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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Detalles Bibliográficos
Autores principales: Cerro-Herreros, Estefanía, González-Martínez, Irene, Moreno-Cervera, Nerea, Overby, Sarah, Pérez-Alonso, Manuel, Llamusí, Beatriz, Artero, Rubén
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
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

Internet

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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