Targeting the 5' untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy by Audrey M. Winkelsas, Christopher Grunseich, George G. Harmison, Katarzyna Chwalenia, Carlo Rinaldi, Suzan M. Hammond, Kory Johnson, Melissa Bowerman, Sukrat Arya, Kevin Talbot, Matthew J. Wood, Kenneth H. Fischbeck

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Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model by Sarah J. Overby, Estefanía Cerro-Herreros, Irene González-Martínez, Miguel A. Varela, David Seoane-Miraz, Yahya Jad, Richard Raz, Thorleif Møller, Manuel Pérez-Alonso, Matthew J. Wood, Beatriz Llamusí, Rubén Artero

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Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression by Irene González-Martínez, Estefanía Cerro-Herreros, Nerea Moreno, Andrea García-Rey, Jorge Espinosa-Espinosa, Marc Carrascosa-Sàez, Diego Piqueras-Losilla, Andrey Arzumanov, David Seoane-Miraz, Yahya Jad, Richard Raz, Matthew J. Wood, Miguel A. Varela, Beatriz Llamusí, Rubén Artero

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Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy by Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno, Jorge Espinosa-Espinosa, Juan M. Fernández-Costa, Anna Colom-Rodrigo, Sarah J. Overby, David Seoane-Miraz, Javier Poyatos-García, Juan J. Vilchez, Adolfo López de Munain, Miguel A. Varela, Matthew J. Wood, Manuel Pérez-Alonso, Beatriz Llamusí, Rubén Artero

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