WO2004042046A3 - Wirksame und stabile dna-enzyme - Google Patents

Wirksame und stabile dna-enzyme Download PDF

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Publication number
WO2004042046A3
WO2004042046A3 PCT/EP2003/012413 EP0312413W WO2004042046A3 WO 2004042046 A3 WO2004042046 A3 WO 2004042046A3 EP 0312413 W EP0312413 W EP 0312413W WO 2004042046 A3 WO2004042046 A3 WO 2004042046A3
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WO
WIPO (PCT)
Prior art keywords
host cells
sirna
dna enzymes
dna
medicaments
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/EP2003/012413
Other languages
English (en)
French (fr)
Other versions
WO2004042046A2 (de
Inventor
Steffen Schubert
Jens Kurreck
Arnold Gruenweller
Volker Erdmann
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.)
Grünenthal GmbH
Original Assignee
Grünenthal GmbH
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 Grünenthal GmbH filed Critical Grünenthal GmbH
Priority to EP03779858A priority Critical patent/EP1556486B1/de
Priority to AU2003288006A priority patent/AU2003288006A1/en
Priority to DE50312344T priority patent/DE50312344D1/de
Priority to AT03779858T priority patent/ATE455170T1/de
Publication of WO2004042046A2 publication Critical patent/WO2004042046A2/de
Publication of WO2004042046A3 publication Critical patent/WO2004042046A3/de
Priority to US11/123,097 priority patent/US20060122136A1/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • C12N15/1138—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 receptors or cell surface proteins
    • 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
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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

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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)
  • Enzymes And Modification Thereof (AREA)

Abstract

Die vorliegende Erfindung betrifft DNA-Enzyme vom Typ 10-23, die durch Modifizierung bestimmter Nukleotide in der Kernsequenz besonders stabil sind und darüber hinaus im wesentlichen die gleiche oder eine höhere Spalteffizienz gegenüber ihrem Substrat aufweisen, wie die entsprechenden nicht-modifizierten DNA-Enzyme. Weitere Gegenstände der vorliegenden Anmeldung betreffen Wirtszellen, welche die erfindungsgemäßen DNA-Enzyme enthalten. Darüber hinaus wird ein Arzneimittel bereitgestellt, welches die erfindungsgemäßen DNA-Enzyme bzw. Wirtszellen enthalten. Die DNA-Enzyme und weiteren Gegenstände sind insbesondere gegen den Vanilloid-Rezeptor 1 (VR1) bzw. Picornaviren gerichtet. Weitere Gegenstände der vorliegenden Erfindung sind kleine Interferenz-RNA-Moleküle (siRNA), die gegen VR1 gerichtet sind, und die siRNA enthaltende Wirtszellen. Die siRNA und entsprechende Wirtszellen eignen sich als Arzneimittel bzw. zur Herstellung von Arzneimitteln, insbesondere zur Behandlung von Schmerzen und anderen mit VR1 zusammenhängenden krankhaften Zuständen.
PCT/EP2003/012413 2002-11-06 2003-11-06 Wirksame und stabile dna-enzyme Ceased WO2004042046A2 (de)

Priority Applications (5)

Application Number Priority Date Filing Date Title
EP03779858A EP1556486B1 (de) 2002-11-06 2003-11-06 Wirksame und stabile dna-enzyme
AU2003288006A AU2003288006A1 (en) 2002-11-06 2003-11-06 Effective and stable dna enzymes
DE50312344T DE50312344D1 (de) 2002-11-06 2003-11-06 Wirksame und stabile dna-enzyme
AT03779858T ATE455170T1 (de) 2002-11-06 2003-11-06 Wirksame und stabile dna-enzyme
US11/123,097 US20060122136A1 (en) 2002-11-06 2005-05-06 Effective and stable DNA enzymes

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
DE10251682.0 2002-11-06
DE10251682 2002-11-06
DE10322662A DE10322662A1 (de) 2002-11-06 2003-05-15 Wirksame und stabile DNA-Enzyme
DE10322662.1 2003-05-15

Related Child Applications (1)

Application Number Title Priority Date Filing Date
US11/123,097 Continuation US20060122136A1 (en) 2002-11-06 2005-05-06 Effective and stable DNA enzymes

Publications (2)

Publication Number Publication Date
WO2004042046A2 WO2004042046A2 (de) 2004-05-21
WO2004042046A3 true WO2004042046A3 (de) 2004-09-30

Family

ID=32313543

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2003/012413 Ceased WO2004042046A2 (de) 2002-11-06 2003-11-06 Wirksame und stabile dna-enzyme

Country Status (7)

