WO2008109378A3 - Nucleic acid compounds for inhibiting pdgfr gene expression and uses thereof - Google Patents

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

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Publication number
WO2008109378A3
WO2008109378A3 PCT/US2008/055381 US2008055381W WO2008109378A3 WO 2008109378 A3 WO2008109378 A3 WO 2008109378A3 US 2008055381 W US2008055381 W US 2008055381W WO 2008109378 A3 WO2008109378 A3 WO 2008109378A3
Authority
WO
WIPO (PCT)
Prior art keywords
pdgfr
nucleic acid
gene expression
acid compounds
pdgfr gene
Prior art date
Application number
PCT/US2008/055381
Other languages
French (fr)
Other versions
WO2008109378A2 (en
WO2008109378A4 (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 WO2008109378A2 publication Critical patent/WO2008109378A2/en
Publication of WO2008109378A3 publication Critical patent/WO2008109378A3/en
Publication of WO2008109378A4 publication Critical patent/WO2008109378A4/en
Priority to US12/552,082 priority Critical patent/US20100105134A1/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
    • C12N15/1138—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
    • C12N2310/14—Type of nucleic acid interfering N.A.

Abstract

The present disclosure provides meroduplex ribonucleic acid molecules (mdRNA) capable of decreasing or silencing PDGFR 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 PDGFR 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 PDGFR gene in a cell or in a subject to treat a PDGFR-related disease.
PCT/US2008/055381 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting pdgfr gene expression and uses thereof WO2008109378A2 (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 (8)

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
US93495407P 2007-06-06 2007-06-06
US60/934,954 2007-06-06
US93492907P 2007-06-14 2007-06-14
US60/934,929 2007-06-14

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055378 Continuation-In-Part WO2008109376A1 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting bcr-abl gene expression and uses thereof

Related Child Applications (3)

Application Number Title Priority Date Filing Date
PCT/US2008/055357 Continuation-In-Part WO2008109359A1 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting pdgfr family 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 (3)

Publication Number Publication Date
WO2008109378A2 WO2008109378A2 (en) 2008-09-12
WO2008109378A3 true WO2008109378A3 (en) 2009-01-29
WO2008109378A4 WO2008109378A4 (en) 2009-03-26

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2008/055381 WO2008109378A2 (en) 2007-03-02 2008-02-28 Nucleic acid compounds for inhibiting pdgfr gene expression and uses thereof

Country Status (1)

Country Link
WO (1) WO2008109378A2 (en)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210292768A1 (en) * 2018-08-08 2021-09-23 Arcturus Therapeutics, Inc. Compositions and agents against nonalcoholic steatohepatitis

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994015943A1 (en) * 1992-12-31 1994-07-21 Texas Biotechnology Corporation Antisense molecules directed against a platelet derived growth factor receptor related gene
WO2003072704A2 (en) * 2002-02-20 2003-09-04 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) 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
WO2008049078A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994015943A1 (en) * 1992-12-31 1994-07-21 Texas Biotechnology Corporation Antisense molecules directed against a platelet derived growth factor receptor related gene
WO2003072704A2 (en) * 2002-02-20 2003-09-04 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED INHIBITION OF PLATELET DERIVED GROWTH FACTOR (PDGF) AND PLATELET DERIVED GROWTH FACTOR RECEPTOR (PDGFR) 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
WO2008049078A1 (en) * 2006-10-18 2008-04-24 Nastech Pharmaceutical Company Inc. Nicked or gapped nucleic acid molecules and uses thereof

Non-Patent Citations (10)

* Cited by examiner, † Cited by third party
Title
BRAMSEN JESPER 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, XP002458184, ISSN: 0305-1048 *
CHEN Y.-X. ET AL.: "Effects of ribozyme targeting Platelet-Derived Growth Factor Receptor beta subunit gene on the proliferation and apoptosis of hepatic stellate cells in vitro", CHINESE MEDICAL JOURNAL, vol. 118, no. 12, 20 June 2005 (2005-06-20), pages 982 - 988, XP002485304 *
COHEN-SACKS H ET AL: "Novel PDGFbetaR antisense encapsulated in polymeric nanospheres for the treatment of restenosis", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 9, 1 December 2002 (2002-12-01), pages 1607 - 1616, XP002970353, ISSN: 0969-7128 *
FUNA K. AND AHGREN A.: "Characterization of Platelet-Derived Growth Factor (PDGF) action on mouse neuroblastoma cell line, NB41, by introduction of an antisense PDGF beta-Receptor RNA", CELL GROWTH AND DIFFERENTIATION, vol. 8, August 1997 (1997-08-01), pages 861 - 869, XP002485306 *
GHIL JUNG-SUN AND CHUNG HAE-MOON: "Evidence that Platelet Derived Growth Factor (PDGF) action is required for mesoderm patterning in early amphibian (Xenopus laevis) embryogenesis", INTERNATIONAL JOURNAL OF DEVELOPMENTAL BIOLOGY, vol. 43, no. 4, July 1999 (1999-07-01), pages 329 - 334, XP002485303 *
HAGERMAN K.R. AND HAGERMAN P.J.: "Helix rigidity of DNA: the meroduplex as an experimental paradigm", JOURNAL OF MOLECULAR BIOLOGY, vol. 260, 1996, pages 207 - 223, XP002484999 *
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 *
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 *
NOISEUX N ET AL: "A bolus endovascular treatment with a PDGFR-beta antisense is sufficient to suppress intimal thickening in a rat carotid injury model", CIRCULATION, AMERICAN HEART ASSOCIATION, DALLAS, TX, vol. 100, no. 18, 2 November 1999 (1999-11-02), pages I.816, XP002967164, ISSN: 0009-7322 *
NOISEUX N. ET AL.: "Bolus endovascular PDGFR-beta antisense treatment suppressed intimal hyperplasia in a rat carotid injury model", CIRCULATION, vol. 102, 2000, pages 1330 - 1336, XP002485305 *

Also Published As

Publication number Publication date
WO2008109378A2 (en) 2008-09-12
WO2008109378A4 (en) 2009-03-26

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