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  1. patents.google.com

    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 ...
  2. data.epo.org

    Thus, oligonucleotides with reduced toxicity but increased stability need to be developed. [0007] The present invention overcomes these limitations by providing improved antimiRs conjugated to oleic acid. DESCRIPTION OF THE FIGURES [0008] Figure 1. Evaluation of toxicity and efficacy on DM1 cells. Representation of toxicity (percentage of cell ...
  3. patentscope.wipo.int

    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. ... WO2023227622 - OLIGONUCLEOTIDES CONJUGATED TO OLEIC ACID AND USES THEREOF. Publication Number WO/2023/227622 Publication Date 30.11 ...
  4. The gene and the mutation implicated in DM1 were described more than 30 years ago. The genetics of DM1 is an intriguing example, firstly because of the mutation, which is a large expansion of the trinucleotide motif in DMPK gene, and secondly due to the location of the mutation in the 3′ UTR. The expansion in DM1 reaches thousands of CTG repeats in the mutant DMPK gene and is transcribed ...
  5. sciencedirect.com

    The limitations associated with mini-nucleic acid drugs have led to a new concept: combining antibodies with targeted functions with oligonucleotides to form AOCs, which can be linked directly or through a linker. Initially used as diagnostic tools, AOCs are increasingly being used as targeted therapies for many diseases.
  6. pmc.ncbi.nlm.nih.gov

    Palmitic acid conjugation enhances potency of Malat-1 ASO in mouse heart and quadriceps. Mice (C57BL/6, n = 4/group) were injected subcutaneously Malat-1 ASO 1 and 5′-palmitoyl conjugated Malat-1 ASO 2 at 0.4, 1.2 and 3.6 μmol/kg once a week for 3 weeks for a total of three doses and sacrificed after 5 days. (A) Malat-1 RNA expression analyzed in mice heart, quadriceps, liver and kidney ...
  7. mysciencework.com

    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.
  8. pubmed.ncbi.nlm.nih.gov

    Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including myotonic dystrophy (DM1). Thus, ASO strategies for DM1 can abolish the toxic RNA gain-of-function mechanism caused by nucleus-retained mutant DMPK (DM1 protein …
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