Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy

Preclinical characterization of antagomiR-218 as a potential treatment for myotonic dystrophy

Myotonic dystrophy type 1 (DM1) is a rare neuromuscular disease caused by expansion of unstable CTG repeats in a non-coding region of the DMPK gene. CUG expansions in mutant DMPK transcripts sequester MBNL1 proteins in ribonuclear foci. Depletion of this protein is a primary contributor to disease s...

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Main Authors: Estefanía Cerro-Herreros (Author), Irene González-Martínez (Author), Nerea Moreno (Author), Jorge Espinosa-Espinosa (Author), Juan M. Fernández-Costa (Author), Anna Colom-Rodrigo (Author), Sarah J. Overby (Author), David Seoane-Miraz (Author), Javier Poyatos-García (Author), Juan J. Vilchez (Author), Adolfo López de Munain (Author), Miguel A. Varela (Author), Matthew J. Wood (Author), Manuel Pérez-Alonso (Author), Beatriz Llamusí (Author), Rubén Artero (Author)
Format: Book
Published: Elsevier, 2021-12-01T00:00:00Z.
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3rd Floor Main Library

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