WO2008109498A3 - Nucleic acid compounds for inhibiting hdac gene expression and uses thereof - Google Patents

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

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Publication number
WO2008109498A3
WO2008109498A3 PCT/US2008/055612 US2008055612W WO2008109498A3 WO 2008109498 A3 WO2008109498 A3 WO 2008109498A3 US 2008055612 W US2008055612 W US 2008055612W WO 2008109498 A3 WO2008109498 A3 WO 2008109498A3
Authority
WO
WIPO (PCT)
Prior art keywords
hdac
nucleic acid
gene expression
acid compounds
mdrna
Prior art date
Application number
PCT/US2008/055612
Other languages
French (fr)
Other versions
WO2008109498A2 (en
WO2008109498A8 (en
WO2008109498A4 (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 WO2008109498A2 publication Critical patent/WO2008109498A2/en
Publication of WO2008109498A3 publication Critical patent/WO2008109498A3/en
Publication of WO2008109498A4 publication Critical patent/WO2008109498A4/en
Priority to US12/552,082 priority Critical patent/US20100105134A1/en
Publication of WO2008109498A8 publication Critical patent/WO2008109498A8/en
Priority to US13/327,545 priority patent/US20130011922A1/en

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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • C12N2310/30—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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • 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

Abstract

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing HDAC (e.g., HDAC1, HDAC2, HDAC3, HDAC4, HDAC5, HDAC6, HDAC7A, HDAC8, HDAC9, HDAC10, HDAC11) 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 an HDAC1, HDAC2, HDAC3, HDAC4, HDAC5, HDAC6, HDAC7A, HDAC8, HDAC9, HDAC10, or HDAC11 1 mRNA. In addition, the meroduplex may have at least one uridine substituted with a 5-methyluridine, a nucleoside replaced with a locked nucleic acid, or optionally other modifications, and any combination thereof. Also provided are methods of decreasing expression of an HDAC gene in a cell or in a subject to treat an HDAC-related disease.
PCT/US2008/055612 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting hdac gene expression and uses thereof WO2008109498A2 (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
US1240407P 2007-12-07 2007-12-07
US61/012,404 2007-12-07

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055622 Continuation-In-Part WO2008109503A1 (en) 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting ms4a1 gene expression and uses thereof

Related Child Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2008/055563 Continuation-In-Part WO2008109475A1 (en) 2007-03-02 2008-02-29 Nucleic acid compounds for inhibiting sirt2 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
US12/552,082 Continuation-In-Part US20100105134A1 (en) 2007-03-02 2009-09-01 Nucleic acid compounds for inhibiting gene expression and uses thereof

Publications (4)

Publication Number Publication Date
WO2008109498A2 WO2008109498A2 (en) 2008-09-12
WO2008109498A3 true WO2008109498A3 (en) 2009-03-12
WO2008109498A4 WO2008109498A4 (en) 2009-04-30
WO2008109498A8 WO2008109498A8 (en) 2009-12-10

Family

ID=39720233

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055612 WO2008109498A2 (en) 2007-03-02 2008-03-03 Nucleic acid compounds for inhibiting hdac gene expression and uses thereof

Country Status (1)

Country Link
WO (1) WO2008109498A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9074205B2 (en) 2006-10-18 2015-07-07 Marina Biotech, Inc. Nicked or gapped nucleic acid molecules and uses thereof
US8664182B2 (en) 2008-11-12 2014-03-04 Duke University Methods of inhibiting cancer cell growth with HDAC inhibitors and methods of screening for HDAC10 inhibitors
EP3099811B1 (en) * 2014-01-28 2018-09-12 Qiagen GmbH Method of amplification of a short tandem repeat locus
CN111840311B (en) * 2020-07-31 2021-11-12 上海交通大学医学院附属第九人民医院 Use of siRNA molecule composition for preparing medicament for inhibiting scar formation against HDAC5

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060142230A1 (en) * 2003-08-25 2006-06-29 Nastech Pharmaceutical Company Inc. Double-stranded ribonucleic acid molecules having ribothymidine
US20060148743A1 (en) * 2001-05-18 2006-07-06 Vasant Jadhav RNA interference mediated inhibition of histone deacetylase (HDAC) 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
WO2008030239A1 (en) * 2006-09-05 2008-03-13 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HISTONE DEACETYLASE (HDAC) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2008049078A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060148743A1 (en) * 2001-05-18 2006-07-06 Vasant Jadhav RNA interference mediated inhibition of histone deacetylase (HDAC) gene expression using short interfering nucleic acid (siNA)
US20060142230A1 (en) * 2003-08-25 2006-06-29 Nastech Pharmaceutical Company Inc. Double-stranded ribonucleic acid molecules having ribothymidine
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
WO2008030239A1 (en) * 2006-09-05 2008-03-13 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF HISTONE DEACETYLASE (HDAC) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2008049078A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof

Non-Patent Citations (8)

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Title
ADCOCK I M ET AL: "Abnormal histone acetylase and deacetylase expression and function in lung inflammation", INFLAMMATION RESEARCH, vol. 55, no. 8, 1 August 2006 (2006-08-01), pages 311 - 321, XP019417186, ISSN: 1420-908X *
BRAMSEN J B ET AL: "Improved silencing properties using small internally segmented interfering RNAs", NUCLEIC ACIDS RESEARCH, vol. 35, no. 17, 28 July 2007 (2007-07-28), pages 5886 - 5897, XP002467844, ISSN: 0305-1048 *
GLASER K B ET AL: "Role of Class I and Class II histone deacetylases in carcinoma cells using siRNA", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 310, no. 2, 17 October 2003 (2003-10-17), pages 529 - 536, XP004458975, ISSN: 0006-291X *
INOUE SATOSHI ET AL: "Inhibition of histone deacetylase class I but not class II is critical for the sensitization of leukemic cells to tumor necrosis factor-related apoptosis-inducing ligand-induced apoptosis", CANCER RESEARCH, vol. 66, no. 13, July 2006 (2006-07-01), pages 6785 - 6792, XP002494873, ISSN: 0008-5472 *
LEUSCHNER P J F ET AL: "Cleavage of the siRNA passenger strand during RISC assembly in human cells", EMBO REPORTS, vol. 7, no. 3, 1 March 2006 (2006-03-01), pages 314 - 320, XP002467845 *
LIU ET AL: "Histone deacetylase inhibitors: Multifunctional anticancer agents", CANCER TREATMENT REVIEWS, vol. 32, no. 3, 1 May 2006 (2006-05-01), pages 157 - 165, XP005473682, ISSN: 0305-7372 *
MATRANGA CHRISTIAN ET AL: "Passenger-strand cleavage facilitates assembly of siRNA into Ago2-containing RNAi enzyme complexes", CELL, vol. 123, no. 4, November 2005 (2005-11-01), pages 607 - 620, XP002484663, ISSN: 0092-8674 *
MILLS JANINE B ET AL: "Origin of the intrinsic rigidity of DNA", NUCLEIC ACIDS RESEARCH, vol. 32, no. 13, 2004, pages 4055 - 4059, XP002484664, ISSN: 0305-1048 *

Also Published As

Publication number Publication date
WO2008109498A2 (en) 2008-09-12
WO2008109498A8 (en) 2009-12-10
WO2008109498A4 (en) 2009-04-30

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