WO2007032794A3 - Inhibition of viral gene expression using small interfering rna - Google Patents

Inhibition of viral gene expression using small interfering rna Download PDF

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Publication number
WO2007032794A3
WO2007032794A3 PCT/US2006/021253 US2006021253W WO2007032794A3 WO 2007032794 A3 WO2007032794 A3 WO 2007032794A3 US 2006021253 W US2006021253 W US 2006021253W WO 2007032794 A3 WO2007032794 A3 WO 2007032794A3
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WO
WIPO (PCT)
Prior art keywords
small interfering
inhibition
gene expression
interfering rna
viral gene
Prior art date
Application number
PCT/US2006/021253
Other languages
French (fr)
Other versions
WO2007032794A2 (en
Inventor
Roger L Kaspar
Heini Ilves
Attila A Seyhan
Alexander V Vlassov
Brian H Johnston
Original Assignee
Somagenics Inc
Roger L Kaspar
Heini Ilves
Attila A Seyhan
Alexander V Vlassov
Brian H Johnston
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 PCT/US2005/032768 external-priority patent/WO2006031901A2/en
Application filed by Somagenics Inc, Roger L Kaspar, Heini Ilves, Attila A Seyhan, Alexander V Vlassov, Brian H Johnston filed Critical Somagenics Inc
Priority to CA002622242A priority Critical patent/CA2622242A1/en
Priority to CN2006800418459A priority patent/CN101305095B/en
Priority to BRPI0615717-3A priority patent/BRPI0615717A2/en
Priority to AU2006291568A priority patent/AU2006291568A1/en
Priority to JP2008529981A priority patent/JP2009521207A/en
Priority to EP06784525A priority patent/EP1979480A2/en
Publication of WO2007032794A2 publication Critical patent/WO2007032794A2/en
Publication of WO2007032794A3 publication Critical patent/WO2007032794A3/en
Priority to IL190114A priority patent/IL190114A0/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
    • 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
    • C12N15/1131—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 against viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • 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
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/24011—Flaviviridae
    • C12N2770/24211—Hepacivirus, e.g. hepatitis C virus, hepatitis G virus
    • 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
    • C12N2770/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses positive-sense
    • C12N2770/00011—Details
    • C12N2770/36011—Togaviridae
    • C12N2770/36111—Alphavirus, e.g. Sindbis virus, VEE, EEE, WEE, Semliki

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Communicable Diseases (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Oncology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

The invention provides methods, compositions, and kits comprising small interfering RNA (shRNA or siRNA) that are useful for inhibition of viral-mediated gene expression. Small interfering RNAs as described herein can be used in methods of treatment of HCV infection. ShRNA and siRNA constructs targetING the internal ribosome entry site (IRES) sequence of HCV are described.
PCT/US2006/021253 2004-09-10 2006-06-01 Inhibition of viral gene expression using small interfering rna WO2007032794A2 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
CA002622242A CA2622242A1 (en) 2005-09-12 2006-06-01 Inhibition of viral gene expression using small interfering rna
CN2006800418459A CN101305095B (en) 2005-09-12 2006-06-01 Inhibition of viral gene expression using small interfering RNA
BRPI0615717-3A BRPI0615717A2 (en) 2005-09-12 2006-06-01 inhibition of viral gene expression using small interfering rna
AU2006291568A AU2006291568A1 (en) 2005-09-12 2006-06-01 Inhibition of viral gene expression using small interfering RNA
JP2008529981A JP2009521207A (en) 2005-09-12 2006-06-01 Inhibition of viral gene expression using small interfering RNA
EP06784525A EP1979480A2 (en) 2005-09-12 2006-06-01 Inhibition of viral gene expression using small interfering rna
IL190114A IL190114A0 (en) 2004-09-10 2008-03-11 Inhibition of viral gene expression using small interfering rna

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
USPCT/US2005/032768 2005-09-12
PCT/US2005/032768 WO2006031901A2 (en) 2004-09-10 2005-09-12 SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF

Publications (2)

Publication Number Publication Date
WO2007032794A2 WO2007032794A2 (en) 2007-03-22
WO2007032794A3 true WO2007032794A3 (en) 2007-08-30

Family

ID=37708176

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/021253 WO2007032794A2 (en) 2004-09-10 2006-06-01 Inhibition of viral gene expression using small interfering rna

Country Status (9)

