WO2006033756A3 - Multiple rna polymerase iii promoter expression constructs - Google Patents

Multiple rna polymerase iii promoter expression constructs Download PDF

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Publication number
WO2006033756A3
WO2006033756A3 PCT/US2005/029976 US2005029976W WO2006033756A3 WO 2006033756 A3 WO2006033756 A3 WO 2006033756A3 US 2005029976 W US2005029976 W US 2005029976W WO 2006033756 A3 WO2006033756 A3 WO 2006033756A3
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WIPO (PCT)
Prior art keywords
polymerase iii
expression constructs
rna polymerase
iii promoter
promoter expression
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/US2005/029976
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French (fr)
Other versions
WO2006033756A2 (en
Inventor
Catherine J Pachuk
Daniel E Mccallus
Chandrasekhar Satishchandran
Martin D Sigg
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Nucleonics Inc
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Nucleonics Inc
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Filing date
Publication date
Priority to JP2007530052A priority Critical patent/JP4804467B2/en
Application filed by Nucleonics Inc filed Critical Nucleonics Inc
Priority to US11/660,937 priority patent/US7985581B2/en
Priority to CA002577519A priority patent/CA2577519A1/en
Priority to CN2005800362406A priority patent/CN101189343B/en
Priority to DE602005024015T priority patent/DE602005024015D1/en
Priority to AU2005287365A priority patent/AU2005287365B2/en
Priority to AT05810305T priority patent/ATE483815T1/en
Priority to EP05810305A priority patent/EP1784492B1/en
Publication of WO2006033756A2 publication Critical patent/WO2006033756A2/en
Publication of WO2006033756A3 publication Critical patent/WO2006033756A3/en
Anticipated expiration legal-status Critical
Priority to US13/160,774 priority patent/US20120028348A1/en
Ceased legal-status Critical Current

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    • 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
    • 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
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
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    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • 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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  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
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  • General Health & Medical Sciences (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
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  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Expression constructs comprising at least two different RNA polymerase III promoters, wherein each promoter is operably linked to a nucleic acid sequence encoding an RNA effector molecule, are disclosed herein. Further provided are expression constructs comprising multiple polymerase III promoters operably linked to sequences encoding short hairpin RNA molecules, which may comprise single and/or multiple fingers. The provided constructs are useful for in vivo delivery of RNA molecules effective in gene silencing, including of viral genes including HBV and HCV.
PCT/US2005/029976 2004-08-23 2005-08-23 Multiple rna polymerase iii promoter expression constructs Ceased WO2006033756A2 (en)

Priority Applications (9)

Application Number Priority Date Filing Date Title
EP05810305A EP1784492B1 (en) 2004-08-23 2005-08-23 Multiple rna polymerase iii promoter expression constructs
US11/660,937 US7985581B2 (en) 2004-08-23 2005-08-23 Multiple RNA polymerase III promoter expression constructs
CA002577519A CA2577519A1 (en) 2004-08-23 2005-08-23 Multiple rna polymerase iii promoter expression constructs
CN2005800362406A CN101189343B (en) 2004-08-23 2005-08-23 Multiple rna polymerase iii promoter expression constructs
DE602005024015T DE602005024015D1 (en) 2004-08-23 2005-08-23 EXPRESSION CONSTRUCTS WITH SEVERAL RNA POLYMERASE III PROMOTERS
JP2007530052A JP4804467B2 (en) 2004-08-23 2005-08-23 Multiple RNA polymerase III promoter expression construct
AT05810305T ATE483815T1 (en) 2004-08-23 2005-08-23 EXPRESSION CONSTRUCTS WITH MULTIPLE RNA POLYMERASE III PROMOTERS
AU2005287365A AU2005287365B2 (en) 2004-08-23 2005-08-23 Multiple RNA polymerase III promoter expression constructs
US13/160,774 US20120028348A1 (en) 2004-08-23 2011-06-15 Multiple rna polymerase iii promoter expression constructs

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US60362204P 2004-08-23 2004-08-23
US60/603,622 2004-08-23
US62994204P 2004-11-22 2004-11-22
US60/629,942 2004-11-22

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US13/160,774 Continuation US20120028348A1 (en) 2004-08-23 2011-06-15 Multiple rna polymerase iii promoter expression constructs

