WO2007002904A3 - Methods and sequences to preferentially suppress expression of mutated huntingtin - Google Patents

Methods and sequences to preferentially suppress expression of mutated huntingtin Download PDF

Info

Publication number
WO2007002904A3
WO2007002904A3 PCT/US2006/025638 US2006025638W WO2007002904A3 WO 2007002904 A3 WO2007002904 A3 WO 2007002904A3 US 2006025638 W US2006025638 W US 2006025638W WO 2007002904 A3 WO2007002904 A3 WO 2007002904A3
Authority
WO
WIPO (PCT)
Prior art keywords
methods
expression
sequences
htt
preferentially suppress
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.)
Ceased
Application number
PCT/US2006/025638
Other languages
French (fr)
Other versions
WO2007002904A2 (en
Inventor
Bilsen Paul H J Van
Leonie Jaspers
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.)
Medtronic Inc
Original Assignee
Medtronic Inc
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
Application filed by Medtronic Inc filed Critical Medtronic Inc
Priority to EP06785990A priority Critical patent/EP1896586A2/en
Publication of WO2007002904A2 publication Critical patent/WO2007002904A2/en
Publication of WO2007002904A3 publication Critical patent/WO2007002904A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Pathology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Investigating Or Analysing Biological Materials (AREA)

Abstract

Disclosed herein are methods and sequences to preferentially suppress the expression of the mutated huntingtin ('htt') protein over expression of the normal htt protein. Also disclosed are methods comprising screening an individual for the heterozygous presence of one or more single nucleotide polymorphisms within the individual's Huntington's genes; administering nucleic acid molecules comprising nucleotide sequences that preferentially suppress the expression of amino acid sequences encoding for mutated huntingtin ('htt') over suppressing the expression of amino acid sequences encoding for normal htt by targeting an area of a Huntington's disease gene that is heterozygous for the presence of one or more single nucleotide polymorphisms.
PCT/US2006/025638 2005-06-28 2006-06-28 Methods and sequences to preferentially suppress expression of mutated huntingtin Ceased WO2007002904A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP06785990A EP1896586A2 (en) 2005-06-28 2006-06-28 Methods and sequences to preferentially suppress expression of mutated huntingtin

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US69507805P 2005-06-28 2005-06-28
US60/695,078 2005-06-28

Publications (2)

Publication Number Publication Date
WO2007002904A2 WO2007002904A2 (en) 2007-01-04
WO2007002904A3 true WO2007002904A3 (en) 2007-08-02

Family

ID=37461493

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/025638 Ceased WO2007002904A2 (en) 2005-06-28 2006-06-28 Methods and sequences to preferentially suppress expression of mutated huntingtin

Country Status (4)

Country Link
US (1) US20070161590A1 (en)
EP (4) EP2062980B1 (en)
AT (1) ATE522626T1 (en)
WO (1) WO2007002904A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005027980A1 (en) * 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
US9133517B2 (en) * 2005-06-28 2015-09-15 Medtronics, Inc. Methods and sequences to preferentially suppress expression of mutated huntingtin
EP2239328A3 (en) 2005-08-18 2011-01-05 Alnylam Pharmaceuticals Inc. Methods and compositions for treating neurological disease
US9273356B2 (en) * 2006-05-24 2016-03-01 Medtronic, Inc. Methods and kits for linking polymorphic sequences to expanded repeat mutations
EP2046993A4 (en) * 2006-07-07 2010-11-17 Univ Massachusetts RNA INTERFERENCE COMPOSITIONS AND METHOD FOR TREATING MORBUS HUNTINGTON
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
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
WO2009008727A2 (en) 2007-07-12 2009-01-15 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
JP2011510678A (en) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Methods and means for treating DNA repeat instability related genetic disorders
AU2009244013B2 (en) * 2008-05-09 2015-06-25 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
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
BRPI0923225A2 (en) 2008-12-02 2016-10-04 Chiralgen Ltd Phosphorus-modified nucleic acid synthesis method
CA2756670A1 (en) * 2009-03-26 2010-09-30 The Regents Of The University Of California Mesenchymal stem cells producing inhibitory rna for disease modification
CN102459595A (en) 2009-04-24 2012-05-16 普罗森那技术公司 Inosine-containing oligonucleotides for the treatment of DMD
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRECURSORS AND THEIR METHODS OF USE
ES2733708T3 (en) * 2010-02-08 2019-12-02 Ionis Pharmaceuticals Inc Selective reduction of allelic variants
JP6018506B2 (en) 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． Selective reduction of allelic variants
DK2620428T3 (en) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetric help group
CN107365339A (en) 2011-07-19 2017-11-21 波涛生命科学有限公司 Methods of Synthesizing Functionalized Nucleic Acids
US20140303235A1 (en) 2011-08-11 2014-10-09 Isis Pharmaceuticals, Inc. Linkage modified gapped oligomeric compounds and uses thereof
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
DK2872147T3 (en) 2012-07-13 2023-02-20 Wave Life Sciences Ltd METHOD FOR PREPARATION OF CHIRAL OLIGONUCLEOTIDES
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
EP4086347A3 (en) 2012-10-12 2023-01-11 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
WO2015108046A1 (en) 2014-01-15 2015-07-23 株式会社新日本科学 Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
CN117965536A (en) 2015-07-22 2024-05-03 波涛生命科学有限公司 Oligonucleotide compositions and methods thereof
AU2017220774B2 (en) * 2016-02-15 2022-12-08 Istituti Clinici Scientifici Maugeri Spa Sb Method of allele specific silencing for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT)
EP3452596A4 (en) 2016-05-04 2020-03-18 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
US12275999B2 (en) 2016-11-10 2025-04-15 Talis Biomedical Corporation Polynucleotides for the amplification and detection of chlamydia trachomatis
CN106884047B (en) * 2017-02-15 2021-03-23 济南大学 A method for detecting miRNA-155 based on nucleic acid aptamer
US12264365B2 (en) 2020-03-23 2025-04-01 Talis Biomedical Corporation Polynucleotides for amplification and detection of SARS-CoV-2

