BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSA<sup>LR</sup> Mice

BlockmiR AONs as Site-Specific Therapeutic MBNL Modulation in Myotonic Dystrophy 2D and 3D Muscle Cells and HSA<sup>LR</sup> Mice

The symptoms of Myotonic Dystrophy Type 1 (DM1) are multi-systemic and life-threatening. The neuromuscular disorder is rooted in a non-coding CTG microsatellite expansion in the DM1 protein kinase (<i>DMPK</i>) gene that, upon transcription, physically sequesters the Muscleblind-like (MB...

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Main Authors: Sarah J. Overby (Author), Estefanía Cerro-Herreros (Author), Jorge Espinosa-Espinosa (Author), Irene González-Martínez (Author), Nerea Moreno (Author), Juan M. Fernández-Costa (Author), Jordina Balaguer-Trias (Author), Javier Ramón-Azcón (Author), Manuel Pérez-Alonso (Author), Thorleif Møller (Author), Beatriz Llamusí (Author), Rubén Artero (Author)
Format: Book
Published: MDPI AG, 2023-03-01T00:00:00Z.
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