Publications (2)

Publication Number Publication Date
WO2006033756A2 WO2006033756A2 (en) 2006-03-30
WO2006033756A3 true WO2006033756A3 (en) 2007-01-18

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PCT/US2005/029976 Ceased WO2006033756A2 (en) 2004-08-23 2005-08-23 Multiple rna polymerase iii promoter expression constructs

Country Status (8)

Country Link
US (2) US7985581B2 (en)
EP (3) EP2169072A1 (en)
JP (1) JP4804467B2 (en)
AT (1) ATE483815T1 (en)
AU (1) AU2005287365B2 (en)
CA (1) CA2577519A1 (en)
DE (1) DE602005024015D1 (en)
WO (1) WO2006033756A2 (en)

Families Citing this family (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2284268A3 (en) * 2003-06-12 2012-05-30 Alnylam Pharmaceuticals Inc. Conserved HBV and HCV sequences useful for gene silencing
US8575327B2 (en) 2003-06-12 2013-11-05 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
AU2016202897A1 (en) * 2004-12-22 2016-05-19 Alnylam Pharmaceuticals, Inc. Conserved HBV and HCV sequences useful for gene silencing
DE602005027747D1 (en) * 2004-12-22 2011-06-09 Alnylam Pharmaceuticals Inc GENSILENCING SUITABLE CONSERVED HBV AND HCV SEQUENCES
JP2008237023A (en) * 2005-07-15 2008-10-09 Univ Of Tokyo Multiple shRNA expression vector
WO2008147430A2 (en) * 2006-10-11 2008-12-04 Nucleonics, Inc. Microrna-formatted multitarget interfering rna vector constructs and methods of using the same
DK2548438T3 (en) 2006-11-08 2015-10-19 Veritas Bio LLC IN VIVO SUBMITTING DOUBLE-STRENGTH RNA TO A CELL
CA2688061A1 (en) * 2007-05-23 2008-12-04 Mannkind Corporation Multicistronic vectors and methods for their design
EP2606134B1 (en) 2010-08-17 2019-04-10 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HEPATITIS B VIRUS (HBV) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
EP2723865B1 (en) 2011-06-21 2019-03-27 Alnylam Pharmaceuticals, Inc. METHODS FOR DETERMINING ACTIVITY OF RNAi IN A SUBJECT
US20130064881A1 (en) * 2011-09-08 2013-03-14 Gradalis, Inc. Compositions and methods for treating prostate cancer
EP2773777B1 (en) 2011-10-31 2020-05-13 University of Utah Research Foundation Genetic alterations in glioblastoma
US10227603B2 (en) * 2012-05-15 2019-03-12 Micromedmark Biotech Co., Ltd. Plants as functional microRNA and/or functional siRNA carriers, preparation methods and uses thereof
WO2015048690A1 (en) 2013-09-27 2015-04-02 The Regents Of The University Of California Optimized small guide rnas and methods of use
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) * 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
CA2948728A1 (en) 2014-03-10 2015-09-17 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2016030863A1 (en) 2014-08-29 2016-03-03 Glaxosmithkline Intellectual Property Development Limited Compounds and methods for treating viral infections
US20180291382A1 (en) * 2014-11-05 2018-10-11 The Regents Of The University Of California Methods for Autocatalytic Genome Editing and Neutralizing Autocatalytic Genome Editing
SG11201703638XA (en) 2014-11-10 2017-06-29 Alnylam Pharmaceuticals Inc Hepatitis b virus (hbv) irna compositions and methods of use thereof
WO2016141243A1 (en) * 2015-03-03 2016-09-09 The Regents Of The University Of California Enzymatic modification of nucleic acids
TWI554608B (en) * 2015-03-27 2016-10-21 Univ China Medical A nucleic acid sequence segment for enhancing protein expression, an expression vector comprising a sequence segment thereof, and a kit comprising its expression vector
WO2017177029A1 (en) * 2016-04-06 2017-10-12 Duke University Compositions and methods for enhanced gene expression and viral replication
CA3032822A1 (en) 2016-08-02 2018-02-08 Editas Medicine, Inc. Compositions and methods for treating cep290 associated disease
WO2018031751A1 (en) * 2016-08-12 2018-02-15 The Regents Of The University Of California Constructs and methods for signal amplification
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic 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
BR112020002394A2 (en) 2017-08-09 2020-07-28 Bioverativ Therapeutics Inc. nucleic acid molecules and uses thereof
AU2019240068B2 (en) 2018-03-21 2025-03-13 Aldevron, L.L.C. Viral and non-viral nanoplasmid vectors with improved production
KR102925832B1 (en) * 2018-08-09 2026-02-11 바이오버라티브 테라퓨틱스 인크. Nucleic acid molecules and their use in non-viral gene therapy
MA70658B1 (en) 2018-08-13 2026-04-30 Alnylam Pharmaceuticals, Inc. Compositions of hepatitis B virus (HBV) RNA/DB agents and their methods of use
JP2022523320A (en) * 2019-01-29 2022-04-22 ザ ジェネラル ホスピタル コーポレイション Oligonucleotides and methods for the treatment of age-related macular degeneration
CN114277030B (en) * 2021-12-28 2024-07-16 滨州医学院 A pri-miRNA improved sequence and a vector expressing the sequence

