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BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSA<sup>LR</sup> Mice by Sarah J. Overby, Estefanía Cerro-Herreros, Jorge Espinosa-Espinosa, Irene González-Martínez, Nerea Moreno, Juan M. Fernández-Costa, Jordina Balaguer-Trias, Javier Ramón-Azcón, Manuel Pérez-Alonso, Thorleif Møller, Beatriz Llamusí, Rubén Artero
Published 2023-03-01
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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
Published 2023-12-01
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