Country Link
US (1) US20060122136A1 (de)
EP (1) EP1556486B1 (de)
AT (1) ATE455170T1 (de)
AU (1) AU2003288006A1 (de)
DE (2) DE10322662A1 (de)
ES (1) ES2339654T3 (de)
WO (1) WO2004042046A2 (de)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2004221760B2 (en) 2003-03-21 2010-03-18 Roche Innovation Center Copenhagen A/S Short interfering RNA (siRNA) analogues
DK1781787T3 (en) * 2004-08-23 2017-07-31 Sylentis Sau TREATMENT OF EYE DISORDERS FEATURED BY AN INCREASED INTRAOCULAR PRESSURE WITH SIRNAS
GB0521351D0 (en) * 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
ES2556128T3 (es) 2006-03-23 2016-01-13 Roche Innovation Center Copenhagen A/S ARN interfiriente pequeño internamente segmentado
EP2390327A1 (de) 2010-05-27 2011-11-30 Sylentis S.A. siRNA und deren Verwendung in Verfahren und Zusammensetzungen zur Behandlung und/oder Prävention von Augenleiden
AU2012389270B2 (en) 2012-09-05 2018-11-08 Sylentis S.A.U. siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
GB201215857D0 (en) 2012-09-05 2012-10-24 Sylentis Sau siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
EP3066114B1 (de) * 2013-11-07 2019-11-13 Agilent Technologies, Inc. Mehrere transposaseadapter für dna-manipulationen
WO2015132303A1 (en) 2014-03-04 2015-09-11 Sylentis Sau Sirnas and their use in methods and compositions for the treatment and/or prevention of eye conditions
DK3095866T3 (da) 2015-05-20 2019-06-24 Secarna Pharmaceuticals Gmbh & Co Kg Middel til profylakse og terapi af virusinfektioner
EP3263115A1 (de) * 2016-06-30 2018-01-03 Technische Hochschule Mittelhessen Erfindung betreffend wirkstoff gegen pseudomonas aeruginosa
WO2022232683A2 (en) * 2021-04-30 2022-11-03 The Regents Of The University Of California Multicomponent xnazyme-based nucleic acid detection system

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002018407A2 (de) * 2000-09-02 2002-03-07 Grünenthal GmbH Antisense oligonukleotide gegen vanilloid rezeptor 1
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002018407A2 (de) * 2000-09-02 2002-03-07 Grünenthal GmbH Antisense oligonukleotide gegen vanilloid rezeptor 1
WO2002044321A2 (en) * 2000-12-01 2002-06-06 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Rna interference mediating small rna molecules

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
BEIGELMAN L ET AL: "CHEMICAL MODIFICATION OF HAMMERHEAD RIBOZYMES CATALYTIC ACTIVITY AND NUCLEASE RESISTANCE", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY OF BIOLOGICAL CHEMISTS, BALTIMORE, MD, US, vol. 270, no. 43, 27 October 1995 (1995-10-27), pages 25702 - 25708, XP000601905, ISSN: 0021-9258 *
BRUMMELKAMP T R ET AL: "STABLE SUPPRESSION OF TUMORIGENICITY BY VIRUS-MEDIATED RNA INTERFERENCE", CANCER CELL, XX, US, vol. 2, no. 3, September 2002 (2002-09-01), pages 243 - 247, XP009006464, ISSN: 1535-6108 *
GRÜNWELLER ARNOLD ET AL: "Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2'-O-methyl RNA, phosphorothioates and small interfering RNA.", NUCLEIC ACIDS RESEARCH. 15 JUN 2003, vol. 31, no. 12, 15 June 2003 (2003-06-15), pages 3185 - 3193, XP002286510, ISSN: 1362-4962 *
NOVINA C D ET AL: "siRNA directed inhibition of HIV-1 infection", NATURE MEDICINE, NATURE PUBLISHING, CO, US, vol. 7, no. 8, July 2002 (2002-07-01), pages 681 - 686, XP002955298, ISSN: 1078-8956 *
SCHUBERT STEFFEN ET AL: "RNA cleaving '10-23' DNAzymes with enhanced stability and activity.", NUCLEIC ACIDS RESEARCH. ENGLAND 15 OCT 2003, vol. 31, no. 20, 15 October 2003 (2003-10-15), pages 5982 - 5992, XP002272988, ISSN: 1362-4962 *
SIOUD M ET AL: "Design of nuclease resistant protein kinase calpha DNA enzymes with potential therapeutic application", JOURNAL OF MOLECULAR BIOLOGY, LONDON, GB, vol. 296, no. 3, 25 February 2000 (2000-02-25), pages 937 - 947, XP004461588, ISSN: 0022-2836 *
WARASHINA M ET AL: "Extremely high and specific activity of DNA enzymes in cells with a Philadelphia chromosome.", CHEMISTRY & BIOLOGY. ENGLAND APR 1999, vol. 6, no. 4, April 1999 (1999-04-01), pages 237 - 250, XP002272987, ISSN: 1074-5521 *

Also Published As

Publication number Publication date
US20060122136A1 (en) 2006-06-08
DE10322662A1 (de) 2004-10-07
AU2003288006A1 (en) 2004-06-07
AU2003288006A8 (en) 2004-06-07
WO2004042046A2 (de) 2004-05-21
ATE455170T1 (de) 2010-01-15
DE50312344D1 (de) 2010-03-04
ES2339654T3 (es) 2010-05-24
EP1556486A2 (de) 2005-07-27
EP1556486B1 (de) 2010-01-13

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