Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

Therapeutic Potential of AntagomiR-23b for Treating Myotonic Dystrophy

Myotonic dystrophy type 1 (DM1) is a chronically debilitating, rare genetic disease that originates from an expansion of a noncoding CTG repeat in the dystrophia myotonica protein kinase (DMPK) gene. The expansion becomes pathogenic when DMPK transcripts contain 50 or more repetitions due to the seq...

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Bibliographic Details
Main Authors: Estefanía Cerro-Herreros, Irene González-Martínez, Nerea Moreno-Cervera, Sarah Overby, Manuel Pérez-Alonso, Beatriz Llamusí, Rubén Artero
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
DM1
myotonic dystrophy
miRNA
antagomiR
HSALR mice
antisense oligonucleotides
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120302109

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http://www.sciencedirect.com/science/article/pii/S2162253120302109

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