CA2596588C - Silencage nucleique de gene de maladie d'huntington - Google Patents

Silencage nucleique de gene de maladie d'huntington Download PDF

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Publication number
CA2596588C
CA2596588C CA2596588A CA2596588A CA2596588C CA 2596588 C CA2596588 C CA 2596588C CA 2596588 A CA2596588 A CA 2596588A CA 2596588 A CA2596588 A CA 2596588A CA 2596588 C CA2596588 C CA 2596588C
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Prior art keywords
seq
sirna
rna
sequence
strand
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CA2596588A
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English (en)
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CA2596588A1 (fr
Inventor
Beverly L. Davidson
Scott Harper
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University of Iowa Research Foundation UIRF
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University of Iowa Research Foundation UIRF
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Priority claimed from US11/048,627 external-priority patent/US20050255086A1/en
Application filed by University of Iowa Research Foundation UIRF filed Critical University of Iowa Research Foundation UIRF
Publication of CA2596588A1 publication Critical patent/CA2596588A1/fr
Application granted granted Critical
Publication of CA2596588C publication Critical patent/CA2596588C/fr
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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
    • 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

L'invention concerne des molécules d'ARN interférentes de petite taille (ARNsi) ciblées contre un gène de la maladie de Huntington, ainsi que des procédés d'utilisation de ces molécules d'ARNsi.
CA2596588A 2005-01-31 2006-01-31 Silencage nucleique de gene de maladie d'huntington Active CA2596588C (fr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US11/048,627 2005-01-31
US11/048,627 US20050255086A1 (en) 2002-08-05 2005-01-31 Nucleic acid silencing of Huntington's Disease gene
PCT/US2006/003298 WO2006083800A2 (fr) 2005-01-31 2006-01-31 Silençage nucleique de gene de maladie d'huntington

Publications (2)

Publication Number Publication Date
CA2596588A1 CA2596588A1 (fr) 2006-08-10
CA2596588C true CA2596588C (fr) 2017-06-27

Family

ID=36777811

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2596588A Active CA2596588C (fr) 2005-01-31 2006-01-31 Silencage nucleique de gene de maladie d'huntington

Country Status (4)

Country Link
EP (1) EP1844148A2 (fr)
AU (1) AU2006210973A1 (fr)
CA (1) CA2596588C (fr)
WO (1) WO2006083800A2 (fr)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2002166817A (ja) 1999-10-01 2002-06-11 Daicel Chem Ind Ltd ハイブリッドインフレータ
US20040241854A1 (en) 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20080274989A1 (en) 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
EP2049664B1 (fr) 2006-08-11 2011-09-14 Prosensa Technologies B.V. Oligonucléotides monocaténaires complémentaires des elements repetitifs pour le traitement de troubles génétiques associés a l'instabilité des répétitions de l'ADN
US20080039415A1 (en) * 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
US20090036395A1 (en) * 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
AU2008260103B2 (en) 2007-05-31 2014-04-03 University Of Iowa Research Foundation Reduction of off-target RNA interference toxicity
AU2008273096B2 (en) 2007-07-12 2013-05-02 Academisch Ziekenhuis Leiden Molecules for targeting compounds to various selected organs or tissues
EP2167135A2 (fr) 2007-07-12 2010-03-31 Prosensa Technologies B.V. Molécules pour cibler les composants vers plusieurs organes sélectionnés, tissus ou cellules de tumeurs
ES2914775T3 (es) 2007-10-26 2022-06-16 Academisch Ziekenhuis Leiden Medios y métodos para contrarresta trastornos del músculo
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
NZ587178A (en) * 2008-02-08 2011-11-25 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
EP2119783A1 (fr) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
CA2759899A1 (fr) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprenant une inosine pour traiter une dystrophie musculaire de duchenne (dmd)
WO2012109667A1 (fr) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Composés thérapeutiques
CA2862628C (fr) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Oligonucleotides a modulation d'arn dotes de caracteristiques ameliorees pour le traitement de la dystrophie musculaire de duchenne et de becker
CA3103101C (fr) 2012-04-23 2023-01-17 Biomarin Technologies B.V. Oligonucleotides de modulation d'arn ayant des caracteristiques perfectionnees pour le traitement de troubles neuromusculaires
CA2904960A1 (fr) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Proteines contenant des repetitions d'acides carboxyliques diamines associees a la sla
RU2711147C2 (ru) 2014-05-20 2020-01-15 Юниверсити Оф Айова Рисерч Фаундейшн Терапевтические соединения для лечения болезни хантингтона
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
EP3440100A4 (fr) 2016-04-04 2020-01-22 University of Florida Research Foundation, Incorporated Manipulation d'eif3 pour moduler une traduction non-atg associée à une répétition (ran)
MX2018014152A (es) 2016-05-18 2019-03-28 Voyager Therapeutics Inc Composiciones y métodos para tratar la enfermedad de huntington.
WO2018195110A1 (fr) 2017-04-17 2018-10-25 University Of Florida Research Foundation, Incorporated Régulation de la traduction de ran par les voies pkr et eif2a-p
AR113134A1 (es) * 2017-09-22 2020-01-29 Genzyme Corp Arni variante
US11903910B2 (en) 2017-09-26 2024-02-20 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce RAN protein levels in the treatment of neurological disorders
AU2018354195A1 (en) * 2017-10-23 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
CN112980837B (zh) * 2019-12-13 2023-07-04 深圳艾码生物科技有限公司 一种抑制HTT基因表达的siRNA及其前体和应用

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
WO2005105995A2 (fr) * 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. Traitement de maladies a expansion de sequence repetee de polyglutamine (polyq) a mediation d'arn interferent mettant en oeuvre un acide nucleique court interferent (sina)

Also Published As

Publication number Publication date
EP1844148A2 (fr) 2007-10-17
WO2006083800A3 (fr) 2007-03-01
WO2006083800A2 (fr) 2006-08-10
AU2006210973A1 (en) 2006-08-10
CA2596588A1 (fr) 2006-08-10

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