WO2001061030A3 - LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION - Google Patents

LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION Download PDF

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Publication number
WO2001061030A3
WO2001061030A3 PCT/US2001/004732 US0104732W WO0161030A3 WO 2001061030 A3 WO2001061030 A3 WO 2001061030A3 US 0104732 W US0104732 W US 0104732W WO 0161030 A3 WO0161030 A3 WO 0161030A3
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WIPO (PCT)
Prior art keywords
mrna
antisense
libraries
human
antisense oligonucleotides
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Application number
PCT/US2001/004732
Other languages
French (fr)
Other versions
WO2001061030A2 (en
Inventor
Arthur P Bollon
Donald M Gray
Lee Ju-Seog
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Arthur P Bollon
Donald M Gray
Lee Ju-Seog
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Publication date
Application filed by Arthur P Bollon, Donald M Gray, Lee Ju-Seog filed Critical Arthur P Bollon
Priority to EP01907219A priority Critical patent/EP1257665A2/en
Priority to AU2001235019A priority patent/AU2001235019A1/en
Priority to CA002400028A priority patent/CA2400028A1/en
Publication of WO2001061030A2 publication Critical patent/WO2001061030A2/en
Publication of WO2001061030A3 publication Critical patent/WO2001061030A3/en

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1131Non-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 viruses
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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 oncogenes or tumor suppressor genes
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

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  • Life Sciences & Earth Sciences (AREA)
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  • Oncology (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Abstract

The invention discloses a method for selecting optimal subsequence antisense targets for preparing an antisense oligonucleotide capable of inhibiting mRNA expression of target mRNA sequences, as well as antisense oligonucleotides capable of binding DNA. These libraries of antisense molecules may then be used for preparing therapeutic agents for the treatment of genetic disease. Antigene sequences specific for a desired target gene sequence and having mRNA, protein or cell growth inhibition efficiencies may then be used to control DNA expression. By in no way intending to be limited to any particular gene, examples of these human genes of interest in which mRNA subsequence targets will be identified and antisense molecules prepared are: c-Myc, c-Myb, Bcl-2, c-Raf, Cyclin D1, IGF-IR, PKCα, or CA12 genes. Antisense oligonucleotides of the invention may in some embodiments be at least 50 nucleotides in length. Examples include antisense oligonucleotides that specifically bind human protein kinase C-alpha and human C-Raf protein kinase.
PCT/US2001/004732 2000-02-14 2001-02-14 LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION WO2001061030A2 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP01907219A EP1257665A2 (en) 2000-02-14 2001-02-14 LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION
AU2001235019A AU2001235019A1 (en) 2000-02-14 2001-02-14 Libraries of optimum subsequence regions of mRNA and genomic DNA for control of gene expression
CA002400028A CA2400028A1 (en) 2000-02-14 2001-02-14 Libraries of optimum subsequence regions of mrna and genomic dna for control of gene expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US50465300A 2000-02-14 2000-02-14
US09/504,653 2000-02-14

Publications (2)

Publication Number Publication Date
WO2001061030A2 WO2001061030A2 (en) 2001-08-23
WO2001061030A3 true WO2001061030A3 (en) 2002-08-29

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PCT/US2001/004732 WO2001061030A2 (en) 2000-02-14 2001-02-14 LIBRARIES OF OPTIMUM SUBSEQUENCE REGIONS OF mRNA AND GENOMIC DNA FOR CONTROL OF GENE EXPRESSION

