OLIGONUCLEOTIDES CONJUGATED TO OLEIC ACID AND USES THEREOF

The invention provides oligonucleotide and/or oligonucleotide analogue molecules that are antagonists of a microRNA, preferably antagonists of human microRNAs hsa-miR-23b-3p and hsa-miR-218-5p, that comprise a mixture of phosphorothioate and phosphodiester linkages, and that are conjugated to at lea...

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Hauptverfasser: ARTERO ALLEPUZ, Rubén, MORENO CERVERA, Nerea, LLAMUSÍ TROISI, María Beatriz, MARCUSSON, Eric, CERRO HERREROS, Estefanía, GONZÁLEZ MARTÍNEZ, Irene
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creator ARTERO ALLEPUZ, Rubén
MORENO CERVERA, Nerea
LLAMUSÍ TROISI, María Beatriz
MARCUSSON, Eric
CERRO HERREROS, Estefanía
GONZÁLEZ MARTÍNEZ, Irene
description The invention provides oligonucleotide and/or oligonucleotide analogue molecules that are antagonists of a microRNA, preferably antagonists of human microRNAs hsa-miR-23b-3p and hsa-miR-218-5p, that comprise a mixture of phosphorothioate and phosphodiester linkages, and that are conjugated to at least one oleic acid molecule. Inhibiting these microRNAs allows to increase the endogenous levels of the corresponding proteins MBNL1 and/or MBNL2. The present invention further provides compositions comprising said oligonucleotides and/or oligonucleotide analogue molecules and their uses for the treatment and prevention of DM in a subject in need thereof. L'invention concerne des oligonucléotides et/ou molécules analogues d'oligonucléotides qui sont des antagonistes d'un microARN, de préférence des antagonistes de microARN humains hsa-miR-23b-3p et hsa-miR-218-5p, qui comprennent un mélange de liaisons phosphorothioate et phosphodiester, et qui sont conjugués à au moins une molécule d'acide oléique. L'inhibition de ces microARN permet d'augmenter les niveaux endogènes des protéines MBNL1 et/ou MBNL2 correspondantes. La présente invention concerne en outre des compositions comprenant lesdits oligonucléotides et/ou molécules analogues d'oligonucléotides et leurs utilisations pour le traitement et la prévention de la dystrophie myotonique chez un sujet en ayant besoin.
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Inhibiting these microRNAs allows to increase the endogenous levels of the corresponding proteins MBNL1 and/or MBNL2. The present invention further provides compositions comprising said oligonucleotides and/or oligonucleotide analogue molecules and their uses for the treatment and prevention of DM in a subject in need thereof. L'invention concerne des oligonucléotides et/ou molécules analogues d'oligonucléotides qui sont des antagonistes d'un microARN, de préférence des antagonistes de microARN humains hsa-miR-23b-3p et hsa-miR-218-5p, qui comprennent un mélange de liaisons phosphorothioate et phosphodiester, et qui sont conjugués à au moins une molécule d'acide oléique. L'inhibition de ces microARN permet d'augmenter les niveaux endogènes des protéines MBNL1 et/ou MBNL2 correspondantes. 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La présente invention concerne en outre des compositions comprenant lesdits oligonucléotides et/ou molécules analogues d'oligonucléotides et leurs utilisations pour le traitement et la prévention de la dystrophie myotonique chez un sujet en ayant besoin.</abstract><oa>free_for_read</oa></addata></record>
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subjects BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
title OLIGONUCLEOTIDES CONJUGATED TO OLEIC ACID AND USES THEREOF
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