Publications by the researcher in collaboration with M BEATRIZ LLAMUSI TROISI (13)

2024

  1. AntimiR treatment corrects myotonic dystrophy primary cell defects across several CTG repeat expansions with a dual mechanism of action

    Science advances, Vol. 10, Núm. 41, pp. eadn6525

2023

  1. BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSALR Mice

    Pharmaceutics, Vol. 15, Núm. 4

  2. Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression

    Molecular Therapy - Nucleic Acids, Vol. 34

2022

  1. Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model

    Molecular Therapy - Nucleic Acids, Vol. 27, pp. 1146-1155

2021

  1. Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy

    Molecular Therapy - Nucleic Acids, Vol. 26, pp. 174-191

2020

  1. Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

    Molecular Therapy - Nucleic Acids, Vol. 21, pp. 837-849

2018

  1. MiR-23b and miR-218 silencing increase Muscleblind-like expression and alleviate myotonic dystrophy phenotypes in mammalian models

    Nature Communications, Vol. 9, Núm. 1

  2. RNA-mediated therapies in myotonic dystrophy

    Drug Discovery Today, Vol. 23, Núm. 12, pp. 2013-2022

  3. RbFOX1/MBNL1 competition for CCUG RNA repeats binding contributes to myotonic dystrophy type 1/type 2 differences

    Nature Communications, Vol. 9, Núm. 1

2017

  1. Expanded CCUG repeat RNA expression in Drosophila heart and muscle trigger Myotonic Dystrophy type 1-like phenotypes and activate autophagocytosis genes

    Scientific Reports, Vol. 7, Núm. 1

2016

  1. Derepressing muscleblind expression by miRNA sponges ameliorates myotonic dystrophy-like phenotypes in Drosophila

    Scientific Reports, Vol. 6

2015

  1. Increased autophagy and apoptosis contribute to muscle atrophy in a myotonic dystrophy type 1 Drosophila model

    DMM Disease Models and Mechanisms, Vol. 8, Núm. 7, pp. 679-690

2014

  1. Two enhancers control transcription of Drosophila muscleblind in the embryonic somatic musculature and in the central nervous system

    PLoS ONE, Vol. 9, Núm. 3