Defined d-hexapeptides bind CUG repeats and rescue phenotypes of myotonic dystrophy myotubes in a Drosophila model of the disease
Abstract In Myotonic Dystrophy type 1 (DM1), a non-coding CTG repeats rare expansion disease; toxic double-stranded RNA hairpins sequester the RNA-binding proteins Muscleblind-like 1 and 2 (MBNL1 and 2) and trigger other DM1-related pathogenesis pathway defects. In this paper, we characterize four d...
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2021-09-01
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Series: | Scientific Reports |
Online Access: | https://doi.org/10.1038/s41598-021-98866-0 |