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WO2006135436A3 - Inhibition of gene expression and therapeutic uses thereof - Google Patents

Inhibition of gene expression and therapeutic uses thereof Download PDF

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Publication number
WO2006135436A3
WO2006135436A3 PCT/US2005/037814 US2005037814W WO2006135436A3 WO 2006135436 A3 WO2006135436 A3 WO 2006135436A3 US 2005037814 W US2005037814 W US 2005037814W WO 2006135436 A3 WO2006135436 A3 WO 2006135436A3
Authority
WO
WIPO (PCT)
Prior art keywords
rna
expression
gene expression
sirnas
sirna
Prior art date
Application number
PCT/US2005/037814
Other languages
French (fr)
Other versions
WO2006135436A2 (en
Inventor
Alfred S Lewin
Sergei Zolotukhin
Original Assignee
Univ Florida
Alfred S Lewin
Sergei Zolotukhin
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 Univ Florida, Alfred S Lewin, Sergei Zolotukhin filed Critical Univ Florida
Publication of WO2006135436A2 publication Critical patent/WO2006135436A2/en
Publication of WO2006135436A3 publication Critical patent/WO2006135436A3/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
    • C12N15/111—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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—Hairpin
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/127—DNAzymes
    • 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
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

RNA interference (RNAi) has become a powerful tool for blocking gene expression in mammals and mammalian cells. This approach requires the delivery of small interfering RNA (siRNA) either as RNA itself or as DNA, using an expression plasmid or virus and the coding sequence for small hairpin RNAs that are processed to siRNAs. Current expression systems for the production of siRNA in vivo rely on RNA polymerase III promoters. These are difficult to regulate and leave most of the RNA in the nucleus of the cell where it is inactive for RNA interference. The invention provides a DNA cassette for the cloning of small hairpin sequences which permit their expression and processing using RNA polymerase II. This system enables efficient transport of the pre-siRNAs to the cytoplasm where they are active and permits the use of regulated and tissue specific promoters for gene expression.
PCT/US2005/037814 2004-10-22 2005-10-21 Inhibition of gene expression and therapeutic uses thereof WO2006135436A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US62155404P 2004-10-22 2004-10-22
US60/621,554 2004-10-22

Publications (2)

Publication Number Publication Date
WO2006135436A2 WO2006135436A2 (en) 2006-12-21
WO2006135436A3 true WO2006135436A3 (en) 2009-06-04

Family

ID=37532749

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2005/037814 WO2006135436A2 (en) 2004-10-22 2005-10-21 Inhibition of gene expression and therapeutic uses thereof

Country Status (1)

Country Link
WO (1) WO2006135436A2 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102851292B (en) * 2011-03-23 2014-04-02 中国农业科学院哈尔滨兽医研究所 siRNA sequences specifically silencing gI and gE gene of Marek's disease virus, and vector and preparation thereof

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EP2096174A1 (en) 2008-02-28 2009-09-02 Centro De Investigación Cooperativa En Biociencias CiC bioGune Ubiquitin binding polypeptides
WO2009150284A2 (en) 2008-06-13 2009-12-17 Proyecto De Biomedicina Cima, S.L. Apo-a conjugates for the administration of biologically active compounds
CN102325790A (en) 2008-12-03 2012-01-18 西马生物医学计划公司 Vaccine Development Using Phenol-Soluble Regulatory Protein
EP2208785A1 (en) 2009-01-15 2010-07-21 Newbiotechnic, S.A. Methods and kits to generate miRNA- and smallRNA-expressing vectors, and its application to develop lentiviral expression libraries
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RU2597989C2 (en) 2009-12-11 2016-09-20 Проекто Де Биомедисина Сима С.Л. Conjugates and compositions for immunotherapy and anticancer treatment
US20120308517A1 (en) 2010-02-09 2012-12-06 Digna Biotech, S.L. Compositions for the treatment of infectious and tumoural diseases
WO2011101332A1 (en) 2010-02-16 2011-08-25 Proyecto De Biomedicina Cima, S.L. Compositions based on the fibronectin extracellular domain a for the treatment of melanoma
WO2011154308A1 (en) 2010-06-08 2011-12-15 Proyecto De Biomedicina Cima, S.L. New compositions and cell therapy methods for the treatment of cirrhosis
WO2012001196A2 (en) 2010-06-28 2012-01-05 Proyecto De Biomedicina Cima, S.L. Alphaviral vectors and the uses thereof for heterologous gene expression
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WO2012123269A1 (en) 2011-03-11 2012-09-20 Proyecto De Biomedicina Cima, S.L. Immunogenic compositions and methods for their use
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JP7032329B2 (en) 2016-05-25 2022-03-08 インバイオモーション エセ．エレ． Therapeutic treatment of breast cancer based on the condition of c-MAF
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Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030157691A1 (en) * 2001-09-13 2003-08-21 Xiao-Feng Qin Method for expression of small antiviral RNA molecules within a cell
US20040219671A1 (en) * 2002-02-20 2004-11-04 Sirna Therapeutics, Inc. RNA interference mediated treatment of parkinson disease using short interfering nucleic acid (siNA)
US20030180756A1 (en) * 2002-03-21 2003-09-25 Yang Shi Compositions and methods for suppressing eukaryotic gene expression

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102851292B (en) * 2011-03-23 2014-04-02 中国农业科学院哈尔滨兽医研究所 siRNA sequences specifically silencing gI and gE gene of Marek's disease virus, and vector and preparation thereof

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