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: | , , , , , |
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| 格式: | Journal article |
| 語言: | English |
| 出版: |
MDPI
2023
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