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Country Link
EP (1) EP1257665A2 (en)
AU (1) AU2001235019A1 (en)
CA (1) CA2400028A1 (en)
WO (1) WO2001061030A2 (en)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003072705A2 (en) * 2002-02-20 2003-09-04 Sirna Therapeutics, Inc. Rna interference mediated inhibition of cyclin d1 gene expression using short interfering nucleic acid (sina)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
EP1816191A4 (en) * 2004-11-19 2009-03-11 Takeda Pharmaceutical METHOD OF SCREENING COMPOUND REGULATING THE TRANSLATION OF SPECIFIC mRNA
US20070275029A1 (en) * 2006-05-26 2007-11-29 Ico Therapeutics Inc. Therapeutic drug combinations and delivery systems comprising c-raf kinase antisense polynucleotides for treating ocular diseases and disorders
EP2632472B1 (en) 2010-10-29 2017-12-13 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using short interfering nucleic acids (sina)
US20120302503A1 (en) * 2011-05-23 2012-11-29 AML Therapeutics, LLC PEPTIDES FOR PREVENTING OR TREATING A DISEASE OR DISORDER ASSOCIATED WITH CBP OR p300 MISREGULATION, AND METHODS FOR USE AND IDENTIFICATION THEREOF
ITMI20120275A1 (en) 2012-02-24 2013-08-25 Biogenera Societa A Responsabilita Limitata OLIGONUCLEOTIDS FOR THE MODULATION OF GENE EXPRESSION AND THEIR USES

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994023755A1 (en) * 1993-04-09 1994-10-27 Board Of Regents Of The University Of Nebraska Novel methods and compositions for the treatment of ras-activated cancer with heterotypic anti-raf antisense oligonucleotides
WO1995002069A1 (en) * 1993-07-09 1995-01-19 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of protein kinase c
US5856103A (en) * 1994-10-07 1999-01-05 Board Of Regents The University Of Texas Method for selectively ranking sequences for antisense targeting

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994023755A1 (en) * 1993-04-09 1994-10-27 Board Of Regents Of The University Of Nebraska Novel methods and compositions for the treatment of ras-activated cancer with heterotypic anti-raf antisense oligonucleotides
WO1995002069A1 (en) * 1993-07-09 1995-01-19 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of protein kinase c
US5856103A (en) * 1994-10-07 1999-01-05 Board Of Regents The University Of Texas Method for selectively ranking sequences for antisense targeting

Non-Patent Citations (7)

* Cited by examiner, † Cited by third party
Title
ARIMA HIDETOSHI ET AL: "Design of potent phosphorothioate antisense oligonucleotides directed to human interleukin 10 gene product and their evaluation of antisense activity in U937 cells.", PHARMACEUTICAL RESEARCH (NEW YORK), vol. 16, no. 8, August 1999 (1999-08-01), pages 1163 - 1171, XP001053078, ISSN: 0724-8741 *
CROOKE S T: "BASIC PRINCIPLES OF ANTISENSE THERAPEUTICS", ANTISENSE RESEARCH AND APPLICATIONS, CRC PRESS, GB, 1998, pages 1 - 50, XP000900999 *
HUNG WEN-CHUN ET AL: "Antisense oligodeoxynucleotides targeted against different regions of cyclin D1 mRNA may exert different inhibitory effects on cell growth and gene expression.", BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS, vol. 220, no. 3, 1996, pages 719 - 723, XP002188470, ISSN: 0006-291X *
IVANOV S V ET AL: "Down-regulation of transmembrane carbonic anhydrases in renal cell carcinoma cell lines by wild-type von Hippel-Lindau transgenes", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, vol. 95, 13 October 1998 (1998-10-13), pages 12596 - 12601, XP000941355, ISSN: 0027-8424 *
MERCOLA D ET AL: "ANTISENSE APPROACHES TO CANCER GENE THERAPY", CANCER GENE THERAPY, vol. 2, no. 1, 1995, pages 47 - 59, XP002911890, ISSN: 0929-1903 *
MITSUHASHI MASATO: "Strategy for designing specific antisense oligonucleotide sequences.", JOURNAL OF GASTROENTEROLOGY, vol. 32, no. 2, 1997, pages 282 - 287, XP001053065, ISSN: 0944-1174 *
SONG HAI-FENG ET AL: "Application of secondary structure prediction in antisense drug design targeting protein kinase C-alpha mRNA and QSAR analysis.", ACTA PHARMACOLOGICA SINICA, vol. 21, no. 1, January 2000 (2000-01-01), pages 80 - 86, XP001053096, ISSN: 0253-9756 *

Also Published As

Publication number Publication date
WO2001061030A2 (en) 2001-08-23
CA2400028A1 (en) 2001-08-23
AU2001235019A1 (en) 2001-08-27
EP1257665A2 (en) 2002-11-20

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