Application of Antisense Conjugates for the Treatment of Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is one of the most common muscular dystrophies and can be potentially treated with antisense therapy decreasing mutant DMPK, targeting miRNAs or their binding sites or via a blocking mechanism for MBNL1 displacement from the repeats. Unconjugated antisense molecules a...

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Detalles Bibliográficos
Autores principales: Stoodley, Jessica, Vallejo-Bedia, Francisco, Seone-Miraz, David, Debasa-Mouce, Manuel, Wood, Matthew J. A., Varela, Miguel A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9916419/
https://www.ncbi.nlm.nih.gov/pubmed/36769018
http://dx.doi.org/10.3390/ijms24032697

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