462787T1/de

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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)
  • 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)
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 (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
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 (cg-RX-API-DMAC7.html)
EP (1) EP2014769B1 (cg-RX-API-DMAC7.html)
JP (1) JP5401451B2 (cg-RX-API-DMAC7.html)
AT (1) ATE462787T1 (cg-RX-API-DMAC7.html)
CA (1) CA2690730C (cg-RX-API-DMAC7.html)
DE (1) DE602007005629D1 (cg-RX-API-DMAC7.html)
WO (1) WO2009007855A2 (cg-RX-API-DMAC7.html)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009135322A1 (en) * 2008-05-09 2009-11-12 The Universtity 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
AU2009323766B2 (en) 2008-12-02 2016-10-06 Wave Life Sciences Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
SG177564A1 (en) 2009-07-06 2012-02-28 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
EP3321361B1 (en) 2010-02-08 2019-03-27 Ionis 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
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
DK2742135T4 (da) 2011-08-11 2020-07-13 Ionis Pharmaceuticals Inc Bindingsmodificerede gapped oligomeriske forbindelser og anvendelser deraf
JP6490426B2 (ja) 2012-02-29 2019-03-27 サンガモ セラピューティクス, インコーポレイテッド ハンチントン病を治療するための方法および組成物
JP6246121B2 (ja) 2012-07-13 2017-12-13 株式会社新日本科学 キラル核酸アジュバント
JP6268157B2 (ja) 2012-07-13 2018-01-24 株式会社Wave Life Sciences Japan 不斉補助基
SG10201912895PA (en) 2012-07-13 2020-02-27 Wave Life Sciences Ltd Chiral control
WO2014062691A2 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP2906697A4 (en) 2012-10-15 2016-06-22 Ionis Pharmaceuticals Inc METHOD FOR MONITORING THE C9ORF72 EXPRESSION
MX2015014666A (es) 2013-04-17 2016-03-01 Pfizer Derivados de n-piperidin-3-ilbenzamida para tratar enfermedades cardiovasculares.
CN112080502A (zh) 2013-10-11 2020-12-15 Ionis制药公司 用于调节c9orf72表达的组合物
MX391904B (es) * 2013-11-11 2025-03-12 Sangamo Biosciences Inc Un represor genetico para usarse en el tratamiento de la enfermedad de huntington.
EP3095460A4 (en) 2014-01-15 2017-08-23 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
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
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
JP2010530239A (ja) 2010-09-09
WO2009007855A3 (en) 2009-04-02
US8217018B2 (en) 2012-07-10
US20100299768A1 (en) 2010-11-25
JP5401451B2 (ja) 2014-01-29
EP2014769A1 (en) 2009-01-14
EP2014769B1 (en) 2010-03-31
CA2690730C (en) 2018-07-31
CA2690730A1 (en) 2009-01-15
DE602007005629D1 (de) 2010-05-12
WO2009007855A2 (en) 2009-01-15

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