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
WO2005027980A1 (en) * 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
WO2005078096A2 (en) * 2004-02-09 2005-08-25 University Of Massachusetts Dual functional oligonucleotides for use in repressing mutant gene expression

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) * 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4965188A (en) * 1986-08-22 1990-10-23 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences using a thermostable enzyme
US4800159A (en) * 1986-02-07 1989-01-24 Cetus Corporation Process for amplifying, detecting, and/or cloning nucleic acid sequences
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (en) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog SYNTHETIC CATALYTIC OLIGONUCLEOTIDE STRUCTURES
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US5350674A (en) 1992-09-04 1994-09-27 Becton, Dickinson And Company Intrinsic factor - horse peroxidase conjugates and a method for increasing the stability thereof
US5354326A (en) * 1993-01-27 1994-10-11 Medtronic, Inc. Screening cable connector for interface to implanted lead
WO1995005864A1 (en) * 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
US5849995A (en) * 1993-09-27 1998-12-15 The University Of British Columbia Mouse model for Huntington's Disease and related DNA sequences
US5624803A (en) * 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US6087171A (en) * 1993-12-17 2000-07-11 Spinal Cord Society Method for inducing DNA synthesis in neurons
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US7069634B1 (en) * 1995-04-28 2006-07-04 Medtronic, Inc. Method for manufacturing a catheter
AU5545596A (en) * 1995-04-28 1996-11-18 Medtronic, Inc. Intraparenchymal infusion catheter system
AU1430097A (en) 1996-01-16 1997-08-11 Ribozyme Pharmaceuticals, Inc. Synthesis of methoxy nucleosides and enzymatic nucleic acid molecules
US7189222B2 (en) * 1996-04-30 2007-03-13 Medtronic, Inc. Therapeutic method of treatment of alzheimer's disease
US5735814A (en) * 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
AU4266597A (en) * 1996-09-11 1998-04-14 General Hospital Corporation, The Use of a non-mammalian dna virus to express an exogenous gene in a mammalian cell
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
GB9701684D0 (en) * 1997-01-28 1997-03-19 Smithkline Beecham Plc Novel compounds
GB9706463D0 (en) * 1997-03-27 1997-05-14 Medical Res Council A model of inflamation in the central nervous system for use in the study of disease
US5782892A (en) * 1997-04-25 1998-07-21 Medtronic, Inc. Medical lead adaptor for external medical device
US6042579A (en) * 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US6187906B1 (en) * 1997-08-11 2001-02-13 Aukland Uniservices Limited Methods to improve neural outcome
WO1999018792A1 (en) * 1997-10-10 1999-04-22 Johns Hopkins University Gene delivery compositions and methods
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6436392B1 (en) * 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6245884B1 (en) * 1998-10-16 2001-06-12 Vivian Y. H. Hook Secretases related to alzheimer's dementia
US6313268B1 (en) * 1998-10-16 2001-11-06 Vivian Y. H. Hook Secretases related to Alzheimer's dementia
US6319905B1 (en) * 1998-12-29 2001-11-20 Cell Genesys, Inc. Method of controlling L-Dopa production and of treating dopamine deficiency
US6310048B1 (en) * 1999-12-09 2001-10-30 St. Louis University Antisense modulation of amyloid beta protein expression
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6551290B1 (en) * 2000-03-31 2003-04-22 Medtronic, Inc. Catheter for target specific drug delivery
US6866859B2 (en) * 2000-08-30 2005-03-15 Biocoat Incorporated Bi-laminar, hyaluronan coatings with silver-based anti-microbial properties
US20050209179A1 (en) * 2000-08-30 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
CA2477014A1 (en) 2002-02-20 2003-08-28 Sirna Therapeutics, Inc. Rna interference mediated inhibition of tnf and tnf receptor superfamily gene expression using short interfering nucleic acid (sina)
US20030224512A1 (en) * 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of beta-site APP-cleaving enzyme expression
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20040023390A1 (en) * 2002-08-05 2004-02-05 Davidson Beverly L. SiRNA-mediated gene silencing with viral vectors
US20040241854A1 (en) * 2002-08-05 2004-12-02 Davidson Beverly L. siRNA-mediated gene silencing
US20050106731A1 (en) * 2002-08-05 2005-05-19 Davidson Beverly L. siRNA-mediated gene silencing with viral vectors
US7829694B2 (en) 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US20050048641A1 (en) * 2002-11-26 2005-03-03 Medtronic, Inc. System and method for delivering polynucleotides to the central nervous system
US20050079512A1 (en) * 2003-02-26 2005-04-14 Emerson Beverly M. Methods of modulating gene expression
US20050202075A1 (en) * 2004-03-12 2005-09-15 Pardridge William M. Delivery of genes encoding short hairpin RNA using receptor-specific nanocontainers
US8240498B2 (en) 2006-10-31 2012-08-14 Crown Packaging Technology, Inc. Resealable closure