Country Link
EP (1) EP1979480A2 (en)
JP (1) JP2009521207A (en)
KR (1) KR20090003147A (en)
CN (2) CN102827841A (en)
AU (1) AU2006291568A1 (en)
BR (1) BRPI0615717A2 (en)
CA (1) CA2622242A1 (en)
RU (1) RU2008114304A (en)
WO (1) WO2007032794A2 (en)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2009042115A2 (en) * 2007-09-24 2009-04-02 Rosetta Inpharmatics Llc Methods of designing short hairpin rnas (shrnas) for gene silencing
ES2708944T3 (en) * 2008-09-22 2019-04-12 Dicerna Pharmaceuticals Inc Compositions and methods for the specific inhibition of gene expression by DSRNA having modifications
US8283460B2 (en) * 2008-10-15 2012-10-09 Somagenics, Inc. Short hairpin RNAs for inhibition of gene expression
US8513207B2 (en) * 2008-12-18 2013-08-20 Dicerna Pharmaceuticals, Inc. Extended dicer substrate agents and methods for the specific inhibition of gene expression
EP2454371B1 (en) 2009-07-13 2021-01-20 Somagenics, Inc. Chemical modification of small hairpin rnas for inhibition of gene expression
SI2606134T1 (en) * 2010-08-17 2019-08-30 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
JOP20200092A1 (en) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc HEPATITIS B VIRUS (HBV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
KR101668954B1 (en) 2015-05-29 2016-10-24 연세대학교 산학협력단 Therapeutic HCV IRES-silencing siRNA for the treatment of hepatitis C virus infection
WO2018112124A1 (en) * 2016-12-15 2018-06-21 Meharry Medical College Antiviral agents
US11324820B2 (en) 2017-04-18 2022-05-10 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (HBV) infection
MX2021001056A (en) 2018-08-13 2021-04-12 Alnylam Pharmaceuticals Inc HEPATITIS B VIRUS (HBV) dsRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF.

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070750A2 (en) * 2002-02-20 2003-08-28 Sirna Therapeutics, Inc Rna interference mediated inhibition of hepatitis c virus
WO2006031901A2 (en) * 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN100344331C (en) * 2004-09-22 2007-10-24 广州拓谱基因技术有限公司 Targeted small interference RNA formulation for preventing and treating Hepatitis C and its preparation method

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070750A2 (en) * 2002-02-20 2003-08-28 Sirna Therapeutics, Inc Rna interference mediated inhibition of hepatitis c virus
WO2006031901A2 (en) * 2004-09-10 2006-03-23 Somagenics, Inc. SMALL INTERFERING RNAs THAT EFFICIENTLY INHIBIT VIRAL GENE EXPRESSION AND METHODS OF USE THEREOF

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
ILVES HEINI ET AL: "Inhibition of hepatitis C IRES-mediated gene expression by small hairpin RNAs in human hepatocytes and mice.", ANNALS OF THE NEW YORK ACADEMY OF SCIENCES, vol. 1082, October 2006 (2006-10-01), pages 52 - 55, XP002420574, ISSN: 0077-8923 *
KAWASAKI H ET AL: "Short hairpin type of dsRNAs that are controlled by tRNA Val promoter significantly induce RNAi-mediated gene silencing in the cytoplasm of human cells", NUCLEIC ACIDS RESEARCH, vol. 31, no. 2, January 2003 (2003-01-01), pages 700 - 707, XP002965487, ISSN: 0305-1048 *
RADHAKRISHNAN S K ET AL: "RNA interference as a new strategy against viral hepatitis", VIROLOGY, vol. 323, no. 2, 1 June 2004 (2004-06-01), pages 173 - 181, XP004515292, ISSN: 0042-6822 *
SEYHAN ATTILA A ET AL: "Complete, gene-specific siRNA libraries: Production and expression in mammalian cells", RNA (COLD SPRING HARBOR), vol. 11, no. 5, May 2005 (2005-05-01), pages 837 - 846, XP002420573, ISSN: 1355-8382 *
WANG ET AL: "Small Hairpin RNAs Efficiently Inhibit Hepatitis C IRES-Mediated Gene Expression in Human Tissue Culture Cells and a Mouse Model", MOLECULAR THERAPY, vol. 12, no. 3, September 2005 (2005-09-01), pages 562 - 568, XP005472392, ISSN: 1525-0016 *
YOKOTA TAKANORI ET AL: "Inhibition of intracellular hepatitis C virus replication by synthetic and vector-derived small interfering RNAs", EMBO REPORTS, vol. 4, no. 6, June 2003 (2003-06-01), pages 602 - 608, XP002300964 *

Also Published As

Publication number Publication date
CN102827841A (en) 2012-12-19
RU2008114304A (en) 2009-11-20
EP1979480A2 (en) 2008-10-15
CN101305095B (en) 2012-09-26
CN101305095A (en) 2008-11-12
BRPI0615717A2 (en) 2011-05-24
JP2009521207A (en) 2009-06-04
KR20090003147A (en) 2009-01-09
WO2007032794A2 (en) 2007-03-22
CA2622242A1 (en) 2007-03-22
AU2006291568A1 (en) 2007-03-22

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