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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...
Autores principales: | Cerro-Herreros, Estefanía, González-Martínez, Irene, Moreno-Cervera, Nerea, Overby, Sarah, Pérez-Alonso, Manuel, Llamusí, Beatriz, Artero, Rubén |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7452101/ https://www.ncbi.nlm.nih.gov/pubmed/32805487 http://dx.doi.org/10.1016/j.omtn.2020.07.021 |
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