WO2006083800A3 - Nucleic acid silencing of huntington's disease gene - Google Patents

Nucleic acid silencing of huntington's disease gene Download PDF

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Publication number
WO2006083800A3
WO2006083800A3 PCT/US2006/003298 US2006003298W WO2006083800A3 WO 2006083800 A3 WO2006083800 A3 WO 2006083800A3 US 2006003298 W US2006003298 W US 2006003298W WO 2006083800 A3 WO2006083800 A3 WO 2006083800A3
Authority
WO
WIPO (PCT)
Prior art keywords
huntington
nucleic acid
disease gene
acid silencing
silencing
Prior art date
Application number
PCT/US2006/003298
Other languages
French (fr)
Other versions
WO2006083800A2 (en
Inventor
Beverly L Davidson
Scott Harper
Original Assignee
Univ Iowa Res Found
Beverly L Davidson
Scott Harper
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Priority claimed from US11/048,627 external-priority patent/US20050255086A1/en
Application filed by Univ Iowa Res Found, Beverly L Davidson, Scott Harper filed Critical Univ Iowa Res Found
Priority to AU2006210973A priority Critical patent/AU2006210973A1/en
Priority to CA2596588A priority patent/CA2596588C/en
Priority to EP06734091A priority patent/EP1844148A2/en
Publication of WO2006083800A2 publication Critical patent/WO2006083800A2/en
Publication of WO2006083800A3 publication Critical patent/WO2006083800A3/en

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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

The present invention is directed to small interfering RNA molecules (siRNA) targeted against a Huntington's Disease gene, and methods of using these siRNA molecules.
PCT/US2006/003298 2005-01-31 2006-01-31 Nucleic acid silencing of huntington's disease gene WO2006083800A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2006210973A AU2006210973A1 (en) 2005-01-31 2006-01-31 Nucleic acid silencing of Huntington's Disease gene
CA2596588A CA2596588C (en) 2005-01-31 2006-01-31 Nucleic acid silencing of huntington's disease gene
EP06734091A EP1844148A2 (en) 2005-01-31 2006-01-31 Nucleic acid silencing of huntington's disease gene

Applications Claiming Priority (2)

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

Publications (2)

Publication Number Publication Date
WO2006083800A2 WO2006083800A2 (en) 2006-08-10
WO2006083800A3 true WO2006083800A3 (en) 2007-03-01

Family

ID=36777811

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/003298 WO2006083800A2 (en) 2005-01-31 2006-01-31 Nucleic acid silencing of huntington's disease gene

Country Status (4)

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

Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9139828B2 (en) 2008-05-14 2015-09-22 Prosensa Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
US9243245B2 (en) 2007-10-26 2016-01-26 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing

Families Citing this family (24)

* Cited by examiner, † Cited by third party
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JP2002166817A (en) 1999-10-01 2002-06-11 Daicel Chem Ind Ltd Hybrid inflator
US20050042646A1 (en) 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
SI2049664T1 (en) 2006-08-11 2012-04-30 Prosensa Technologies Bv Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
US20080039415A1 (en) * 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
CA2688514A1 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
EP2164967B1 (en) 2007-05-31 2015-07-08 University of Iowa Research Foundation Reduction of off-target rna interference toxicity
JP2010533170A (en) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー．ブイ． Molecules for targeting compounds to various selected organs, tissues or tumor cells
JP5706157B2 (en) 2007-07-12 2015-04-22 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． Molecules for targeting compounds to various selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
AU2009210872A1 (en) * 2008-02-08 2009-08-13 Prosensa Holding Bv Methods and means for treating DNA repeat instability associated genetic disorders
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
EP2673286B1 (en) 2011-02-12 2019-07-03 University of Iowa Research Foundation Therapeutic compounds
BR112014018427B1 (en) 2012-01-27 2021-11-03 Biomarin Technologies B.V. RNA MODULATOR OLIGONUCLEOTIDES WITH IMPROVED FEATURES FOR THE TREATMENT OF DUCHENNE AND BECKER'S MUSCULAR DYSTROPHY
CN104271741A (en) 2012-04-23 2015-01-07 普罗森萨科技有限公司 RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
JP6628285B2 (en) 2013-03-14 2020-01-08 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド ALS-related diamino acid repeat-containing protein
US9523093B2 (en) 2014-05-20 2016-12-20 University Of Iowa Research Foundation Huntington's disease therapeutic compounds
WO2016196324A1 (en) * 2015-05-29 2016-12-08 University Of Floridia Research Foundation, Inc. Methods for diagnosing huntington's disease
EP3440100A4 (en) 2016-04-04 2020-01-22 University of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
US11951121B2 (en) 2016-05-18 2024-04-09 Voyager Therapeutics, Inc. Compositions and methods for treating Huntington's disease
CA3060250A1 (en) 2017-04-17 2018-10-25 University Of Florida Research Foundation, Incorporated Regulation of ran protein translation by pkr and eif2a-p pathways
AR113134A1 (en) * 2017-09-22 2020-01-29 Genzyme Corp ARNI VARIANT
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 (en) * 2019-12-13 2023-07-04 深圳艾码生物科技有限公司 siRNA for inhibiting HTT gene expression, precursor and application thereof

Citations (5)

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Publication number Priority date Publication date Assignee Title
WO2004013280A2 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
WO2005105995A2 (en) * 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
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WO2004058940A2 (en) * 2002-08-05 2004-07-15 University Of Iowa Research Foundation 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
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
WO2005105995A2 (en) * 2004-04-14 2005-11-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
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Non-Patent Citations (4)

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Title
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LIU W ET AL: "Specific inhibition of Huntington's disease gene expression by siRNAs in cultures cells", PROCEEDINGS OF THE JAPAN ACADEMY. SERIES B, PHYSICAL AND BIOLOGICAL SCIENCES, vol. 79B, no. 10, December 2003 (2003-12-01), pages 293 - 298, XP002983007, ISSN: 0386-2208 *
XIA H ET AL: "RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia", NATURE MEDICINE, vol. 10, no. 8, August 2004 (2004-08-01), pages 816 - 820, XP002311180, ISSN: 1078-8956 *

Cited By (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9260716B2 (en) 2002-08-05 2016-02-16 University Of Iowa Research Foundation RNA interference suppression of neurodegenerative diseases and methods of use thereof
US9487779B2 (en) 2002-08-05 2016-11-08 University Of Iowa Research Foundation siRNA-mediated gene silencing
US9243245B2 (en) 2007-10-26 2016-01-26 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
US9499818B2 (en) 2007-10-26 2016-11-22 BioMarin Technologies, B.V. Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene
US9528109B2 (en) 2007-10-26 2016-12-27 Biomarin Technologies B.V. Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA
US9139828B2 (en) 2008-05-14 2015-09-22 Prosensa Technologies B.V. Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means

Also Published As

Publication number Publication date
AU2006210973A1 (en) 2006-08-10
CA2596588C (en) 2017-06-27
WO2006083800A2 (en) 2006-08-10
CA2596588A1 (en) 2006-08-10
EP1844148A2 (en) 2007-10-17

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