EP1581635A4 - Durch sirna vermitteltes gen-silencing - Google Patents

Durch sirna vermitteltes gen-silencing

Info

Publication number
EP1581635A4
EP1581635A4 EP03814141A EP03814141A EP1581635A4 EP 1581635 A4 EP1581635 A4 EP 1581635A4 EP 03814141 A EP03814141 A EP 03814141A EP 03814141 A EP03814141 A EP 03814141A EP 1581635 A4 EP1581635 A4 EP 1581635A4
Authority
EP
European Patent Office
Prior art keywords
sirna
gene silencing
mediated gene
molecules
allele
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.)
Withdrawn
Application number
EP03814141A
Other languages
English (en)
French (fr)
Other versions
EP1581635A2 (de
Inventor
Henry Paulson
Victor Miller
Cynthia Gouvion
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.)
University of Iowa Research Foundation UIRF
Original Assignee
University of Iowa Research Foundation UIRF
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 US10/430,351 external-priority patent/US20040023390A1/en
Priority claimed from PCT/US2003/016887 external-priority patent/WO2004013280A2/en
Application filed by University of Iowa Research Foundation UIRF filed Critical University of Iowa Research Foundation UIRF
Publication of EP1581635A2 publication Critical patent/EP1581635A2/de
Publication of EP1581635A4 publication Critical patent/EP1581635A4/de
Withdrawn legal-status Critical Current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
    • C12N15/113—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
    • C12N15/1137—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 enzymes
    • 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
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus
EP03814141A 2002-12-17 2003-12-16 Durch sirna vermitteltes gen-silencing Withdrawn EP1581635A4 (de)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US32208602A 2002-12-17 2002-12-17
US322086 2002-12-17
US430351 2003-05-05
US10/430,351 US20040023390A1 (en) 2002-08-05 2003-05-05 SiRNA-mediated gene silencing with viral vectors
WOPCT/US03/16887 2003-05-26
PCT/US2003/016887 WO2004013280A2 (en) 2002-08-05 2003-05-26 ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
PCT/US2003/040292 WO2004058940A2 (en) 2002-08-05 2003-12-16 Sirna-mediated gene silencing

Publications (2)

Publication Number Publication Date
EP1581635A2 EP1581635A2 (de) 2005-10-05
EP1581635A4 true EP1581635A4 (de) 2007-10-17

Family

ID=32685878

Family Applications (1)

Application Number Title Priority Date Filing Date
EP03814141A Withdrawn EP1581635A4 (de) 2002-12-17 2003-12-16 Durch sirna vermitteltes gen-silencing

Country Status (2)

Country Link
EP (1) EP1581635A4 (de)
AU (1) AU2003301030A1 (de)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004013355A1 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation Sirna-mediated gene silencing with viral vectors
WO2004013280A2 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004013355A1 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation Sirna-mediated gene silencing with viral vectors
WO2004013280A2 (en) * 2002-08-05 2004-02-12 University Of Iowa Research Foundation ALLELE-SPECIFIC siRNA-MEDIATED GENE SILENCING
WO2004047872A2 (en) * 2002-11-26 2004-06-10 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of short interfering rna (sirna)

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
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 *
DING H ET AL: "Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis", AGING CELL, BLACKWELL PUBLISHING,, GB, vol. 2, 2003, pages 209 - 217, XP002389534, ISSN: 1474-9718 *
DONZE O ET AL: "RNA interference in mammalian cells using siRNAs synthesized with T7 RNA polymerase", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 30, no. 10, 15 May 2002 (2002-05-15), pages e46 - 1, XP002279045, ISSN: 0305-1048 *
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 *
MILLER V M ET AL: "Targeting Alzheimer's disease genes with RNA interference: An efficient strategy for silencing mutant alleles", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 32, no. 2, 2004, pages 661 - 668, XP002315762, ISSN: 0305-1048 *
SCHWARZ D.S. ET AL: "Asymmetry in the assembly of the RNAi enzyme complex", CELL, vol. 115, 17 October 2003 (2003-10-17), pages 199 - 208, XP002521543, DOI: 10.1016/S0092-8674(03)00759-1 *

Also Published As

Publication number Publication date
AU2003301030A1 (en) 2004-07-22
AU2003301030A8 (en) 2004-07-22
EP1581635A2 (de) 2005-10-05

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