WO2008109547A3 - Nucleic acid compounds for inhibiting tymp gene expression and uses thereof - Google Patents

Nucleic acid compounds for inhibiting tymp gene expression and uses thereof Download PDF

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Publication number
WO2008109547A3
WO2008109547A3 PCT/US2008/055697 US2008055697W WO2008109547A3 WO 2008109547 A3 WO2008109547 A3 WO 2008109547A3 US 2008055697 W US2008055697 W US 2008055697W WO 2008109547 A3 WO2008109547 A3 WO 2008109547A3
Authority
WO
WIPO (PCT)
Prior art keywords
tymp
nucleic acid
gene expression
inhibiting
acid compounds
Prior art date
Application number
PCT/US2008/055697
Other languages
French (fr)
Other versions
WO2008109547A9 (en
WO2008109547A4 (en
WO2008109547A2 (en
Inventor
Steven C Quay
James Mcswiggen
Narendra K Vaish
Mohammad Ahmadian
Original Assignee
Mdrna Inc
Steven C Quay
James Mcswiggen
Narendra K Vaish
Mohammad Ahmadian
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 Mdrna Inc, Steven C Quay, James Mcswiggen, Narendra K Vaish, Mohammad Ahmadian filed Critical Mdrna Inc
Publication of WO2008109547A2 publication Critical patent/WO2008109547A2/en
Publication of WO2008109547A3 publication Critical patent/WO2008109547A3/en
Publication of WO2008109547A9 publication Critical patent/WO2008109547A9/en
Publication of WO2008109547A4 publication Critical patent/WO2008109547A4/en
Priority to US12/552,082 priority Critical patent/US20100105134A1/en
Priority to US13/327,545 priority patent/US20130011922A1/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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.

Landscapes

  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing TYMP gene expression. An mdRNA of this disclosure comprises at least three strands that combine to form at least two non-overlapping double-stranded regions separated by a nick or gap wherein one strand is complementary to a TYMP mRNA. In addition, the meroduplex may have at least one uridine is a 5-methyluridine, a nucleoside is a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of a TYMP gene in a cell or in a subject to treat a TYMP-related disease.
PCT/US2008/055697 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting tymp gene expression and uses thereof WO2008109547A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US12/552,082 US20100105134A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof
US13/327,545 US20130011922A1 (en) 2007-03-02 2011-12-15 Nucleic acid compounds for inhibiting gene expression and uses thereof

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US93494007P 2007-03-02 2007-03-02
US60/934,940 2007-03-02
US93493007P 2007-03-16 2007-03-16
US60/934,930 2007-03-16
US1240107P 2007-12-07 2007-12-07
US61/012,401 2007-12-07

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055662 Continuation-In-Part WO2008109526A1 (en) 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting egr gene expression and uses thereof

Related Child Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2008/055627 Continuation-In-Part WO2008109506A1 (en) 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting jun gene expression and uses thereof
US12/552,082 Continuation-In-Part US20100105134A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof
AU2009212920A Division AU2009212920A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof

Publications (4)

Publication Number Publication Date
WO2008109547A2 WO2008109547A2 (en) 2008-09-12
WO2008109547A3 true WO2008109547A3 (en) 2008-11-06
WO2008109547A9 WO2008109547A9 (en) 2008-12-24
WO2008109547A4 WO2008109547A4 (en) 2009-02-19

Family

ID=39639020

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055697 WO2008109547A2 (en) 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting tymp gene expression and uses thereof

Country Status (1)

Country Link
WO (1) WO2008109547A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2007310982A1 (en) 2006-10-18 2008-04-24 Marina Biotech, Inc. Nicked or gapped nucleic acid molecules and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004111237A1 (en) * 2003-04-16 2004-12-23 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET-DERIVED ENDOTHELIAL CELL GROWTH FACTOR (ECGF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2007056153A2 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004111237A1 (en) * 2003-04-16 2004-12-23 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET-DERIVED ENDOTHELIAL CELL GROWTH FACTOR (ECGF1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2007056153A2 (en) * 2005-11-04 2007-05-18 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
WO2007107162A2 (en) * 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BRAMSEN J B ET AL: "Improved silencing properties using small internally segmented interfering RNAs", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 35, no. 17, 28 July 2007 (2007-07-28), pages 5886 - 5897, XP002467844, ISSN: 0305-1048 *
LEUSCHNER P J F ET AL: "Cleavage of the siRNA passenger strand during RISC assembly in human cells", EMBO REPORTS, XX, XX, vol. 7, no. 3, 1 March 2006 (2006-03-01), pages 314 - 320, XP002467845 *
MATRANGA CHRISTIAN ET AL: "Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes", CELL,, vol. 123, no. 4, 1 November 2005 (2005-11-01), pages 607 - 620, XP002484663 *
MILLS JANINE B ET AL: "Origin of the intrinsic rigidity of DNA", NUCLEIC ACIDS RESEARCH,, vol. 32, no. 13, 1 January 2004 (2004-01-01), pages 4055 - 4059, XP002484664 *

Also Published As

Publication number Publication date
WO2008109547A9 (en) 2008-12-24
WO2008109547A4 (en) 2009-02-19
WO2008109547A2 (en) 2008-09-12

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