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...
Main Authors: | Jessica Stoodley, Francisco Vallejo-Bedia, David Seone-Miraz, Manuel Debasa-Mouce, Matthew J. A. Wood, Miguel A. Varela |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2023-01-01
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Series: | International Journal of Molecular Sciences |
Subjects: | |
Online Access: | https://www.mdpi.com/1422-0067/24/3/2697 |
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