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005035718A2 (en) * 2003-10-03 2005-04-21 Welgen, Inc. Biderectional promoters for small rna expression
WO2005111219A1 (en) * 2004-04-16 2005-11-24 University Of Washington METHODS AND VECTORS FOR EXPRESSING siRNA

Family Cites Families (35)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5643771A (en) 1980-05-19 1997-07-01 The Board Of Trustees Of The Leland Stanford Junior University Non-reverting live bacterial vaccines
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5279833A (en) 1990-04-04 1994-01-18 Yale University Liposomal transfection of nucleic acids into animal cells
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
US5547862A (en) 1993-07-29 1996-08-20 Ambion Inc. Vectors containing multiple promoters in the same orientation
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
JPH09510601A (en) 1993-11-12 1997-10-28 ケース・ウエスタン・リザーブ・ユニバーシティ Episomal expression vector for human gene therapy
US5739118A (en) 1994-04-01 1998-04-14 Apollon, Inc. Compositions and methods for delivery of genetic material
US5585263A (en) 1994-05-20 1996-12-17 University Of Alabama At Birmingham Research Foundation Purified retroviral constitutive transport enhancer and its use to facilitate mRNA transport, and to produce recombinant, attenuated HIV
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5877159A (en) 1995-05-03 1999-03-02 University Of Maryland At Baltimore Method for introducing and expressing genes in animal cells and live invasive bacterial vectors for use in the same
US5932241A (en) 1995-06-07 1999-08-03 Valentis, Incorporated Cationic lipid DNA complexes for gene targeting
US5824538A (en) 1995-09-06 1998-10-20 The United States Of America As Represented By The Secretary Of The Army Shigella vector for delivering DNA to a mammalian cell
WO1997014431A1 (en) 1995-10-20 1997-04-24 Collagen Corporation Production of recombinant procollagen in yeast
US5851804A (en) 1996-05-06 1998-12-22 Apollon, Inc. Chimeric kanamycin resistance gene
ATE386126T1 (en) 1996-11-08 2008-03-15 Neose Technologies Inc IMPROVED EXPRESSION VECTORS
US5874242A (en) 1997-03-19 1999-02-23 University Of Georgia Research Foundation, Inc. Efficient translation in eukaryotic and prokaryotic systems
AU745805B2 (en) 1997-10-28 2002-04-11 Wyeth Compositions and methods for delivery of genetic material
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
BR9908223A (en) 1998-03-16 2000-10-24 Procter & Gamble Method to reduce cellulite on the skin of mammals
CA2370628A1 (en) 1999-04-21 2000-10-26 American Home Products Corporation Methods and compositions for inhibiting the function of polynucleotide sequences
CA2369944A1 (en) * 2001-01-31 2002-07-31 Nucleonics Inc. Use of post-transcriptional gene silencing for identifying nucleic acid sequences that modulate the function of a cell
JP2003269028A (en) 2002-03-18 2003-09-25 Aisin Seiki Co Ltd Door unlocking device
AU2003266014B2 (en) 2002-05-06 2009-05-14 Alnylam Pharmaceuticals, Inc. Methods for delivery of nucleic acids
WO2004011624A2 (en) 2002-07-31 2004-02-05 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
US7303906B2 (en) 2002-09-06 2007-12-04 Wisconsin Alumni Research Foundation Competent bacteria
CA2502649A1 (en) 2002-10-18 2004-04-29 Nucleonics Inc. Double-stranded rna structures and constructs, and methods for generating and using the same
EP1597351B1 (en) 2003-02-27 2012-04-18 Alnylam Pharmaceuticals Inc. METHODS AND CONSTRUCTS FOR EVALUATION OF RNAi TARGETS AND EFFECTOR MOLECULES
EP2284268A3 (en) 2003-06-12 2012-05-30 Alnylam Pharmaceuticals Inc. Conserved HBV and HCV sequences useful for gene silencing
WO2005007877A2 (en) 2003-07-18 2005-01-27 University Of Massachusetts Regulatable promoters for synthesis of small hairpin rna
WO2005007875A2 (en) 2003-07-18 2005-01-27 University Of Massachusetts Enhanced promoters for synthesis of small hairpin rna
WO2005040388A2 (en) 2003-08-22 2005-05-06 Nucleonics Inc. Eukariotic expression systems for expression of inhibitory rna in multiple intracellular compartments

