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WO2013013165A3 - Utilisation de composés d'acide nucléique bloqué (lna) pour l'inhibition thérapeutique de mir-21 dans le lupus érythémateux systémique - Google Patents

Utilisation de composés d'acide nucléique bloqué (lna) pour l'inhibition thérapeutique de mir-21 dans le lupus érythémateux systémique Download PDF

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Publication number
WO2013013165A3
WO2013013165A3 PCT/US2012/047638 US2012047638W WO2013013165A3 WO 2013013165 A3 WO2013013165 A3 WO 2013013165A3 US 2012047638 W US2012047638 W US 2012047638W WO 2013013165 A3 WO2013013165 A3 WO 2013013165A3
Authority
WO
WIPO (PCT)
Prior art keywords
mir
lupus erythematosus
systemic lupus
therapeutic inhibition
lna compounds
Prior art date
Application number
PCT/US2012/047638
Other languages
English (en)
Other versions
WO2013013165A2 (fr
Inventor
Marianthi Kiriakidou
Barry GARCHOW
Sakari Kauppinen
Original Assignee
The Trustees Of The University Of Pennsylvania
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 The Trustees Of The University Of Pennsylvania filed Critical The Trustees Of The University Of Pennsylvania
Publication of WO2013013165A2 publication Critical patent/WO2013013165A2/fr
Publication of WO2013013165A3 publication Critical patent/WO2013013165A3/fr

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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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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

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)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La présente invention concerne des compositions de LNA antimiR ainsi que des procédés d'utilisation associés pour la régulation de l'expression de miR-21. La présente invention concerne des procédés de traitement de maladies dans lesquelles miR-21 est régulé à la hausse. Un exemple d'une telle maladie est le lupus érythémateux systémique (LES).
PCT/US2012/047638 2011-07-21 2012-07-20 Utilisation de composés d'acide nucléique bloqué (lna) pour l'inhibition thérapeutique de mir-21 dans le lupus érythémateux systémique WO2013013165A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161510283P 2011-07-21 2011-07-21
US61/510,283 2011-07-21
US201161511854P 2011-07-26 2011-07-26
US61/511,854 2011-07-26

Publications (2)

Publication Number Publication Date
WO2013013165A2 WO2013013165A2 (fr) 2013-01-24
WO2013013165A3 true WO2013013165A3 (fr) 2013-04-11

Family

ID=47558744

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2012/047638 WO2013013165A2 (fr) 2011-07-21 2012-07-20 Utilisation de composés d'acide nucléique bloqué (lna) pour l'inhibition thérapeutique de mir-21 dans le lupus érythémateux systémique

Country Status (1)

Country Link
WO (1) WO2013013165A2 (fr)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2013012452A (es) 2011-04-25 2014-11-21 Regulus Therapeutics Inc Compuestos de microarn y metodos para modular la actividad de mir-21.
PL398796A1 (pl) * 2012-04-11 2013-10-14 Krzysztof Selmaj Zastosowanie czasteczek miRNA oraz antagomiru dla miRNA w diagnostyce, zapobieganiu i leczeniu stwardnienia rozsianego
UA117098C2 (uk) 2012-04-25 2018-06-25 Рег'Юлес Терап'Ютікс Інк. Сполука, що містить модифікований олігонуклеотид
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
WO2019051355A1 (fr) 2017-09-08 2019-03-14 Ohio State Innovation Foundation Nouvelle thérapie par inhibiteur de microarn pour le lupus érythémateux systémique

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100286234A1 (en) * 2006-04-03 2010-11-11 Joacim Elmen Pharmaceutical Composition Comprising Anti-Mirna Antisense Oligonucleotides
US20100298410A1 (en) * 2007-10-04 2010-11-25 Santaris Pharma A/S MICROMIRs
US20100322909A1 (en) * 2009-06-17 2010-12-23 The University Of Pittsburgh - Of The Commonwealth System Of Higher Education Th1-associated micrornas and their use for tumor immunotherapy
US20110118339A1 (en) * 2008-01-18 2011-05-19 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20100286234A1 (en) * 2006-04-03 2010-11-11 Joacim Elmen Pharmaceutical Composition Comprising Anti-Mirna Antisense Oligonucleotides
US20100298410A1 (en) * 2007-10-04 2010-11-25 Santaris Pharma A/S MICROMIRs
US20110118339A1 (en) * 2008-01-18 2011-05-19 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides and uses thereof
US20100322909A1 (en) * 2009-06-17 2010-12-23 The University Of Pittsburgh - Of The Commonwealth System Of Higher Education Th1-associated micrornas and their use for tumor immunotherapy

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
FLUITER ET AL.: "Filling the gap in LNA antisense oligo gapmers: the effects of unlocked nucleic acid (UNA) and 4'-C-hydroxymethyl-DNA modifications on RNase H recruitment and efficacy of an LNA gapmer.", MOL. BIOSYST., vol. 5, no. 8, 2009, pages 838 - 843 *
GARCHOW ET AL.: "Silencing of microRNA-21 in vivo ameliorates autoimmune splenomegaly in lupus mice.", EMBO MOL MED., vol. 3, no. 10, 1 September 2011 (2011-09-01), pages 605 - 615 *
OBAD ET AL.: "Silencing of microRNA families by seed-targeting tiny LNAs.", NAT GENET., vol. 43, no. 4, 20 March 2011 (2011-03-20), pages 371 - 378 *
QIAGEN.: "hsa-mir-21", 2008, Retrieved from the Internet <URL:https://www.qiagen.com/geneglobe/miprimerview.aspx?keepresult=true&producttype=88ID=MS00009086#ProductList> [retrieved on 20121217] *
ROSSI.: "Stopping RNA interference at the seed.", NAT GENET., vol. 43, no. 4, 29 March 2011 (2011-03-29), pages 288 - 289 *
STAGAKIS ET AL.: "Identification of novel microRNA signatures linked to human lupus disease activity and pathogenesis: miR-21 regulates aberrant T cell responses through regulation of PDCD4 expression.", ANN RHEUM DIS., vol. 70, no. 8, 20 May 2011 (2011-05-20), pages 1496 - 1506 *

Also Published As

Publication number Publication date
WO2013013165A2 (fr) 2013-01-24

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