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

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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Bibliographic Details
Main Authors: Jessica Stoodley, Francisco Vallejo-Bedia, David Seone-Miraz, Manuel Debasa-Mouce, Matthew J. A. Wood, Miguel A. Varela
Format: Article
Language:English
Published: MDPI AG 2023-01-01
Series:International Journal of Molecular Sciences
Subjects:
antibody
bridged nucleic acids
cell-penetrating peptide
lipids
muscle
myotonic dystrophy
Online Access:https://www.mdpi.com/1422-0067/24/3/2697

Internet

https://www.mdpi.com/1422-0067/24/3/2697

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