ESTEFANIA CERRO HERREROS-rekin lankidetzan egindako argitalpenak (15)

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

  2. Rapid Determination of MBNL1 Protein Levels by Quantitative Dot Blot for the Evaluation of Antisense Oligonucleotides in Myotonic Dystrophy Myoblasts

    Methods in molecular biology (Clifton, N.J.), Vol. 2434, pp. 207-215

2021

  1. Bioengineered in vitro 3D model of myotonic dystrophy type 1 human skeletal muscle

    Biofabrication, Vol. 13, Núm. 3

  2. 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