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AssigneesUniversitat De Valencia, Arthex Biotech S.L.
InventorsRubén ARTERO ALLEPUZ, Nerea MORENO CERVERA, Irene GONZÁLEZ MARTÍNEZ, Estefanía CERRO HERREROS, Eric G. MARCUSSON, María Beatriz LLAMUSÍ TROISI
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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.
An oligonucleotide molecule consisting of SEQ ID NOs:
The method of claim 13, wherein the disease is myotonic dystrophy. The method of claim 14, wherein the myotonic dystrophy is type 1 myotonic dystrophy.
The method of claim 15, wherein the pharmaceutical composition comprises an oligonucleotide molecule consisting of SEQ ID NO:
The method of claim 15, wherein the pharmaceutical composition comprises an oligonucleotide molecule consisting of SEQ ID NO:
The method of claim 15, wherein the pharmaceutical composition comprises an oligonucleotide molecule consisting of SEQ ID NO:
Dependencies are inferred from the claim text.