WO2010091878A3 - Means for inhibiting the expression of opa1 - Google Patents

Means for inhibiting the expression of opa1 Download PDF

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Publication number
WO2010091878A3
WO2010091878A3 PCT/EP2010/000888 EP2010000888W WO2010091878A3 WO 2010091878 A3 WO2010091878 A3 WO 2010091878A3 EP 2010000888 W EP2010000888 W EP 2010000888W WO 2010091878 A3 WO2010091878 A3 WO 2010091878A3
Authority
WO
WIPO (PCT)
Prior art keywords
duplex region
antisense
sense
opa1
inhibiting
Prior art date
Application number
PCT/EP2010/000888
Other languages
French (fr)
Other versions
WO2010091878A2 (en
Inventor
Jörg Kaufmann
Ansgar Santel
Original Assignee
Silence Therapeutics Ag
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 Silence Therapeutics Ag filed Critical Silence Therapeutics Ag
Publication of WO2010091878A2 publication Critical patent/WO2010091878A2/en
Publication of WO2010091878A3 publication Critical patent/WO2010091878A3/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
    • 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 invention is related to an siRNA comprising an antisense strand and a sense strand, wherein all or a portion of said antisense strand comprises an antisense duplex region, wherein all or a portion of said sense strand comprises a sense duplex region, wherein said antisense duplex region is at least partially complementary to said sense duplex region, wherein said siRNA comprises a duplex region consisting of said antisense duplex region and said sense duplex region, and wherein: a) said antisense strand comprises SEQ ID NO: 3, 5, 7 or 9; or b) said antisense strand comprises an antisense duplex region, all or a portion of which, is complementary to a portion of SEQ ID NOs: 1, 10, 11, 12, 13, 14, 15 and/or 16.
PCT/EP2010/000888 2009-02-13 2010-02-12 Means for inhibiting the expression of opa1 WO2010091878A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP09002077.7 2009-02-13
EP09002077 2009-02-13

Publications (2)

Publication Number Publication Date
WO2010091878A2 WO2010091878A2 (en) 2010-08-19
WO2010091878A3 true WO2010091878A3 (en) 2010-11-25

Family

ID=42111739

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2010/000888 WO2010091878A2 (en) 2009-02-13 2010-02-12 Means for inhibiting the expression of opa1

Country Status (1)

Country Link
WO (1) WO2010091878A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
EP3996708A1 (en) * 2019-07-12 2022-05-18 Università Degli Studi Di Padova Pharmaceutical compositions comprising inhibitors of opa1 for use in the treatment of cancer

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070031844A1 (en) * 2002-11-14 2007-02-08 Anastasia Khvorova Functional and hyperfunctional siRNA
WO2008103135A2 (en) * 2007-02-16 2008-08-28 The Johns Hopkins University The micrornaome

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US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
ATE515510T1 (en) 1991-12-24 2011-07-15 Isis Pharmaceuticals Inc OLIGONUCLEOTIDES MODIFIED BY DNA SECTIONS
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5753613A (en) 1994-09-30 1998-05-19 Inex Pharmaceuticals Corporation Compositions for the introduction of polyanionic materials into cells
US6001311A (en) 1997-02-05 1999-12-14 Protogene Laboratories, Inc. Apparatus for diverse chemical synthesis using two-dimensional array
US6395713B1 (en) 1997-07-23 2002-05-28 Ribozyme Pharmaceuticals, Inc. Compositions for the delivery of negatively charged molecules
JP2003525017A (en) 1998-04-20 2003-08-26 リボザイム・ファーマシューティカルズ・インコーポレーテッド Nucleic acid molecules with novel chemical composition that can regulate gene expression
JP2002537828A (en) 1999-03-10 2002-11-12 フォゲン リミティド Delivery of substances to cells
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070031844A1 (en) * 2002-11-14 2007-02-08 Anastasia Khvorova Functional and hyperfunctional siRNA
WO2008103135A2 (en) * 2007-02-16 2008-08-28 The Johns Hopkins University The micrornaome

Non-Patent Citations (12)