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005035718A2 (en) * 2003-10-03 2005-04-21 Welgen, Inc. Biderectional promoters for small rna expression
WO2005111219A1 (en) * 2004-04-16 2005-11-24 University Of Washington METHODS AND VECTORS FOR EXPRESSING siRNA

Non-Patent Citations (11)

* Cited by examiner, † Cited by third party
Title
BODEN D. ET AL.: "Promoter choice affects the potency of HIV-1 specific RNA interference", NUCLEIC ACIDS RESEARCH, vol. 31, no. 7, 2003, pages 5033 - 5038, XP002383800 *
CZAUDERNA F. ET AL.: "Inducible shRNA expression for application in a prostate cancer mouse model", NUCLEIC ACIDS RESEARCH, vol. 31, no. 21, 2003, pages E127, XP002341775 *
DATABASE EMBL-EBI 20 May 1992 (1992-05-20), MURPHY S. ET AL.: "7SK RNA; Alu repetitive sequence; small cytoplasmic RNA", XP002404574, Database accession no. X04992 *
GILADI H. ET AL.: "Small interfering RNA inhibits Hepatitis B virus replication in mice", MOLECULAR THERAPY, ACADEMIC PRESS, SAN DIEGO, CA,, US, vol. 8, no. 5, November 2003 (2003-11-01), pages 769 - 776, XP008040415, ISSN: 1525-0016 *
KRÖNKE J. ET AL.: "Alternative approaches for efficient inhibition of Hepatitis C virus RNA replication by small interfering RNAs", JOURNAL OF VIROLOGY, vol. 78, no. 7, April 2004 (2004-04-01), pages 3436 - 3446, XP002300966 *
LI M.-J. ET AL.: "Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs", MOLECULAR THERAPY, vol. 8, no. 2, August 2003 (2003-08-01), pages 196 - 206, XP002982612 *
MCCAFFREY A.P. ET AL.: "Inhibition of hepatitis B virus in mice by RNA interference", NATURE BIOTECHNOLOGY, vol. 21, no. 6, June 2003 (2003-06-01), pages 639 - 644, XP002311189 *
MURPHY S. ET AL.: "A sequence upstream from the coding region is required for the transcription of the 7SK RNA genes", NUCLEIC ACIDS RESEARCH, vol. 14, no. 23, 1986, pages 9243 - 9260, XP002383798 *
PEBERNARD S. AND IGGO R.D.: "Determinants of interferon-stimulated gene induction by RNAi vectors", DIFFERENTIATION, vol. 72, March 2004 (2004-03-01), pages 103 - 111, XP002383799 *
TALLET-LOPEZ B. ET AL.: "Antisense oligonucleotides targeted to the domain IIId of the hepatitis C virus IRES compete with 40S ribosomal subunit binding and prevent in vitro translation", NUCLEIC ACIDS RESEARCH, vol. 31, no. 2, 2003, pages 734 - 742, XP002383801 *
YING CHEN ET AL.: "Inhibition of hepatitis B virus replication by stably expressed shRNA", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 311, 2003, pages 398 - 404, XP004468647 *

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