Publicaciones en las que colabora con MANUEL PEREZ ALONSO (28)

2024

  1. Msi2 enhances muscle dysfunction in a myotonic dystrophy type 1 mouse model

    Biomedical Journal, Vol. 47, Núm. 4

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. Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSALR Mice

    International Journal of Molecular Sciences, Vol. 24, Núm. 12

  3. Quantitative magnetic resonance imaging assessment of muscle composition in myotonic dystrophy mice

    Scientific Reports, Vol. 13, Núm. 1

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. Defined d-hexapeptides bind CUG repeats and rescue phenotypes of myotonic dystrophy myotubes in a Drosophila model of the disease

    Scientific Reports, Vol. 11, Núm. 1

  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

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

  2. In silico discovery of substituted pyrido[2,3-d] pyrimidines and pentamidine-like compounds with biological activity in myotonic dystrophy models

    PLoS ONE, Vol. 12, Núm. 6

  3. Myotonic dystrophy: candidate small molecule therapeutics

    Drug Discovery Today, Vol. 22, Núm. 11, pp. 1740-1748

2016

  1. Six serum miRNAs fail to validate as myotonic dystrophy type 1 biomarkers

    PLoS ONE, Vol. 11, Núm. 2

2015

  1. Pentamidine rescues contractility and rhythmicity in a drosophila model of myotonic dystrophy heart dysfunction

    DMM Disease Models and Mechanisms, Vol. 8, Núm. 12, pp. 1569-1578

2014

  1. Development of a Drosophila melanogaster spliceosensor system for in vivo high-throughput screening in myotonic dystrophy type 1

    DMM Disease Models and Mechanisms, Vol. 7, Núm. 11, pp. 1297-1306

2013

  1. Expanded CTG repeats trigger miRNA alterations in Drosophila that are conserved in myotonic dystrophy type 1 patients

    Human Molecular Genetics, Vol. 22, Núm. 4, pp. 704-716

  2. In vivo strategies for drug discovery in myotonic dystrophy disorders

    Drug Discovery Today: Technologies, Vol. 10, Núm. 1

2009

  1. A FRET-based assay for characterization of alternative splicing events using peptide nucleic acid fluorescence in situ hybridization

    Nucleic Acids Research, Vol. 37, Núm. 17

2005

  1. Myotonic dystrophy associated expanded CUG repeat muscleblind positive ribonuclear foci are not toxic to Drosophila

    Human Molecular Genetics, Vol. 14, Núm. 6, pp. 873-883

2002

  1. Generation of GAL4-responsive muscleblind constructs

    Genesis, Vol. 34, Núm. 1-2, pp. 111-114