* Cited by examiner, † Cited by third party
Title
ARNOULT DAMIEN ET AL: "Release of OPA1 during apoptosis participates in the rapid and complete release of cytochrome c and subsequent mitochondrial fragmentation.", THE JOURNAL OF BIOLOGICAL CHEMISTRY 21 OCT 2005 LNKD- PUBMED:16115883, vol. 280, no. 42, 21 October 2005 (2005-10-21), pages 35742 - 35750, XP002580230, ISSN: 0021-9258 *
CHEN HSIUCHEN ET AL: "Disruption of fusion results in mitochondrial heterogeneity and dysfunction", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, INC, US LNKD- DOI:10.1074/JBC.M503062200, vol. 280, no. 28, 17 May 2005 (2005-05-17), pages 26185 - 26192, XP009088993, ISSN: 0021-9258 *
CZAUDERNA FRANK ET AL: "Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells", NUCLEIC ACIDS RESEARCH 1 JUN 2003 LNKD- PUBMED:12771196,, vol. 31, no. 11, 1 June 2003 (2003-06-01), pages 2705 - 2716, XP002579231 *
DATABASE Geneseq [online] 30 October 2008 (2008-10-30), "Human miRNA related target gene fragment, SEQ ID 1760.", XP002580657, retrieved from EBI accession no. GSN:ATF56731 Database accession no. ATF56731 *
DATABASE Probe [online] 8 July 2006 (2006-07-08), "Small hairpin RNA (shRNA) probe for Homo sapiens gene optic atrophy 1 (OPA1)", XP002580232, Database accession no. PR195954.1 *
DATABASE Probe [online] NCBI; 8 July 2006 (2006-07-08), "Small interfering RNA (siRNA) probe for Homo sapiens gene optic atrophy 1 (OPA1)", XP002580231, Database accession no. Pr003104593.1 *
DELIVANI P ET AL: "Mitochondrial membrane remodeling in apoptosis: an inside story.", CELL DEATH AND DIFFERENTIATION DEC 2006 LNKD- PUBMED:17039250, vol. 13, no. 12, December 2006 (2006-12-01), pages 2007 - 2010, XP002580626, ISSN: 1350-9047 *
GOTTLIEB EYAL: "OPA1 and PARL keep a lid on apoptosis.", CELL 14 JUL 2006 LNKD- PUBMED:16839872, vol. 126, no. 1, 14 July 2006 (2006-07-14), pages 27 - 29, XP002580625, ISSN: 0092-8674 *
LEE SEUNGMIN ET AL: "Mitochondrial fission and fusion mediators, hFis1 and OPA1, modulate cellular senescence.", THE JOURNAL OF BIOLOGICAL CHEMISTRY 3 AUG 2007 LNKD- PUBMED:17545159, vol. 282, no. 31, 3 August 2007 (2007-08-03), pages 22977 - 22983, XP002580233, ISSN: 0021-9258 *
LEE YANG-JA ET AL: "Roles of the mammalian mitochondrial fission and fusion mediators Fis1, Drp1, and Opa1 in apoptosis", MOLECULAR BIOLOGY OF THE CELL, AMERICAN SOCIETY FOR CELL BIOLOGY, US, vol. 15, no. 11, 1 November 2004 (2004-11-01), pages 5001 - 5011, XP002404054, ISSN: 1059-1524 *
OLICHON A ET AL: "LOSS OF OPA1 PERTURBATES THE MITOCHONDRIAL INNER MEMBRANE STRUCTURE AND INTEGRITY, LEADING TO CYTOCHROME C RELEASE AND APOPTOSIS", JOURNAL OF BIOLOGICAL CHEMISTRY, AMERICAN SOCIETY FOR BIOCHEMISTRY AND MOLECULAR BIOLOGY, INC, US LNKD- DOI:10.1074/JBC.C200677200, vol. 278, no. 10, 31 December 2002 (2002-12-31), pages 7743 - 7746, XP001188969, ISSN: 0021-9258 *
SANTEL A ET AL: "A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB LNKD- DOI:10.1038/SJ.GT.3302777, vol. 13, no. 16, 20 April 2006 (2006-04-20), pages 1222 - 1234, XP002459352, ISSN: 0969-7128 *

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