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)
WO2005027980A1 (en) * 2003-09-12 2005-03-31 University Of Massachusetts Rna interference for the treatment of gain-of-function disorders
WO2005078096A2 (en) * 2004-02-09 2005-08-25 University Of Massachusetts Dual functional oligonucleotides for use in repressing mutant gene expression

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LI SHI-HUA ET AL: "Interaction of Huntington disease protein with transcriptional activator Sp1.", MOLECULAR AND CELLULAR BIOLOGY MAR 2002, vol. 22, no. 5, March 2002 (2002-03-01), pages 1277 - 1287, XP002421817, ISSN: 0270-7306 *
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, TOKYO, JP, vol. 79B, no. 10, December 2003 (2003-12-01), pages 293 - 298, XP002983007, ISSN: 0386-2208 *
MILLER V M ET AL: "Allele-specific silencing of dominant disease genes", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE, WASHINGTON, DC, US, vol. 100, no. 12, 10 June 2003 (2003-06-10), pages 7195 - 7200, XP002278730, ISSN: 0027-8424 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Also Published As

Publication number Publication date
EP2322657A1 (en) 2011-05-18
EP2062980A2 (en) 2009-05-27
EP2322657B1 (en) 2014-11-05
EP2316967A1 (en) 2011-05-04
ATE522626T1 (en) 2011-09-15
EP1896586A2 (en) 2008-03-12
WO2007002904A2 (en) 2007-01-04
EP2062980B1 (en) 2011-08-31
US20070161590A1 (en) 2007-07-12
EP2062980A3 (en) 2009-08-26

Similar Documents

Publication Publication Date Title
EP2062980A3 (en) Methods and sequences to preferentially suppress expression of mutated huntingtin gene.
WO2008005562A3 (en) Rna silencing compositions and methods for the treatment of huntington's disease
WO2007117438A3 (en) Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith
WO2001077375A8 (en) Diagnosis of diseases associated with gene regulation
WO2006119266A3 (en) Human micrornas and methods for inhibiting same
WO2004066941A3 (en) Expression profiles for colon cancer and methods of use
WO2005081714A3 (en) Use of interfering rna in the production of transgenic animals
WO2004024757A3 (en) Modified pna molecules
WO2007134272A3 (en) Humanized models via targeted mtagenesis with zinc finger nuclease
WO2007051045A3 (en) Compositions and methods for inhibiting expression of huntingtin gene
WO2004035803A3 (en) Method and nucleic acids for the treatment of breast cell proliferative disorders
WO2005059108A3 (en) Gene expression profiles and methods of use
WO2010022328A3 (en) Genetic loci associated with head smut resistance in maize
TNSN08252A1 (en) Sirna anti myosine va et depigmentation de la peau
WO2005032495A8 (en) Gene expression profiles and methods of use
WO2008147887A8 (en) Methods and kits for linking polymorphic sequences to expanded repeat mutations
WO2009007934A3 (en) Cd44 splice variants in neurodegenerative diseases
WO2005112619A3 (en) Novel gene disruptions, compositions and methods relating thereto
WO2007100684A3 (en) Extracellular matrix/metastasis modifer genes for the prevention or inhibition of metastasis or growth of tumor and for characterization of tumor
WO2007120955A3 (en) Genes affecting human memory performance
WO2008052016A3 (en) The sortilin-related receptor sorl1 is functionally and genetically associated with alzheimer's disease
WO2002101048A3 (en) NEW POLYNUCLEOTIDES AND POLYPEPTIDES OF THE IFNα-7 GENE
WO2006016172A3 (en) Cell surface glycoprotein
WO2005113815A3 (en) Polymorphisms in the fcgr2b promoter and uses thereof
AU2003253197A1 (en) Targeted gene modification by single-stranded dna oligonucleotides

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application
DPE1 Request for preliminary examination filed after expiration of 19th month from priority date (pct application filed from 20040101)
WWE Wipo information: entry into national phase

Ref document number: 2006785990

Country of ref document: EP

NENP Non-entry into the national phase

Ref country code: DE