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WO2011150921A3 - Biologically active molecules that are based on sirna and that can be activated in a cell-specific manner, method for activating same, and application kit for administration - Google Patents

Biologically active molecules that are based on sirna and that can be activated in a cell-specific manner, method for activating same, and application kit for administration Download PDF

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Publication number
WO2011150921A3
WO2011150921A3 PCT/DE2011/001224 DE2011001224W WO2011150921A3 WO 2011150921 A3 WO2011150921 A3 WO 2011150921A3 DE 2011001224 W DE2011001224 W DE 2011001224W WO 2011150921 A3 WO2011150921 A3 WO 2011150921A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
sirna
target cells
administration
activated
Prior art date
Application number
PCT/DE2011/001224
Other languages
German (de)
French (fr)
Other versions
WO2011150921A2 (en
Inventor
Tobias PÖHLMANN
Rolf Günther
Original Assignee
Friedrich-Schiller-Universität Jena
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 Friedrich-Schiller-Universität Jena filed Critical Friedrich-Schiller-Universität Jena
Publication of WO2011150921A2 publication Critical patent/WO2011150921A2/en
Publication of WO2011150921A3 publication Critical patent/WO2011150921A3/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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 nucleic acids [NA]
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Agricultural Chemicals And Associated Chemicals (AREA)

Abstract

The aim of the invention was to create biological active substances that can be transfected into or onto target cells in living beings, plants, and fungi in vitro as well as in vivo and only there inhibit the expression of genes highly effectively without affecting the target-gene expression specific to the active agent and thus the formation of proteins in other cells of the organism. According to the invention, the siRNA (1) has at least one covalent bond (6) to one or more oligosaccharides (3), which each have one or more specific chemical structures selected for enzymes typical of the target cells. By means of said bond (6), which is broken only by said enzyme or enzymes typical of the target cells in order to biologically activate the active substance, the molecules remain biologically inactive in cells other than the target cells. Said molecules are used, for example, to influence the gene expression of preferably diseased organs, cells, or plants that are infected and afflicted with parasites or fungi.
PCT/DE2011/001224 2010-06-04 2011-06-01 Biologically active molecules that are based on sirna and that can be activated in a cell-specific manner, method for activating same, and application kit for administration WO2011150921A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
DE102010022937A DE102010022937A1 (en) 2010-06-04 2010-06-04 Cell-specific activatable biologically active molecules based on siRNA, methods for their activation and application kit for administration
DE102010022937.7 2010-06-04

Publications (2)

Publication Number Publication Date
WO2011150921A2 WO2011150921A2 (en) 2011-12-08
WO2011150921A3 true WO2011150921A3 (en) 2012-04-19

Family

ID=44946904

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/DE2011/001224 WO2011150921A2 (en) 2010-06-04 2011-06-01 Biologically active molecules that are based on sirna and that can be activated in a cell-specific manner, method for activating same, and application kit for administration

Country Status (2)

Country Link
DE (1) DE102010022937A1 (en)
WO (1) WO2011150921A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE102012022596B4 (en) * 2012-11-15 2017-05-04 Friedrich-Schiller-Universität Jena New cell-specific effective nucleotide molecules and application kit for their application
CN104177450A (en) * 2014-09-17 2014-12-03 江苏天晟药业有限公司 Oligosaccharide for preventing and treating fungous plant diseases and insect pests of liquorice and preparation method and application of oligosaccharide

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060008907A1 (en) * 2004-06-09 2006-01-12 The Curators Of The University Of Missouri Control of gene expression via light activated RNA interference
WO2008098569A2 (en) * 2007-02-15 2008-08-21 Friedrich-Schiller-Universität Jena Biologically active molecules, particularly based on pna and sirna, method for the cell-specific activation thereof, and application kit to be administered

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
MXPA03004836A (en) 2000-12-01 2005-09-08 Max Planck Gesellschaft SMALL RNA MOLECULES THAT MEDIATE RNA INTERFERENCE.
JP4597976B2 (en) * 2003-04-17 2010-12-15 アルナイラム ファーマシューティカルズ インコーポレイテッド Modified iRNA agent
WO2007021142A1 (en) * 2005-08-17 2007-02-22 Bioneer Corporation Sirna-hydrophilic polymer conjugates for intracellular delivery of sirna and method thereof
EP2395012B8 (en) * 2005-11-02 2018-06-06 Arbutus Biopharma Corporation Modified siRNA molecules and uses thereof

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060008907A1 (en) * 2004-06-09 2006-01-12 The Curators Of The University Of Missouri Control of gene expression via light activated RNA interference
WO2008098569A2 (en) * 2007-02-15 2008-08-21 Friedrich-Schiller-Universität Jena Biologically active molecules, particularly based on pna and sirna, method for the cell-specific activation thereof, and application kit to be administered

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
AKASAKA T ET AL: "Transformation from block-type to graft-type oligonucleoitde-glycopolymer conjugates by self-organization with half-sliding complementary oligonucleoitdes and theri lectin recognition", BIOCONJUGATE CHEMISTRY, ACS, WASHINGTON, DC, US, vol. 12, no. 5, 1 January 2001 (2001-01-01), pages 776 - 785, XP002471391, ISSN: 1043-1802, DOI: 10.1021/BC0100152 *
HYEJUNG MOK ET AL: "Antisense Oligodeoxynucleotide-Conjugated Hyaluronic Acid/Protamine Nanocomplexes for Intracellular Gene Inhibition", BIOCONJUGATE CHEMISTRY, vol. 18, no. 5, 1 September 2007 (2007-09-01), pages 1483 - 1489, XP055019244, ISSN: 1043-1802, DOI: 10.1021/bc070111o *
VIMARY VÁZQUEZ-DORBATT ET AL: "Synthesis of a Pyridyl Disulfide End-Functionalized Glycopolymer for Conjugation to Biomolecules and Patterning on Gold Surfaces", BIOMACROMOLECULES, vol. 10, no. 8, 10 August 2009 (2009-08-10), pages 2207 - 2212, XP055019504, ISSN: 1525-7797, DOI: 10.1021/bm900395h *
ZATSEPIN TIMOFEI S ET AL: "Synthesis and applications of oligonucleotide-carbohydrate conjugates", CHEMISTRY & BIODIVERSITY, HELVETICA CHIMICA ACTA, ZUERICH, CH, vol. 1, no. 10, 1 October 2004 (2004-10-01), pages 1401 - 1417, XP002551178, ISSN: 1612-1872, [retrieved on 20041021], DOI: 10.1002/CBDV.200490104 *

Also Published As

Publication number Publication date
WO2011150921A2 (en) 2011-12-08
DE102010022937A1 (en) 2011-12-08

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