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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Bibliographische Detailangaben
Hauptverfasser:	ARTERO ALLEPUZ, Rubén, MORENO CERVERA, Nerea, LLAMUSÍ TROISI, María Beatriz, MARCUSSON, Eric, CERRO HERREROS, Estefanía, GONZÁLEZ MARTÍNEZ, Irene
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	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
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Beschreibung
Zusammenfassung:	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.