Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model

Proof of concept of peptide-linked blockmiR-induced MBNL functional rescue in myotonic dystrophy type 1 mouse model

Myotonic dystrophy type 1 is a debilitating neuromuscular disease causing muscle weakness, myotonia, and cardiac dysfunction. The phenotypes are caused by muscleblind-like (MBNL) protein sequestration by toxic RNA in the DM1 protein kinase (DMPK) gene. DM1 patients exhibit a pathogenic number of rep...

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Main Authors: Sarah J. Overby (Author), Estefanía Cerro-Herreros (Author), Irene González-Martínez (Author), Miguel A. Varela (Author), David Seoane-Miraz (Author), Yahya Jad (Author), Richard Raz (Author), Thorleif Møller (Author), Manuel Pérez-Alonso (Author), Matthew J. Wood (Author), Beatriz Llamusí (Author), Rubén Artero (Author)
Format: Book
Published: Elsevier, 2022-03-01T00:00:00Z.
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3rd Floor Main Library

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