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WO2004038019A3 - Dnazyme cleaving mutant polynucleotides - Google Patents

Dnazyme cleaving mutant polynucleotides Download PDF

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Publication number
WO2004038019A3
WO2004038019A3 PCT/GB2003/004614 GB0304614W WO2004038019A3 WO 2004038019 A3 WO2004038019 A3 WO 2004038019A3 GB 0304614 W GB0304614 W GB 0304614W WO 2004038019 A3 WO2004038019 A3 WO 2004038019A3
Authority
WO
WIPO (PCT)
Prior art keywords
dnazyme
mutant polynucleotides
dnazymes
cleaving
flanking regions
Prior art date
Application number
PCT/GB2003/004614
Other languages
French (fr)
Other versions
WO2004038019A2 (en
Inventor
David Beeson
Matthew Wood
Amr Abdelgany
Original Assignee
Isis Innovation
David Beeson
Matthew Wood
Amr Abdelgany
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 Isis Innovation, David Beeson, Matthew Wood, Amr Abdelgany filed Critical Isis Innovation
Priority to AU2003274382A priority Critical patent/AU2003274382A1/en
Publication of WO2004038019A2 publication Critical patent/WO2004038019A2/en
Publication of WO2004038019A3 publication Critical patent/WO2004038019A3/en

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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

The present invention relates to DNAzymes that are designed to target mutant alleles and cleave at a site remote from the mutation site. The DNAzymes bind selectively to a mutant allele and comprise a central catalytic motif and two flanking regions, wherein at least one of the flanking regions has a polynucleotide sequence complementary to a region that includes the mutation in the mutant allele.
PCT/GB2003/004614 2002-10-23 2003-10-23 Dnazyme cleaving mutant polynucleotides WO2004038019A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2003274382A AU2003274382A1 (en) 2002-10-23 2003-10-23 Dnazyme cleaving mutant polynucleotides

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
GB0224663.5 2002-10-23
GB0224663A GB0224663D0 (en) 2002-10-23 2002-10-23 DNAzyme

Publications (2)

Publication Number Publication Date
WO2004038019A2 WO2004038019A2 (en) 2004-05-06
WO2004038019A3 true WO2004038019A3 (en) 2004-08-19

Family

ID=9946431

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/GB2003/004614 WO2004038019A2 (en) 2002-10-23 2003-10-23 Dnazyme cleaving mutant polynucleotides

Country Status (3)

Country Link
AU (1) AU2003274382A1 (en)
GB (1) GB0224663D0 (en)
WO (1) WO2004038019A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9856480B2 (en) * 2013-01-14 2018-01-02 National Taiwan University Dnazyme for silencing the expression of EGFR

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997037013A1 (en) * 1996-04-02 1997-10-09 Commonwealth Scientific And Industrial Research Organisation Asymmetric hammerhead ribozymes

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997037013A1 (en) * 1996-04-02 1997-10-09 Commonwealth Scientific And Industrial Research Organisation Asymmetric hammerhead ribozymes

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
CAIRNS M J ET AL: "Nucleic acid mutation analysis using catalytic DNA", NUCLEIC ACIDS RESEARCH, vol. 28, no. 3, 1 February 2000 (2000-02-01), pages E9, XP002233619, ISSN: 0305-1048 *
PHYLACTOU L A ET AL: "HAMMERHEAD RIBOZYMES TARGETED TO THE FBN1 MRNA CAN DISCRIMINATE A SINGLE BASE MISMATCH BETWEEN RIBOZYME AND TARGET", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 249, no. 3, 1998, pages 804 - 810, XP000914845, ISSN: 0006-291X *
PHYLACTOU L A ET AL: "Potential therapy paradigms for Marfan syndrome", EXPERT OPINION ON INVESTIGATIONAL DRUGS, vol. 8, no. 7, 1999, pages 983 - 993, XP008031688, ISSN: 1354-3784 *
PHYLACTOU LEONIDAS A ET AL: "Ribozymes as therapeutic tools for genetic disease", HUMAN MOLECULAR GENETICS, vol. 7, no. 10, 1998, pages 1649 - 1653, XP002285064, ISSN: 0964-6906 *
SANTORO S W ET AL: "MECHANISM AND UTILITY OF AN RNA-CLEAVING DNA ENZYME", BIOCHEMISTRY, vol. 37, no. 22, September 1998 (1998-09-01), pages 13330 - 13342, XP001009842, ISSN: 0006-2960 *
SZYRACH M ET AL: "Cleavage of AML1/MTG8 by asymmetric hammerhead ribozymes", EUROPEAN JOURNAL OF BIOCHEMISTRY, vol. 268, no. 12, June 2001 (2001-06-01), pages 3550 - 3557, XP002251944, ISSN: 0014-2956 *
WOOD M J ET AL: "RIBOZYMES AND SIRNA FOR THE TREATMENT OF DISEASES OF THE NERVOUS SYSTEM", CURRENT OPINION IN MOLECULAR THERAPEUTICS, vol. 5, no. 4, 2003, pages 383 - 388, XP009029174, ISSN: 1464-8431 *

Also Published As

Publication number Publication date
WO2004038019A2 (en) 2004-05-06
GB0224663D0 (en) 2002-12-04
AU2003274382A8 (en) 2004-05-13
AU2003274382A1 (en) 2004-05-13

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