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
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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 EP06734091A priority Critical patent/EP1844148A2/en
Priority to CA2596588A priority patent/CA2596588C/en
Priority to AU2006210973A priority patent/AU2006210973A1/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
EP06734091A EP1844148A2 (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
AU2006210973A AU2006210973A1 (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 US20050255086A1 (en) 2002-08-05 2005-01-31 Nucleic acid silencing of Huntington's Disease gene
US11/048,627 2005-01-31

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
US20080039415A1 (en) * 2006-08-11 2008-02-14 Gregory Robert Stewart Retrograde transport of sirna and therapeutic uses to treat neurologic disorders
EP2049664B1 (en) 2006-08-11 2011-09-14 Prosensa Technologies B.V. Single stranded oligonucleotides complementary to repetitive elements for treating DNA repeat instability associated genetic disorders
US20090036395A1 (en) * 2007-04-26 2009-02-05 Davidson Beverly L Rna interference suppression of neurodegenerative diseases and methods of use thereof
ES2549122T3 (en) 2007-05-31 2015-10-23 University Of Iowa Research Foundation Reduction of toxicity by RNA interference diverted from its target
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
NZ582521A (en) 2007-07-12 2011-09-30 Prosensa Technologies Bv A conjugate comprising the amino acid sequence LGAQSNF for targeting compounds to muscle tissue
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
WO2010123369A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
CN104203289B (en) 2012-01-27 2020-11-03 比奥马林技术公司 RNA-regulatory oligonucleotides with improved properties for the treatment of duchenne muscular dystrophy and becker muscular dystrophy
RS56319B1 (en) 2012-04-23 2017-12-29 Biomarin Tech Bv Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
CA2904960A1 (en) 2013-03-14 2014-10-02 University Of Florida Research Foundation, Inc. Di-amino acid repeat-containing proteins associated with als
EP3146051B8 (en) 2014-05-20 2019-11-27 University of Iowa Research Foundation Huntington's disease therapeutic compounds
US10509045B2 (en) 2015-05-29 2019-12-17 University Of Florida Research Foundation, Incorporated Methods for diagnosing Huntington's disease
CA3019847A1 (en) 2016-04-04 2017-10-12 University Of Florida Research Foundation, Incorporated Manipulation of eif3 to modulate repeat associated non-atg (ran) translation
SG11201809643UA (en) * 2016-05-18 2018-12-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
US11345911B2 (en) 2017-04-17 2022-05-31 University Of Florida Research Foundation, Incorporated Regulation of RAN translation by PKR and eIF2A-P pathways
CA3076191A1 (en) * 2017-09-22 2019-03-28 Genzyme Corporation Variant rnai
AU2018342105B2 (en) 2017-09-26 2023-11-16 University Of Florida Research Foundation, Incorporated Use of metformin and analogs thereof to reduce ran protein levels in the treatment of neurological disorders
WO2019084068A1 (en) * 2017-10-23 2019-05-02 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
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Patent Citations (6)

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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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DAVIDSON B L ET AL: "VIRAL VECTORS FOR GENE DELIVERY TO THE NERVOUS SYSTEM", NATURE REVIEWS NEUROSCIENCE, vol. 4, no. 5, May 2003 (2003-05-01), pages 353 - 364, XP001180914, ISSN: 1471-0048 *
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 *
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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
CA2596588A1 (en) 2006-08-10
AU2006210973A1 (en) 2006-08-10
CA2596588C (en) 2017-06-27
EP1844148A2 (en) 2007-10-17
WO2006083800A2 (en) 2006-08-10

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